MedicalNews

Positive results from ALS safety trial

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.”

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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.”

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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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