Orchard Eyes 2020 for Filing BLA for ADA-SCOD Gene Therapy

Shares of Orchard Therapeutics are climbing this morning after the company announced positive results from a two-year follow up of the company’s stem cell gene therapy for the treatment of severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). This morning the London-based company announced the follow-up data from its registrational trial of OTL-101, an ex vivo, autologous, gene therapy candidate. The trial hit its primary endpoints, Orchard said. The company said the data in the 20 patient study showed 100 percent overall survival and 100 percent event-free survival in comparison to a historical standard of care from a historical control group. These durable results continue to be seen with some of these patients who are approaching five years of follow up. Additionally, the company said overall survival was 12 percent better than hematopoietic stem cell transplantation (HSCT), while event-free survival was 44 percent better than (HSCT). As a result, Orchard is preparing to file for a Biologics License Application in 2020.