Smiths 2005 stem cell law to be reauthorized by House

Rep. Chris Smith (R-NJ)statement submitted during debate in the House of Representatives

on the Stem Cell Therapeutic and Research ActSeptember 29, 2020

Margaret Hahnmy mother-in-lawpassed away on Friday and a Mass of Christian burial will be held today at St. Mary Church in South Amboy, New Jersey. She was 96 and was deeply loved and will be deeply missed.

MargaretPegwas a great womanwife, mother, grandmother, and great-grandmother. She selflessly devoted her life to public service including her amazing work as Sayreville Borough Clerk for twenty years. She had an incredible reputation for getting things done for the people. No matter who served as mayor or on Council, everyone knew she was the power.

My wife Marie and I will join family and friends today at her funeral and internment making it impossible for me to speak today during the debate on the reauthorization of a law I originally authored fifteen years agothe Stem Cell Therapeutic and Research Act of 2005and the Stem Cell Therapeutic and Research Act of 2015.

So, I submit these comments for the Congressional Record.

Madam Speaker, today the House of Representatives will vote to reauthorize the Stem Cell Therapeutic and Research Act.

This was an original idea of mine 20 years ago. Joined by 70 cosponsors, I introduced it in 2001 and again in 2003.

After five long years of hard work and numerous setbacks, my bill was finally enacted into law in 2005.

Beginning in 2001, Dr. Joanne Kurtzberg, who is President of the Cord Blood Association, helped draft my original law.Dr. Kurtzberg has said, Cord blood transplantation is now an established field with enormous potential. In the future, it may emerge as a source of cells for cellular therapies focused on tissue repair and regeneration.

The new law created a nationwide umbilical cord blood stem cell program, designed to collect, derive, type, and freeze cord blood units for transplantation into patients to mitigate and to even cure serious disease. Pursuant to the law, it also provided stem cells for research. The new cord blood program was combined in our 2005 law with an expanded bone marrow initiative, which was crafted over several years by our distinguished colleague, CongressmanBill Young.

I was the prime sponsor again when it was reauthorized in 2015.

Umbilical cord blood stem cells, obtained after the birth of a child, have proved highly efficacious in treating 70 diseases, including sickle-cell disease, lymphoma, and leukemia. And scientists are continuing to study and better understand the regenerative effects of cord blood cell therapies for other diseases and conditions. Bone marrow donations provide lifesaving transplants to treat diseases like blood cancer, sickle cell anemia, or inherited metabolic or immune system disorders.

The National Cord Blood Inventory (NCBI) provides funding to public cord blood banks participating in the program to allow them to expand the national inventory of cord blood units available for transplant. These units are then listed on the registry by the Be the Match Program. The funds appropriated thus far have led to an important increase in the overall number of high-quality cord blood units available through the national registry, including 150,000 NCBI units. Within the Be the Match registry, there are more than 783,000 NCBI units worldwide.

The Program registry allows patients and physicians to locate matching cord blood units, as well as adult donors for marrow and peripheral blood stem cells, when a family donor is not available. The Program is the worlds largest, most diverse donor registry, with more than 22 million volunteers and more than 300,000 public cord blood units. To date, the National Marrow Donor Program/Be The Match (NMDP), through its operation of the Program, has facilitated more than 100,000 transplants. More than 45,000 patients have receivedcord bloodtransplants, according Dr. Joanne Kurtzberg.

The reauthorization before us authorizes $23 million to be appropriated for fiscal year 2021 through fiscal year 2025. It also authorizes $30 million to be appropriated for fiscal years 2021 through 2025 for the bone marrow transplant program. This continues funding at the same levels authorized in the 2015 authorization bill.

Madam Speaker, each year nearly 4 million babies are born in America. In the past, virtually every placenta and umbilical cord was tossed as medical waste. Today, doctors have turned this medical waste into medical miracles.

Not only has God in His wisdom and goodness created a placenta and umbilical cord to nurture and protect the precious life of an unborn child, but now we know that another gift awaits us immediately after birth. Something very special is left behindcord blood that is teeming with lifesaving stem cells. Indeed, it remains one of the best kept secrets in America that umbilical cord blood stem cells and adult stem cells in general are curing people of a myriad of terrible conditions and diseasesover 70 diseases in adults as well as in children.

The legislation that is before us will enable even more patients to receive the treatments that they so desperately need.

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Smith's 2005 stem cell law to be reauthorized by House - InsiderNJ

Next, to accommodate the donors immune cells, they had to wipe out Mr. Browns own immune system by bombarding him with chemotherapy and radiation. Next came the transplant procedure itself. On that same February day, Mr. Brown stopped taking his antiretroviral medication. Three months later, after a grueling recovery in which he almost died, he was H.I.V.-free.

For Mr. Brown, the epiphany came one day in the gym, when he found that he was developing muscles again after years of wasting away. That was kind of my proof that it was gone, he said.

Many hurdles remained. A recurrence of leukemia required a second transplant a year later. A brain biopsy left Mr. Brown temporarily paralyzed and nearly blind. He had to be taught how to walk and talk again. His recovery, complicated by injuries from a 2009 mugging in Berlin, left him with a stiff shoulder, limited vision and neurological damage, which prevented him from resuming his work as a translator.

My life is far from perfect, he said in 2015, but it is still my life.

He was living in Nevada in 2013 when he met Mr. Hoeffgen on the Scruff dating app. They moved to Southern California in 2015. In April, Mr. Brown was admitted to a cancer hospital; his leukemia, unrelated to H.I.V., had returned. Covid-19 restrictions kept the couple together on the medical campus for weeks.

This month, Mr. Hoeffgen told Mark S. King, a blogger and AIDS activist, that Mr. Brown had terminal cancer and had been receiving home hospice care. Mr. Brown was aware that he was dying.

I have asked him what he wants me to tell people when we make his situation public, Mr. Hoeffgen said. He said: Tell people to keep fighting. Fight for a cure for H.I.V. that works for everyone. I never wanted to be the only one.

In addition to Mr. Hoeffgen, Mr. Brown is survived by his mother.

One researcher asked whether the couple would consider donating Mr. Browns body to science.

I said, Thank you, but no, Mr. Hoeffgen said. I think hes done enough.

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Timothy Ray Brown, First Patient Cured of H.I.V., Dies at 54 - The New York Times

Newswise In a world first, a young man suffering from severe aplastic anemia who could not be helped by standard treatments has been given a life-saving blood transplant with the made-in-Canada UM171 molecule.

The procedure was done by a medical team at the Institute of Hemato-oncology and Cellular Therapy (iHOTC) of Maisonneuve-Rosemont Hospital and the Institute for Research in Immunology and Cancer, both affiliated with Universit de Montral.

The young mans case history, including the lifesaving transplant, was recently published in the scientific journal European Journal of Haematology, highlighting the unique and revolutionary properties of the UM171 molecule.

An autoimmune disease, severe aplastic anemia destroys stem cells in bone marrow and leads to a halt in the production of red blood cells, white blood cells and platelets. For allografting (grafting between individuals) for this disease, the donor's stem cells must be as compatible as possible with those of the recipient to avoid the risk of immunological complications.

No donor option

If no compatible family or unrelated donor can be found, stem cells from a semi-identical family donor, also known as a haplo-identical donor, may be considered, under certain conditions, as an alternative source of cells. However, a family member must be healthy and available for such a procedure; the young man in this case did not have that option.

Cord blood transplantation, which is less demanding in terms of compatibility, is a good option for many patients requiring a stem cell transplant. On the other hand, cord blood generally does not contain enough stem cells for an adult patient weighing more than 70 kg; it produces a slow rise in white blood cells with an increased risk of often fatal infections.

In addition, the rate of graft rejection the destruction of infused cord cells by the recipient's immune system is very high in patients with severe aplastic anemia who have received multiple blood transfusions.

It was after having exhausted all our treatment options that UM171, which had already proven itself in a clinical trial in blood cancer patients, came into play, said UdeM medical professor Jean Roy, a hematologist and clinical researcher at the MRH.

35-fold increase

As well as increasing the number of stem cells in a unit of umbilical cord blood by an average of 35 times, it greatly reduces the risk of a frequent long-term immunological complication (graft-versus-host disease) requiring years of use of toxic immunosuppressive drugs.

The researchers success confirms the excellent performance of UM171, which has already been demonstrated in two other studies conducted by iHOTC research teams with very encouraging results. A third study is now underway.

This young man's story and the other studies with UM171 clearly demonstrate how innovative clinical research, set up by local investigators, can create a culture of excellence and improve care to save more lives, said IHOTC director Denis Claude Roy.

The future will certainly bring us more such accomplishments, and thats very encouraging.

Maisonneuve-Rosemont Hospital is one of 26 institutions that are part of the Centre intgr universitaire de sant et de services sociaux de l'Est-de-l'le-de-Montral, serving over half a million Montrealers.

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UM171 saves another life - Newswise

When 5-year-old Sawyer was diagnosed with sickle cell at a young age, his fraternal twin, Saxton, turned out to be a perfect match.

ATLANTA This is not just a birthday party, its a celebration of second chances.

We are so blessed, said OShea Guillory. We want to share this.

It is also a commitment to help others, too.

Mikari Tarpley is 16. She is at the small birthday celebration and sang her heart out for 5-year-old Sawyer and his brother Saxton. She sings from a place of understanding.

She and Sawyer, her neighbor, have both been through a lot this year.

We found out about Sawyers sickle cell disease at a very early age, Guillory said. He was about three weeks old.

