Orchard Therapeutics Presents Positive Results from Gene Therapy Trial for Beta Thalassemia

May 1, 2019 – This past Monday at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Washington, D.C., Orchard Therapeutics presented positive results from its gene therapy trial for the treatment of beta thalassemia.

During this study, nine transfusion-dependent beta thalassemia patents received OTL-300 gene therapy treatment, and all nine treated patients met the safety endpoint of survival over follow-up periods ranging from 16-43 months with no reports of treatment-related adverse events.

Dr. Giuliana Ferrari, head of the gene transfer into stem cell unit at San Raffaele Scientific Institute and San Raffaele-Telethon Institute for Gene Therapy, explained, “Patients with transfusion-dependent beta-thalassemia are not able to make enough hemoglobin to survive due to a mutation in the beta-globin gene which causes ineffective red blood cell production. Beta-thalassemia patients, especially those with severe phenotypes such as the β0/β0 genotype, rely on frequent blood transfusions and iron chelation therapy to survive. They may be treated with a bone marrow transplant, however this carries significant risk and can lead to early mortality.”

Dr. Ferrari continued by stating that the results shared during Orchard Therapeutics’ presentation at the ASGCT meeting “demonstrate that HSC gene therapy with OTL-300 has the potential to provide clinical benefit and a new, life-changing treatment for patients burdened with this devastating condition.”

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