Exa-Cel Therapy'Transformative' in SCD, Beta-Thalassemia Exa-Cel Therapy'Transformative' in SCD, Beta-Thalassemia
The investigational gene therapy exagamglogene autotemcel shows encouraging efficacy, safety, and patient-reported outcomes for patients with transfusion-dependent beta-thalassemia and sickle cell disease.MDedge News (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - July 12, 2023 Category: Cancer & Oncology Tags: Hematology-Oncology News Source Type: news
Dr Reddy's to enhance presence in emerging segments like nutraceuticals, gene therapy
Dr Reddy's Laboratories plans to expand its presence in nutraceuticals, cell and gene therapy and new chemical entities, as well as exploring integrated digital healthcare services, digital therapeutics and disease management, according to its annual report. The pharmaceuticals firm also stated that increases in access to medicines was a driving philosophy, with efficient and effective operations, processes and practices being key to future productivity. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - July 11, 2023 Category: Pharmaceuticals Source Type: news
The Future of Gene Therapy for a Rare Pediatric Autoimmune Disease
By editing a mutated immune regulatory gene in patient cells, Rosa Bacchetta brings hope to those suffering from IPEX syndrome. (Source: The Scientist)
Source: The Scientist - July 10, 2023 Category: Science Tags: News News & Opinion Source Type: news
Bayer extends partnership with Peking University to foster pharmaceutical innovation in China
Bayer and Peking University (PKU) will collaborate to foster the translation of basic pharmaceutical research into drug discovery and development, while accelerating scientific research on cutting-edge technologies across the pharmaceutical value chain.
The collaboration will focus on selected key areas of interests, such as oncology, cardiorenal, immunology, as well as cell and gene therapy. (Source: World Pharma News)
Source: World Pharma News - July 10, 2023 Category: Pharmaceuticals Tags: Featured Bayer Business and Industry Source Type: news
Cheaper, more accessible cell, gene therapies? Nobel winner's task force points the way.
As more gene and cell therapies work their way through clinical trials — and some, like BioMarin's hemophilia A gene therapy costing millions of dollars — now is the time to look at new ways to develop, manufacturing and pay for those treatments, a task force says. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - July 7, 2023 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news
First Gene Therapy for Deadly Form of Muscular Dystrophy Gets FDA Approval for Young Kids
The FDA OK'd the treatment only for children ages 4 and 5, based on study results showing the therapy helped produce a protein needed for muscle growth, which is missing in boys with the condition. (Source: Arkansas Business - Health Care)
Source: Arkansas Business - Health Care - July 6, 2023 Category: American Health Source Type: news
Cell And Gene Therapies Face Persistent Manufacturing Capacity Constraints
The increasing numbers of cell and gene therapy pipeline candidates, along with a steady growth of new approvals, are stretching manufacturing capacity to its limits. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - July 6, 2023 Category: Pharmaceuticals Authors: Joshua Cohen, Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
Skyline Therapeutics Receives FDA Clearance of IND for SKG0106, a Novel Intravitreally Delivered AAV Gene Therapy Candidate for Neovascular Age-related Macular Degeneration
SHANGHAI and BOSTON, July 3, 2023 -- (Healthcare Sales & Marketing Network) -- Skyline Therapeutics, an innovation-driven gene therapy company committed to developing unique and novel solutions for rare and severe diseases, today announced that the U.S. F... Regenerative Medicine, Ophthalmology, FDA Skyline Therapeutics, macular degeneration, gene therapy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 3, 2023 Category: Pharmaceuticals Source Type: news
BioMarin ’s Next Roctavian Challenge Is How To Successfully Commercialize Its Gene Therapy: Warranties May Be An Option
As BioMarin turns to commercialization of Roctavian, we will likely hear more about how warranties could help gene therapies overcome payer reimbursement hurdles. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - July 1, 2023 Category: Pharmaceuticals Authors: Joshua Cohen, Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
What is gene therapy, and how does it work?
The Food and Drug Administration on Thursday approved a treatment that uses gene therapy to treat severe hemophilia A, a rare and sometimes fatal blood disorder. The new drug, Roctavian, could save people with the severe form of the disease from a lifetime of frequent injections. The drug’s maker…#roctavian #hemophilia #fda #hemgenix (Source: Reuters: Health)
Source: Reuters: Health - July 1, 2023 Category: Consumer Health News Source Type: news
The FDA just approved another gene therapy. Here’s what to know about them.
Gene therapy could treat a wide variety of medical problems. It's expensive, though, and not every use is approved. Here's what to know. (Source: Washington Post: To Your Health)
Source: Washington Post: To Your Health - July 1, 2023 Category: Consumer Health News Authors: Shera Avi-Yonah Source Type: news
FDA Approves First Gene Therapy for Severe Hemophilia A, Roctavian
FRIDAY, June 30, 2023 -- The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A. The treatment, Roctavian (valoctocogene roxaparvovec), will cost $2.9 million for a single... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - June 30, 2023 Category: General Medicine Source Type: news
Lab Notes: FDA to review Pfizer's application for gene therapy licensed from Spark Therapeutics
This week's Lab Notes has updates on a Spark-developed gene therapy candidate licensed to Pfizer, a laboratory equipment supplier's new program to benefit the environment, some funding news for two local life sciences companies and more. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 30, 2023 Category: Biotechnology Authors: John George Source Type: news
FDA Approves First Gene Therapy for Severe Hemophilia A
FRIDAY, June 30, 2023 -- The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder.
The treatment is not cheap: Roctavian will... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - June 30, 2023 Category: General Medicine Source Type: news
FDA Approves First Gene Therapy for Hemophilia A FDA Approves First Gene Therapy for Hemophilia A
Most patients benefit from valoctocogene roxaparvovec for at least 2 years, but it ' s not known how long the effect lasts.Medscape Medical News (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - June 30, 2023 Category: Cancer & Oncology Tags: Hematology-Oncology News Alert Source Type: news