Sawyers mom said she was absolutely devastated. She refused to accept there was nothing she could do to help her son.

I did a ton of research," she said. I found an organization called Be The Match.

She discovered information about transplants that could be a potential cure. Doctors take stem cells from the bone marrow of a donor and transplant it into the recipient.

They just needed a donor.

It turns out Sawyer was born with his cure, Guillory said. His twin, Saxton, was a perfect sibling match.

Across the cul-de-sac, Mikari Tarpley, an actress, was fighting Hodgkins Lymphoma and finishing chemotherapy.

She knew Sawyer has been battling sickle cell. Theyd both lost their hair from treatment. She decided she wanted to use her Sweet 16th birthday to raise money for children like Sawyer.

We couldnt do much for my sixteenth because of COVID, so we thought it would be a great idea to do a fundraiser to help others, Mikari said.

She reached her goal of $16,000 to help children being treated for sickle cell disease at the Aflac Cancer and Blood Disorders Center.

Five-year-old Sawyer received a bone marrow transplant from his fraternal twin, Saxton. It was a success. Before long, Sawyer was riding a tricycle around Childrens Healthcare of Atlanta, racing his nurses and giggling along the way.

I truly have little warriors, Guillory said. My son, who helped save his brothers life - and my son - who was able to go through all of that and come out even stronger.

Now Sawyer, Saxton and their parents are spending their 5th birthday following Mikaris lead by paying it forward.

Sawyers mom OShea is starting a nonprofit, Sawyers Sickle Circle, to spread awareness and knowledge about sickle cell, Beads of Courage and Be the Match program. They launched a fundraiser on Sawyer and Saxtons birthday.

The Guillory family said the best present they could wish for is for other kids to have the chance to celebrate many more birthdays to come.

Continued here:
A perfect match: Bone marrow transplant saves twin brother's life. Now their family pays it forward - 11Alive.com WXIA

By Claudia Paba Prada, MD

For more than 30 years, oncologists have used immunotherapy to treat cancer, harnessing the strength of the patients own immune system to fight the disease. For many, it has been a welcome alternative or supplement to more traditional chemotherapy, radiation, and surgical options.

The disease I specialize in, multiple myeloma, has no cure, but patients can maintain quality of life with treatment combinations. These individuals have cancer cells form in plasma cells within bone marrow, crowding out healthy (red and white) blood cells and damaging bones, the immune system, and kidneys. We use immunotherapy in combination with chemotherapy to treat cancerous plasma cells, transitioning to different drugs when the cancer mutates and becomes resistant to the previously prescribed treatment. Our goal is to get patients to a stem cell transplant or, if they arent an appropriate candidate, to utilize a combination of drugs to kill myeloma cells. We then continue maintenance therapy to keep cancer cells dormant and preserve their existing lifestyle.

Its critical that we never stop studying the biology of the diseases we see, since each patient is different and there is no one-size-fits-all treatment. What approaches have been used previously, their toxicity, and the patients comorbidities (diabetes, heart issues, etc.) all factor into what may or may not be the appropriate next step. With younger, newly diagnosed patients, a more aggressive approach to get to transplantation may be pursued. In older patients, the goal is usually to get to some level of remission, even if that isnt a permanent solution.

Some myeloma patients, however, dont respond to any of the available chemotherapy drugs or may have a cancer relapse after their transplant. Thats why there is excitement within the cancer community about clinical trials were participating in at the

Moffitt Malignant Hematology and Cellular Therapy Program at Memorial Hospital West that have increased what were able to accomplish through immunotherapy.

The new approach is called CAR-T cell therapy and its administered like a blood transfusion after the patients own T cells are reprogrammed to attack the cancer cells. This is done by genetically altering T cells so they produce synthetic molecules called chimeric antigen receptors, or CARs, which enable T cells to recognize and attach to a certain protein in tumor cells and kill them.

We see 70-80 new multiple myeloma cases each year and more than 300 with relapse disease so, while not every patient will be a CAR-T candidate, were hoping many more will be as the trial progresses. Were using drugs under research that are unavailable anywhere else in Florida for myeloma and expect to expand to include leukemia and lymphoma patients in the coming year.

All this work is being done as we establish a myeloma-specific institute at Moffitt/Memorial that will be the only one of its type in Broward and Palm Beach counties. Bringing specialists together and providing South Floridians access to clinical trials is part of what were planning, but its also important to address the whole person and not just the disease. Thats why were already collaborating with the Leukemia and Lymphoma Society of Broward County and have established a support group for myeloma patients and their caregivers. The group will address issues related to a cancer diagnosis and provide opportunities for attendees to discuss concerns, anxieties, feelings related to their illness, treatment, and connected issues. Meetings, even the virtual ones were having during COVID-19, are designed to offer mutual support and information to members by connecting them to others whose situations are similar to their own.

My own journey has taken me from my home country of Colombia to an internal medicine residency in Philadelphia, hematology/oncology fellowship in Memphis, and an advanced fellowship in hematologic malignancies at Dana Farber Cancer Institute in Boston. I was at Dana Farber for seven years before relocating to South Florida in 2017. I joined the Moffitt team at Memorial Hospital West in July and am anxious to further the research and treatment of multiple myeloma at one of the nations leading cancer centers. CAR-T cellular therapy is one of the ways we can get there together.

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New Weapons in the Battle Against Blood Cancers: Current Articles - South Florida Hospital News

LONDON (Reuters) - Timothy Ray Brown, the first person known to be cured of HIV when he had a unique type of bone marrow transplant, has died in California after relapsing with cancer, his partner said.

FILE PHOTO: Timothy Ray Brown, also known as "The Berlin Patient," smiles during a news conference held by the World AIDS Institute in Washington July 24, 2012. Brown is thought to be the first patient ever to be cured of HIV infection. REUTERS/Kevin Lamarque

It is with great sadness that I announce that Timothy passed away ... this afternoon surrounded by myself and friends, after a 5 month battle with leukaemia, his partner, Tim Hoeffgen, said in a post on Facebook. He said Brown was his hero and the sweetest person in the world.

Brown, born on March 11, 1966, became known as the Berlin Patient after his HIV was cleared by treatment there in 2007.

The Americans case fascinated and inspired a generation of HIV doctors as well as patients infected with the virus that causes AIDS, offering a glimmer of hope that one day a cure will be found that eventually ends the AIDS pandemic.

We owe Timothy and his doctor, Gero Huetter, a great deal of gratitude for opening the door for scientists to explore the concept that a cure for HIV is possible, said Adeeba Kamarulzaman, president of the International AIDS Society (IAS).

Brown was diagnosed with HIV in 1995 while living in the German capital, and in 2006 was also diagnosed with a type of blood cancer known as acute myeloid leukaemia.

While Brown remained clear of HIV for more than a decade after being treated, he had suffered a relapse of the leukaemia in the past year. His doctors said the blood cancer had spread to his spine and brain, and he had recently been in hospice care in his home town of Palm Springs, California.

Im heartbroken that my hero is now gone. Tim was truly the sweetest person in the world, Hoeffgen said in the Facebook post. He said Brown had made it his lifes work to tell the story of his HIV cure and became an ambassador of hope.

For Huetter, the German doctor caring for him in 2007, Browns case was a shot in the dark. The treatment involved the destruction of Browns immune system and the transplanting of stem cells with a gene mutation called CCR5 that resists HIV.

Only a tiny proportion of people most of them of northern European descent have the CCR5 mutation that makes them resistant to the AIDS-causing virus.

This and other factors made the treatment Brown had expensive, complex and highly risky. Most experts say it could never become a way to cure all HIV patients, since many of them would risk death from the procedure itself.

More than 37 million people worldwide are currently infected with HIV, and the AIDS pandemic has killed about 35 million people since it began in the 1980s.

Medical advances over the past three decades have led to the development of drug combinations known as antiretroviral therapies that can keep the virus in check, allowing many HIV positive people to live with the virus for years.

A second HIV patient, Adam Castillejo, who was known as the London patient until he revealed his identity this year, is also thought to be in remission from HIV after having a transplant in 2016 similar to the one Brown had.

Sharon Lewin, president-elect of the IAS and an HIV specialist at Australias Doherty Institute, said Brown was a champion and advocate for keeping an HIV cure on the agenda.

It is the hope of the scientific community that one day we can honour his legacy with a safe, cost-effective and widely accessible strategy to achieve HIV remission and cure.

Reporting by Kate Kelland, editing by Gareth Jones and Nick Macfie

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World's first patient cured of HIV dies after cancer returns - Reuters India

CLR 131, a phospholipid ether molecule, is showing promising disease control in an ongoing phase 2 study (CLOVER-1, NCT02952508) as treatment of patients with relapsed or refractory lymphoplasmacytic lymphoma (LPL) and Waldenstrm macroglobulinemia (WM), according to initial study results.

The findings were presented in a poster during the American Association for Cancer Research (AACR) Virtual Meeting: Advances in Malignant Lymphoma by Jarrod Longcor, chief business officer of Cellectar Biosciences, Inc, the developer of CLR 131.

Prior to the initiation of the phase 2 study, the potential of CLR 131 was demonstrated throughin vitro, in vivo, and preclinical studies. The agent works by binding to and entering the tumor via lipid rafts, which stabilize tumor cells. This mechanism of action is particularly effective in hematologic malignancies.

Read earlier data on CLR 131 here.

