18 months into the first serious clinical trials of CRISPR gene therapy for sickle cell disease and beta-thalassemiaand all patients are free from symptoms and have not needed blood transfusions.

Sickle cell disease (SCD) can cause a variety of health problems including episodes of severe pain, called vaso-occlusive crises, as well as organ damage and strokes.

Patients with transfusion-dependent thalassemia (TDT) are dependent on blood transfusions from early childhood.

The only available cure for both diseases is a bone marrow transplant from a closely related donor, an option that is not available for the vast majority of patients because of difficulty locating matched donors, the cost, and the risk of complications.

In the studies, the researchers goal is to functionally cure the blood disorders using CRISPR/Cas9 gene-editing by increasing the production of fetal hemoglobin, which produces normal, healthy red blood cells as opposed to the misshapen cells produced by faulty hemoglobin in the bodies of individuals with the disorders.

The clinical trials involve collecting stem cells from the patients. Researchers edit the stem cells using CRISPR-Cas9 and infuse the gene-modified cells into the patients. Patients remain in the hospital for approximately one month following the infusion.

Prior to receiving their modified cells, the seven patients with beta thalassemia required blood transfusions approximately every three to four weeks and the three patients with SCD suffered episodes of severe pain roughly every other month.

All the individuals with beta thalassemia have been transfusion independent since receiving the treatment, a period ranging between two and 18 months.

Similarly, none of the individuals with SCD have experienced vaso-occlusive crises since CTX001 infusion. All patients showed a substantial and sustained increase in the production of fetal hemoglobin.

15 months on, and the first patient to receive the treatment for SCD, Victoria Gray, has even been on a plane for the first time.

Before receiving CRISPR gene therapy, Gray worried that the altitude change would cause an excruciating pain attack while flying. Now she no longer worries about such things.

She told NPR of her trip to Washington, D.C: It was one of those things I was waiting to get a chance to do It was exciting. I had a window. And I got to look out the window and see the clouds and everything.

MORE: MIT Researchers Believe Theyve Developed a New Treatment for Easing the Passage of Kidney Stones

This December, theNew England Journal of Medicinepublishedthe first peer-reviewed research paperfrom the studyit focuses on Gray and the first TDT patient who was treated with an infusion of billions of edited cells into their body.

There is a great need to find new therapies for beta thalassemia and sickle cell disease, saidHaydar Frangoul, MD,Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center. What we have been able to do through this study is a tremendous achievement. By gene editing the patients own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases.

READ: For the First Time in the US, Surgeons Pump New Life into Dead Donor Heart for Life-Saving Transplant

Because of the precise way CRISPR-Cas9 gene editing works, Dr. Frangoul suggested the technique could potentially cure or ameliorate a variety of diseases that have genetic origins.

As GNN has reported, researchers are already using CRISPR to try and treat cancer, Parkinsons, heart disease, and HIV, as well.

Source: American Society of Hematology

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Every Patient Treated With CRISPR Gene Therapy for Blood Diseases Continues to Thrive, More Than a Year On - Good News Network

CAMBRIDGE, Mass., Dec. 18, 2020 (GLOBE NEWSWIRE) -- Vericel Corporation (NASDAQ:VCEL), a leader in advanced therapies for the sports medicine and severe burn care markets, today announced that Nick Colangelo, President and CEO, will present the latest company overview at the 39th Annual J.P. Morgan Healthcare Conference. The conference is being conducted in a virtual format and the presentation will take place on Thursday, January 14, 2021 at 11:40 a.m. Eastern Time.

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Vericel to Present at the 39th Annual J.P. Morgan Healthcare Conference on Thursday, January 14, 2021

AUSTIN, Texas, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, announced that the company will participate in the virtual H.C. Wainwright BioConnect 2021 Conference to be held January 11-14, 2021.

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Lumos Pharma to Participate in the H.C. Wainwright BioConnect 2021 Conference

NEW YORK, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer announced today that it has entered into a distribution agreement with Swixx BioPharma AG (“Swixx”) to be the exclusive distributor of the Company’s antibodies, DANYELZA® (naxitamab-gqgk) for the treatment of patients with relapsed/refractory high-risk neuroblastoma and omburtamab, if approved, for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma in Eastern Europe, including Russia. DANYELZA (naxitamab-gqgk) 40mg/10mL was approved by the U.S. Food and Drug Administration (“FDA”) on November 25, 2020 and is indicated, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. The Company plans to resubmit its (“BLA”) to the FDA for omburtamab by the end of 2020 or in early 2021.

