Cell therapy weekly: US FDA priority review for Biologics License Application for sickle cell disease gene therapy
This week: the US FDA has accepted a Biologics License Application for a sickle cell disease gene therapy for priority review; a landmark study detailing the first successful clinical trial of an RNA cell therapy was published; and Gamida Cell (MA, USA) announced it will host an Investor Day on Thursday 29 June 2023 at 08:00 [EDT].
The news highlights:
FDA priority review for Biologics License Application for sickle cell disease gene therapy
The US FDA has accepted a Biologics License Application for priority review of bluebird bio’s (MA, USA) lovotibeglogene autotemcel (lovo-cel). Lovo-cel is an investigational gene therapy for the treatment of sickle cell disease (SCD) in patients aged 12 and older who have a history of vaso-occlusive events. It is designed to deliver functional copies of a modified form of the β-globin gene to patients’ hematopoietic stem cells to enable the production of anti-sickling hemoglobin.
“The burden that people living with SCD and their families live with today is staggering. Beyond extreme pain crises that send patients to the hospital, SCD progression is associated with grave long-term consequences,” stated Andrew Obenshain, CEO of bluebird bio. “The FDA’s acceptance of our BLA for lovo-cel moves us one step closer in bringing a potentially transformative therapy to the [SCD] community that is long overdue, and we are grateful to the patients, caregivers, researchers, clinicians, and community leaders who have enabled this exciting milestone. We look forward to working with the agency on its review.”
First successful clinical trial of RNA cell therapy for autoimmunity
A landmark paper has been published describing the administration of a first-in-class RNA CAR-T (rCAR-T) therapy, Cartesian Therapeutics’ (MD, USA) Descartes-08, to patients with generalized myasthenia gravis (MG). There is currently no cure for MG and, as it is an autoimmune disorder, treatment requires chronic immunosuppression, which has numerous risks and side effects. The clinical improvements demonstrated in the study were marked and long-lasting across four MG disease scoring systems.
“Currently, the mainstay of [MG] is chronic use of broad immunosuppression, which has many drawbacks,” said senior author of the paper James Howard, Professor of Neurology at the University of North Carolina at Chapel Hill (NC, USA). “The prospect of inducing potent, durable responses and reducing or eliminating use of immunosuppressive therapy is very appealing to the myasthenia community. Based on the compelling results of this paper, we are now enrolling patients with MG into a randomized placebo-controlled study, the first study of its kind for an engineered adoptive cell therapy.”
Gamida Cell will host an Investor Day
Gamida Cell, a cell therapy company harnessing NAM-enabled technology to create powerful therapeutics, will host an Investor Day featuring presentations by two stem cell transplant thought leaders. Steven M. Devine, Chief Medical Officer at the National Marrow Donor Program®/Be The Match® (MN, USA), will discuss the current unmet needs in stem cell transplant. Usama Gergis, Director of Stem Cell Transplant and Immune Cellular Therapy, Department of Medical Oncology, Sidney Kimmel Cancer Center, Thomas Jefferson University in Philadelphia (PA, USA), will discuss the clinical landscape for patients requiring stem cell transplants.
The Investor Day will take place on Thursday 29 June 2023 at 08:00 [EDT] in New York City and will also be webcast live. Presentations by the Gamida executive team regarding the company’s recently FDA-approved allogeneic cell therapy, Omisirge (omidubicel-onlv), will also feature on the program.