April 27, 2026 —
Astellas Pharma is reshaping its pipeline and doubling down on next-generation AAV gene therapy, halting development of AT132 (resamirigene bilparvovec) while advancing a newer muscle-targeted program, ASP2957, for X-linked myotubular myopathy (XLMTM).
The strategic move marks a notable pivot in one of gene therapy’s most closely watched rare disease programs. While Astellas is taking an approximately $103 million impairment related to AT132, the company emphasized the decision reflects portfolio prioritization rather than a shift away from the biology or indication itself.
AT132, originally acquired through Astellas’ $3 billion purchase of Audentes Therapeutics, had shown promise but was weighed down by a difficult clinical history, including safety concerns that placed the program under intense scrutiny.
Now Astellas is moving resources toward ASP2957, a next-generation muscle-targeting Adeno-associated virus (AAV) gene therapy licensed from Kate Therapeutics. The program is designed with improved tissue specificity and reduced liver targeting—an important evolution in systemic AAV delivery.
According to the company, preclinical data suggest ASP2957 may enable dosing roughly 100-fold lower than AT132 while maintaining effective muscle targeting, a potentially meaningful advancement for safety, manufacturability, and therapeutic index.
The move reflects a broader trend across the AAV field: not retreat from gene therapy, but progression toward next-generation capsids and smarter vector engineering.
Astellas is also reinforcing that strategy externally. The company recently partnered with Dyno Therapeutics to access novel AAV capsids designed to improve delivery to muscle, an area historically challenging for systemic gene transfer.
Taken together, the changes suggest Astellas is repositioning around a newer generation of gene therapies focused on precision targeting rather than abandoning the modality.
Beyond gene therapy, Astellas also discontinued two Phase 1 assets—ASP5502 and ASP1570—as part of broader pipeline prioritization.