Four patients with LPL/WM were enrolled to assess interim efficacy and safety in part A of the trial. The median age of the population was 70 years (range, 54-81). Three of the patients had an ECOG performance status of 0. The mean prior number of therapies received was 2.5 with a median of 2 (range, 1-5). All patients had received prior rituximab (Rituxan); ibrutinib (Imbruvica) and autologous stem cell transplant were received by 1 patient each. In addition, 75% of patients received other agents.

Efficacy in the Overall Population

The objective response rate (ORR) observed with CLR 131 in the LPL/WM population treated in part A of CLOVER-1 was 100% with a major response to treatment observed in 3 patients after 2 to 4 doses of the drug. Despite the characteristics of these heavily pretreated patients, CLR 131 is the only monotherapy that has achieved responses in the relapsed or refractory population.

In the overall population of 4 patients, survival and duration of response (DOR) were also assessed. Two patients in the study received 1 cycle of CLR 131 (2 doses total) and the other 2 received 2 cycles (4 doses total).

Of the 2 patients receiving 1 cycles of therapy, patient 1 had an overall survival (OS) duration 16.4 month, a progression-free survival (PFS) of 16.4 months, and a DOR of 15 months. The same patient had a major response to therapy. The second patients had an OS and PFS of 13.8 months and a DOR of 13.1 months. The response observed in this patient was a partial response (PR).

Of the 2 patients receiving 2 cycles of therapy, patient 3 had the longest OS and PFS at 33.2 with a DOR of 31.7 months and a complete response to treatment. Finally, patient 4 had an OS and PFS of 10.1 months with a DOR of 8.4 months. The type of response shown with patient 4 was a PR.

In all patients, the median DOR was not reached and the ongoing mean was calculated as 17.1 months. Evaluation of all patient responses is ongoing.

Longer-Term Response in Elderly Patient

One patient in the study was a 66-year-old female who, at baseline, presented with pleural effusion and 5 large extra-medullary nodules in the third-line setting. She was refractory to all of the prior treatment she received. CLR 131 in this patient, however, led to 100% overall tumor burden reduction as well as complete resolution of all tumors. The tumor locations resolved included a subdiaphragmatic mass, left epicardial mass, aortic bifurcation, right ovary, and left ovary. This result occurred by day 187. In terms of the duration of complete response, CT and bone marrow biopsy performed at day 406 revealed the patient had a duration of response of over 31 months.

Safety in the Overall Population

The safety analysis in all patients with non-Hodgkin lymphoma treated on the trial (n = 19) revealed treatment-emergent adverse events (TEAEs) that occurred in 15% or more included thrombocytopenia (83%), lymphocyte count decreased (25%), decreased white blood cell count (58%), anemia (58%), and neutropenia (50%). In the LPL population alone, the most common events observed were neutropenia (100%), thrombocytopenia (100%), and fatigue (75%). The analysis demonstrated that patients with extramedullary disease have lower rates of cytopenias at lower grades. The drug was overall well-tolerated in patients.

CLOVER-1 is an open-label, multicenter, 2-part study. In part A, which is now completed, patients with multiple myeloma, diffuse large B-cell lymphoma, LPL/WM, mantle cell lymphoma, chronic lymphocytic leukemia/small lymphocytic leukemia, and marginal zone lymphoma were assessed for interim CLR 131 efficacy. Patients in part A received a starting dose of less than 50 mCi total body dose of CLR131, following by 50 mCi then 75 mCi. Part B is actively enrolling patients with multiple myeloma and LPL/WM to assess the clinical benefit rate, ORR, time to response, time to progression, and OS. Patients in part B will receive either 1 or 2 cycles of CLR 131 100 mCi total body dose.

Read more about CLR 131 for the treatment of hematologic malignancies.

Reference

Ailwadhi S, Longcor, J Oliver K, and Grachev I. CLR 131 demonstrates 100% overall response rate in relapsed or refractory lymphoplasmacytic lymphoma (LPL)/Waldenstroms macroglobulinemia (WM): initial results from ongoing phase 2 trial, CLOVER-1 study. Presented at: American Association for Cancer Research Virtual Meeting: Advances in Malignant Lymphoma; Aug 1719, 2020. Abstract PO-25

Continued here:
Encouraging ORR Observed With CLR 131 as CLOVER-1 Heads To Pivotal Stage - Targeted Oncology

The global stem cell banking market was valued at $1,986 million in 2016, and is estimated to reach $6,956 million by 2023, registering a CAGR of 19.5% from 2017 to 2023. Stem cell banking is a process where the stem cell care isolated from different sources such as umbilical cord and bone marrow that is stored and preserved for future use. These cells can be cryo-frozen and stored for decades. Private and public banks are different types of banks available to store stem cells.

Top Companies Covered in this Report: Cord Blood Registry,ViaCord,Cryo-Cell, China Cord Blood Corporation, Cryo-Save, New York Cord Blood Program, CordVida, Americord, CryoHoldco, Vita34

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Increase in R&D activities in regards with applications of stem cells and increase in prevalence of fatal chronic diseases majorly drive the growth of the global stem cell banking market. Moreover, the large number of births occurring globally and growth in GDP & disposable income help increase the number of stem cell units stored, which would help fuel the market growth. However, legal and ethical issues related to stem cell collections and high processing & storage cost are projected to hamper the market growth. The initiative taken by organizations and companies to spread awareness in regards with the benefits of stem cells and untapped market in the developing regions help to open new avenues for the growth of stem cell banking market in the near future.

The global stem cell banking market is segmented based on cell type, bank type, service type, utilization, and region. Based on cell type, the market is classified into umbilical cord stem cells, adult stem cells, and embryonic stem cells. Depending on bank type, it is bifurcated into public and private. By service type, it is categorized into collection & transportation, processing, analysis, and storage. By utilization, it is classified into used and unused. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

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Table Of Content

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: STEM CELL BANKING MARKET, BY CELL TYPE

CHAPTER 5: STEM CELL BANKING MARKET, BY BANK TYPE

CHAPTER 6: STEM CELL BANKING MARKET, BY SERVICE TYPE

CHAPTER 7: STEM CELL BANKING MARKET, BY UTILIZATION

CHAPTER 8: STEM CELL BANKING MARKET, BY REGION

CHAPTER 9: COMPANY PROFILES

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Stem Cell Banking Market is forecast to reach $6,956 million by 2023 | ViaCord,Cryo-Cell, China Cord Blood Corporation, Cryo-Save - The Daily...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-02, the companys investigational gene therapy for the treatment of Gaucher disease. AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. AVROBIO recently dosed the first patient in the GuardOne Phase 1/2 clinical trial to evaluate the safety and efficacy of AVR-RD-02.

Like many lysosomal disorders, Gaucher disease can lead to debilitating complications throughout the body and brain. The standard of care does not address all these symptoms and may not be able to halt progression of the disease, said Geoff MacKay, AVROBIOs president and CEO. Our investigational gene therapy is designed to address the head-to-toe manifestations of Gaucher disease with a single dose. Were pleased to receive orphan drug designation, which recognizes the potential of our approach to transform the standard of care and, we hope, the quality of life for people living with this rare genetic disorder.

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

AVR-RD-02 has also received orphan drug designation from the U.S. Food and Drug Administration.

About Gaucher Disease

Gaucher disease is a rare, inherited lysosomal storage disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages bloated with these fatty substances are called Gaucher cells which amass primarily in the spleen, liver and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts and an adjusted lifetime relative risk of developing Parkinson's disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy (ERT) the current standard of care people with Gaucher disease type 1 typically have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life. An estimated 1 in 44,000 people are diagnosed with Gaucher disease.

About AVR-RD-02

AVR-RD-02 is an investigational lentiviral gene therapy designed to provide a durable therapeutic benefit for people living with Gaucher disease. The therapy starts with the patients own hematopoietic stem cells, which are genetically modified to express functional glucocerebrosidase (GCase). Functional GCase reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease. AVROBIO is currently evaluating AVR-RD-02 in GuardOne, a Phase 1/2 clinical trial.

About lentiviral gene therapy

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the therapeutic genes presence as the patients cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated into the patients own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the vector.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, including AVR-RD-02 for the treatment of Gaucher disease; the anticipated benefits of the European Commissions grant of orphan drug designation for AVR-RD-02; the design, commencement, enrollment and timing of ongoing or planned clinical trials and regulatory pathways; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals; the anticipated benefits of our gene therapy platform including the potential impact on our commercialization activities, timing and likelihood of success; the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs; and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in pre-clinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized; the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators; the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials; the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform; the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate; the risk that prior results, such as signals of safety, activity or durability of effect, observed from pre-clinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates; the risk that we will be unable to obtain and maintain regulatory approval for our product candidates; the risk that the size and growth potential of the market for our product candidates will not materialize as expected; risks associated with our dependence on third-party suppliers and manufacturers; risks regarding the accuracy of our estimates of expenses and future revenue; risks relating to our capital requirements and needs for additional financing; risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises; and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease -...

Hematopoietic stem cells are young or immature blood cells found to be living in bone marrow. These blood cells on mature in bone marrow and only a small number of these cells get to enter blood stream. These cells that enter blood stream are called as peripheral blood stems cells. Hematopoietic stem cells transplantation is replacement of absent, diseased or damaged hematopoietic stem cells due to chemotherapy or radiation, with healthy hematopoietic stem cells. Over last 30 years hematopoietic stem cells transplantation market seen rapid expansion and constant expansion with lifesaving technological advances. Hematopoietic stem cells transplantation is also known blood and marrow transplantation which brings about reestablishment of the patients immune and medullary function while treating varied range of about 70 hematological and non-hematological disorders. In general hematopoietic stem cells transplantation is used in treatment of hereditary, oncological, immunological and malignant and non-malignant hematological diseases.

