Orchard Therapeutics

Data Supports Achievement of Proof-of-Concept Milestone for OTL-300

Orchard Therapeutics has announced the achievement of clinical proof-of-concept based on data from the trial of OTL-300, for the treatment of transfusion-dependent beta-thalassemia (TDT).

The primary endpoint of transfusion reduction was achieved after one year of follow-up in eight out of nine TDT patients treated with OTL-300. All nine patients met the safety endpoints with no adverse events related to the therapy.

"We are pleased OTL-300 has shown clinical benefit in severe transfusion-dependent beta-thalassemia and has the potential to be a part of a new wave of treatment options for these patients,” said Andrea Spezzi, MBBS, FFPM, chief medical officer at Orchard. “We believe these data support proof-of-concept and the further development of this therapeutic approach. This marks the fifth program from Orchard’s dedicated gene therapy portfolio to achieve clinical proof-of-concept and further supports our belief that a single administration of gene-modified autologous hematopoietic stem cells can lead to durable engraftment and potential disease correction.”

You can read the full press release on the Orchard Therapeutics website.