Spark Therapeutics (ONCE) Announces New Data for Lead Investigational Gene Therapy, FDA Amends Orphan Drug … – StreetInsider.com
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Spark Therapeutics (ONCE) Announces New Data for Lead Investigational Gene Therapy, FDA Amends Orphan Drug …
StreetInsider.com … Phase 1 clinical trial and natural history study findings that enhance the understanding of investigational voretigene neparvovec, a one-time adeno-associated viral (AAV) gene therapy for Inherited Retinal Disease (IRD) caused by biallelic RPE65 … |