There are two types of peripheral blood stem cell transplants mainly autologous and allogeneic transplantation. In autologous transplants patients own hematopoietic stem cells are harvested or removed before the high-dose treatment that might destroy the patients hematopoietic stem cells. While in allogeneic transplants stem cells are obtained from a tissue type of matched or mismatched donor. Hematopoietic stem cells are harvested from blood or bone marrow and is then frozen to use later. Depending upon the source of hematopoietic stem cells, worldwide there are three types of hematopoietic stem cells transplants namely bone marrow transplant (BMT), peripheral blood stem cell transplant and cord blood transplant. Major drivers in the hematopoietic stem cells transplantation market are establishment of strong and well developed network of hematopoietic stem cells transplantation organizations having global reach and presence has recognized NGO named Worldwide Network for Blood and Marrow Transplantation Group (WBMT) in official relation with World Health Organization (WHO) and rapid increase in number of transplants. Major restraints in hematopoietic stem cells transplantation market is high cost of transplantation and lack of funding for WBMT and other organizations such as regional, national and donor.

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The global market for Hematopoietic stem cells transplantation market is segmented on basis of transplant type, application, disease indication, end user and geography:

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Based on transplantation type, hematopoietic stem cells transplantation market is segmented into allogeneic and autologous. Hematopoietic stem cells transplantation market is also segmented by application type into bone marrow transplant (BMT), peripheral blood stem cell transplant and cord blood transplant. The market for hematopoietic stem cells transplantation is majorly driven by bone marrow transplant (BMT) segment. Based on end user hematopoietic stem cells transplantation market is segmented into hospitals and specialty centers. Peripheral blood stem cell transplant type holds the largest market for hematopoietic stem cells transplantation. Hematopoietic stem cells transplantation market is further segmented by disease indication into three main categories i.e. lymphoproliferative disorders, leukemia, and non-malignant disorders. Segment lymphoproliferative disorder holds largest share amongst the three in Hematopoietic stem cells transplantation market. On the basis of regional presence, global hematopoietic stem cells transplantation market is segmented into five key regions viz. North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. Europe leads the global hematopoietic stem cells transplantation market followed by U.S. due to easy technological applications, funding and high income populations. Other reasons for rise in hematopoietic stem cells transplantation market is high prevalence of lymphoproliferative disorders and leukemia; demand for better treatment options; and easy accessibility and acceptance of population to new technological advances. Transplantation rates in high income countries are increasing at a greater extent but continued rise is also seen in low income countries and expected to rise more. Hematopoietic stem cells transplantation market will have its potential in near future as being a perfect alternative to traditional system in many congenital and acquired hematopoietic disorders management. While India, China and Japan will be emerging as potential markets. An excellent and long term alternative to relief by side effects of chemotherapy, radiotherapy and immune-sensitive malignancies is another driver for hematopoietic stem cells transplantation market. The key players in global hematopoietic stem cells transplantation market are Lonza, Escape Therapeutics, Cesca Therapeutics Inc., Regen BioPharma, Inc., Invitrx Inc, StemGenex, Lion Biotechnologies, Inc., CellGenix GmbH, Actinium Pharmaceuticals, Inc., Pluristem, Kite Pharma, Novartis AG.You Can Request for TOC Here @https://www.persistencemarketresearch.com/toc/14563

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The Hematopoietic Stem Cells Transplantation market to Undergo positive Transformation between 2017 and 2025 - Crypto Daily

Fort Collins, Colorado The Stem Cell Therapy Market is growing at a rapid pace and contributes significantly to the global economy in terms of turnover, growth rate, sales, market share and size. The Stem Cell Therapy Market Report is a comprehensive research paper that provides readers with valuable information to understand the basics of the Stem Cell Therapy Report. The report describes business strategies, market needs, dominant market players and a futuristic view of the market.

The report has been updated to reflect the most recent economic scenario and market size regarding the ongoing COVID-19 pandemic. The report looks at the growth outlook as well as current and futuristic earnings expectations in a post-COVID scenario. The report also covers changing market trends and dynamics as a result of the pandemic and provides an accurate analysis of the impact of the crisis on the market as a whole.

Global Stem Cell TherapyMarketwas valued at 117.66 million in 2019 and is projected to reach USD255.37 million by 2027, growing at a CAGR of 10.97% from 2020 to 2027.

Industry Stem Cell Therapy Study provides an in-depth analysis of key market drivers, opportunities, challenges and their impact on market performance. The report also highlights technological advancements and product developments that drive market needs.

The report contains a detailed analysis of the major players in the market, as well as their business overview, expansion plans and strategies. Key players explored in the report include:

The report provides comprehensive analysis in an organized manner in the form of tables, graphs, charts, pictures and diagrams. Organized data paves the way for research and exploration of current and future market outlooks.

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The report provides comprehensive data on the Stem Cell Therapy market and its trends to help the reader formulate solutions to accelerate business growth. The report provides a comprehensive overview of the economic scenario of the market, as well as its benefits and limitations.

The Stem Cell Therapy Market Report includes production chain analysis and value chain analysis to provide a comprehensive picture of the Stem Cell Therapy market. The research consists of market analysis and detailed analysis of application segments, product types, market size, growth rates, and current and emerging industry trends.

1.Stem Cell Therapy Market, By Cell Source:

Adipose Tissue-Derived Mesenchymal Stem Cells Bone Marrow-Derived Mesenchymal Stem Cells Cord Blood/Embryonic Stem Cells Other Cell Sources

2.Stem Cell Therapy Market, By Therapeutic Application:

Musculoskeletal Disorders Wounds and Injuries Cardiovascular Diseases Surgeries Gastrointestinal Diseases Other Applications

3.Stem Cell Therapy Market, By Type:

Allogeneic Stem Cell Therapy Market, By Application Musculoskeletal Disorders Wounds and Injuries Surgeries Acute Graft-Versus-Host Disease (AGVHD) Other Applications Autologous Stem Cell Therapy Market, By Application Cardiovascular Diseases Wounds and Injuries Gastrointestinal Diseases Other Applications

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The market is geographically spread across several key geographic regions and the report includes regional analysis as well as production, consumption, revenue and market share in these regions for the 2020-2027 forecast period. Regions include North America, Latin America, Europe, Asia Pacific, the Middle East, and Africa.

Radical Coverage of the Stem Cell Therapy Market:

Key Questions Addressed in the Report:

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Stem Cell Therapy Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 - The Daily Chronicle

Watch here: https://www.youtube.com/watch?feature=youtu.be&v=VVkQU91KbEs

press release: The UW has a long history of pioneering medical advancements that have transformed the world. From performing the first bone marrow transplant in the United States to cultivating the first laboratory-derived human embryonic stem cells. Now, where will UW medical research go next?

On the next Wisconsin Medicine Livestream, meet trailblazing doctors, researchers, and medical leaders who are charting a bold course to completely alter the health care landscape. During this insightful panel discussion, well explore how gene therapy and cell replacements could hold the keys to treating inherited and acquired blindness. Youll also discover the remarkable potential in xenotransplantation where nonhuman animal source organs are transplanted into human recipients. In addition, you will learn about UW Healths journey to build a multidisciplinary program to serve the community. These, and other, fascinating developments in treatment and care are happening right now at the UW and are the future of medicine. The presentation will be moderated by Robert Golden, the dean of the University of WisconsinMadisons School of Medicine and Public Health.

Our Guests:

David Gamm, professor, Department of Ophthalmology and Visual Sciences; Emmett A. Humble Distinguished Director, McPherson Eye Research Institute; Sandra Lemke Trout Chair in Eye Research

Dr. Gamms lab is at the forefront in developing cell-based therapies to combat retinal degenerative diseases (RDDs). As the director of the McPherson Eye Research Institute and a member of the Waisman Center Stem Cell Research Program, the UW Stem Cell and Regenerative Medicine Center, and the American Society for Clinical Investigation, his efforts are directed toward basic and translational retinal stem cell research. The Gamm Lab uses induced pluripotent stem cells to create retinal tissues composed of authentic human photoreceptor cells rods and cones that can detect light and initiate visual signals in a dish. The aims of his laboratory are to investigate the cellular and molecular events that occur during human retinal development and to generate cells for use in retinal disease modeling and cell replacement therapies. In collaboration with other researchers at UWMadison and around the world, the lab is developing methods to produce and transplant photoreceptors and/or retinal pigment epithelium (RPE) in preparation for future clinical trials. At the same time, the Gamm Lab uses lab-grown photoreceptor and RPE cells to test and advance a host of other experimental treatments, including gene therapies. In so doing, the lab seeks to delay or reverse the effects of blinding disorders, such as retinitis pigmentosa and age-related macular degeneration, and to develop or codevelop effective interventions for these RDDs at all stages of disease.

Dhanansayan Shanmuganayagam, assistant professor, Department of Surgery, School of Medicine and Public Health; Department of Animal and Dairy Sciences, UWMadison; director, Biomedical, and Genomic Research Group

Dr. Shanmuganayagams research focuses on the development and utilization of pigs as homologous models to close the translational gap in human disease research, taking advantage of the overwhelming similarities between pigs and humans in terms of genetics, anatomy, physiology, and immunology. He and his colleagues created the human-sized Wisconsin Miniature Swine breed that is unique to the university. The breed exhibits greater physiological similarity to humans, particularly in vascular biology and in modeling metabolic disorders and obesity. He currently leads genetic engineering of swine at the UW. His team has created more than 15 genetic porcine models including several of pediatric genetic cancer-predisposition disorders such as neurofibromatosis type 1 (NF1). In the context of NF1, his lab is studying the role of alternative splicing of the nf1 gene on the tissue-specific function of neurofibromin and whether gene therapy to modulate the regulation of this splicing can be used as a viable treatment strategy for children with the disorder.

