The adaptable FLASH-CAR platform can be used to create personalized cell therapy from a patients own cells, as well as off-the-shelf cell therapy from a universal donor

(NASDAQ: AVCO) announced Friday that it is advancing its next-generation immune cell therapy to treat blood cancers using FLASH-CAR technology co-developed with strategic partner Arbele Limited.

The adaptable FLASH-CAR platform can be used to create personalized cell therapy from a patients own cells, as well as off-the-shelf cell therapy from a universal donor.

Currently, the Chimeric Antigen Receptor T (CAR-T) cellular immunotherapy platform is available. It involves a patients own T-cells a type of white blood cell that protects against infections and other diseases including cancer that are turned into personalized cancer-fighting cells.

The T-cells are removed from the patient, reprogrammed in the lab using a viral vector to target cancer cells, and infused back into the patient as a cancer immunotherapy.

But in contrast to these existing therapies, Avalon said its FLASH-CAR platform uses next-generation CAR technology to modify patients T-cells and natural killer (NK) cells using a ribonucleic acid (RNA)-based platform rather than a viral vector.

Similar to T-cells, NK cells are a type of white blood cell, also able to attack cancer cells, but utilize different mechanisms. By using RNA molecules rather than a viral vector, Avalons RNA-based CAR technology is designed to rapidly create personalized CAR therapies in 1 to 2 days compared to the 10- to 14-day bio-manufacturing time necessary to generate currently available CAR-T cellular immunotherapy.

Avalon said its FLASH-CAR technology is also designed to reprogram the immune cells to hone in on multiple crucial cancer cell targets, called tumor antigens, to potentially achieve superior therapeutic effect. Avoiding the use of viral vectors and complicated bio-processing procedures significantly reduces manufacturing costs, resulting in a more affordable and potentially breakthrough therapy for cancer patients.

The FLASH-CAR technology can also be used to generate off-the-shelf,universal cell therapy that has the potential to reach even more patients.

Avalons first FLASH-CAR platform candidate, AVA-011, targets both CD19 and CD22 tumor antigens on cancer cells.

Pre-clinical research on AVA-011, including tumor cytotoxicity studies, has been successfully completed and Avalon said it is immediately entering the process development stage to generate clinical-grade CAR-T and CAR-NK cells for use in human clinical trials.

Avalon and Arbele have jointly filed for US patents for this RNA-based CAR platform cellular therapy and for other applications.

Avalon expects to begin a first-in-human clinical trial with AVA-011 for the treatment of relapsed or refractory B-cell lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma in the first quarter of 2021. The goal is to use AVA-011 as a bridge to bone marrow stem cell transplant therapy, currently the only curative approach for patients with these blood cancers.

Avalon GloboCare is committed to decreasing the time it takes to deliver cellular immunotherapies to cancer patients, as well as lowering the cost of manufacturing by building on our unique RNA-based CAR platform that does not require using a viral vector, said CEO Dr David Jin.

We are accelerating our innovative discovery and development plan, as well as delivering precise clinical execution and leadership in cellular immunotherapy. Our pre-clinical studies are encouraging and we are excited for AVA-011 to enter the clinical development stage, including multi-center clinical trials following completion of process development to generate the cell therapy.

Arbele CEO John Luk added: Through this strategic partnership with Avalon GloboCare, we envision an accelerated scientific and clinical development of the RNA-based FLASH-CAR technology platform with great potential to generate 'off-the-shelf'immune effector cell therapies to treat both hematologic and solid malignancies.

Avalon, based in Freehold, New Jersey, specializes in developing cell-based technologies and is involved in the management of stem-cell banks and clinical laboratories.

The companys stock recently traded up 10% to $1.99a share in New York.

--UPDATES stock price--

Contact the author: [emailprotected]

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Thalassemia is a type of inherited blood disorder. It is passed from parents to children through genes. This disorder involves lack of oxygen-carrying protein called hemoglobin (an important part of red blood cells). When there is insufficiency of hemoglobin in the body, the red blood cells dont function properly. It also reduces the life of RBC, which means fewer healthy RBC travel in the blood.

RBC carries oxygen to all the cells of the body. Oxygen acts as food, which is used by cells to function. Shortage of healthy RBC means shortage in supply of oxygen to all other cells of the body. This may lead to lethargy in a person. The person may feel tired, weak or short of breath. This condition is termed as Anaemia.

People with thalassemia may suffer from mild or acute Anaemia. Acute Anaemia can be very severe and can lead to damage of major organs. It can even cause death.

Thalassemia major babies are born to parents who are carriers of thalassemia gene. According to rough estimates, each year some 10000 babies are born in India with thalassemia. Best way to prevent or eliminate thalassemia is screening of all pregnant women between 9 to 12 weeks.

Thalassemia is diagnosed through blood tests which include doing a complete blood count (CBC) and special hemoglobin tests. Through a sample of blood, CBC measures the amount of hemoglobin and the different kind of blood cells, such as red blood cells. Hemoglobin tests measure the types of hemoglobin in blood.

Moderate and acute thalassemia is usually diagnosed in childhood. This is because signs and symptoms, such as acute Anemia usually occur at an early age of 2 years. People who have mild form of thalassemia may get diagnosed after a routine blood test, as it will detect if they have anemia.

Here's Dr. Rahul Bhargava, Director and Head, Hematology, Haemato- Oncology and Bone Marrow Transplant, Fortis Memorial Research Institute, Gurugram has to say about the treatments:

Blood Transfusion

Treatment of thalassemia major relies on regular blood transfusion at regular intervals, to keep Hb above 9 gm. percent. It will help prevent form short stature and other skeletal and facial deformities. Recurrent lifelong blood transfusion since 6 months of birth is necessary.

Iron Chelation Therapy

With transfusion comes the problem of iron deposition, as each blood transfusion lead to incremental iron deposition in various tissues like pituitary gland liver and heart leading to early death. So along with transfusion patient also needs iron chelation therapy. It can be either oral (defriprone and defreseirox) or IV desferoxmine. Serum ferritin is one of the surrogate markers of iron overload in thalassemia patients. It needs to be done every 3 months. Gov.s efforts of providing free blood products and iron chelators is bearing fruits as life expectancy has shown an upward trend.

Bone Marrow or Stem Cell Transplant

As it is commonly known, bone marrow or stem cell transplant is the only curative modality for thalassemia. If done at an early age, 80 percent patients can be cured. Source of stem cell could be either brother or sister whose HLA is a complete match. Otherwise fully matched HLA donor can be tried in various international registries. This process is called as match unrelated donor transplant.

Gene Therapy

Gene therapy is gaining lot of traction in field of hemoglobinopathies. It has shown remarkable result with minimum toxicities and sustained haemoglobin production in various trials. There has been no major risk of cancer or other late effects.

We have come a long way and probably this decade will bring the much awaited cheers to thalassaemics. Till then in India, prevention is the only strategy to reduce the burden on already stretched health care system.

Better rate of blood transfusion

Regular Blood screening has significantly impacted reduction of infections due to blood transfusion

Significant improvement in treatment

Bone Marrow Transplant and Stem cell transplantation has led to patients having a good quality of life

Read more| 10 things to keep in mind while travelling with Asthma

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A Washington based biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announcedthe availability of thepre-print version of the manuscriptdescribing the immunological mechanism by whichleronlimabrestores immune function and impacts disease in COVID-19 patients.

This manuscript,Disruption of the CCL5/RANTES-CCR5 Pathway Restores Immune Homeostasis and Reduces Plasma Viral Load in Critical COVID-19, has been shared with the World Health Organization and is currently under peer review, said CytoDyn, Inc., as of May 6, 2020.

As described in thepre-print, in a cohort of 10critically ill patients, after treatment with leronlimab, these critically ill patients experienced reversed hyperimmune activation and inflammation, as well as reversed immunosuppression, therebyfacilitating a more effective immune responsecorrelated with decreases in SARS-CoV-2 level in blood.

These results demonstrate a novel approach to resolving unchecked inflammation whilerestoring immunologic deficiencies.

This is an important finding since according to various studies, a major driver of severe COVID-19 disease is excessive inflammation.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn said in a press statement, We are now most hopeful the entire medical community will understand the potential benefit leronlimab can provide critically ill COVID-19 patients."

"Moreover, this discovery by Dr. Bruce Patterson that leronlimab decreases plasma viral load may have tremendous long-term positive ramifications to bring this pandemic under control. We are grateful that we are able to release this research at such a critical time for patients throughout the world.

Leronlimab (PRO 140) is being used as a treatment for severe COVID-19 under the emergency Investigational New Drug (IND) recently granted by the U.S. Food and Drug Administration (FDA).

Leronlimab is a drug candidate that is aCCR5 antagonist with the potential for multiple therapeutic indications.Leronlimab belongs to a group of HIV drugs calledCCR5antagonists.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions.

Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells.

Previously, CytoDyn announced it is seeking a compassionate use designation for leronlimabfor the treatment of COVID-19 patients who are ineligible for participation in its two existing clinical trials. If this request is granted by the FDA, it will significantly expand the pool of patients who would be eligible to receive leronlimabtherapy.

CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor.

Precision Vaccinations publishes developing COVID-19 therapeutic drug news.

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A 7-year-old boy in Marylandwho suffers from sickle cell anemiais on his way to a full recovery after being hospitalized with a case of the coronavirus, according to reports.

Nasir Striggs was first hospitalized at Sinai Hospital in Baltimore in early April. His mother, Deshannon Striggs, brought him in for an examination after she noticed her son was experiencing trouble breathing.

He tested positive for COVID-19 at the hospital. An X-ray revealed he also had pneumonia in both lungs. The child, diagnosed with sickle cell disease, an inherited red blood cell disorder, underwent several blood transfusions at the hospital before his release.

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Nasir Striggs, 7, who has sickle cell anemia, is home from the hospital after recovering from the coronavirus. (Courtesy: Deshannon Striggs)

He had to keep getting stuck by the needle because the needle kept coming out, the mother told WBAL. To watch him go through that, it was really scary.

After undergoing treatment for several days, his condition began to improve, Deshannon said. She said prayers and support, as well as the dedicated care from the hospitals medical team, have helped her sons recovery.

Just keep the faith. Thats the message: keep the faith, she said.

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Deshannon said doctors have been monitoringNasirs conditionvia virtual check-ups since he was discharged from the hospital. Photos she shared with Fox News show the boy at home smiling,his face mask pulled beneath his chin.

Sickle cell disease is usually diagnosed shortly after birth. The genetic disorder results in oxygen-carrying red blood cells taking on a C or sickle shape, instead of round,often getting stuck in small blood vessels and clogging blood flow, according to the Centers for Disease Control and Prevention.

Children with the disease are at an increased risk of infection and other health problems. The only known cure is a bone marrow or stem cell transplant.

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VANCOUVER, Washington, May 08, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, today further clarified the status of the Companys submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for leronlimab as a combination therapy with HAART for highly treatment experienced HIV patients, filed on April 27, 2020 with the FDA. The BLA will not be considered completed until the Company submits to the FDA clinical datasets required to address FDA comments it received in March 2020, as described in the Companys press releases on May 4 and May 6, 2020. CytoDyn expects to submit these clinical datasets on May 11, 2020.

After the BLA submission is deemed completed, FDA makes a filing decision and sets a PDUFA goal date. CytoDyn has Fast Track designation for leronlimab and a rolling review for its BLA, as previously assigned by the FDA and the Company plans to request a priority review for the BLA. A priority review designation, if granted, means the FDAs goal is to take action on the application within six months of receipt (compared with 10 months under standard review).

About Coronavirus Disease 2019CytoDyn is currently enrolling patients in two clinical trials for COVID-19, a Phase 2 randomized clinical trial for mild-to-moderate COVID-19 population in the U.S. and a Phase 2b/3 randomized clinical trial for severe and critically ill COVID-19 population in several hospitals throughout the country.

SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140) and BLA Submission for the HIV Combination TherapyThe FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer.Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

The Company filed its BLA for Leronlimab as a Combination Therapy for Highly Treatment Experienced HIV Patients to the FDA on April 27, 2020. The BLA will not be considered completed until the Company submits required clinical datasets to the FDA. The Company expects to submit the required datasets on May 11, 2020. After the BLA submission is considered completed, FDA will make a filing decision and set a PDUFA goal date. CytoDyn has Fast Track designation for leronlimab and a rolling review for its BLA, as previously assigned by the FDA and the Company plans to request a priority review for the BLA. A priority review designation means the FDAs goal is to take action on the marketing application within six months of receipt (compared with 10 months under standard review).

