Orchard Therapeutics achieves 100% survival rate during trial treatment for ADA-SCID
- On February 9, 2019
- By GrowthInvest Admin
Orchard Therapeutics, a commercial-stage biopharmaceutical company, presented their two-year follow-up data in 20 patients from the registrational trial evaluating OTL-101, aimed at treating those with adenosine deaminase deficiency (ADA-SCID). They achieved a 100% Overall Survival and 100% Event Free Survival in Patients Treated with OTL-101; compared to 88% OS and 56% EvFS with Historical HSCT Overall at 24 months
ADA-SCID is a rare, life-threatening, inherited disease caused by mutations in the ADA gene. Patients with ADA-SCID are unable to fight off and frequently succumb to complications from bacterial, viral and fungal infections.
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