Dr. Shanmuganayagam is also currently leading the efforts to establish the University of Wisconsin Center for Biomedical Swine Research and Innovation (CBSRI) that will leverage the translatability of research in pig models and UWMadisons unique swine and biomedical research infrastructure, resources, and expertise to conduct innovative basic and translational research on human diseases. The central mission of CBSRI is to innovate and accelerate the discovery and development of clinically relevant therapies and technologies. The center will also serve to innovate graduate and medical training. As the only center of its kind in the United States, CBSRI will make UWMadison a hub of translational research and industry-partnered biomedical innovation.

Petros Anagnostopoulos, surgeon in chief, American Family Childrens Hospital; chief, Section of Pediatric Cardiothoracic Surgery; professor, Department of Surgery, Division of Cardiothoracic Surgery

Dr. Anagnostopoulos is certified by the American Board of Thoracic Surgery and the American Board of Surgery. He completed two fellowships, one in cardiothoracic surgery at the University of Pittsburgh School of Medicine and a second in pediatric cardiac surgery at the University of California, San Francisco School of Medicine. He completed his general surgery residency at Henry Ford Hospital in Detroit. Dr. Anagnostopoulos received his MD from the University of Athens Medical School, Greece. His clinical interests include pediatric congenital heart surgery and minimally invasive heart surgery.

Dr. Anagnostopoulos specializes in complex neonatal and infant cardiac reconstructive surgery, pediatric heart surgery, adult congenital cardiac surgery, single ventricle palliation, extracorporeal life support, extracorporeal membrane oxygenation, ventricular assist devices, minimally invasive cardiac surgery, hybrid surgical-catheterization cardiac surgery, off-pump cardiac surgery, complex mitral and tricuspid valve repair, aortic root surgery, tetralogy of Fallot, coronary artery anomalies, Ross operations, obstructive cardiomyopathy, and heart transplantation.

When: Tuesday, Sept. 29, at 7 p.m. CDT

Where: Wisconsin Medicine Livestream: wiscmedicine.org/programs/ending-alzheimers

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ONLINE: The Future of Medicine - Isthmus

COMEDIAN Al Murray told Lorraine Kelly today his seven-year-old nephew is "very ill" but is "hanging in there" amid his battle with leukemia.

The Pub Landlord star, 52, appeared on the ITV lunchtime show to urge viewers to donate the price of a round of drinks to cancer charity DKMS, and help them boost their stem cell register.

3

Finley Relf has a rare and aggressive form ofleukaemiaand his only hope of survival is a bone marrow transplant.

Al's partner Eleanor Relf is the sister of Finley's dad Ben. Finley's family had been tested to be a donor, but are sadly not a match.

Speaking to Lorraine, 60, Al said: "Hes an incredibly brave little lad.

"I cant tell you hes well, hes very, very ill, but hes hanging in there.

"They load him up with treatments and he takes it. But he's seven, he wants to be out there playing footballwith his friends."

3

Al went on: "People can get on this register and then be a life saver in waiting. That's what we're trying to do.

"Theres been a decline in donors and contributions. The thing about blood stem cell donations is its dead easy.

"They send you a pack with swabs and you swab some cheek tissue and send it back to them."

And Lorraine joked: "It's just like picking your nose which we all do!"

It takes just a few minutes to become a donorbut Al explained that processing the swabs to add people to the register costs DKMS 40 per person.

3

Finley, who lives in Haywards Heath, West Sussex, with four-year-old brother Jacob, is having chemotherapy at Londons Great Ormond Street Hospital.

Finleys family are hoping another stranger will be identified as his genetic twin with similar tissue. Al said Finley has tried two donors but their stem cells didn't work.

He said: Its not like blood types. Its much more fundamental to your DNA."

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DONAL'S BACKFans say it's 'great to see' Donal Maher back on Fair City as Paul helps Ruth

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Baby prepGlenda Gilson reveals she's 'packing hospital bag' as due date fast approaches

"Its not quite fingerprints but its on that level, so thats why we need more people to join the register."

Finleys ordeal started last year when his parents Ben and Nicky noticed he was losing weight.

Doctors discovered his spleen was enlarged and he underwent numerous tests, which led them to diagnosing him with blood cancer.

Visit link:
Al Murray says his seven-year-old nephew is very ill but hanging on in there amid battle with rare form of L - The Irish Sun

AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-02, the companys investigational gene therapy for the treatment of Gaucher disease. AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. AVROBIO recently dosed the first patient in the GuardOne Phase 1/2 clinical trial to evaluate the safety and efficacy of AVR-RD-02.

"Like many lysosomal disorders, Gaucher disease can lead to debilitating complications throughout the body and brain. The standard of care does not address all these symptoms and may not be able to halt progression of the disease," said Geoff MacKay, AVROBIOs president and CEO. "Our investigational gene therapy is designed to address the head-to-toe manifestations of Gaucher disease with a single dose. Were pleased to receive orphan drug designation, which recognizes the potential of our approach to transform the standard of care and, we hope, the quality of life for people living with this rare genetic disorder."

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

AVR-RD-02 has also received orphan drug designation from the U.S. Food and Drug Administration.

About Gaucher Disease

Gaucher disease is a rare, inherited lysosomal storage disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages bloated with these fatty substances are called Gaucher cells which amass primarily in the spleen, liver and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts and an adjusted lifetime relative risk of developing Parkinson's disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy (ERT) the current standard of care people with Gaucher disease type 1 typically have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life. An estimated 1 in 44,000 people are diagnosed with Gaucher disease.

Story continues

About AVR-RD-02

AVR-RD-02 is an investigational lentiviral gene therapy designed to provide a durable therapeutic benefit for people living with Gaucher disease. The therapy starts with the patients own hematopoietic stem cells, which are genetically modified to express functional glucocerebrosidase (GCase). Functional GCase reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease. AVROBIO is currently evaluating AVR-RD-02 in GuardOne, a Phase 1/2 clinical trial.

About lentiviral gene therapy

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the therapeutic genes presence as the patients cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated into the patients own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the vector.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as "aims," "anticipates," "believes," "could," "designed to," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, including AVR-RD-02 for the treatment of Gaucher disease; the anticipated benefits of the European Commissions grant of orphan drug designation for AVR-RD-02; the design, commencement, enrollment and timing of ongoing or planned clinical trials and regulatory pathways; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals; the anticipated benefits of our gene therapy platform including the potential impact on our commercialization activities, timing and likelihood of success; the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs; and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in pre-clinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized; the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators; the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials; the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform; the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate; the risk that prior results, such as signals of safety, activity or durability of effect, observed from pre-clinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates; the risk that we will be unable to obtain and maintain regulatory approval for our product candidates; the risk that the size and growth potential of the market for our product candidates will not materialize as expected; risks associated with our dependence on third-party suppliers and manufacturers; risks regarding the accuracy of our estimates of expenses and future revenue; risks relating to our capital requirements and needs for additional financing; risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises; and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled "Risk Factors" in AVROBIOs most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200928005176/en/

Contacts

Investor Contact: Christopher F. BrinzeyWestwicke, an ICR Company339-970-2843chris.brinzey@westwicke.com

Media Contact: Tom DonovanTen Bridge Communications857-559-3397tom@tenbridgecommunications.com

See the article here:
AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease - Yahoo...

MINNEAPOLIS, Sept. 24, 2020 /PRNewswire/ --Today, Senator Jack Reed (D-RI) introduced the TRANSPLANT Act (S. 4762), which will provide for the timely reauthorization of the C.W. Bill Young Transplantation Program (Program). The National Marrow Donor Program(NMDP)/Be The Match applauds the leadership of Senator Reed and urges Congress to act quickly to reauthorize the Program, which expires on September 30th.

"We appreciate the leadership of the bill's sponsor Sen. Jack Reed (D-RI), and the bipartisan support of the bill's lead cosponsors Sens. Richard Burr (R-NC), Tina Smith (D-MN), and Tim Scott (R-SC)," said NMDP/Be The Match Chief Policy Officer Brian Lindberg.

The Program provides access to live-saving bone marrow, peripheral blood stem cell (PBSC) and cord blood transplants to those living with one of the more than 70 blood cancers or blood disorders for which a transplant is the only curative option.

Since its inception in the mid-1980s, NMDP/Be The Match has been entrusted by Congress to operate the Program and has facilitated more than 100,000 life-saving transplants to give patients with otherwise fatal blood cancers or blood disorders a second chance at life.

The COVID-19 pandemic with increasingly restrictive travel bans, border closings, and the declining availability of scheduled commercial passenger aviation service on international and domestic routes - has created unprecedented obstacles for NMDP/ Be The Match in delivering life-saving cells to the patients who need them.

This new reality creates a web of increasingly complex challenges in facilitating the timely collection and delivery of bone marrow products to patients who are in the midst of treatment protocols or whose conditions have deteriorated to the point that a bone marrow transplant is the only course of treatment that will save their life.

