Astellas Pharma Inc. and Kate Therapeutics entered an exclusive license agreement to develop and commercialize KT430. KT430 is a preclinical next-generation investigational gene therapy that delivers a functional copy of the MTM1 gene via a novel MyoAAV capsid to treat X-linked myotubular myopathy (XLMTM), a life-threatening, rare neuromuscular disease characterized by extreme muscle weakness, respiratory failure and early death.
 
Astellas will make an undisclosed upfront payment to KateTx, which is also eligible to receive development, regulatory and commercial milestones, plus royalties on worldwide sales. Astellas will receive an exclusive worldwide license to develop, manufacture and commercialize KT430.
 
“This agreement brings together Astellas and KateTx’s collective patient-focused missions, allowing us to evaluate how to advance this new potential therapy for people diagnosed with XLMTM,” stated Adam Pearson, Chief Strategy Officer at Astellas. “The combination of Kate’s unique scientific approach and Astellas’ depth of experience in developing gene therapies for XLMTM provides a solid foundation for the advancement of KT430 as it progresses towards the clinic. The addition of this new potential gene therapy treatment for XLMTM along with our current AT132 program further enhances our commitment to this patient community and dedication to delivering transformative medicines.”
 
“We are very enthusiastic that Astellas has acquired worldwide rights to KT430 and what it means for the patients and families waiting for a potential new therapy for XLMTM,” said Kevin Forrest, Ph.D., president and CEO of KateTx. “Our company uses novel technology platforms that directly address the key limitations of current gene therapies, including tissue-specific delivery and gene regulation, with the potential to improve efficacy and safety. We see tremendous opportunities to bring important new therapies to patients with very serious conditions like XLMTM, as well as for other genetic disorders that are the focus of our internal efforts.”