Genetic Disorders

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VY-AADC01 Gene Therapy Demonstrates Safety, Improved Motor Function in Phase 1 Trial
VY-AADC01 Gene Therapy Demonstrates Safety, Improved Motor Function in Phase 1 Trial

October 22nd 2021

The trial provided Class IV evidence that VY-AADC01 and the associated surgical delivery procedure were well-tolerated in patients aged 40 to 70 years with moderately advanced Parkinson disease.

SMA Gene Therapy Cleared to Proceed Following FDA Clinical Hold
SMA Gene Therapy Cleared to Proceed Following FDA Clinical Hold

August 9th 2021

FDA Grants Orphan, Rare Pediatric Designations to Gene Therapies
FDA Grants Orphan, Rare Pediatric Designations to Gene Therapies

August 5th 2021

scAAV9 Gene Therapy Safe, Effective in Preclinical Study in Epileptic SLC13A5 Deficiency
scAAV9 Gene Therapy Safe, Effective in Preclinical Study in Epileptic SLC13A5 Deficiency

May 13th 2021

FDA Clears IND Application for Huntington Disease Therapy VY-HTT01
FDA Clears IND Application for Huntington Disease Therapy VY-HTT01

May 5th 2021

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