"During the pandemic, we have been able to mitigate any disruption to our mission and complete more than 3,000 lifesaving therapies without missing a single delivery," continued Lindberg. "We have been able to do that, in large part, because of our designation in Federal law as the nation's bone marrow registry. Unfortunately, that designation will lapse with the expiration of our Federal authorization on September 30, 2020."

This designation has helped Be The Match to care for our patients during the pandemic by:

"Our ability to call upon our Federal partners in these extreme circumstances, and our standing in the eyes of foreign governments where we must operate, could be compromised after September 30th if our authorization expires and we are no longer technically designated as the Nation's registry," said Lindberg. "This could put at risk our ability to continue to ensure the timely delivery of life-saving cellular products to otherwise terminally ill patients here and throughout the world.

"Ensuring access to cellular therapy is critical for the patients we serve," continued Lindberg. "Congress must move immediately to reauthorize these programs to expand the number of adult volunteer donors and cord blood units on the national registry so that every American who needs a transplant can find a match."

About Be The MatchFor people with life-threatening blood cancerslike leukemia and lymphomaor other diseases, a cure exists. Be The Match connects patients with their donor match for a life-saving marrow or umbilical cord blood transplant. People can contribute to the cure as a member of the Be The Match Registry, financial contributor or volunteer. Be The Match provides patients and their families one-on-one support, education, and guidancebefore, during and after transplant.

Be The Match is operated by the National Marrow Donor Program (NMDP), a nonprofit organization that matches patients with donors, educates health care professionals and conducts research through its research program, CIBMTR (Center for International Blood and Marrow Transplant Research), so more lives can be saved. To learn more about the cure, visit BeTheMatch.org or call 1 (800) MARROW-2.

SOURCE Be The Match

https://bethematch.org

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National Marrow Donor Program/Be The Match Urges Congress to Quickly Pass Reauthorization Bill to Protect Access to Life Saving Bone Marrow and Cord...

Latest Research Study on Stem Cell Therapy Market published by The Insight Partners, offers a detailed overview of the factors influencing the global business scope. The research report provides deep insights into the global market revenue, parent market trends, macro-economic indicators, and governing factors, along with market attractiveness per market segment. The report provides an overview of the growth rate of the Stem Cell Therapy market during the forecast period, i.e., 20202027. Most importantly, the report further identifies the qualitative impact of various market factors on market segments and geographies. The research segments the market on the basis of product type, application, technology, and region. To offer more clarity regarding the industry, the report takes a closer look at the current status of various factors including but not limited to supply chain management, niche markets, distribution channel, trade, supply, and demand and production capability across different countries. Some of the key players profiled in the study are MEDIPOST, Pharmicell Co., Inc., RichSource, BioTime Inc. (Lineage Cell Therapeutics, Inc.), Mesoblast Limited, Holostem Terapie Avanzate Srl, U.S. Stem Cell, Inc., Caladrius Biosciences, Inc., TiGenix NV, AlloSource, etc.

The stem cell therapy marketwas valued at US$ 1,534.55 million in 2019 and is expected to grow at a CAGR of 16.7% from 2020to 2027 to reach US$ 5,129.66 million by 2027.

Request Sample Copy of Stem Cell Therapy Market at: https://www.theinsightpartners.com/sample/TIPHE100000991/

Stem Cell Therapy Market In-Depth Analysis

Stem cells are preliminary body cells from which all other cells with specialized functions are generated. Under controlled environment in the body or a clinical laboratory, these cells divide to form more cells called daughter cells. Due to the advent of modern health science, these cells play a major role in understanding the occurrence of diseases, generation of advanced regenerative medicines, and drug discovery. There are certain sources such as embryo, bone marrow, body fats, and umbilical cord blood amongst others, where stem cells are generated. The global stem cell therapy market is driven by factors such asincreasing awareness related to the stem cells therapy in effective disease management and growing demand for regenerative medicines. However, high cost related with stem cell therapy is likely to obstruct the growth of the stem cell therapymarket during the forecast period. The growing research and development activities in Asia Pacific region is expected to offer huge growth opportunity for stem cell therapy market.

End User Insights

Based on end user, the Stem Cell Therapy market is segmented into academic and research institutes, hospitals and specialty clinics. The academic and research institutes held the largest share of end user segment in the global market and is expected to grow at the fastest rate during the forecast period.

Key Benefits

The report profiles the key players in the industry, along with a detailed analysis of their individual positions against the global landscape. The study conducts SWOT analysis to evaluate strengths and weaknesses of the key players in the Stem Cell Therapy market. The researcher provides an extensive analysis of the Stem Cell Therapy market size, share, trends, overall earnings, gross revenue, and profit margin to accurately draw a forecast and provide expert insights to investors to keep them updated with the trends in the market.

Competitive scenario:

The study assesses factors such as segmentation, description, and applications of Stem Cell Therapy industries. It derives accurate insights to give a holistic view of the dynamic features of the business, including shares, profit generation, thereby directing focus on the critical aspects of the business.

Scope of the Report

The research on the Stem Cell Therapy market focuses on mining out valuable data on investment pockets, growth opportunities, and major market vendors to help clients understand their competitors methodologies. The research also segments the Stem Cell Therapy market on the basis of end user, product type, application, and demography for the forecast period 20212027. Comprehensive analysis of critical aspects such as impacting factors and competitive landscape are showcased with the help of vital resources, such as charts, tables, and infographics.

Global Stem Cell Therapy Market By Type

Global Stem Cell Therapy Market By Treatment

Global Stem Cell Therapy Market ByApplication

Global Stem Cell Therapy Market By End User

Promising Regions & Countries Mentioned in TheStem Cell Therapy Market Report:

Major highlights of the report:

All-inclusive evaluation of the parent market

Evolution of significant market aspects

Industry-wide investigation of market segments

Assessment of market value and volume in past, present, and forecast years

Evaluation of market share

Study of niche industrial sectors

Tactical approaches of market leaders

Lucrative strategies to help companies strengthen their position in the market

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Massive Growth Seen in Stem Cell Therapy Market 2020-2027 | In Depth Analysis with Top Key Players RichSource, Mesoblast Limited, TiGenix NV,...

Alyssa Mulvey in the Great North Children's Hospital at the RVI.

The total brought in by the man who walked 21 laps around Elizabethan walls earlier this month reached 2,000 earlier this week.

He was accompanied by Stewart Brown for the whole 26.2-mile marathon distance and family, friends and other supporters did some of the laps during the event in aid of wards in the Great North Children's Hospital (GNCH) at the RVI in Newcastle.

On his fund-raising page, Derek who lives in Highcliffe, Spittal specifically mentioned two local girls from Berwick (Alyssa Mulvey and Kelis Bloomfield) who are currently receiving treatment at the GNCH.

Alyssa has been poorly since birth and was diagnosed with A20 haploinsufficiency, a very rare auto-immune condition, when she was 10.

She has been on ward 3, a bone marrow transplant ward that deals with children who were born with zero immune systems or severe immune conditions. She had recovered well from a stem cell transplant last year, using stem cells donated by her dad Shane, but then contracted Covid-19.

Fortunately, Alyssa made an excellent recovery. However, a check-up weeks later revealed her blood pressure was extremely high and tests found that she had a serious kidney disease called thrombotic microangiopathy (TMA).

Although the condition was not caused by Covid-19, medics believe the virus ignited it in her system and that it had been dormant beforehand. She has now been at the GNCH for 21 consecutive weeks.

Kelis receives treatment on ward 2a. She has eosinophilic esophagitis (EoE), which is a chronic allergic inflammatory disease of the esophagus. She is allergic to things such as grass and pollen, which can cause respiratory failure.

Ward 2a specialises in respiratory and long-term ventilation, and diabetes.

Derek has previously supported the GNCH by doing the Great North Run half marathon his way of giving back to the team who saved the life of his daughter, Saskia, in 2018.

As the run was not held this year due to the coronavirus pandemic, he still wanted to do something in 2020 and so came up with the local challenge, which took place earlier this month before the extra local and national restrictions were announced.

He said: We set off just before 6am and after 8am, Deva (his wife) and Saskia joined us and walked the opposite way. Friends and supporters did some of the laps with us.

"We kept a brisk pace and when we had a break, we had a bacon roll and coffee courtesy of The Mule on Rouge.

"Many of the visitors on the day asked us why were doing the walk and a combination of visitors, friends and supporters saw a lot of people put money into the charity bucket that we had with us when we counted it later, the total in it was 255.

In the afternoon, Deva and Saskia completed half marathon distance and with six laps to go we were joined by the Plundering Pirates of the North East, dressed in costume, who raised money for their own charities. The last lap was done with Stewarts wife Amanda and their children, Mathew and Mia. Me and Stewart were pleased to complete the marathon distance in 10 hours.

Also thanks to all those who have donated online gf.me/u/yny5p2 its great that Ill be giving more than 1,000 to the two wards.

Alyssas mum Kirstie said that her daughter, 14, has infusions meds going in through a central line every day and has the dialysis procedure four to five times a week.

In relation to Dereks fund-raising, she said: He is amazing and we think he is an absolute superhero for helping to raise funds for and awareness of these wards.

The money will be vital for them because they are not as well known as others such as the cancer wards.

Other support for Alyssa and her family includes Kirsties best friend Julie Newton organising a raffle (1,000) and piping events on Thursday evenings at Mordington Avenue until last week (about 700 in total from the bucket donations each week).

Denise Lody from Facebook group Isolation Berwick upon Tweed and Surrounding Area set-up a GoFundMe page that raised 2,651.

Sean Ryan and Dave Smith of 2SPT raised 2,623.55 by running private physical training sessions through the lockdown period.