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells.The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn filed its BLA in April 2020 to seek FDA approval for leronlimab as a combination therapy for highly treatment experienced HIV patients, and plans to submit additional datasets needed to complete the BLA on May 11, 2020. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors: Dave Gentry, CEORedChip CompaniesOffice: 1.800.RED.CHIP (733.2447)Cell: 407.491.4498dave@redchip.com

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You should read the following discussion and analysis of our financial conditionand results of operations together with the condensed consolidated financialstatements and related notes that are included elsewhere in this QuarterlyReport on Form 10-Q and our Annual Report on Form 10-K for the fiscal year endedDecember 31, 2019 filed with the U.S. Securities and Exchange Commission, or theSEC, on March 6, 2020, or our 2019 Form 10-K. This discussion containsforward-looking statements based upon current plans, expectations and beliefsthat involve risks and uncertainties. Our actual results may differ materiallyfrom those anticipated in these forward-looking statements as a result ofvarious factors, including, but not limited to, those discussed in the sectionentitled "Risk Factors" and elsewhere in this Quarterly Report on Form 10-Q. Inpreparing this MD&A, we presume that readers have access to and have read theMD&A in our 2019 Form 10-K, pursuant to Instruction 2 to paragraph (b) of Item303 of Regulation S-K. Unless stated otherwise, references in this QuarterlyReport on Form 10-Q to "us," "we," "our," or our "Company" and similar termsrefer to Rocket Pharmaceuticals, Inc.

We are a clinical-stage, multi-platform biotechnology company focused on thedevelopment of first, only and best-in-class gene therapies, with directon-target mechanism of action and clear clinical endpoints, for rare anddevastating diseases. We currently have three clinical-stage ex vivo lentiviralvector ("LVV") programs currently enrolling patients in the US and EU forFanconi Anemia ("FA"), a genetic defect in the bone marrow that reducesproduction of blood cells or promotes the production of faulty blood cells,Leukocyte Adhesion Deficiency-I ("LAD-I"), a genetic disorder that causes theimmune system to malfunction and Pyruvate Kinase Deficiency ("PKD"), a rare redblood cell autosomal recessive disorder that results in chronic non-spherocytichemolytic anemia. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-Iprogram are in registration-enabling studies in the US and EU. In addition, inthe US we have a clinical stage in vivo adeno-associated virus ("AAV") programfor Danon disease, a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Finally, we have a pre-clinical stage LVV programfor Infantile Malignant Osteopetrosis ("IMO"), a genetic disorder characterizedby increased bone density and bone mass secondary to impaired bone resorption -this program is anticipated to enter the clinic in 2020. We have globalcommercialization and development rights to all of these product candidatesunder royalty-bearing license agreements. Additional work in the discovery stagefor an FA CRISPR/CAS9 program as well as a gene therapy program for the lesscommon FA subtypes C and G is ongoing.

Recent Developments

On February 20, 2020, we entered into separate, privately negotiated exchangeagreements (the "Exchange Agreements") with certain holders of our outstanding5.75% Convertible Senior Notes due 2021 (the "2021 Convertible Notes") to extendthe maturity date by one year. Pursuant to the Exchange Agreements, we exchangedapproximately $39.35 million aggregate principal amount of the 2021 ConvertibleNotes (which represents approximately 76% of the aggregate outstanding principalamount of the 2021 Convertible Notes) for (a) approximately $39.35 millionaggregate principal amount of 6.25% Convertible Senior Notes due August 2022(the "2022 Convertible Notes") (an exchange ratio equal to 1.00 2022 ConvertibleNote per exchanged 2021 Convertible Note) and (b) $119,416 in cash to pay theaccrued and unpaid interest on the exchanged 2021 Convertible Notes from, andincluding, February 1, 2020 to February 20, 2020. The 2022 Convertible Noteswere issued in private placements exempt from registration in reliance onSection 4(a) (2) of the Securities Act of 1933, as amended (the "SecuritiesAct"). Upon completion of the exchange transactions, approximately $12.65million aggregate principal amount of 2021 Convertible Notes remainedoutstanding.

Gene Therapy Overview

Genes are composed of sequences of deoxyribonucleic acid ("DNA"), which code forproteins that perform a broad range of physiologic functions in all livingorganisms. Although genes are passed on from generation to generation, geneticchanges, also known as mutations, can occur in this process. These changes canresult in the lack of production of proteins or the production of alteredproteins with reduced or abnormal function, which can in turn result in disease.

Gene therapy is a therapeutic approach in which an isolated gene sequence orsegment of DNA is administered to a patient, most commonly for the purpose oftreating a genetic disease that is caused by genetic mutations. Currentlyavailable therapies for many genetic diseases focus on administration of largeproteins or enzymes and typically address only the symptoms of the disease. Genetherapy aims to address the disease-causing effects of absent or dysfunctionalgenes by delivering functional copies of the gene sequence directly into thepatient's cells, offering the potential for curing the genetic disease, ratherthan simply addressing symptoms.

We are using modified non-pathogenic viruses for the development of our genetherapy treatments. Viruses are particularly well suited as delivery vehiclesbecause they are adept at penetrating cells and delivering genetic materialinside a cell. In creating our viral delivery vehicles, the viral (pathogenic)genes are removed and are replaced with a functional form of the missing ormutant gene that is the cause of the patient's genetic disease. The functionalform of a missing or mutant gene is called a therapeutic gene, or the"transgene." The process of inserting the transgene is called "transduction."Once a virus is modified by replacement of the viral genes with a transgene, themodified virus is called a "viral vector." The viral vector delivers thetransgene into the targeted tissue or organ (such as the cells inside apatient's bone marrow). We have two types of viral vectors in development, LVVand AAV. We believe that our LVV and AAV-based programs have the potential tooffer a long-lasting and significant therapeutic benefit to patients.

Gene therapies can be delivered either (1) ex vivo (outside the body), in whichcase the patient's cells are extracted and the vector is delivered to thesecells in a controlled, safe laboratory setting, with the modified cells thenbeing reinserted into the patient, or (2) in vivo (inside the body), in whichcase the vector is injected directly into the patient, either intravenously("IV") or directly into a specific tissue at a targeted site, with the aim ofthe vector delivering the transgene to the targeted cells.

We believe that scientific advances, clinical progress, and the greaterregulatory acceptance of gene therapy have created a promising environment toadvance gene therapy products as these products are being designed to restorecell function and improve clinical outcomes, which in many cases includeprevention of death at an early age.

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The chart below shows the current phases of development of Rocket's programs andproduct candidates:

LVV Programs. Rocket's LVV-based programs utilize third-generation,self-inactivating lentiviral vectors to target selected rare diseases.Currently, Rocket is developing LVV programs to treat FA, LAD-I, PKD, and IMO.

Fanconi Anemia Complementation Group A (FANCA):

FA, a rare and life-threatening DNA-repair disorder, generally arises from amutation in a single FA gene. An estimated 60 to 70% of cases arise frommutations in the Fanconi-A ("FANCA") gene, which is the focus of our program. FAresults in bone marrow failure, developmental abnormalities, myeloid leukemiaand other malignancies, often during the early years and decades of life. Bonemarrow aplasia, which is bone marrow that no longer produces any or very few redand white blood cells and platelets leading to infections and bleeding, is themost frequent cause of early morbidity and mortality in FA, with a median onsetbefore 10 years of age. Leukemia is the next most common cause of mortality,ultimately occurring in about 20% of patients later in life. Solid organmalignancies, such as head and neck cancers, can also occur, although at lowerrates during the first two to three decades of life.

Although improvements in allogeneic (donor-mediated) hematopoietic stem celltransplant ("HSCT"), currently the most frequently utilized therapy for FA, haveresulted in more frequent hematologic correction of the disorder, HSCT isassociated with both acute and long-term risks, including transplant-relatedmortality, graft versus host disease ("GVHD"), a sometimes fatal side effect ofallogeneic transplant characterized by painful ulcers in the GI tract, livertoxicity and skin rashes, as well as increased risk of subsequent cancers. Ourgene therapy program in FA is designed to enable a minimally toxic hematologiccorrection using a patient's own stem cells during the early years of life. Webelieve that the development of a broadly applicable autologous gene therapy canbe transformative for these patients.

Each of our LVV-based programs utilize third-generation, self-inactivatinglentiviral vectors to correct defects in patients' HSCs, which are the cellsfound in bone marrow that are capable of generating blood cells over a patient'slifetime. Defects in the genetic coding of HSCs can result in severe, andpotentially life-threatening anemia, which is when a patient's blood lacksenough properly functioning red blood cells to carry oxygen throughout the body.Stem cell defects can also result in severe and potentially life-threateningdecreases in white blood cells resulting in susceptibility to infections, and inplatelets responsible for blood clotting, which may result in severe andpotentially life-threatening bleeding episodes. Patients with FA have a geneticdefect that prevents the normal repair of genes and chromosomes within bloodcells in the bone marrow, which frequently results in the development of acutemyeloid leukemia ("AML"), a type of blood cancer, as well as bone marrow failureand congenital defects. The average lifespan of an FA patient is estimated to be30 to 40 years. The prevalence of FA in the US and EU is estimated to be about4,000, and given the efficacy seen in non-conditioned patients, the addressableannual market opportunity is now thought to be in the 400 to 500 range.

We currently have one LVV-based program targeting FA, RP-L102. RP-L102 is ourlead lentiviral vector based program that we in-licensed from Centro deInvestigaciones Energticas, Medioambientales y Tecnolgicas ("CIEMAT"), whichis a leading research institute in Madrid, Spain. RP-L102 is currently beingstudied in our sponsored Phase 2 registrational enabling clinical trialstreating FA patients initially at the Center for Definitive and CurativeMedicine at Stanford University School of Medicine ("Stanford") and HospitalInfantil de Nino Jesus ("HNJ") in Spain. The Phase 2 portion of the trial isexpected to enroll ten patients total from the U.S. and EU. Patients willreceive a single IV infusion of RP-L102 that utilizes fresh cells and "ProcessB" which incorporates a modified stem cell enrichment process, transductionenhancers, as well as commercial-grade vector and final drug product.

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Table of ContentsIn October 2019, at the European Society of Cell and Gene Therapy ("ESGCT") 2019Annual Congress, long-term Phase 1/2 clinical data of RP-L102, from the clinicaltrial sponsored by CIEMAT, for FA "Process A", without the use of myeloablativeconditioning was presented demonstrating evidence of increasing and durableengraftment leading to bone marrow restoration exceeding the 10% thresholdagreed to by the FDA and EMA for the ongoing registration-enabling Phase 2trial. In patient 02002, who received what we consider adequate drug product,hemoglobin levels are now similar to those in the first year after birth,suggesting hematologic correction over the long term.

During the third quarter of 2019, we received alignment from the FDA on thetrial design and the primary endpoint. This alignment was similar to thatpreviously received from the European Medicines Agency ("EMA"). Resistance tomitomycin-C, a DNA damaging agent, in bone marrow stem cells at a minimum timepoint of one year to serve as the primary endpoint for our Phase II study. InDecember 2019, we announced that the first patient of the global Phase 2 studyfor RP-L102 "Process B" for FA received investigational therapy. There will betotal of 10 patients enrolled in the global Phase 2 studies.

In December 2019, we also announced preliminary results from two pediatricpatients treated with "Process B" RP-L102 prior to development of severe bonemarrow failure in our Phase 1 trial of RP-L102 for FA. To evaluate transductionefficiency, an analysis of the proportion of the MMC-resistant colony formingcells was conducted and both patients have thus far exhibited early signs ofengraftment, including increases in blood cell lineages in one patient. Nodrug-related safety or tolerability issues have been reported.

Leukocyte Adhesion Deficiency-I (LAD-I):

LAD-I is a rare autosomal recessive disorder of white blood cell adhesion andmigration, resulting from mutations in the ITGB2 gene encoding for the Beta-2Integrin component, CD18. Deficiencies in CD18 result in an impaired ability forneutrophils (a subset of infection-fighting white blood cells) to leave bloodvessels and enter into tissues where these cells are needed to combatinfections. As is the case with many rare diseases, true estimates of incidenceare difficult; however, several hundred cases have been reported to date.

Most LAD-I patients are believed to have the severe form of the disease. SevereLAD-I is notable for recurrent, life-threatening infections and substantialinfant mortality in patients who do not receive an allogeneic HSCT. Mortalityfor severe LAD-I has been reported as 60 to 75% by age two in the absence ofallogeneic HCST.

We currently have one program targeting LAD-I, RP-L201. RP-L201 is a clinicalprogram that we in-licensed from CIEMAT. We have partnered with UCLA to leadU.S. clinical development efforts for the LAD-I program. UCLA and its Eli andEdythe Broad Center of Regenerative Medicine and Stem Cell Research is servingas the lead U.S. clinical research center for the registrational clinical trialfor LAD-I, and HNJ is serving as the lead clinical site in Spain.

