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. 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.

Andrea Spezzi, MBBS, FFPM, Chief Medical Officer, Orchard

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

Related Content

We make UCL’s research physical and life sciences ideas happen.