Further funds came from a beautiful cake raffled by Ashley McKnight and Berwick landscape postcards sold by Julie.

Kirstie said: The response has been unbelievable. Alyssa said to me why are they doing this for me, but I reminded her of how brave she is and told her how much people love her and they wanted to show just how much they care.

We were unable to speak in depth with Kelis mum Stacey, but she did send us a message that said: What Derek has done for these wards is absolutely amazing.

"Kelis has helped out by doing an online raffle, which raised 70 to the cause.

Originally posted here:
Marathon effort in aid of hospital wards - Berwick Advertiser

Los Angeles, United State, The report titledGlobal Hematopoietic Stem Cell Transplantation (HSCT) Marketis one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Hematopoietic Stem Cell Transplantation (HSCT) market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Hematopoietic Stem Cell Transplantation (HSCT) market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Hematopoietic Stem Cell Transplantation (HSCT) market is carefully analyzed and researched about by the market analysts.

Global Hematopoietic Stem Cell Transplantation (HSCT) Market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

Top Key Players of the Global Hematopoietic Stem Cell Transplantation (HSCT) Market : Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT)

>>>Download Full PDF Sample Copy of Report: (Including Full TOC, List of Tables & Figures, Chart) :

https://www.qyresearch.com/sample-form/form/1528548/global-hematopoietic-stem-cell-transplantation-hsct-market

The Essential Content Covered in the Global Hematopoietic Stem Cell Transplantation (HSCT) Market Report :

Top Key Company Profiles.Main Business and Rival InformationSWOT Analysis and PESTEL AnalysisProduction, Sales, Revenue, Price and Gross MarginMarket Size And Growth RateCompany Market Share

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Segmentation By Product:, Allogeneic, Autologous Hematopoietic Stem Cell Transplantation (HSCT)

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Segmentation By Application: Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT)

In terms of region, this research report covers almost all the major regions across the globe such asNorth America, Europe, South America, the Middle East, and Africa and the Asia Pacific. Europe and North America regions are anticipated to show an upward growth in the years to come. WhileHematopoietic Stem Cell Transplantation (HSCT) Market in Asia Pacific regions is likely to show remarkable growth during the forecasted period. Cutting edge technology and innovations are the most important traits of the North America region and thats the reason most of the time the US dominates the global markets.Hematopoietic Stem Cell Transplantation (HSCT) Market in South, America region is also expected to grow in near future.

Key questions answered in the report

*What will be the market size in terms of value and volume in the next five years?*Which segment is currently leading the market?*In which region will the market find its highest growth?*Which players will take the lead in the market?*What are the key drivers and restraints of the markets growth?

We provide detailed product mapping and analysis of various market scenarios. Our analysts are experts in providing in-depth analysis and breakdown of the business of key market leaders. We keep a close eye on recent developments and follow latest company news related to different players operating in the global Hematopoietic Stem Cell Transplantation (HSCT) market. This helps us to deeply analyze companies as well as the competitive landscape. Our vendor landscape analysis offers a complete study that will help you to stay on top of the competition.

Why to Buy this Report?

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Table of Contents

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by Hematopoietic Stem Cell Transplantation (HSCT) Revenue1.4 Market by Type1.4.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size Growth Rate by Type: 2020 VS 20261.4.2 Allogeneic1.4.3 Autologous1.5 Market by Application1.5.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Share by Application: 2020 VS 20261.5.2 Peripheral Blood Stem Cells Transplant (PBSCT)1.5.3 Bone Marrow Transplant (BMT)1.5.4 Cord Blood Transplant (CBT)1.6 Study Objectives1.7 Years Considered 2 Global Growth Trends2.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Perspective (2015-2026)2.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Growth Trends by Regions2.2.1 Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Share by Regions (2015-2020)2.2.3 Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 Hematopoietic Stem Cell Transplantation (HSCT) Market Growth Strategy2.3.6 Primary Interviews with Key Hematopoietic Stem Cell Transplantation (HSCT) Players (Opinion Leaders) 3 Competition Landscape by Key Players3.1 Global Top Hematopoietic Stem Cell Transplantation (HSCT) Players by Market Size3.1.1 Global Top Hematopoietic Stem Cell Transplantation (HSCT) Players by Revenue (2015-2020)3.1.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Revenue Market Share by Players (2015-2020)3.1.3 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Concentration Ratio3.2.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by Hematopoietic Stem Cell Transplantation (HSCT) Revenue in 20193.3 Hematopoietic Stem Cell Transplantation (HSCT) Key Players Head office and Area Served3.4 Key Players Hematopoietic Stem Cell Transplantation (HSCT) Product Solution and Service3.5 Date of Enter into Hematopoietic Stem Cell Transplantation (HSCT) Market3.6 Mergers & Acquisitions, Expansion Plans 4 Market Size by Type (2015-2026)4.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Size by Type (2015-2020)4.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Type (2021-2026) 5 Market Size by Application (2015-2026)5.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020)5.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Application (2021-2026) 6 North America6.1 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)6.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in North America (2019-2020)6.3 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)6.4 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 7 Europe7.1 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)7.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Europe (2019-2020)7.3 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)7.4 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 8 China8.1 China Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)8.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in China (2019-2020)8.3 China Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)8.4 China Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 9 Japan9.1 Japan Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)9.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Japan (2019-2020)9.3 Japan Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)9.4 Japan Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 10 Southeast Asia10.1 Southeast Asia Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)10.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Southeast Asia (2019-2020)10.3 Southeast Asia Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)10.4 Southeast Asia Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 11 India11.1 India Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)11.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in India (2019-2020)11.3 India Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)11.4 India Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 12 Central & South America12.1 Central & South America Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)12.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Central & South America (2019-2020)12.3 Central & South America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)12.4 Central & South America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 13 Key Players Profiles13.1 Regen Biopharma Inc13.1.1 Regen Biopharma Inc Company Details13.1.2 Regen Biopharma Inc Business Overview13.1.3 Regen Biopharma Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.1.4 Regen Biopharma Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020))13.1.5 Regen Biopharma Inc Recent Development13.2 China Cord Blood Corp13.2.1 China Cord Blood Corp Company Details13.2.2 China Cord Blood Corp Business Overview13.2.3 China Cord Blood Corp Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.2.4 China Cord Blood Corp Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.2.5 China Cord Blood Corp Recent Development13.3 CBR Systems Inc13.3.1 CBR Systems Inc Company Details13.3.2 CBR Systems Inc Business Overview13.3.3 CBR Systems Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.3.4 CBR Systems Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.3.5 CBR Systems Inc Recent Development13.4 Escape Therapeutics Inc13.4.1 Escape Therapeutics Inc Company Details13.4.2 Escape Therapeutics Inc Business Overview13.4.3 Escape Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.4.4 Escape Therapeutics Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.4.5 Escape Therapeutics Inc Recent Development13.5 Cryo-Save AG13.5.1 Cryo-Save AG Company Details13.5.2 Cryo-Save AG Business Overview13.5.3 Cryo-Save AG Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.5.4 Cryo-Save AG Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.5.5 Cryo-Save AG Recent Development13.6 Lonza Group Ltd13.6.1 Lonza Group Ltd Company Details13.6.2 Lonza Group Ltd Business Overview13.6.3 Lonza Group Ltd Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.6.4 Lonza Group Ltd Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.6.5 Lonza Group Ltd Recent Development13.7 Pluristem Therapeutics Inc13.7.1 Pluristem Therapeutics Inc Company Details13.7.2 Pluristem Therapeutics Inc Business Overview13.7.3 Pluristem Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.7.4 Pluristem Therapeutics Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.7.5 Pluristem Therapeutics Inc Recent Development13.8 ViaCord Inc13.8.1 ViaCord Inc Company Details13.8.2 ViaCord Inc Business Overview13.8.3 ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.8.4 ViaCord Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.8.5 ViaCord Inc Recent Development 14 Analysts Viewpoints/Conclusions 15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Disclaimer15.3 Author Details

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Hematopoietic Stem Cell Transplantation (HSCT) Market is Thriving Worldwide with Top Companies like: China Cord Blood Corp, CBR Systems Inc, Escape...

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, today announced that it has licensed intellectual property relating to its pioneering chlorotoxin chimeric antigen receptor (CLTX-CAR) T cell therapy to Chimeric Therapeutics Limited, an Australian biotechnology company.

The therapy is currently being used in a phase 1 clinical trial at City of Hope to treat glioblastoma (GBM), a type of brain tumor. The first patient in the trial was recently dosed; Behnam Badie, M.D., chief of City of Hopes Division of Neurosurgery and The Heritage Provider Network Professor in Gene Therapy, is leading this innovative, first-of-its-kind trial.

Chimeric has acquired the exclusive worldwide rights to develop and commercialize certain patents relating to City of Hopes CLTX-CAR T cells, as well as to further develop the therapy for other cancers.

City of Hope is excited to enter into this agreement with Chimeric as it supports our innovative research in CAR T cell therapy and our commitment to extend these therapies to more patients, particularly those with GBM and other solid tumors that are difficult to treat, said Christine Brown, Ph.D., The Heritage Provider Network Professor in Immunotherapy and deputy director of City of Hopes T Cell Therapeutics Research Laboratory. Chimeric shares our goal of providing effective CAR T cell therapies to more patients with current unmet medical needs.

Led by Brown and Michael Barish, Ph.D., chair of City of Hopes Department of Developmental and Stem Cell Biology, and Dongrui Wang, Ph.D., a recent graduate of City of Hopes Irell & Manella Graduate School of Biological Sciences, the team developed and tested the first CAR T cell therapy using CLTX, a component of scorpion venom, to direct T cells to target brain tumor cells. The research was published this past March in Science Translational Medicine.