The ongoing open-label, single-arm, Phase 1/2 registration enabling clinicaltrial of RP-L201 has dosed one severe LAD-I patient in the U.S. to assess thesafety and tolerability of RP-L201. The first patient was treated with RP-L201in third quarter 2019. This study has received $6.5 million CLIN2 grant awardfrom the California Institute for Regenerative Medicine ("CIRM") to support theclinical development of gene therapy for LAD-I.

In December 2019, we announced initial results from the first pediatric patienttreated with RP-L201, demonstrating early evidence of safety. Analyses ofperipheral vector copy number ("VCN"), and CD18-expressing neutrophils wereperformed through three months after infusion of RP-L201 to evaluate engraftmentand phenotypic correction. The patient exhibited early signs of engraftment withVCN myeloid levels at 1.5 at three months and CD-18 expression of 45%. No safetyor tolerability issues related to RP-L201 administration (or investigationalproduct) had been identified as of that date. The study is expected to enrollnine patients globally.

Pyruvate Kinase Deficiency (PKD):

Red blood cell PKD is a rare autosomal recessive disorder resulting frommutations in the pyruvate kinase L/R ("PKLR") gene encoding for a component ofthe red blood cell ("RBC") glycolytic pathway. PKD is characterized by chronicnon-spherocytic hemolytic anemia, a disorder in which RBCs do not assume anormal spherical shape and are broken down, leading to decreased ability tocarry oxygen to cells, with anemia severity that can range from mild(asymptomatic) to severe forms that may result in childhood mortality or arequirement for frequent, lifelong RBC transfusions. The pediatric population isthe most commonly and severely affected subgroup of patients with PKD, and PKDoften results in splenomegaly (abnormal enlargement of the spleen), jaundice andchronic iron overload which is likely the result of both chronic hemolysis andthe RBC transfusions used to treat the disease. The variability in anemiaseverity is believed to arise in part from the large number of diverse mutationsthat may affect the PKLR gene. Estimates of disease incidence have rangedbetween 3.2 and 51 cases per million in the white U.S. and EU population.Industry estimates suggest at least 2,500 cases in the U.S. and EU have alreadybeen diagnosed despite the lack of FDA-approved molecularly targeted therapies.Enrollment is currently ongoing and we anticipate treating the first patient inthe third quarter of 2020.

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Table of ContentsWe currently have one LVV-based program targeting PKD, RP-L301. RP-L301 is aclinical stage program that we in-licensed from CIEMAT. The IND for RP-L301 toinitiate a global Phase 1 study was cleared by the FDA in October 2019. Thisprogram has been granted EMA orphan drug disease designation and FDA orphan drugdisease designation ("ODD").

This global Phase 1 open-label, single-arm, clinical trial is expected to enrollsix adult and pediatric transfusion-dependent PKD patients in the U.S. andEurope. Lucile Packard Children's Hospital Stanford will serve as the lead sitein the U.S. for adult and pediatric patients, and Hospital InfantilUniversitario Nio Jess will serve as the lead site in Europe for pediatricsand Hospital Universitario Fundacin Jimnez Daz will serve as the lead site inEurope for adult patients.

Infantile Malignant Osteopetrosis (IMO):

IMO is a genetic disorder characterized by increased bone density and bone masssecondary to impaired bone resorption. Normally, small areas of bone areconstantly being broken down by special cells called osteoclasts, then madeagain by cells called osteoblasts. In IMO, the cells that break down bone(osteoclasts) do not work properly, which leads to the bones becoming thickerand not as healthy. Untreated IMO patients may suffer from a compression of thebone-marrow space, which results in bone marrow failure, anemia and increasedinfection risk due to the lack of production of white blood cells. Untreated IMOpatients may also suffer from a compression of cranial nerves, which transmitsignals between vital organs and the brain, resulting in blindness, hearing lossand other neurologic deficits.

We currently have one LVV-based program targeting IMO, RP-L401. RP-L401 is apreclinical program that we in-licensed from Lund University, Sweden. Thisprogram has been granted ODD and Rare Pediatric Disease designation from theFDA. The FDA defines a "rare pediatric disease" as a serious andlife-threatening disease that affects less than 200,000 people in the U.S. thatare aged between birth to 18 years. The Rare Pediatric Disease designationprogram allows for a sponsor who receives an approval for a product topotentially qualify for a voucher that can be redeemed to receive a priorityreview of a subsequent marketing application for a different product. We havepartnered with UCLA to lead U.S. clinical development efforts for the IMOprogram and anticipate that UCLA will serve as the lead U.S. clinical site forIMO. We intend to file an IND for IMO and commence our clinical trial in thefourth quarter of 2020.

Danon disease is a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Danon disease is caused by mutations in the geneencoding lysosome-associated membrane protein 2 ("LAMP-2"), a mediator ofautophagy. This mutation results in the accumulation of autophagic vacuoles,predominantly in cardiac and skeletal muscle. Male patients often require hearttransplantation and typically die in their teens or twenties from progressiveheart failure. Along with severe cardiomyopathy, other Danon disease symptomscan include skeletal muscle weakness, liver disease, and intellectualimpairment. There are no specific therapies available for the treatment of Danondisease. RP-A501 is in clinical trials as an in vivo therapy for Danon disease,which is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S.and the EU, however new market research is being performed and the prevalence ofpatients may be updated in the future.

In January 2019, we announced the clearance of our IND application by the FDAfor RP-A501, and in February 2019, we were notified by the FDA that we weregranted Fast Track designation for RP-A501. University of California San DiegoHealth is the initial and lead center for our Phase 1 clinical trial.

On May 2, 2019, we presented additional preclinical data at the ASCGT annualmeeting, indicating that high VCN, in Danon disease-relevant organs in both miceand non-human primates ("NHN's"), with high concentrations in heart and livertissue (for NHP, cardiac VCN was approximately 10 times higher on average thanin skeletal muscle and central nervous system), which is consistent withreported results in several studies of heart tissue across different species.There were no treatment-related adverse events or safety issues up to thehighest dose. We have dosed three patients in the RP-A501 phase 1 clinicaltrial. We will continue further enrollment with clinical data read-outs in thefourth quarter of 2020.

As of March 2020, we have dosed three patients in the RP-A501 phase 1 clinicaltrial. This completes the first low dose cohort of the Phase 1 study. Based onthe preliminary safety and efficacy data review of this completed cohort, boththe FDA and IDMC has provided clearance to advance to a higher dose cohort inPhase 1 Trial of RP-A501 for Danon Disease. We will continue further enrollmentwith clinical data read-outs in the second half of 2020.

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In addition to its LVV and AAV programs, we also have a program evaluatingCRISPR/Cas9-based gene editing for FA. This program is currently in thediscovery phase. CRISPR/Cas9-based gene editing is a different method ofcorrecting the defective genes in a patient, where the editing is very specificand targeted to a particular gene sequence. "CRISPR/Cas9" stands for Clustered,Regularly Interspaced Short Palindromic Repeats ("CRISPR") Associated protein-9.The CRISPR/Cas9 technology can be used to make "cuts" in DNA at specific sitesof targeted genes, making it potentially more precise in delivering genetherapies than traditional vector-based delivery approaches. CRISPR/Cas9 canalso be adapted to regulate the activity of an existing gene without modifyingthe actual DNA sequence, which is referred to as gene regulation.

Strategy

We seek to bring hope and relief to patients with devastating, undertreated,rare pediatric diseases through the development and commercialization ofpotentially curative first-in-class gene therapies. To achieve these objectives,we intend to develop into a fully-integrated biotechnology company. In the near-and medium-term, we intend to develop our first-in-class product candidates,which are targeting devastating diseases with substantial unmet need, developproprietary in-house analytics and manufacturing capabilities and continue tocommence registration trials for our currently planned programs. In the mediumand long-term, we expect to submit our first biologics license applications("BLAs"), and establish our gene therapy platform and expand our pipeline totarget additional indications that we believe to be potentially compatible withour gene therapy technologies. In addition, during that time, we believe thatour currently planned programs will become eligible for priority review vouchersfrom the FDA that provide for expedited review. We have assembled a leadershipand research team with expertise in cell and gene therapy, rare disease drugdevelopment and commercialization.

We believe that our competitive advantage lies in our disease-based selectionapproach, a rigorous process with defined criteria to identify target diseases.We believe that this approach to asset development differentiates us as a genetherapy company and potentially provides us with a first-mover advantage.

Financial Overview

Since our inception, we have devoted substantially all of our resources toorganizing and staffing the Company, business planning, raising capital,acquiring or discovering product candidates and securing related intellectualproperty rights, conducting discovery, research and development activities forthe programs and planning for potential commercialization. We do not have anyproducts approved for sale and have not generated revenue from product sales.From inception through March 31, 2020, we raised net cash proceeds ofapproximately $373.1 million from investors through both equity and convertibledebt financing to fund operating activities. As of March 31, 2020, we had cash,cash equivalents and investments of $275.9 million.

Since inception, we have incurred significant operating losses. Our ability togenerate product revenue sufficient to achieve profitability will depend heavilyon the successful development and eventual commercialization of one or more ofthe current or future product candidates and programs. We had net losses of$24.7 million for the three months ended March 31, 2020 and $77.3 million forthe year ended December 31, 2019. As of March 31, 2020, we had an accumulateddeficit of $207.8 million. We expect to continue to incur significant expensesand higher operating losses for the foreseeable future as we advance our currentproduct candidates from discovery through preclinical development and clinicaltrials and seek regulatory approval of our product candidates. In addition, ifwe obtain marketing approval for any of their product candidates, we expect toincur significant commercialization expenses related to product manufacturing,marketing, sales and distribution. Furthermore, we expect to incur additionalcosts as a public company. Accordingly, we will need additional financing tosupport continuing operations and potential acquisitions of licensing or otherrights for product candidates.

Until such a time as we can generate significant revenue from product sales, ifever, we will seek to fund our operations through public or private equity ordebt financings or other sources, which may include collaborations with thirdparties and government programs or grants. Adequate additional financing may notbe available to us on acceptable terms, or at all. We can make no assurancesthat we will be able to raise the cash needed to fund our operations and, if wefail to raise capital when needed, we may have to significantly delay, scaleback or discontinue the development and commercialization of one or more productcandidates or delay pursuit of potential in-licenses or acquisitions.

Because of the numerous risks and uncertainties associated with productdevelopment, we are unable to predict the timing or amount of increased expensesor when or if we will be able to achieve or maintain profitability. Even if weare able to generate product sales, we may not become profitable. If we fail tobecome profitable or are unable to sustain profitability on a continuing basis,then we may be unable to continue our operations at planned levels and be forcedto reduce or terminate our operations.

Revenue

To date, we have not generated any revenue from any sources, including fromproduct sales, and we do not expect to generate any revenue from the sale ofproducts in the near future. If our development efforts for product candidatesare successful and result in regulatory approval or license agreements withthird parties, we may generate revenue in the future from product sales.

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Research and Development Expenses

Our research and development program ("R&D") expenses consist primarily ofexternal costs incurred for the development of our product candidates. Theseexpenses include:

expenses incurred under agreements with research institutions that conduct

research and development activities including, process development,

preclinical, and clinical activities on Rocket's behalf;

costs related to process development, production of preclinical and clinical

materials, including fees paid to contract manufacturers and manufacturing

input costs for use in internal manufacturing processes;

consultants supporting process development and regulatory activities; and

costs related to in-licensing of rights to develop and commercialize our

product candidate portfolio.

We recognize external development costs based on contractual payment schedulesaligned with program activities, invoices for work incurred, and milestoneswhich correspond with costs incurred by the third parties. Nonrefundable advancepayments for goods or services to be received in the future for use in researchand development activities are recorded as prepaid expenses.

Our direct research and development expenses are tracked on a program-by-programbasis for product candidates and consist primarily of external costs, such asresearch collaborations and third party manufacturing agreements associated withour preclinical research, process development, manufacturing, and clinicaldevelopment activities. Our direct research and development expenses by programalso include fees incurred under license agreements. Our personnel, non-programand unallocated program expenses include costs associated with activitiesperformed by our internal research and development organization and generallybenefit multiple programs. These costs are not separately allocated by productcandidate and consist primarily of:

Our research and development activities are central to our business model.Product candidates in later stages of clinical development generally have higherdevelopment costs than those in earlier stages of clinical development. As aresult, we expect that research and development expenses will increasesubstantially over the next several years as we increase personnel costs,including stock-based compensation, support ongoing clinical studies, seek toachieve proof-of-concept in one or more product candidates, advance preclinicalprograms to clinical programs, and prepare regulatory filings for productcandidates.