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Y-mAbs Signs Distribution Agreement with Swixx for DANYELZA® (naxitamab-gqgk) and Omburtamab in Eastern Europe

TORONTO, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Unifor, Canada's largest private-sector union and Aleafia Health Inc. (TSX: AH, OTC: ALEAF), have entered a long term partnership that through collective bargaining will seek to provide access for its members to medical cannabis coverage as part of their benefit and insurance coverage.

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Unifor and Aleafia Health partner on groundbreaking medical cannabis coverage

NEW YORK, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer announced today that it has entered into a license agreement with SciClone Pharmaceuticals International Ltd (“SciClone”) to be the exclusive co-development and commercialization partner of the Company’s antibodies, DANYELZA® (naxitamab-gqgk) for the treatment of patients with relapsed/refractory high-risk neuroblastoma and omburtamab, if approved, for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma in China. DANYELZA (naxitamab-gqgk) 40mg/10mL was approved by the U.S. Food and Drug Administration (“FDA”) on November 25, 2020 and is indicated, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. The Company plans to resubmit its Biologics License Application (“BLA”) to the FDA for omburtamab by the end of 2020 or in early 2021.

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Y-mAbs Signs License Agreement with SciClone for DANYELZA® (naxitamab-gqgk) and Omburtamab in China

Acquisition Enhances Company’s Decentralized Solutions Offering by Strengthening Patient Focus Acquisition Enhances Company’s Decentralized Solutions Offering by Strengthening Patient Focus

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Syneos Health Acquires Illingworth Research Group, a Leading Provider of Clinical Research Home Health Services

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Immunicum AB (publ) Publishes Prospectus for Admission to Trading of Newly Issued Shares on Nasdaq Stockholm

CAMBRIDGE, Mass. and ROSTOCK, Germany and BERLIN, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG) (“CENTOGENE” or the “Company”), a commercial-stage company focused on rare diseases that transforms real-world clinical and genetic data into actionable information for patients, physicians and pharmaceutical companies, today announced the voting results of the Company’s Extraordinary General Meeting. The shareholders of the Company voted in favor of all proposals, which included the appointments of Dr. Andrin Oswald as Managing Director and Dr. Jonathan Sheldon as Supervisory Director, the reappointment of Mr. Richard Stoffelen as Managing Director, and adjustments to the compensation of the Supervisory Board.

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CENTOGENE Announces Voting Results of Extraordinary General Meeting

VANCOUVER, British Columbia, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Qu Biologics Inc., a private clinical stage biopharmaceutical company developing Site Specific Immunomodulators (SSIs), a novel platform of immunotherapies designed to restore innate immune function, is pleased to announce it is receiving advisory services and research and development funding from the National Research Council of Canada Industrial Research Assistance Program (NRC IRAP) to quickly move forward the potential application of Qu’s lung-directed SSI, QBKPN, to prevent and treat infection with SARS-CoV-2, the virus causing COVID-19. Unlike traditional vaccines that direct the host adaptive immune response to a very specific antigen, Qu’s immunotherapy platform engages and trains the innate arm of the immune system that is responsible for providing the body’s first line of defense. Optimal innate immune defenses can efficiently contain and clear infection before it spreads and plays an essential role in also directing the adaptive immune response against new infections. By training the innate immune system to protect the lungs, QBKPN is designed to protect the lungs not only against COVID-19, but also against a wide range of other bacterial and viral respiratory infections, like influenza. Importantly, since QBKPN activates innate immunity, the immune protection it provides would not lose efficacy if SARS-CoV-2 mutates, which is a risk with traditional vaccines that rely on antigen specificity.

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Qu Biologics Receives Funding for COVID-19 Prevention/Treatment Research with Qu’s Novel Immunotherapy Platform

The Company’s Proposed Pharma Grade Version of Tauri-Gum™ is Being Developed Specifically to Target: Patients Subjected to Ongoing Chemotherapy Treatment (the “Indication”)

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Tauriga Sciences Inc. Enters into Master Services Agreement with CSTI to Resume the Clinical Development of its Anti-Nausea Pharmaceutical Grade...

