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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