We cannot determine with certainty the duration and costs to complete current orfuture clinical studies of product candidates or if, when, or to what extent wewill generate revenues from the commercialization and sale of any of our productcandidates that obtain regulatory approval. We may never succeed in achievingregulatory approval for any of our product candidates. The duration, costs, andtiming of clinical studies and development of product candidates will depend ona variety of factors, including:

the scope, rate of progress, and expense of ongoing as well as any future

clinical studies and other research and development activities that we

undertake;

future clinical trial results;

uncertainties in clinical trial enrollment rates;

changing standards for regulatory approval; and

the timing and receipt of any regulatory approvals.

We expect research and development expenses to increase for the foreseeablefuture as we continue to invest in research and development activities relatedto developing product candidates, including investments in manufacturing, as ourprograms advance into later stages of development and as we conduct additionalclinical trials. The process of conducting the necessary clinical research toobtain regulatory approval is costly and time-consuming, and the successfuldevelopment of product candidates is highly uncertain. As a result, we areunable to determine the duration and completion costs of research anddevelopment projects or when and to what extent we will generate revenue fromthe commercialization and sale of any of our product candidates.

Our future research and development expenses will depend on the clinical successof our product candidates, as well as ongoing assessments of the commercialpotential of such product candidates. In addition, we cannot forecast with anydegree of certainty which product candidates may be subject to futurecollaborations, when such arrangements will be secured, if at all, and to whatdegree such arrangements would affect our development plans and capitalrequirements. We expect our research and development expenses to increase infuture periods for the foreseeable future as we seek to complete development ofour product candidates.

The successful development and commercialization of our product candidates ishighly uncertain. This is due to the numerous risks and uncertainties associatedwith product development and commercialization, including the uncertainty of:

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Table of Contents

the scope, progress, outcome and costs of our clinical trials and other

research and development activities;

the efficacy and potential advantages of our product candidates compared to

alternative treatments, including any standard of care;

the market acceptance of our product candidates;

obtaining, maintaining, defending and enforcing patent claims and other

intellectual property rights;

significant and changing government regulation; and

the timing, receipt and terms of any marketing approvals.

A change in the outcome of any of these variables with respect to thedevelopment of our product candidates that we may develop could mean asignificant change in the costs and timing associated with the development ofour product candidates. For example, if the FDA or another regulatory authoritywere to require us to conduct clinical trials or other testing beyond those thatwe currently contemplate for the completion of clinical development of any ofour product candidates that we may develop or if we experience significantdelays in enrollment in any of our clinical trials, we could be required toexpend significant additional financial resources and time on the completion ofclinical development of that product candidate.

General and Administrative Expenses

General and administrative ("G&A") expenses consist primarily of salaries andrelated benefit costs for personnel, including stock-based compensation andtravel expenses for our employees in executive, operational, finance, legal,business development, and human resource functions. In addition, othersignificant general and administrative expenses include professional fees forlegal, patents, consulting, investor and public relations, auditing and taxservices as well as other expenses for rent and maintenance of facilities,insurance and other supplies used in general and administrative activities. Weexpect general and administrative expenses to increase for the foreseeablefuture due to anticipated increases in headcount to support the continuedadvancement of our product candidates. We also anticipate that we will incurincreased accounting, audit, legal, regulatory, compliance and director andofficer insurance costs as well as investor and public relations expenses.

Interest Expense

Interest expense is related to the 2021 Convertible Notes, which mature inAugust 2021, and the 2022 Convertible Notes, which mature in August 2022.

Interest Income

Interest income is related to interest earned from investments.

Critical Accounting Policies and Significant Judgments and Estimates

Our consolidated financial statements are prepared in accordance with generallyaccepted accounting principles in the U.S. The preparation of our financialstatements and related disclosures requires us to make estimates and judgmentsthat affect the reported amounts of assets, liabilities, costs and expenses, andthe disclosure of contingent assets and liabilities in our financial statements.We base our estimates on historical experience, known trends and events andvarious other factors that we believe are reasonable under the circumstances,the results of which form the basis for making judgments about the carryingvalues of assets and liabilities that are not readily apparent from othersources. We evaluate estimates and assumptions on an ongoing basis. Actualresults may differ from these estimates under different assumptions orconditions.

Our significant accounting policies are described in more detail in our 2019Form 10-K, except as otherwise described below.

Results of Operations

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(MENAFN - iCrowdNewsWire) May 8, 2020

According to the new market research report " Cell Therapy Technologies Market by Product (Consumables, Equipment, Software), Cell Type (Human Stem & Differentiated, Animal), Process Stages (Cell Processing, Distribution, Handling, QC), End User, and Region - Global Forecast to 2023, , published by MarketsandMarkets, The global cell therapy technologies market is projected to reach USD 19.9 billion by 2023 from USD 10.2 billion in 2018, at a CAGR of 14.4% during the forecast period.

Browse in-depth TOC on 'Cell Therapy Technologies Market" 75 - Table30 Figures116 Pages

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Rising government investments for cell-based research, the increasing number of GMP-certified production facilities, and the large number of oncology-oriented cell-based therapy clinical trials are the key factors driving the growth of this market. China, India, Japan, Korea, and Brazil are emerging markets for cell therapy instruments. These markets boast comparatively lenient standards and government regulations as opposed to developed markets in North America and the EU, and thus offer significant growth potential for providers. However, the high cost of cell-based research and the low success rate is expected to restrain market growth to some extent during the forecast period.

Consumables are expected to account for the largest cell therapy technologies market share in 2018 : By product, the cell therapy technologies market is segmented into consumables, equipment, and systems & software. The consumables segment is expected to account for the largest share of the market in 2018. Factors such as increasing investments by companies to develop advanced products as well as government initiatives for enhancing cell-based research are contributing to the growth of the cell therapy consumables market.

Cell processing segment to witness the highest growth during the forecast period :

Based on process, the cell therapy technologies market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control. The cell processing segment is expected to account for the largest market share in 2018 and is projected to witness the highest CAGR during the forecasted period.

Human cells segment accounts for the large share of the cell therapy instruments market, by cell type :

Based on cell type, the market is segmented into human cells and animal cells. In 2018, the human cells segment is expected to account for the largest share of the cell therapy technologies market. The rising adoption of human cells over animal cells for cell therapeutics research, technological advancements, and the rising incidence of diseases such as cancer and cardiac abnormalities are the key factors driving the growth of this segment.

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North America to dominate the cell therapy technologies market during the forecast period : The market is segmented into four major regions, namely, North America, Europe, Asia Pacific, and the Rest of the World (RoW). North America is expected to dominate the market in 2018 owing to the high burden of chronic diseases and increasing R & D activities in the pharmaceutical and biotechnology industries. The Asia Pacific region is expected to register the highest CAGR during the forecast period.

The major players in the western blotting market are Beckman Coulter (US), Becton, Dickinson and Company (US), GE Healthcare (US), Lonza (Switzerland), Merck KGaA (Germany), Miltenyi Biotec (Germany), STEMCELL Technologies, Inc. (Canada), Terumo BCT (US), and Thermo Fisher Scientific (US).

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Korean researchers have found a signal transduction system that modulates the treatment of mesenchymal stem cells and immune control functions, opening the way for treating graft-versus-host disease treatment.

Mesenchymal stem cells divide into various cells, have immunomodulatory functions, and are the primary cell sources for stem cell therapy.

Graft-versus-host disease is a fatal disease that leads to death after an allogeneic blood transfusion or bone marrow transplantation. Although there are many clinical trials underway worldwide to treat the symptom, there are no applicable treatments besides alleviating symptoms with high-dose steroids.

The team, led by Professor Shin Dong-myeong of the Department of Biomedical Sciences at Asan Medical Center, discovered that the CREB1 (CAMP responsive element binding protein 1) signaling system activates the treatment and immune control functions of mesenchymal stem cells.

The team administered a therapeutic agent made by upgrading mesenchymal stem cells to graft-versus-host disease mice, and found that it alleviated anorexia symptoms and reduced the weight loss rate by 30 percent while increasing the survival rate by 30 percent.

When developing a cell therapy product, researchers have to cultivate the stem cells in vitro. Thus it is very likely that it will impair stem cell functions due to free radicals generated in the cells. To prevent the deterioration of stem cell function, it is necessary to improve the stem cell function in vitro culture, prevent stem cell oxidation, and increase the antioxidant capacity of the cell itself.

Until now, there was a lack of specific evidence and understanding of how stem cells regulate glutathione, an indicator of antioxidant capacity. Therefore, it was difficult to prevent stem cell dysfunction and oxidation.

Professor Shin's team developed experimental techniques that can monitor and quantify glutathione in real-time and confirmed that the CREB1 signaling system regulated the amount and activity of glutathione.

By activating the CREB1 signaling system, the team found that the process also activated nuclear factor erythroid 2-related factor 2 (NRF2) protein, which maintains the antioxidant capacity of mesenchymal stem cells and the increase of both the expression levels of peroxiredoxin-1 (PRDX1) and glutamate-cysteine ligase modifier subunit (GCLM) protein, which synthesize glutathione and are antioxidant activity indicators.

As a result, the team confirmed that its method was effective in treating the graft-versus-host disease.

"Based on this study, we have secured a technological foundation to advance stem cell treatment by controlling the antioxidant capacity of stem cells," Professor Shin said.

If this technology makes a high-purity and high-quality stem cell treatment, the team expects that it will be a step toward developing a graft-versus-host disease treatment and overcoming various intractable diseases such as nervous system diseases and inflammatory diseases with high medical demand, Shin added.

The results of the study were published in the journal, Science Advances.

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A group of UB researchers have published a paper that clarifies certain cellular mechanisms that could lead to improved outcomes in patients with globoid cell leukodystrophy, commonly known as Krabbe disease.

The paper, titled Macrophages Expressing GALC Improve Peripheral Krabbe Disease by a Mechanism Independent of Cross-Correction, was published May 5 in the journal Neuron.

The research was led by Lawrence Wrabetz and M. Laura Feltri. Wrabetz and Feltri head the Hunter James Kelly Research Institute and both are professors in the departments of Biochemistry and Neurology in the Jacobs School of Medicine and Biomedical Sciences at UB.

The institute is named for the son of former Buffalo Bills quarterback Jim Kelly. Hunter Kelly died at age 8 in 2005 from complications of Krabbe disease.

Krabbe disease is a progressive and fatal neurologic disorder that usually affects newborns and causes death before a child reaches the age of 2 or 3.

Traditionally, hematopoietic stem cell transplantation, also known as a bone marrow transplant, has improved the long-term survival and quality of life of patients with Krabbe disease, but it is not a cure.

It has long been assumed that the bone marrow transplant works by a process calledcross-correction, in which an enzyme called GALC is transferred from healthy cells to sick cells.

Using a new Krabbe disease animal model and patient samples, the UB researchers determinedthatin reality cross-correctiondoes not occur. Rather, the bone marrow transplant helps patients through a different mechanism.

The researchers first determined which cells are involved in Krabbe disease and by which mechanism. They discovered that both myelin-forming cells, or Schwann cells, and macrophages require the GALC enzyme, which is missing in Krabbe patients due to genetic mutation.

Schwann cells require GALC to prevent the formation of a toxic lipid called psychosine, which causes myelin destruction and damage to neurons. Macrophages require GALC to aid with the degradation of myelin debris produced by the disease.

The research showed that hematopoietic stem cell transplantation does not work bycross-correction, but by providing healthy macrophages with GALC.

According to Feltri, the data reveal that improvingcross-correctionwould be a way to makebone marrow transplants and other experimental therapies such as gene therapy more effective.

Bone marrow transplantation and other treatments for lysosomal storage disorders, such as enzyme replacement therapy, have historically had encouraging but limited therapeutic benefit, says study first author Nadav I. Weinstock, an MD-PhD student in the Jacobs School. Our work defined the precise cellular and mechanistic benefit of bone marrow transplantation in Krabbe disease, while also shedding light on previously unrecognized limitations of this approach.

Future studies, using genetically engineered bone marrow transplantation or other novelapproaches,may one day build on our findings and eventually bridge the gap for effectively treating patients with lysosomal disease, he continues.

UB investigators included Daesung Shin, research assistant professor at the Hunter James Kelly Research Institute; Nicholas Silvestri, clinical associate professor of neurology, Jacobs School; Narayan Dhimal, PhD student; Chelsey B. Reed, MD-PhD student; and undergraduate student Oliver Sampson.

Also participating in the research were Eric E. Irons, MD-PhD student, and Joseph T.Y. Lau, a distinguished faculty member from the Department of Molecular and Cellular Biology at Roswell Park Comprehensive Cancer Center.

The research was funded by multiple grants from the National Institutes of Health awarded to Weinstock, Shin, Wrabetz and Feltri, and also supported by Hunters Hope.

