Form Type:  SC 13G/A
Filing Date:  2/14/2020 
CIK:  0001609351 
Address:  3737 MARKET STREET
SUITE 1300
City, State, Zip:  PHILADELPHIA, Pennsylvania 19104 
Telephone:  888-772-7560 
Fiscal Year:  12/31 
Description of Business
We are a leader in the field of gene therapy, seeking to transform the lives of patients suffering from debilitating genetic diseases by developing potentially one-time, life-altering treatments. The goal of gene therapy is to overcome the effects of a malfunctioning, disease-causing gene. Gene therapies have the potential to provide long-lasting effects, dramatically and positively changing the lives of patients with conditions where no, or only palliative, therapies exist. We have built a pipeline of gene therapy product candidates that are directed to the retina, the liver and the central nervous system, or CNS. In December 2017, the U.S. Food and Drug Administration, or FDA, approved LUXTURNATM (voretigene neparvovec-rzyl) for the treatment of patients with viable retinal cells and confirmed biallelic RPE65 mutation-associated retinal dystrophy, a genetic blinding condition caused by mutations in the RPE65 gene.
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