Chimeric is excited to join City of Hope in its quest to find more effective cancer therapies. This is an exceedingly rare opportunity to acquire a promising technology in one of the most exciting areas of immuno-oncology today, said Paul Hopper, executive chairman of Chimeric. Furthermore, the CLTX-CAR T cell therapy has completed years of preclinical research and development, and recently enrolled its first patient in a phase 1 clinical trial for brain cancer.

CARs commonly incorporate a monoclonal antibody sequence in their targeting domain, enabling CAR T cells to recognize antigens and kill tumor cells. In contrast, the CLTX-CAR uses a synthetic 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain.

In this recent study, City of Hope researchers used tumor cells in resection samples from a cohort of patients with GBM to compare CLTX binding with expression of antigens currently under investigation as CAR T cell targets. They found that CLTX bound to a greater proportion of patient tumors, and cells within these tumors.

CLTX binding included the GBM stem-like cells thought to seed tumor recurrence. Consistent with these observations, CLTX-CAR T cells recognized and killed broad populations of GBM cells while ignoring nontumor cells in the brain and other organs. The study team demonstrated that CLTX-directed CAR T cells are highly effective at selectively killing human GBM cells without off-tumor targeting and toxicity in cell-based assays and in animal models.

City of Hope, a recognized leader in CAR T cell therapies for GBM and other cancers, has treated more than 500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has 30 ongoing CAR T cell clinical trials, including CAR T cell trials for HER-2 positive breast cancer that has spread to the brain, and PSCA-positive bone metastatic prostate cancer. It was the first and only cancer center to treat GBM patients with CAR T cells targeting IL13R2, and the first to administer CAR T cell therapy locally in the brain, either by direct injection at the tumor site, through intraventricular infusion into the cerebrospinal fluid, or both. In late 2019, City of Hope opened a first-in-human clinical trial for patients with recurrent GBM, combining IL13R2-CAR T cells with checkpoint inhibitors nivolumab, an anti-PD1 antibody, and ipilimumab, blocking the CTLA-4 protein.

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"City of Hope is delighted to license this technology to Chimeric, said Sangeeta Bardhan Cook, Ph.D., City of Hope director of the Office of Technology Licensing. We are impressed with the ability of their executive team to push and bring therapies to market expeditiously. At City of Hope, our mission is to transform the future of health care. We believe Chimeric has the vision to offer innovative therapies to cancer patients.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nations Best Hospitals in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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City of Hope Enters Licensing Agreement With Chimeric to Develop Its Pioneering Chlorotoxin CAR T Cell Therapy - Business Wire

NEW YORK, Sept. 23, 2020 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (the "Company" or "Actinium") today announced that it has successfully completed the first dosing cohort in the Actimab-A and venetoclax combination, multi-center Phase 1 trial for patients with Relapsed or Refractory ("R/R") Acute Myeloid Leukemia (AML) age 18 and above. All patients from the first dosing cohort (0.50 uCi/kg of Actimab-A) completed treatment and cleared their initial safety evaluation, thus allowing the study to proceed to the second dose cohort of 1.0 uCi/kg Actimab-A added to venetoclax. In a poster presentation at the American Association of Cancer Research (AACR) Annual Meeting 2019, Actimab-A was shown to be synergistic with venetoclax in venetoclax resistant cell lines, by depleting MCL-1, a protein shown to mediate resistance to venetoclax. The ongoing Phase 1 study was planned to replicate this synergy in a clinical setting. Actinium plans to report study proof of concept results in 2021.

Venetoclax is a B-Cell Lymphoma 2 (BCL-2) inhibitor jointly developed and marketed by AbbVie and Genentech that is approved in combination with hypomethylating agents ("HMAs") for patients with AML. The use of venetoclax has become widespread in the treatment of fit and unfit patients with R/R AML following its inclusion in the recently expanded National Comprehensive Cancer Network ("NCCN") guidelines. Actinium's preclinical research has demonstrated that by adding Actimab-A to venetoclax, the targeted internalized radiation from Actimab-A can deliver potent AML cell killing, as well as effectively deplete MCL-1 levels. The overexpression of MCL-1, a member of the BCL-2 family which venetoclax does not inhibit, promotes resistance to venetoclax. Thus, Actimab-A reverses resistance to venetoclax and has independent anti-leukemic activity mediated by CD33 as well.

"We are pleased to confirm that the second combination trial in our CD33 program is advancing through the dose escalation study as planned. Despite approval in multiple blood cancers, including AML, most AML patients are not cured with venetoclax regimens and eventually relapse. Based on the preclinical data, synergy with venetoclax and Actimab-A should lead to higher remission rates in R/R AML," said Dr. Mark Berger, Actinium's Chief Medical Officer. "We continue to generate promising data from our broader combination program. For example, the Actimab-A combination trial with chemotherapy agent CLAG-M increased the complete response rate compared to CLAG-M alone in R/R AML patients by 60%. We expect to complete the proof of concept Actimab-A venetoclax combination trial in 2021."

This Phase 1 study is a multicenter, open label trial of Actimab-A added to venetoclax for patients with CD33 positive R/R AML. The study will continue to enroll patients that have been previously treated with venetoclax as well as venetoclax nave patients. Gary Schiller, MD, Professor, Hematology-Oncology and Director, Hematologic Malignancy/Stem Cell Transplant Program at the UCLA Medical Center is the Principal Investigator for this study. The trial is also active at the University of Louisville.

Sandesh Seth, Actinium's Chairman and Chief Executive Officer, said, "We continue to advance the CD33 program for fit and unfit R/R AML patients as there is still a significant unmet need despite multiple recently approved agents. These therapeutic agents are not curative and patients continue to experience low response rates and/or high relapse rates. Our CD33 program, which also includes the Actimab-A CLAG-M combination trial, is anchored in leveraging mechanistic synergies of Actimab-A with approved or novel therapeutic agents in order to improve patient outcomes. We look forward to multiple clinical trial updates by year-end from our three ongoing trials in R/R AML, including our Iomab-B SIERRA Phase 3 pivotal trial."

Rationale for Actimab-A Venetoclax Combination Trial

This Phase 1/2 trial is a multicenter, open label trial of Actimab-A (lintuzumab-Ac225) added to venetoclax for patients with CD33 positive relapsed/refractory (R/R) Acute Myeloid Leukemia. The Phase 1 portion of the study is designed to determine the maximum tolerated dose (MTD) of Actimab-A added to venetoclax for R/R AML. The Phase 2 portion of the trial will assess the percentage of patients with CR, CRh, or Overall Response (CR + CRh), up to six months after the start of the treatment without receiving other AML therapies. The trial will enroll R/R AML patients who have been treated with venetoclax as well as venetoclax-nave patients. At the 1.0 uCi/kg dose, Actimab-A is administered on Day 1 of each cycle for four cycles and venetoclax is taken on Days 1-21 of each cycle for up to 4 cycles. Each cycle is 28 days, with a potential to expand to 42 days to allow for full hematologic recovery. Gary Schiller, MD, Professor, Hematology-Oncology and Director, Hematologic Malignancy/Stem Cell Transplant Program at the UCLA Medical Center is the Principal Investigator for this study.

More information on the clinical trial design is available at clinicaltrials.gov (NCT03867682).

About Actinium's CD33 Program (Actimab-A)

Antibody Radiation Conjugate (ARC) Actimab-A targets the CD33 antigen that is expressed on virtually all AML cells with the antibody lintuzumab which delivers potent alpha radiation via its Actinium-225 radioisotope payload. Blood cancers like AML are highly sensitive to radiation but cannot treated with the current standard of external beam delivery because the disease is too widespread throughout the body. The combination of targeted radiation with Actimab-A potentially allows for greater cancer cell death than a standalone chemotherapy regimen such as CLAG-M or venetoclax, which are frequently used in the treatment of fit and unfit patients with relapsed or refractory AML per National Comprehensive Cancer Network (NCCN) guidelines. Prior clinical results in over 100 patients treated with Actimab-A, including a Phase 1/2 trial of 58 patients, demonstrated a safety profile with minimal non-hematologic toxicities and an unmatched ability to deliver attenuated doses of radiation internally to CD33 expressing cancer cells. In the Phase 1/2 trial, Actimab-A as a single agent produced a 69% remission rate (CR, CRi, CRp) at high doses in patients with newly diagnosed AML but Actinium elected to pursue low dose combination trials for therapeutic development based on observed myelosuppression. In the Actimab-A CLAG-M Phase 1 combination trial, the second cohort with CLAG-M plus the 0.50 uCi/kg dose showed that 86% (6/7) of patients achieved complete remission (CR/CRi) after receiving the 0.50 uCi/kg dose of Actimab-A. This is a nearly 60% increase over the remission rate reported in a trial of seventy-four patients with relapsed or refractory AML who received CLAG-M alone. The company expects trial results, including the third dose cohort, in 2020. The Actimab-A Venetoclax Phase 1 trial continues to enroll patients in a maximum tolerated dose and expects to announce proof-of-concept results in 2021.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Clayton RobertsonActinium Pharmaceuticals, Inc.crobertson@actiniumpharma.com

Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

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SOURCE Actinium Pharmaceuticals, Inc.

Company Codes: AMEX:ATNM

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Actinium Pharmaceuticals Successfully Completes First Dosing Cohort in the Phase 1 Study of Actimab-A and Venetoclax Combination Therapy in...