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Developing new stem cell therapies requires more than a solo biologist having a eureka moment alone in the lab. Real progress relies on collaborations between biologists, engineers and physicians. Thats why The Eli and Edythe Broad Foundation has continued its support of two strategic initiatives: innovation awards bringing together teams of engineers and scientists from USC and Caltech, and clinical research fellowships for physician-scientists.

Engineering new approaches: The Broad Innovation Awards

For the fifth consecutive year, the Broad Innovation Awards are providing critical funding to USC-affiliated faculty members pursuing multi-investigator research collaborations related to stem cells. For the first year, these collaborations are also drawing on the expertise of biomedical engineers from Caltech. Each award provides $200,000 of funding for a one-year project.

Were very excited to be joining our colleagues at USC in pioneering new approaches to advancing stem cell research, said Stephen L. Mayo, chair of the Division of Biology and Biological Engineering at Caltech. Were thankful to The Broad Foundation for supporting cross-town collaborations between scientists with different expertise but common goals.

With support from a Broad Innovation Award, Andy McMahon, the director of the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC, is collaborating with Caltech biomedical engineer Long Cai to leverage a new technology for understanding chronic kidney disease. The technology, called seqFISH, provides information about genetic activity taking place in intact tissueenabling the study of the interactions between cells in their native environments.

Dr. Cais seqFISH technology will provide an unprecedented insight into the cellular interplay underlying chronic kidney disease caused by a maladaptive response to acute kidney injury, said McMahon, who is the W.M. Keck Provost and University Professor of Stem Cell Biology and Regenerative Medicine, and Biological Sciences, as well as the chair of the Department of Stem Cell Biology and Regenerative Medicine at USC. We aim to better understand this maladaptive responsewhich is more common in malesin order to find new targets for preventing the progression to chronic kidney disease.

A second Broad Innovation Award brings together USC Stem Cell scientist Rong Lu and Caltech synthetic biologist Michael Elowitz. Their team will study the spatial organization of blood-forming stem and progenitor cells, also called hematopoietic stem and progenitor cells (HSPCs), which reside in the bone marrow. By pinpointing the locations of specific HSPCs, the scientists may find clues to explain why certain HSPCs are so dominantreplenishing the majority of the bodys blood and immune cells after a disruption such as a bone marrow transplantation.

Spatial advantages may be the primary drivers of what we refer to as the clonal dominance of certain HSPCs, said Lu, a Richard N. Merkin Assistant Professor of Stem Cell Biology and Regenerative Medicine, Biomedical Engineering, Medicine, and Gerontology at USC. Understanding the spatial competition between HSPCs could help improve bone marrow transplantation and provide new insights into aging and the development of diseases such as leukemiawhich are associated with clonal dominance.

Elowitz added: Thanks to the Broad Innovation Award and this exciting collaboration with Rong Lu, we will be able to bring a new, synthetic biology approach to record cell histories and read them out in individual cells within their native spatial context, providing new insights into fundamental questions in blood stem cell development.

A third Broad Innovation Award brings together three collaborators at USC: Michael Bonaguidi, an assistant professor of stem cell biology and regenerative medicine, biomedical engineering, and gerontology; Robert Chow, a professor of physiology and neuroscience, and biomedical engineering; and Jonathan Russin, an assistant professor of neurological surgery and associate surgical director for the USC Neurorestoration Center. Their project focuses on finding new approaches to treating epilepsy by studying neural cells called astroglia. These cells perform a variety of key functions that support the health of neurons in the brain, and they may also play a role in modulating epileptic seizures.

Although adults dont tend to generate many new brain cells, humans do produce a limited number of new astroglia, said Bonaguidi. We will examine these newborn astroglia at the single-cell level to better understand their role in epileptic patients, and to lay the groundwork for identifying new treatments.

The doctors are in: The Broad Clinical Research Fellowships

The Broad Clinical Research Fellowships are also entering their fifth consecutive year. These fellowships support stem cell research by physician-scientists and residents who intend to practice medicine in California.

These fellowships provide a very special opportunity for our medical residents to engage deeply in laboratory research, as a complement to their extensive training in patient care, said Laura Mosqueda, Dean of the Keck School of Medicine of USC. This valuable research experience gives them a much more complete perspective on how to meet the challenges of finding the best possible treatments for their patients.

A USC resident physician in general surgery, Kemp Anderson will spend his fellowship studying necrotizing enterocolitis, a very serious intestinal infection that affects nearly 10 percent of premature infants. Specifically, he will explore how a molecule involved in cellular communication, called farnesoid X receptor, or FXR, might contribute to this disease.

If FXR plays a role in compromising intestinal barrier function in these premature infants, then altering the activity of FXR could potentially yield treatment modalities for necrotizing enterocolitis, avoiding the morbidity and mortality associated with surgical intervention, said Anderson, who is performing the research under the mentorship of Christopher Gayer and Mark Frey at Childrens Hospital Los Angeles (CHLA). Im deeply appreciative of the benefactors and the selection committee for awarding me the Broad Clinical Fellowship, as it is allowing me devoted time to focus on this important project, and to become a more well-rounded physician through this academic pursuit.

Brittany Rocque, a resident physician in general surgery, will use her fellowship to seek better ways to predict, detect and diagnose immune rejection in patients who have undergone liver transplantation. Nearly 60 percent of pediatric patients and at least 15 percent of adult patients reject their liver transplants, and this can currently only be confirmed through an invasive surgical biopsy. Rocque is utilizing the technology Imaging Mass Cytometry to identify and analyze the types of immune cells involved in rejection.

My project has the potential to provide a noninvasive option to assess rejection in transplanted patients, and to expand our understanding of immune rejection, said Rocque, who is being co-mentored by Juliet Emamaullee and Shahab Asgharzadeh at CHLA. Im greatly looking forward to applying my passion for transplantation surgery in the context of basic science, and enhancing my appreciation for the nuances of research, thanks to the Broad Clinical Research Fellowship.

A hematology-oncology fellow who will be transitioning to a junior faculty position at USC next year, Caitlin ONeill will study a condition known as clonal hematopoiesis or CH, a phenomenon common in the aging population. CH involves genetic mutations that cause the expansion of a particular population of blood cells without leukemia or related malignancies. CH increases risks for certain health conditions including heart disease.

During her Broad Clinical Research Fellowship, ONeill will look at one mutation seen in patients with CH: a mutation to the gene called Tet methylcytosine dioxygenase 2, or TET2. ONeill will explore if this mutation promotes blood clots, inflammation and heart disease.

The goal is to inform therapies to prevent heart disease and leukemic progression in aging patients with CH, said ONeill, who is working with co-mentors Casey OConnell and Rong Lu at USC. Im very happy to be working on this project, with support from the Broad Clinical Research Fellowship, during my transition to becoming a faculty member at USC.

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Broad Foundation brings together stem cell scientists, engineers and physicians at University of Southern - Mirage News

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Avrobio is working to make conditioning, a necessary step for some gene therapies, safer. But its not stopping at improving current approachesthe company is teaming up with Magenta Therapeutics to see whether an antibody-drug conjugate (ADC) can do the job.

Under the deal, the duo will test Magentas lead conditioning program, MGTA-117, alongside at least one of Avrobios gene therapies. Each company will hold onto the rights for their respective programs, but Avrobio will pick up the tab for clinical trials involving MGTA-117.

We believe targeted ADCs represent the next generation of medicines to prepare patients for gene therapy or transplant in a targeted, precise way This partnership will allow Magenta to validate our conditioning platform in lentiviral gene therapy applications, said Magenta CEO Jason Gardner, D.Phil., in a statement.

Maintaining Momentum: Applying Recent Regulatory Guidance in the Midst of the Coronavirus

A panel of CATO SMS experts will review the key issues contained in the emerging Agency Guidance and offer thoughts on what changes and options for sponsors may be seen in the coming months.

Avrobios lead program is a gene therapy for Fabry disease dubbed AVR-RD-01. It is based on CD34+ stem cells that have been modified using a lentiviral vector to carry and express the gene that codes for the enzyme that is missing in Fabry disease. It is also working on treatments for Gaucher disease, Cystinosis and Pompe disease.

RELATED: Avrobio posts encouraging update for Fabry gene therapy phase 1, 2 trials

Patients undergoing lentiviral gene therapies must first take the chemotherapy drug busulfan in a process called conditioning, which helps the gene-modified stem cells take root in their bone marrow. Avrobio uses therapeutic drug monitoring to tailor busulfan dosing to each patient, to improve the odds of success for its gene therapies while tamping down on side effects. Some patients may be more susceptible to infection and bleeding after conditioning, and they may suffer side effects like nausea, hair loss and mouth sores.

MGTA-117 is made up of an anti-CD117 antibody linked to amanitin, a cell-killing toxin. It is designed to target only hematopoietic, or blood-forming, stem cells and progenitor cells. Animal studies suggest it could clear space in bone marrow for gene-modified stem cells to take root, Magenta said in the statement. The company plans to wrap IND-enabling studies for the antibody-drug conjugate this year.

The deal comes on the heels of a busulfan-focused one for Avrobio. The company joined forces with Saladex Biomedical on Monday to develop a rapid blood test that monitors how quickly patients metabolize the drug. The hope is to get results in minutes, rather than the hours that current methods take, so dosing can be adjusted quickly.

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Avrobio taps Magenta's ADC in ongoing quest to improve gene therapy conditioning - FierceBiotech

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"global Stem Cell Therapy market"

A new market study, titled Covid-19 Impact on Global Stem Cell Therapy Market Size, Status and Forecast 2020-2026 has been featured on WiseGuyReports.

The global Stem Cell Therapy market research offers a comprehensive overall market analysis focused on the latest findings. The introduction portion includes a brief overview of the industry, along with the product and service descriptions. This also includes the main applications for all end-user industries. The report also presents market prospects along with the forecast, with the study covering the period 2020-2026. The report includes an in-depth analysis of the major factors that could decide the market's trajectory in the coming years.

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

In the last several years, global stem cell therapy market developed fast at a average growth rate of 46.81%.

Competition Analysis

In the competitive analysis section of the report, leading as well as prominent players of the global Stem Cell Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.

The following players are covered in this report:

Osiris Therapeutics

NuVasive

Chiesi Pharmaceuticals

JCR Pharmaceutical

Pharmicell

Medi-post

Anterogen

Molmed

Takeda (TiGenix)

This report also analyses the impact of Coronavirus COVID-19 on the Stem Cell Therapy industry.

Regional and Country-level Analysis

The report offers an exhaustive geographical analysis of the global Stem Cell Therapy market, covering important regions, viz, North America, Europe, China, Japan, Southeast Asia, India and Central & South America. It also covers key countries (regions), viz, U.S., Canada, Germany, France, U.K., Italy, Russia, China, Japan, South Korea, India, Australia, Taiwan, Indonesia, Thailand, Malaysia, Philippines, Vietnam, Mexico, Brazil, Turkey, Saudi Arabia, UAE, etc.

The report includes country-wise and region-wise market size for the period 2015-2026. It also includes market size and forecast by each application segment in terms of revenue for the period 2015-2026.

Stem Cell Therapy Breakdown Data by Type

Autologous

Allogeneic

Stem Cell Therapy Breakdown Data by Application

Musculoskeletal Disorder

Wounds & Injuries

Cornea

Cardiovascular Diseases

Others

Request Free Sample Report athttps://www.wiseguyreports.com/sample-request/5252275-covid-19-impact-on-global-stem-cell-therapy

Table of Contents

1 Report Overview

2 Global Growth Trends by Regions

3 Competition Landscape by Key Players

4 Breakdown Data by Type (2015-2026)

5 Stem Cell Therapy Breakdown Data by Application (2015-2026)

6 North America

7 Europe

8 China

9 Japan

10 Southeast Asia

11 India

12 Central & South America

13 Key Players Profiles

14 Analyst's Viewpoints/Conclusions

NOTE: Our team is studying Covid-19 and its impact on various industry verticals and wherever required we will be considering Covid-19 footprints for a better analysis of markets and industries. Cordially get in touch for more details.

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Wise Guy Reports is part of the Wise Guy Research Consultants Pvt. Ltd. and offers premium progressive statistical surveying, market research reports, analysis & forecast data for industries and governments around the globe.

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Impact of Covid-19 Outbreak on Global Stem Cell Therapy Market Future Opportunities and Forecast Analysis 2020-2026 - Press Release - Digital Journal

Bone Marrow Stem Cells Comments Off on Impact of Covid-19 Outbreak on Global Stem Cell Therapy Market Future Opportunities and Forecast Analysis 2020-2026 – Press Release – Digital Journal | May 7th, 2020

One of the most talked-about topics today happens to be Mike Tyson appearing in shape and ready to appear in a boxing ring again. The former heavyweight champion of the world is gearing up for charity exhibition bouts thanks to Kings MMA coach Rafael Cordeiro.

