<a href="https://www.fiercebiotech.com/biotech/astellas-thins-early-pipeline-swaps-out-struggling-rare-disease-gene-therapy" hreflang="en">Astellas thins early pipeline, swaps out struggling rare disease gene therapy</a>
Astellas Pharma has streamlined its early pipeline by halting development of its gene therapy AT132 for X-linked myotubular myopathy, incurring a loss of approximately $103 million, and instead is focusing on a new gene therapy candidate, ASP2957, which shows promise in preclinical studies. Additionally, the company has discontinued two phase 1 candidates, ASP5502 and ASP1570, as part of a strategic decision to concentrate resources on more promising assets.
Astellas Pharma's strategic shift from the troubled gene therapy AT132 to ASP2957, which shows high muscle specificity and reduced liver targeting, highlights a focus on refining delivery mechanisms in gene therapy to enhance safety and efficacy. This move, coupled with their deal with Dyno Therapeutics for advanced AAV capsid technology, signals an investment opportunity in companies developing novel delivery systems for gene therapies targeting muscle tissue.