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Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy

Groundbreaking? Game-changing? Transformational? Historic? None of the buzzwords sound adequate to describe Friday’s FDA approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exa-cel) to treat sickle cell disease (SCD). The therapy is a long-awaited potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black. It’s also the first medicine using the revolutionary CRISPR gene-editing system, which earned its inventors a Nobel Prize in 2020 and holds tantalizing potential to cure diseases for which there is no treatment. Friday, in surprising timing, the FDA approved a second gene therapy for SCD, bluebird bio’s Lyfgenia (lovo-cel), which was due for a decision Dec. 20. Both approvals are for people 12 and older. The FDA has signed off on roughly two dozen cell or gene therapies, beginning with Novartis’ Kymriah in 2017, but none have the potential impact of Casgevy and Lyfgenia. ...
- Source: drugdu
- 112
- December 12, 2023
China Biopharm’s TQA3038 “siRNA” Phase I Clinic Completes Dosing in First Batch of Subjects

On December 5, China Biopharm announced that it is conducting a randomized, double-blind, placebo-controlled Phase I clinical trial to evaluate the safety, tolerability and pharmacokinetic profile of TQA3038, a small interfering RNA (siRNA) targeting the hepatitis B virus (HBV) developed independently by China Biopharm for the indication of chronic hepatitis B (CHB). TQA3038 is a small interfering RNA (siRNA) drug developed independently by China Biopharmaceuticals targeting HBV, with the indication of chronic hepatitis B. The first human clinical trial of TQA3038 has been completed with the enrollment of the first batch of subjects, and the dosing process went smoothly, and post-dosing observation has been completed. Chronic hepatitis B is a serious public health problem worldwide, with approximately 290 million chronically infected patients worldwide. China is a high prevalence area of hepatitis B. It is estimated that there are about 86 million chronic HBV-infected patients, and a large number of patients ...
- Source: drugdu
- 105
- December 12, 2023
Gates Foundation Delegation Visits Zhifei Biological Products Co., Ltd.

On December 1, a delegation of global health experts from the Bill & Melinda Gates Foundation (Gates Foundation) visited Beijing Zhifei Biological Products Co., Ltd., a wholly-owned subsidiary of Zhifei Biologicals, and visited the production workshop and related laboratories, and had an in-depth exchange of views on innovative topics, such as key conjugate vaccine projects. Mr. Du Lin, Executive Director of Beijing Zhifei Biological Products Co., Ltd., and others warmly received the delegation and attended the seminar. The visiting Gates Foundation delegation included clinical experts, vaccinologists and immunologists, and senior strategists. During the meeting, the Zhifei Biologicals team explained the clinical trials of key conjugate vaccine products, domestic and overseas sites and vaccine marketization, etc. The delegation introduced the incidence of relevant diseases and flow-regulation data, and made suggestions on planned clinical studies, etc. The Gates Foundation delegation fully recognized Beijing Zhifei Biological Products Co., Ltd.’s R&D strength and industrialization ...
- Source: drugdu
- 92
- December 12, 2023
Vaccinations still vital to curb illness spread

Known pathogens behind current spate of respiratory infections in parts of nation The recent surge of respiratory illnesses in China is fueled by known pathogens, and vaccination efforts are still vital to stem the spread, medical experts said on Saturday. Mi Feng, National Health Commission spokesman, said the clusters were all caused by recognized pathogens, and medical workers are dealing with them by using mature treatment plans. “Diseases induced by unknown viruses and bacteria have not been detected yet,” he told a news conference in Beijing. Mi said vaccinations must be stepped up for vulnerable groups such as older adults and children to lower the risk of infection. He said their work priorities would be to increase the supply of hospital beds, streamline the outpatient reception process and bolster the supply of online medical services to avoid large gatherings in hospitals. The spokesman urged the public to take personal precautions ...
- Source: drugdu
- 96
- December 12, 2023
Dizal’s Golidocitinib Pivotal Trial Demonstrates Superior and Durable Clinical Benefits for Patients with r/r PTCL in Oral Presentation at 2023 ASH

Shanghai, December 10, 2023 – Dizal today presented the full analysis of the multinational pivotal study of golidocitinib for r/r PTCL (JACKPOT8 PART B) in an oral presentation at the 65th American Society of Hematology Annual Meeting and Exposition (2023 ASH, San Diego). The results were simultaneously published in the prestigious peer-reviewed journal The Lancet Oncology (Impact Factor: 54.4). This follows the publication of the Phase I clinical data of golidocitinib for the treatment of r/r PTCL (JACKPOT8 PART A) in Annals of Oncology (Impact Factor: 51.8) three months ago. Golidocitinib is currently the first and only Janus kinase 1 (JAK1) selective inhibitor being evaluated for the treatment of r/r PTCL. A total of 104 patients with r/r PTCL were enrolled in the JACKPOT8 PART B study to evaluate the efficacy and safety of golidocitinib as a monotherapy. At the cut-off date of August 31, 2023, per independent review committee ...
- Source: drugdu
- 95
- December 12, 2023
FDA Approves Two Landmark Cell-Based Gene Therapies for Sickle Cell Disease