Tyson (50-6, 2NC) wants to help those who are going through a tough time, like drug addicts. Relating to their situation, The 53-year-old is looking to make a difference while staying in shape. How exactly did he quickly get into fighting shape?

You know what I had done? I had stem-cell research therapy, Tyson ended up stating to Shaquille ONeal on Instagram Live, via The Sun. I feel like a different person but I cant comprehend why I feel this way. Its really wild what scientists can do.

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. The process to carry it out is via bone marrow transplantation. Several athletes from all sports have undergone the treatment, including Hines Ward, Alex Rodriguez, Kobe Bryant, Tiger Woods, and Rafael Nadal. There are specific types of therapy treatments, but it is unknown what Tyson went through.

Tyson made his professional debut in 1985 and quickly climbed up the ranks in the boxing world. In his 28th fight, Tyson knocked out Trevor Berbick in the second round to win the WBC Heavyweight Title. He became the youngest world heavyweight champion of all time at 20. Following his last fight in 2005, he ballooned up to 325 pounds.

When Shaq talked about hurting himself while hanging and working out with his kids, Tyson helped explain why. With his new regiment, Tyson is lifting weights constantly and sparring multiple times a day.

Thats just because you havent done it for a while, Tyson went on to say. If you continue to do it consistently youll be back to normal. Its just like me, I havent boxed or hit the bag for 15 years it has been three days so far and I feel incredible.

It is unknown who Tyson will be facing in his return. He has been offered, however, $1 million to fight in Australia to face some of the top stars in rugby and Australian football.

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Tyson Reveals What Helped Him Train For Exhibition Bouts | FIGHT SPORTS - FIGHT SPORTS

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FDA Approves AstraZenecas Farxiga for Heart Failure in Adults with Reduced Ejection Fraction

The U.S. Food and Drug Administration (FDA) announced on Tuesday that it has approved dapagliflozin, also known under the brand name Farxiga, for the treatment of heart failure in adults with reduced ejection fraction. The drug can potentially reduce the risk of cardiovascular death and hospitalization for heart failure.

AstraZenecas Farxiga is now the first in its drug class of sodium-glucose co-transporter 2 (SGLT2) inhibitors to be approved to treat adults with the New York Heart Associations functional class II-IV heart failure with reduced ejection fraction. AstraZeneca was granted with the approval of Farxiga related to heart failure by the FDA.

In a clinical trial, Farxiga appeared to improve survival and reduce the need for hospitalization in adults with heart failure and reduced ejection fraction.

To determine the efficacy of the drug, researchers looked at the number of instances of cardiovascular death, hospitalization for heart failure and urgent heart failure visits. Some trial participants were given a once-daily dose of 10mg of Farxiga, while others were given a placebo. After approximately 18 months, those who were given Farxiga had fewer cardiovascular deaths, hospitalizations for heart failure and urgent heart failure visits compared to their counterparts.

Heart failure is a serious health condition that contributes to one in eight deaths in the U.S. and impacts nearly 6.5 million Americans, said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDAs Center for Drug Evaluation and Research. This approval provides patients with heart failure with reduced ejection fraction an additional treatment option that can improve survival and reduce the need for hospitalization.

Farxiga can cause side effects including dehydration, urinary tract infections and genetical yeast infections. It can also potentially result in serious cases of necrotizing fasciitis of the perineum in people with diabetes and low blood sugar when combined with insulin.

On Tuesday, BioCardia, Inc. also announced positive preclinical datasupporting its new drug application for anti-inflammatory cell therapy for heart failure. BioCardias allogenic neurokinin 1 receptor positive mesenchymal stem cell (NK1R+ MSC) therapy appeared to improve heart function in a study. NK1R+ MSC is being marketed under the name CardiALLO.

Alexanderstock23 / Shutterstock

Researchers looked at 26 animals treated with both low dose and high dose CardiALLO in their study. Echocardiographic measures of cardiac ejection fraction, fractional shortening and cardiac outflow all notably improved in the animals.

In light of these positive data on our allogenic NK1R+ MSC therapy, we expect to meet our internal timeline to complete our submission to the FDA for our first indication for CardiALLO, and potentially receive IND acceptance by the end of the second quarter, said BioCardia Chief Scientific Officer Ian McNiece, PhD. The MSCs that were studied are subtypes of MSC that we have delivered previously in our co-sponsored trials, which we believe have enhanced potency over MSC generated from unselected bone marrow cells. We look forward to seeing additional data from this animal study that are currently being analyzed, including histology and pathology of the heart and lungs.

BioCardia also intends to submit an IND for the use of NK1R+ MSC delivered via intravenous infusion for the treatment of Acute Respiratory Distress Syndrome caused by COVID-19.

Approximately 6.5 million adults in the U.S. are living with heart failure, according to the Centers for Disease Control and Protection. In 2017, it was a contributing cause of death in one out of eight people.

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FDA Approves AstraZeneca's Farxiga for Heart Failure in Adults with Reduced Ejection Fraction - PharmaLive

Bone Marrow Stem Cells Comments Off on FDA Approves AstraZeneca’s Farxiga for Heart Failure in Adults with Reduced Ejection Fraction – PharmaLive | May 7th, 2020

Laila Anderson continues to make an impact in the lives of others one year after she became part of the Blues run to the 2019 Stanley Cup title.

On Tuesday, Anderson, who has battled HLH, a disease that causes the body to make too many immune cells, took part in an all-day livestream event called Couch2Cure to benefit Be the Match. It served as not only a fundraiser, but also a call for people to become donors for patients seeking blood stem cell matches. She was joined by FOX play-by-play man Joe Buck and Blues PA announcer Tom Calhoun.

The event was a success, raising$1.45 million and resulting in 36,000 registries to the Be the Match program.

Laila spoke with NBC Sports Kathryn Tappen on Wednesday to talk about the Couch2Cure event, the importance of the Be the Match registry, and how shes doing one year after the Blues triumph.

MORE LAILA ANDERSON: Laila introduces Blues All-Stars with gusto Blues superfan enjoying life one year after bone marrow transplant Laila meets bone marrow donor Laila gets moment with Stanley Cup

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

British Columbia Premier John Horgan has offered the NHL a place to play if the league can find a way to resume the season.

Speaking at a COVID-19 media briefing Wednesday, Horgan said he has written a letter to both NHL Commissioner Gary Bettman and NHL Players Association head Donald Fehr to let them know B.C. is a place to potentially restart the NHL assuming the games would be played without audiences, but instead played for television.

The NHL suspended its season March 12 with 189 regular-season games left.

Dr. Bonnie Henry, British Columbias provincial health officer, was asked Monday about Vancouver hosting NHL games with no fans and said: These are the types of things that we need to think about.

Ontario Premier Doug Ford said Tuesday the Maple Leafs parent company, MLSE, has been in contact with the province about the possibility of Toronto serving as a hockey pod for teams as well. Alberta Premier Jason Kenney and Bettman spoke last month about Edmonton as another potential hub city.

Welcome to the PHT Morning Skate, a collection of links from around the hockey world. Have a link you want to submit? Email us atphtblog@nbcsports.com.

A really nice read about ex-Blackhawks president John McDonoughs friendship with 11-year-old Cammy Babiarz, who is unable to walk or talk because of Rett Syndrome a rare developmental disorder. [Midway Minute]

It didnt last very long, but at one point in time Michael Jordan was a minority owner of the Capitals. [ESPN]

Economies are beginning to open up again, so too are hockey rinks in the U.S. [The Hockey News]

Georges Laraque opens about his up and down relationship with his father. [Vice]

How the 2011-12 Kings became unlikely Stanley Cup champions. [The Score]

Comparing Brady Tkachuks early days in the NHL to that of Mark Stones. [Silver Seven Sens]

What if some of NHLs all-time best hadnt run into historic dynasties? [Sportsnet]

Looking ahead to whats expected to be an intriguing 2022 NHL draft class. [Stephen Ellis]

Finally, the Wild music video you were looking for:

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

After pulling the plug on the 2019-20 season in March, the KHL has decided there will be no champion and the Gagarin Cup will not be awarded for the first time in league history.

Due to this decision, the league has equally ranked the eight teams that advanced to the second round of the playoffs:Ak Bars Kazan,Barys Nur-Sultan,CSKA Moscow,Dynamo Moscow,Jokerit Helsinki,Salavat Yulaev Ufa,Sibir Novosibirsk, and SKA St. Petersburg.

From the KHL:

With the season incomplete, there is no way that a Gagarin Cup winner and other prize winners can be fairly chosen based on the results of the regular season. To announce a champion based on the regular season and one round of the playoffs would violate the sporting integrity of the competition.

The Russian Hockey Federation has drawn up a separate procedure to determine the Russian Champion for the 2019-20 season, and to award silver and bronze medals to the second and third-placed teams. This proposal will be submitted to the KHL Board of Directors for approval.

The Gagarin Cup playoffs were halted in the conference semifinals after Jokerit and Barys pulled out due to the coronavirus pandemic. Originally, the KHL was planning for a one-week break to come up with a new format for the four remaining teams. They later chose to end the season completely.

Im sure that the league has taken a fair and balanced decision in this difficult situation, said KHL president Alexei Morozov. This was the only choice that respects our sporting principles. For the first time in history, the KHL season had to be interrupted and ultimately curtailed. That was a tough, but essential decision, dictated by the need to protect the health of the nation.

MORE: Bill Peters signs two-year deal to coach KHLs Avtomobilist

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

Brendan Leipsic of the Capitals has apologized for comments made on social media that were leaked online Wednesday. Panthers prospect Jack Rodewald was also in the Instagram group chat where remarks were made about the appearances of Meaghan Pearson, whose husband, Tanner, plays for the Canucks, Lauren Kyle, the girlfriend of Oilers forward Connor McDavid, and other women.

Yesterday my friends Instagram account was hacked and an individual circulated images that are representative of private conversations I was a part of, Leipsic wrote in an apology note posted on Twitter. I fully recognize how inappropriate and offensive these comments are and sincerely apologize to everyone for my actions. I am committed to learning from this and becoming a better person by taking time to determine how to move forward in an accountable, meaningful way. I am truly sorry.

The NHL released a statement of their own stating they will address this with the players involved.

The National Hockey League strongly condemns the misogynistic and reprehensible remarks made by players Brendan Leipsic and Jack Rodewald in a private group chat that has surfaced on social media. There is no place in our League for such statements, attitudes and behavior, no matter the forum. We will address this inexcusable conduct with the clubs and players involved.

Leipsic, who has played 61 games with the Capitals this season, is on his fifth team in five years since entering the NHL. His current team wrote in a statement,We are aware of the unacceptable and offensive comments made by Brendan Leipsic in a private conversation on social media. We will handle this matter internally.

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

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Laila Anderson on Couch2Cure event, importance of Be the Match registry - NBCSports.com

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The global Cell Therapy Manufacturing Market research report thoroughly explains each and every aspect related to the Cell Therapy Manufacturing Market, which facilitates the reports reader to study and evaluate the upcoming market trend and execute the analytical data to promote the business. The growth trend forecasted on account of thorough examination offers in-depth information regarding the global Cell Therapy Manufacturing Market. A pathway of development is offered by the market to the several connected networks of businesses under it, which include different firms, industries, organizations, vendors, distributors, and local manufacturers too. All the key Cell Therapy Manufacturing Market players compete with each other by offering better products and services at a reasonable price in order to grab significant share at the regional and global level market.

Cell therapy is one of the most promising healthcare procedure for restoration of damaged tissue. Cell therapies have huge potential for the wide range of disease treatment including tissue degradation, immune deficiency, metabolic disorders, and cancer. Cell therapy are categorized into two types allogeneic (cells from third party donor) and autologous (cells from ones own body). Cell therapy has gained significant traction in recent past and currently it under commercial development. Main objective of cell therapy is to re-establish the lost function of tissues and cells rather than to produce a new organ. Some cell therapies have been established and permitted for clinical trial. For instance, in 2013 a stage 2 clinical trial was completed for transplantation of bone marrow derived stem cells in affected knee by rheumatoid arthritis. The growth of this technique is also increased due to involvement of the government agencies. For instance, innovate U.K. 2014 report showed that government agreed to fund US$15.3 million in cell therapy manufacturing market.ription

Get a Sample Copy of this Report: https://www.coherentmarketinsights.com/insight/request-sample/1728

This report sample includesBrief Introduction to the research report.Table of Contents (Scope covered as a part of the study)Top players in the marketResearch framework (presentation)Research methodology adopted by Coherent Market Insights

The report incorporates an estimated impact of strict standards and regulations set by the government over the market in the upcoming years. The market report also comprises exhaustive research done using several analytical tools such as SWOT analysis to identify the market growth pattern.

