Search results for " gene therapy"

Article Design Considerations for a Commercial Cell and Gene Therapy Facility
The workflows of a cell therapy or a gene therapy differ from each other and from those of therapeutic antibodies, so the design and layout of the manufacturing facility must take these workflows into…

Article Manufacturing Standards Key to Advancing Cellular and Gene Therapies
Such advances, though, require further clarification of regulatory policies governing gene therapy production and quality assurance. FDA is considering at least three CAR-T applications, and mo…

Article FDA Seeks to Enhance Manufacturing of Cell and Gene Therapies
CBER is receiving dozens of applications to test new gene therapies, Marks reported at the Cell and Gene Therapy symposium in July 2018, sponsored by CASSS. He acknowledged that the logistics of manuf…

Article Bioterminology: A Guide to the Biopharmaceutical Lexicon
(See PAS)  CBER Center for Biologics Evaluation and Research at the FDA; CBER regulates vaccines, gene therapy, cellular products, allergenic extracts, antitoxins, antivenins, venoms, and blood an…

Article Emerging Therapies Test Existing Bioanalytical Methods
Questions include the following: · How much of a dosed gene therapy is present in circulation, in the target tissue, and how long does it stay there? · Is the target protein expression changed…

Article FDA Framework Spurs Advanced Therapies
Three new products were approved in 2017 by FDA, chimeric antigen receptor (CAR) T-cell cancer therapies from Novartis and Kite Pharma and a retinal gene therapy from Spark Therapeutics. Markets and M…

Article Patenting Prospects for Cell-Based Therapies
Cell-based therapies are moving medical treatments forward, but intellectual property uncertainties may delay progress. By Kevin Noonan Treatment using cell-based therapies is a rapidl…

Article Scaling Up Novel Therapies
Although these treatments are in early stages of development, gene therapy has already become a reality, with companies such as Spark Therapeutics. The company received “breakthrough therapy” status f…

Article Science Focus Fuels Successful Process Development for Startups
…prietary systems can provide significant advantages, particularly in nascent fields such as cell and gene therapy,” he remarks. “The systems we have implemented have tremendous advantages over commer…

Article Biopharma Seeks Balance
Developing sufficient quantities of a gene therapy for thousands of patients in a clinical trial would be cost-prohibitive, said Marks. Manufacturing processes need to be more productive for biopharma…

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