Pfizer, Novartis, Janssen and other biotechs sign onto NIH/FDA program to develop new gene therapies for rare diseases
Ten top drugmakers are joining forces with the NIH, the FDA and other nonprofits to streamline the development of new gene therapies for those with rare diseases.
Known as the Bespoke Gene Therapy Consortium, companies including Pfizer, Novartis, Janssen, Biogen, Takeda, Spark Therapeutics, Regenxbio, Thermo Fisher Scientific, and Ultragenyx are teaming up to better understand the basic biology of a common gene delivery vector, known as the adeno-associated virus. The consortium will seek to improve the efficiency of vector manufacturing and enhance the overall therapeutic benefit of AAV-based gene therapies.
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