Biopharma Sarepta Therapeutics and Brammer Bio enter into a long-term strategic partnership to design and develop new manufacturing facilities at CDMO’s plant in Massachusetts. The new partnership is for the development of the production of its microdystrophin, Duchenne Muscular Dystrophy (DMD) therapy and future gene therapies.
A second patient has been treated in a historic gene editing study in California, and no major side effects or safety issues have emerged from the first man’s treatment nearly three months ago, doctors revealed Tuesday.
Spark Therapeutics (ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced it has entered into a licensing agreement with Novartis Pharmaceuticals to develop and commercialize investigational voretigene neparvovec outside the U.S.
Gene therapies are an undeniably exciting new frontier for medicine. From Kymriah's genetically modified, cancer-hunting immune cells, to Luxturna's vision-restoring healthy gene replacement, these therapies are fundamentally unlike 20th-century medicines which generally require ongoing drug treatment. Gene therapies can be one-off doses that in effect, cure a specific disease.
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