Top Manufacturers in GlobalCell Therapy ManufacturingMarket Includes:Pharmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

Regions & Countries Mentioned In The Cell Therapy Manufacturing Market Report:

North America ( United States)

Europe ( Germany, France, UK)

Asia-Pacific ( China, Japan, India)

Latin America ( Brazil)

The Middle East & Africa

Key Highlights of the Table of Contents:

Cell Therapy Manufacturing Market Study Coverage: It includes key manufacturers covered, key market segments, the scope of products offered in the global market, years considered, and study objectives. Furthermore, it tuches the segmentation study provided in the report on the basis of the type of product and applications.

Cell Therapy Manufacturing Market Executive Summary: This section emphasizes on the key studies, market growth rate,Competitive landscape, market drivers, trends, and issues.

Cell Therapy Manufacturing Market Production by Region: The report provides information related to import and export, production, revenue, and key players of all regional markets studied are covered in this section.

Cell Therapy Manufacturing Market Profile of Manufacturers: Analysis of each market player profiled is detailed in this section. This also provides SWOT analysis, products, production, value, capacity, and other vital factors of the individual player.

Buy This Complete A Business Report: https://www.coherentmarketinsights.com/insight/buy-now/1728

Table of Contents

Report Overview:It includes the Cell Therapy Manufacturing market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary:This section of the report gives information about Cell Therapy Manufacturing market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players:Here, key players of the Cell Therapy Manufacturing market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study:All of the regions and countries analyzed in the Cell Therapy Manufacturing market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

An Overview of the Impact of COVID-19 on this Market:

The pandemic of COVID-19 continues to expand and impact over 175 countries and territories. Although the outbreak appears to have slowed in China, COVID-19 has impacted globally. The pandemic could affect three main aspects of the global economy: production, supply chain, and firms and financial markets. National governments have announced largely uncoordinated, country-specific responses to the virus. As authorities encourage social distancing and consumers stay indoors, several businesses are hit. However, coherent, coordinated, and credible policy responses are expected to offer the best chance at limiting the economic fallout.

National governments and international bodies are focused on adopting collaborative efforts to encourage financial institutions to meet the financial needs of customers and members affected by the coronavirus. However, there are some sectors that have remained unscathed from the impact of the pandemic and there are some that are hit the hardest.

We, at Coherent Market Insights, understand the economic impact on various sectors and markets. Using our holistic market research methodology, we are focused on aiding your business sustain and grow during COVID-19 pandemics. With deep expertise across various industries-no matter how large or small- and with a team of highly experienced and dedicated analysts, Coherent Market Insights will offer you an impact analysis of coronavirus outbreak across industries to help you prepare for the future.

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Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

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Cell Therapy Manufacturing Market 2020 Coronavirus (Covid-19) Business Impact Size Will Escalate Rapidly in the Near Future 3w Market News Reports -...

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British Columbia Premier John Horgan has offered the NHL a place to play if the league can find a way to resume the season.

Speaking at a COVID-19 media briefing Wednesday, Horgan said he has written a letter to both NHL Commissioner Gary Bettman and NHL Players Association head Donald Fehr to let them know B.C. is a place to potentially restart the NHL assuming the games would be played without audiences, but instead played for television.

The NHL suspended its season March 12 with 189 regular-season games left.

Dr. Bonnie Henry, British Columbias provincial health officer, was asked Monday about Vancouver hosting NHL games with no fans and said: These are the types of things that we need to think about.

Ontario Premier Doug Ford said Tuesday the Maple Leafs parent company, MLSE, has been in contact with the province about the possibility of Toronto serving as a hockey pod for teams as well. Alberta Premier Jason Kenney and Bettman spoke last month about Edmonton as another potential hub city.

Laila Anderson continues to make an impact in the lives of others one year after she became part of the Blues run to the 2019 Stanley Cup title.

On Tuesday, Anderson, who has battled HLH, a disease that causes the body to make too many immune cells, took part in an all-day livestream event called Couch2Cure to benefit Be the Match. It served as not only a fundraiser, but also a call for people to become donors for patients seeking blood stem cell matches. She was joined by FOX play-by-play man Joe Buck and Blues PA announcer Tom Calhoun.

The event was a success, raising$1.45 million and resulting in 36,000 registries to the Be the Match program.

Laila spoke with NBC Sports Kathryn Tappen on Wednesday to talk about the Couch2Cure event, the importance of the Be the Match registry, and how shes doing one year after the Blues triumph.

MORE LAILA ANDERSON: Laila introduces Blues All-Stars with gusto Blues superfan enjoying life one year after bone marrow transplant Laila meets bone marrow donor Laila gets moment with Stanley Cup

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

Welcome to the PHT Morning Skate, a collection of links from around the hockey world. Have a link you want to submit? Email us atphtblog@nbcsports.com.

A really nice read about ex-Blackhawks president John McDonoughs friendship with 11-year-old Cammy Babiarz, who is unable to walk or talk because of Rett Syndrome a rare developmental disorder. [Midway Minute]

It didnt last very long, but at one point in time Michael Jordan was a minority owner of the Capitals. [ESPN]

Economies are beginning to open up again, so too are hockey rinks in the U.S. [The Hockey News]

Georges Laraque opens about his up and down relationship with his father. [Vice]

How the 2011-12 Kings became unlikely Stanley Cup champions. [The Score]

Comparing Brady Tkachuks early days in the NHL to that of Mark Stones. [Silver Seven Sens]

What if some of NHLs all-time best hadnt run into historic dynasties? [Sportsnet]

Looking ahead to whats expected to be an intriguing 2022 NHL draft class. [Stephen Ellis]

Finally, the Wild music video you were looking for:

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

After pulling the plug on the 2019-20 season in March, the KHL has decided there will be no champion and the Gagarin Cup will not be awarded for the first time in league history.

Due to this decision, the league has equally ranked the eight teams that advanced to the second round of the playoffs:Ak Bars Kazan,Barys Nur-Sultan,CSKA Moscow,Dynamo Moscow,Jokerit Helsinki,Salavat Yulaev Ufa,Sibir Novosibirsk, and SKA St. Petersburg.

From the KHL:

With the season incomplete, there is no way that a Gagarin Cup winner and other prize winners can be fairly chosen based on the results of the regular season. To announce a champion based on the regular season and one round of the playoffs would violate the sporting integrity of the competition.

The Russian Hockey Federation has drawn up a separate procedure to determine the Russian Champion for the 2019-20 season, and to award silver and bronze medals to the second and third-placed teams. This proposal will be submitted to the KHL Board of Directors for approval.

The Gagarin Cup playoffs were halted in the conference semifinals after Jokerit and Barys pulled out due to the coronavirus pandemic. Originally, the KHL was planning for a one-week break to come up with a new format for the four remaining teams. They later chose to end the season completely.

Im sure that the league has taken a fair and balanced decision in this difficult situation, said KHL president Alexei Morozov. This was the only choice that respects our sporting principles. For the first time in history, the KHL season had to be interrupted and ultimately curtailed. That was a tough, but essential decision, dictated by the need to protect the health of the nation.

MORE: Bill Peters signs two-year deal to coach KHLs Avtomobilist

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

Brendan Leipsic of the Capitals has apologized for comments made on social media that were leaked online Wednesday. Panthers prospect Jack Rodewald was also in the Instagram group chat where remarks were made about the appearances of Meaghan Pearson, whose husband, Tanner, plays for the Canucks, Lauren Kyle, the girlfriend of Oilers forward Connor McDavid, and other women.

Yesterday my friends Instagram account was hacked and an individual circulated images that are representative of private conversations I was a part of, Leipsic wrote in an apology note posted on Twitter. I fully recognize how inappropriate and offensive these comments are and sincerely apologize to everyone for my actions. I am committed to learning from this and becoming a better person by taking time to determine how to move forward in an accountable, meaningful way. I am truly sorry.

The NHL released a statement of their own stating they will address this with the players involved.

The National Hockey League strongly condemns the misogynistic and reprehensible remarks made by players Brendan Leipsic and Jack Rodewald in a private group chat that has surfaced on social media. There is no place in our League for such statements, attitudes and behavior, no matter the forum. We will address this inexcusable conduct with the clubs and players involved.

Leipsic, who has played 61 games with the Capitals this season, is on his fifth team in five years since entering the NHL. His current team wrote in a statement,We are aware of the unacceptable and offensive comments made by Brendan Leipsic in a private conversation on social media. We will handle this matter internally.

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

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NHL offered place to play in British Columbia - NBCSports.com

Bone Marrow Stem Cells Comments Off on NHL offered place to play in British Columbia – NBCSports.com | May 7th, 2020

Youve probably seen those before-and-after quarantine memes, the ones featuring faces or more specifically, lips that have undergone a drastic change in shape in the absence of access to an aesthetician. Exaggerated they might be, but it is true that lockdown has left an injectable-shaped hole in some of our lives, and whether youre into them or not, our newly facial-free (and less-than-active) lifestyles are wreaking a certain amount of havoc on our skin anyway. For those who have regular in-clinic treatments, lockdown can mean that lines start to show, full lips diminish, cheeks depress and sunken eye sockets and dark circles re-appear, says aesthetic doctor Dr Sophie Shotter, who has temporarily shuttered her clinic and returned to the NHS frontline to join the fight against Covid-19. And, while the issue might seem trivial in light of everything else going on in the world, it is possible to harness the power of intelligent skincare to help mimic the effects of filler and Botox in lieu of the real thing. Dr Shotter shares her tips here.

Filler is routinely administered to add volume to the skin, usually in the face, says Shotter, who explains that its made from hyaluronic acid, a molecule we should all be utilising in our skincare routines to help soften fine lines. One of the best serums for the job is SkinCeuticals HA Intensifier, which boosts hyaluronic acid levels in the skin, making it look and feel firm, plump and hydrated. Alternatively, try LOrals Revitalift Filler Hyaluronic Acid for a more affordable option.

To smooth skin, Shotter also recommends BioEffect EGF Serum, which boosts skin thickness and collagen levels in the skin, meaning fewer wrinkles and a healthy glow. And finally, look to SkinBetter Science AlphaRet Overnight Cream to reap the benefits of prescription strength vitamin A (otherwise known as retinol): Retinol is the ultimate age management molecule that increases collagen levels, she says.

If youve had Botox before, you will know that its a toxin injected where there are dynamic lines to relax them and remove creases within the skin, says Shotter. There are products that mimic its effects: Meder Skincare Myo-Fix Concentrate, is full of muscle-relaxing peptides that work to block the nerve communicating with the muscle. Although it might sound daunting, its totally safe to use, and Shotter recommends regular and sustained use for the best results. Another great product to use if you usually have Botox administered superficially to regulate oil production is Institut Esthederms Pure System Pore Refiner Concentrate it is silicone based so refines their appearance beautifully.

Used religiously, there are products that can help to lift and plump the lips, although, like all of the products listed here, they wont have effects on a par with the treatment itself. One of the best to try is Fillerinas Lip Volume Grade 3, which contains six different hyaluronic acid molecules of varying weights and sizes, to deeply hydrate lips and give a fuller effect with continual use. Shotter also recommends a brilliant lip balm. Medik8s Mutiny Squalane Lip Balm helps to nourish and heal the lips, drawing moisture to the area to give them a plumper, more hydrated look, she says, adding that its important to use SPF to protect them from collagen-depleting UV rays. Protecting the health of the lips will help them stay plump and full.

Filler is often strategically placed to fill the hollows under the eyes, to refresh and hydrate and to lift dark circles, says Shotter. A (plant-based) stem cell serum, like Dr Levy Intense Stem Cell Eye Booster Concentrate, will boost the thickness of the delicate under eye skin, plus it also contains retinol and antioxidants to smooth fine lines and brighten the area. MGC Dermas CBD Stem Cells and Peptides Eye Cream is a brilliant option too, thanks to firming peptides, hyaluronic acid and calming CBD.

More from British Vogue:

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11 Skincare Products To Try Now Injectables Are Off The Cards - British Vogue

Skin Stem Cells Comments Off on 11 Skincare Products To Try Now Injectables Are Off The Cards – British Vogue | May 7th, 2020

Tabrecta (capmatinib) will treat patients with metastatic non-small cell lung cancer that has a mutation leading to MET exon 14 skipping. The drug is the first targeted option for patients with lung cancer and this type of mutation.

Tabrecta is the first therapy approved by the FDA specifically to treat NSCLC with mutations that lead to epithelial-mesenchymal transition (EMT), which is MET exon 14 skipping.

Tabrecta is approved for patients who are new to treatment and also those who have received previous therapies, regardless of prior treatment type.

