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