Cell therapy weekly: Gene therapy manufacturing collaboration between Charles River and Cure AP-4
This week: Gene therapy manufacturing collaboration between Charles River and Cure AP-4, positive results from amyotrophic lateral sclerosis (ALS) safety trial for combined stem cell and gene therapy and European marketing authorization granted to Tecartus® CAR-T therapy.
The news highlights:
Gene therapy manufacturing collaboration between Charles River and Cure AP-4
Charles River Laboratories (MA, USA) and Cure AP-4 (MA, USA), a non-profit foundation focussed on raising funds and awareness about Adapter-Protein 4 Hereditary Spastic Paraplegia, have entered into a manufacturing collaboration. The collaboration will leverage Charles River’s expertise to manufacture high quality plasmid DNA to be used in Cure AP-4’s Phase I/II gene therapy trials against Adapter-Protein 4 Hereditary Spastic Paraplegia, which currently has no known treatments or cures.
Birgit Girshick, Corporate Vice President and Chief Operating Officer of Charles River stated, “The opportunity to work with a group as driven as Cure AP-4 is exactly why we do the work we do. Playing a small but critical role in delivering a potentially transformative therapy to a severely underserved patient population gives us an incredible sense of pride, and we are excited for the Cure AP-4 team to achieve this next stage.”
Positive results from ALS safety trial for combined stem cell and gene therapy
Positive results have been reported from a Phase I/IIa trial by Cedars-Sinai (CA, USA), investigating the safety of delivering genetically modified neural progenitor cells to the motor cortex for the treatment of ALS. The therapeutic CNS10-NPC-GDNF cells are genetically modified to produce glial cell line-derived neurotrophic factor and will compensate for the decreased function of diseased glial cells in ALS.
Clive Svendsen, the executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, stated: “We were able to show that the engineered stem cell product can be safely transplanted in the human spinal cord. And after a one-time treatment, these cells can survive and produce an important protein for over three years that is known to protect motor neurons that die in ALS.”
European marketing authorization granted to Tecartus® CAR-T therapy
Kite Pharma (CA, USA) announced that the European Commission (Brussels, Belgium) has approved their CAR-T cell therapy, Tecartus®, for the treatment of adult patients aged 26 years and older with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. This approval is supported by data from the ongoing Zuma-3 trial, which demonstrated a median overall survival rate of more than 2 years and overall complete remission in 71% of patients.
“This approval makes Tecartus the first and only CAR T-cell therapy indicated for this population of patients, addressing a significant unmet medical need,” said CEO of Kite, Christi Shaw. “This is also the fourth indication in Europe for which a Kite cell therapy is approved, clearly demonstrating the benefits they offer to patients, especially those with limited treatment options.”
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