Along with the drug approval, the FDA gave the green light to a companion diagnostic, the FoundationOne CDx assay, which can identify these mutations in patients.

In epithelialmesenchymal transition(EMT), the cells that line an organ lose their polarity and ability to adhere to other cells, giving them the ability to invade tissues and organs. MET exon 14 skipping means that a segment of RNA that should prompt the production of a specific protein stops sending those messages.

The spread of cancer consists of a sequential series of events and MET exon 14 skipping is recognized as a critical event in this process, the FDA stated in a press release about the approval. Mutations leading to MET exon 14 skipping are found in 3% to 4% of patients with lung cancer, the agency stated.

Lung cancer is increasingly being divided into multiple subsets of molecularly defined populations with drugs being developed to target these specific groups, said Dr. Richard Pazdur, director of the FDAs Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDAs Center for Drug Evaluation and Research, in the release.

Taken orally, Tabrecta works by blocking a key protein that drives metastatic NSCLC in these patients. The FDA approved it based on the results of a clinical trial involving patients with NSCLC who had mutations leading to MET exon 14 skipping; their tumors did not express the proteins EGFR or ALK.

The evaluated study population included 28 patients who had never undergone treatment for NSCLC and 69 previously treated patients. The overall response rate (ORR; the percentage of participants who experienced a prespecified amount of tumor shrinkage) for the 28 participants was 68%, with 4% having a complete response and 64% having a partial response.

The ORR for the 69 participants was 41%, with all having a partial response. Of the responding participants who had never undergone treatment for NSCLC, 47% had a duration of response lasting 12 months or longer compared with 32.1% of the responding participants who had been previously treated.

Common side effects for patients taking Tabrecta included swelling of the legs, nausea, fatigue, vomiting, shortness of breath and decreased appetite.

Tabrecta may cause serious side effects including scarring or inflammation of the lungs. It may also cause damage to liver cells or harm a developing fetus or newborn baby. Patients may be more sensitive to sunlight when they take Tabrecta and should take precautions to cover their skin and use sunscreen.

Tabrecta was approved under theFDAs accelerated approval, breakthrough designation and priority review programs, which provide for a quicker review of drugs that treat serious or life-threatening diseases and represent a meaningful advantage over existing treatments.

Continued approval for this indication may be contingent upon verification of these results in confirmatory clinical trials.

Check back for what you need to know regarding this approval.

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FDA Approves Tabrecta, the First Targeted Drug for Patients with Non-Small Cell Lung Cancer and MET exon 14 - Curetoday.com

Skin Stem Cells Comments Off on FDA Approves Tabrecta, the First Targeted Drug for Patients with Non-Small Cell Lung Cancer and MET exon 14 – Curetoday.com | May 7th, 2020

Verditek PLC (LON:VDTK), the cleantech play, tumbled 11% to 2.85p after it announced a share subscription to raise 1mln.

The company issued 40mln shares at 2.5p a pop.

The newly-issued shares represent about 14% of the company's enlarged issued share capital.

KRM22 PLC (LON:KRM) plunged 44% to 23.5p after it said it is continuing talks about potential fundraising.

The risk management software specialist put out a statement on Thursday afternoon in response to a fall in its share price.

Discussions with shareholders and certain other investors regarding an equity fundraising or potential alternative financing are continuing, the London-based company said.

Anglo African Oil & Gas PLC (LON:AAOG) shares were up 21% at 0.85p, much to the mystification of the companys management.

The shares started the week at 0.22p and closed last night at 0.7p, prompting the company to issue a statement that essentially said: dont ask me, guv.

On 1 May the company completed the sale of Anglo African Oil & Gas Congo to Zenith Energy Limited on the terms set out in the announcement of 17 April 2020.

AA PLC (LON:AA. shares rallied 13% to 29.8p as its financial results highlighted a resilient, cash generative business, albeit the financials cover the pre-coronavirus period.

The roaside recovery firm's revenue for the twelve months ended 31 January 2020 amounted to 995mln, up 2%, while operating profit increased 17% to 257mln.

Profit before tax rose by 102% to 107mln, with earnings per share at 14.1p from 6.9p in the preceding year.

Crimson Tide PLC (LON:TIDE), the software company, rose 21% to 2.6p after it reported a sharp rise in pre-tax profits for 2019.

The company behind the Mpro5 mobile workforce management app saw profit before tax soar to 352,000 from 69,000 in 2018.

The company said the profit increase was achieved despite a strategy of significant increase in sales and marketing expenditure.

Filta Group Holdings PLC (LON:FLTA) soared 76% to 126p in early trade on Thursday after it launched a new sanitising service to help businesses prevent the spread of coronavirus (COVID-19).

The provider of fryer management and other services to commercial kitchens has launched FiltaShield, which is an anti-viral product developed for use in restaurants, bars, shops and offices (those that are still open) as well as healthcare facilities.

The solution is applied to a surface by spraying or fogging, leaving behind a mono-molecular layer that bonds to the surface and protects surfaces for up to 30 days.

Not just for kitchens, perfect for any public space. Hit me up for the deets #ROC. #FiltaShield #Sanitize #StopTheSpread https://t.co/0KCiqrwV2i

Best of the Best PLC (LON:BOTB) rose 18% to 512.5p as it raised full-year revenue and profit guidance.

The competitions runners switch to an online model it used to waylay travellers at airports to try to persuade them to enter a competition to win a car (tricky as a car would have been hard to fit in the suitcase) looks to have paid off.

The company said the second half of its fiscal year, which runs to the end of April, was particularly strong.

88 Energy Ltd (LON:88E) (ASX:88E) said it has reached an agreement with XCD Energy Ltds (LON:ASX:XCD) board for a unanimously recommended merger. The prior unsolicited takeover bid had achieved backing of about 18.5% of XCDs shareholders, and the recommended merger comes with improved terms. In the all-paper deal, 88 Energy will issue 2.4 new 88 Energy shares and 0.7 for every listed share option held -up from 1.67 per share and 0.5 per option. XCDs board now recommend that its shareholders accept the offer, in the absence of any superior proposals. They will hold around 20% of the enlarged company as a result of the transaction.

C4X Discovery Holdings PLC (LON:C4XD), the AIM-listed drug discovery company, has announced plans to raise at least 1mln by placing shares at 15p a throw. C4X shares closed at 15.25p yesterday. The net proceeds from the placing will be used to further strengthen the companys balance sheet as its partnering and strategic collaborations progressand will boost working capital during the progression of its pipeline portfolio.

MaxCyte Inc (LON:MXCT) has signed a clinical and commercial agreement with a US life sciences group for its cutting edge gene-editing technology. Caribou Biosciences will use MaxCytes flow electroporation systems and the companys ExPERT platform in its allogeneic t-cell therapy programmes. MaxCyte will receive undisclosed development and approval milestones as well as sales-based payments and other licensing fees. This important agreement represents another key expansion for MaxCyte, emphasising the value of our technology platform to companies developing pioneering gene-editing and cell therapies, MaxCytes chief executive, Doug Doerfler said in a statement.

Panther Metals PLC (LON:PALM) has announced the commencement of a three-week field exploration programme from this weekend on the gold-focused Big Bear property in Ontario, Canada. The main market-listed company, which is focused on mineral exploration in Canada and Australia, said the three-week work sampling and mapping programme, which will build its understanding of possible drill and trench targets is commencing on May 10. It noted that the work will target both orogenic gold and volcanogenic massive sulphide (VMS) style mineralisation.

ReNeuron Group PLC (LON:RENE) said new positive data relating to its CTX cell therapy candidate have been published in the peer-reviewed scientific journal Stem Cells. The developer of cell-based therapeutics said the data was included in a paper entitled "Implantation of the clinical-grade human neural stem cell line, CTX0E03, rescues the behavioural and pathological deficits in the quinolinic acid-lesioned rodent model of Huntington's disease". It said the new data show for the first time that ReNeuron's CTX human neural stem cell line can rescue deficits associated with an accepted animal model of Huntington's disease, a progressive genetic brain disorder.

NQ Minerals PLC (LON:NQMI) has unveiled a new resource statement for the Beaconsfield gold mine in Tasmania. Total resources rise to 1.454mln tonnes at a grade of 10.3 grams per tonnes (g/t), for 483,000 ounces gold. There are 354,000 ounces in measured and inferred resources, 485,000 tonnes at 11.4 g/t in measured for 177,000 ounces and 492,000 tonnes at 11.2 g/t in indicated for 177,000 ounces. A further 477,000 tonnes at 8.4 g/t remain in the inferred resource category, and, the company told investors that significant additional gold potential is still to be assessed.

Next Fifteen Communications Group PLC (LON:NFC) has announced that Penny will become the new chair of its board with effect from February 1, 2021, replacing Richard Eyre who will complete nine years as its chairman on May 11, 2020. Ladkin-Brand, who has chaired the Next 15 Audit Committee since 2017, also becomes the companys Senior Independent Director with immediate effect. The group said its board has asked Eyre to continue in the post to the end of the current financial year, and, consequently, he will seek re-election at the AGM and be available to support a smooth transition. Ladkin-Brand is currently chief financial officer at Future PLC (LON:FUTR), the FTSE 250 global multi-platform media company, and she will be moving into a new role of chief strategy officer on June 1, 2020, and will step down from the groups board.

Frontier IP Group PLC (LON:FIPP) said its portfolio firm Exscientia has entered into a collaboration with US research centre SRI International to expedite the discovery of molecules for a high-value oncology target. The IP investor said the agreement will see the two firms implement a new approach to drug discovery using SRIs automated synthetic-chemistry system with Exscientia's Centaur Chemist system. Frontier IP owns a 2.3% stake in Exscientia.

Eden ResearchPLC (LON:EDEN) said it is poised to capitalise on new product and market opportunities in 2020 as it predicted more sales for its Cedroz product. Posting its results for the year endedDecember 31, 2019, the biopesticide specialist said it expected to build on the sales achieved in the territories where it received approvals during 2019 and early 2020, including sales for Cedroz in Spain, Italy, France, Belgium, the Netherlands and the United Kingdom where the applications for registrations have now been outstanding from the early part of 2019. The firm also said it expected US regulators to approve Cedroz and its Mevalone product during 2020, although the pace of approvals hasbeen slowed by the coronavirus (COVID-19) pandemic.

Premier African Minerals Ltd (LON:PREM) has agreed to acquirea further 7% interest in the Otjozondu manganese mining project in Namibia. The transaction sees Premier buy a 7% interest in MN Holdings Limited, the projects owner, for US$700,000 paid in new shares. It will increase Premier Africans stake in MN to 19%. This further proposed increase in our holding in MNH is based on the same valuation formula applied to our initial acquisition, George Roach, Premier African's chief executive said in a statement released after the market close on Wednesday.

Amryt Pharma PLC (LON:AMYT) has taken another important step towards the global launch of its phase III treatment for the rare skin condition epidermolysis bullosa (EB) by announcing its brand name. AP101 will be launched commercially as FILSUVEZ. This, alongside our recent completion of recruitment into the EASE study, represents further progress as we endeavour to develop a therapy for patients with EB, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment," Amryt chief executive, Dr Joe Wiley said in a statement.

Anglo African Oil & Gas PLC (LON:AAOG) said it has noted the recent rise in its share price and confirms that it knows of no reason for this movement. As announced on May 1, 2020, the company has completed the sale of Anglo African Oil & Gas Congo S.A.U to Zenith Energy Limited on the terms set out in the announcement of April 17, 2020.

Silence Therapeutics PLC (LON:SLN), a leader in the discovery, development and delivery of novel RNA therapeutics for the treatment of serious diseases, said its annual general meeting will be held on June 9, 2020, at 10.00am at Herons Ghyll, Tilford Road, Tilford, Surrey GU10 2DD. It added that to comply with the UK Government's Stay at Home measures, shareholders will not be allowed to attend the meeting in person and are strongly encouraged to, therefore, submit their votes, in respect of all matters of business, via proxy as early as possible. If the situation changes then shareholders will be notified via the company's website at https://www.silence-therapeutics.com and via RNS announcement.

Woodbois Limited (LON:WBI),the African focused forestry and timber trading company, has announced that following the release of the audited results for the year ended December 31, 2019, on April 30, the company has made their latest corporate presentation available from the investor centre of the company's website.

Base Resources Limited (LON:BSE) (ASX:BSE), the African mineral sands producer, has announced that the latest investor update presentation is now available from the companys website - http://www.baseresources.com.au - and a pre-recorded webcast of that presentation can be viewed at https://edge.media-server.com/mmc/p/6av8h2ew

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Anglo African Oil & Gas mystified by sharp share price hike - Proactive Investors UK

Skin Stem Cells Comments Off on Anglo African Oil & Gas mystified by sharp share price hike – Proactive Investors UK | May 7th, 2020