GENE ONLINE|News &
10 Top Gene Therapy Companies in 2021
Three years ago, the FDA granted landmark approval to the first in vivo gene therapy for treating an inherited eye disease. Luxturna, an AAV2 based gene therapy developed by Spark Therapeutics, bagged regulatory win to treat this eye disease called Leber congenital amaurosis. Since then, the agency has approved Zolgensma, a spinal muscular atrophy treatment, and has given green light to dozens of small and big biotechs to start clinical trials for their gene therapies.
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