MENLO PARK, Calif., Dec. 18, 2020 (GLOBE NEWSWIRE) -- Pacific Biosciences of California, Inc. (NASDAQ: PACB) (“Pacific Biosciences” or the “Company”), a leading provider of high-quality sequencing of genomes, transcriptomes, and epigenomes, today announced that the Compensation Committee of the Company’s Board of Directors granted non-qualified stock options covering an aggregate of 100,000 shares of Pacific Biosciences common stock and restricted stock units (“RSU”) covering 50,000 shares of Pacific Biosciences common stock to a recently hired non-executive officer employee under the Pacific Biosciences 2020 Inducement Equity Incentive Plan on December 14, 2020 (the “Effective Date”).

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BASEL, Switzerland, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Myovant Sciences (NYSE: MYOV), a healthcare company focused on redefining care for women and for men, today announced that the U.S. Food and Drug Administration (FDA) has approved ORGOVYX™ (relugolix) for the treatment of adult patients with advanced prostate cancer. ORGOVYX, which was granted Priority Review by the FDA, is the first and only oral gonadotropin-releasing hormone (GnRH) receptor antagonist for men with advanced prostate cancer. The approval is based on efficacy and safety data from the Phase 3 HERO study of ORGOVYX in men with advanced prostate cancer. ORGOVYX is expected to be available in January 2021.

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Myovant Sciences Announces FDA Approval of ORGOVYX™ (relugolix), the First and Only Oral Gonadotropin-Releasing Hormone (GnRH) Receptor Antagonist...

Basel, December 18, 2020 — The U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the new drug application (NDA) for inclisiran, a potential treatment for hyperlipidemia in adults who have elevated low-density lipoprotein cholesterol (LDL-C) while being on a maximum tolerated dose of a statin therapy. The FDA stated that the agency cannot approve the NDA by the Prescription Drug User Fee Act (PDUFA) action date of December 23, 2020, due to unresolved facility inspection-related conditions. The conditions will be conveyed to the European manufacturing facility within 10 business days. The third-party facility is responsible for drug product manufacturing. Satisfactory resolution of the unresolved facility inspection-related conditions is required before the Novartis NDA may be approved. No onsite inspection was conducted. If it is determined that a facility inspection is needed to approve the application, the FDA will define an approach for scheduling once safe travel may resume based on public health need and other factors.

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Novartis receives complete response letter from U.S. FDA for inclisiran

SAN FRANCISCO, Dec. 18, 2020 (GLOBE NEWSWIRE) -- FibroGen, Inc. (Nasdaq: FGEN) today announced that the U.S. Food and Drug Administration (FDA) has extended the review period of the New Drug Application (NDA) for roxadustat for the treatment of anemia of chronic kidney disease (CKD) by three months. The updated Prescription Drug User Fee Act (PDUFA) action date is March 20, 2021.

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FibroGen Provides Regulatory Update on Roxadustat

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Hiya Vitamins Reviews - Best Vitamins Supplement for your Kids? - Product Review by Mike Vaughn

AUSTIN, TX and DURHAM, NC, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Shattuck Labs, Inc. (Shattuck) (NASDAQ: STTK), a clinical-stage biotechnology company pioneering the development of bi-functional fusion proteins as a new class of biologic medicine for the treatment of patients with cancer and autoimmune disease, today announced that it will be added to the Russell 2000® and 3000® Indexes effective December 21, 2020, following Russell’s quarterly additions of select initial public offerings.

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Shattuck Labs Added to Russell 2000® and 3000® Indexes

MALVERN, Pa., Dec. 18, 2020 (GLOBE NEWSWIRE) -- Recro (Nasdaq:REPH), a leading contract development and manufacturing organization (CDMO), with integrated solutions for formulation, analytical services, regulatory support, manufacturing and packaging of both commercial and development stage oral solid dose drug products, today announced that it granted an inducement award to its newly-appointed President and Chief Executive Officer, David Enloe.

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Recro Grants Inducement Award to New Chief Executive Officer

COLUMBUS, Ohio, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Green Growth Brands Inc. (CSE: GGB) (OTCQB: GGBXF) (“GGB” or the “Company”) and certain of its direct and indirect wholly owned subsidiaries (collectively, the “Applicants”) today provided an update on their insolvency proceedings under the Companies’ Creditors Arrangement Act (Canada) (“CCAA”).

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Green Growth Brands Provides CCAA and Other Updates

VANCOUVER, British Columbia, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Haltain Developments Corp. (“Haltain” or the “Company”) announces that its previously announced non-binding letter of intent with ScreenPro Security Ltd. has terminated and that the transactions contemplated thereby will not proceed. For more information on the letter of intent and the transactions contemplated thereby, please see the Company's news release dated June 25, 2020.

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Haltain Announces Termination of LOI with ScreenPro Security Ltd.