Japanese pharmaceutical company Astellas Pharma has partnered with Kate Therapeutics to develop a gene therapy for X-linked myotubular myopathy (XLMTM), a rare and life-threatening neuromuscular disease leading to severe muscle weakness. The partnership aims to reduce the likelihood of severe side effects by using significantly lower doses of viral vectors responsible for delivering genes into patients’ cells. Astellas will make an undisclosed upfront payment, as well as milestone payments and royalties on worldwide sales. This announcement comes after issues surrounding Astellas' AT132 gene therapy for XLMTM led to a clinical hold from the FDA following four deaths during clinical trials of a previous treatment from Astellas for the same condition. Keywords: Astellas Pharma, Kate Therapeutics, Gene Therapy, X-linked Myotubular Myopathy (XLMTM), Clinical Trials.
The safety issues surrounding Astellas’ AT132 gene therapy have hindered its progress in treating XLMTM, leading to this partnership with Kate Therapeutics in developing KT430. The latter's approach seeks to deliver a functional copy of the MTM1 gene that targets extreme muscle weakness, respiratory failure and premature mortality that are hallmarks of this condition using novel MyoAAV capsids at lower doses than what is currently available. This collaboration