Pharmaceutical Executive Editorial Staff FDA approval of bluebird bio’s Lyfgenia and Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy marks significant milestone in the treatment of sickle cell disease. The FDA has approved a pair of landmark treatments for sickle cell disease (SCD) in patients 12 years of age and older, marking the first cell-based gene therapies for SCD. As part of today’s regulatory action, both bluebird bio’s Lyfgenia (lovotibeglogene autotemcel [lovo-cel]) and Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]) gained approval for the inherited blood disorders, which affect approximately 100,000 people in the United States. “Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” Nicole Verdun, MD, director of the Office of Therapeutic Products within the ...
- Source: drugdu
- 95
- December 12, 2023
On Heels of ASH Data, Pfizer Eyes FDA Submission for New Hemophilia Drug

Pfizer already reported its experimental drug for hemophilia A and B met a pivotal study’s goals of reducing bleeding episodes. During the annual meeting of the American Society of Hematology, the company presented a full look at the results, which are expected to support regulatory submissions soon. By FRANK VINLUAN Patients with hemophilia A or B can manage the inherited bleeding disorders with regular infusions of the clotting proteins they lack. This treatment helps—until it doesn’t. One problem with these infused factor replacement therapies is patients can develop inhibitors, which are antibodies that render the clotting proteins ineffective. Experimental Pfizer drug marstacimab takes a different approach to both forms of hemophilia. Results from a pivotal study show the subcutaneously injected drug met the main goals of reducing bleeding episodes. The company also has some additional long-term data showing continuing improvement beyond the initial clinical trial evaluation period. The full Phase ...
- Source: drugdu
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- December 12, 2023
BD receives 510(k) clearance for fingerstick blood test sample collection device

Dive Brief Becton Dickinson has received 510(k) clearance for a device that collects blood from a fingerstick instead of from the vein. The MiniDraw Capillary Blood Collection System container is slightly larger than its predicate device, the BD Microtainer MAP Microtube, but is cleared for sample collection in “ancillary healthcare facilities,” positioning BD and its partner Babson Diagnostics to support blood collection from community sites such as pharmacies. When Babson rolls out its BetterWay blood testing service next year, the device could support the collection of samples for lipid panel, selected chemistry tests, and hemoglobin and hematocrit analyses. Dive Insight BD began working with Babson to develop a capillary blood collection and testing system in 2019. The partners expanded their collaboration last year to support work on self-collection, mobile services and at-home collection. Devices that enable the collection of capillary, rather than venous, blood for tests of hemoglobin and hematocrit ...
- Source: drugdu
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- December 12, 2023
Discovery of Rogue Protein Paves Way for Diagnostic Test to Detect Early-Onset Dementia

Neurodegenerative disorders are often marked by the buildup of amyloid filament inclusions of specific proteins in the brain. These proteins are critical in diagnosing and treating the associated diseases. Frontotemporal lobar degeneration (FTLD) is one such disorder, leading to frontotemporal dementia, which ranks just behind Alzheimer’s disease in prevalence. In about 10% of FTLD cases, the identity of the filament-forming protein was previously unknown. The protein FUS was the suspected culprit, given its presence in brain inclusions and its known genetic role in some instances of amyotrophic lateral sclerosis, another neurodegenerative condition. Now, researchers using electron cryo-microscopy have unexpectedly found that the filaments are instead formed by the protein TAF15. The research team from MRC Laboratory of Molecular Biology (Cambridge, UK) extracted amyloid filaments from the brains of four individuals affected by this type of FTLD. They carried out neuropathological examinations on the brain tissues obtained. Employing electron cryo-microscopy, they ...
- Source: drugdu
- 91
- December 12, 2023
Edwards plans to spin off critical care business next year

BY SEAN WHOOLEY Edwards Lifesciences (NYSE: EW)+ announced today that it intends to spin off its critical care business by the end of 2024. The company shared a number of key plans going forward as it outlined its plans for future growth. Among its plans, Edwards expects to spin off the critical care unit and its full range of smart monitoring technologies next year. “During 2024, Edwards plans to support the growth and leadership of innovations in advanced patient monitoring, with the goal of improving the quality of care for millions of patients annually,” Edwards wrote in a news release. Analysts expect shares of Edwards to move up on the news today. Shares of EW rose 1% at $70.17 apiece in early-morning trading today. MassDevice’s MedTech 100 Index — which includes stocks of the world’s largest medical device companies — remained even. Edwards intends to complete the spin-off tax-free. The ...
- Source: drugdu
- 100
- December 11, 2023

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