Cell and Gene Therapy
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As the biotech implements a more focused strategy for its Roctavian hemophilia A gene therapy, BioMarin has recruited two seasoned pharma executives to bolster its C-suite.
With the help of third-party investors, the new venture will focus on three genetic and rare diseases: tuberous sclerosis complex, erythropoietic protoporphyria and alpha-A1 antitrypsin deficiency.
The recent invalidation of an AAV gene therapy patent overlooks the complexity of innovation in biotechnology and could put a broad swath of intellectual property at risk.
Regeneron, Akouos and Mass Eye and Ear are testing therapies that can reverse genetic protein deficiency to restore hearing, with promising early results.
Likely to miss its initiation target, bluebird bio has renegotiated the loan deals of its agreement with Hercules Capital, giving it until June 30 next year—at the latest.
Eli Lilly’s new research and development facility in Boston’s Seaport district will focus on DNA- and RNA-based therapies, as well as other priority areas such as diabetes and obesity.
Patients in the U.K. with transfusion-dependent beta-thalassemia will have access to Vertex Pharmaceuticals’ gene editing therapy Casgevy, thanks to an agreement with the National Health Service on the price.
Experts say the time is now to develop and provide widespread access to genetic medicines for the rarest diseases. What’s more, they say it is a moral imperative.
BioMarin Pharmaceuticals on Monday said it is restricting sales of its hemophilia A gene therapy to three countries in an effort to reduce costs and help the treatment become profitable by 2025.
Longeveron and Lexeo Therapeutics are working on CGT therapies to treat Alzheimer’s disease, but it’s not clear whether they have a better chance of success than traditional approaches.