Hansa Biopharma announces positive data for Duchenne muscular dystrophy gene therapy
Ella Day | August 4, 2025 | News story | Research and Development | Duchenne Muscular Dystophy, Hansa Biopharma, Rare Diseases, Sarepta Therapeutics, clinical trial, genetic disorder
Hansa Biopharma has announced encouraging results from its ongoing SRP-9001-104 trial, which is investigating the use of imlifidase as a pre-treatment to enable gene therapy in patients with Duchenne muscular dystrophy (DMD) who are otherwise ineligible.
The clinical trial includes boys aged four to nine years with high levels of pre-existing antibodies to AAVrh74, the vector used in Sarepta Therapeutics’ approved gene therapy Elevidys (delandistrogene moxeparvovec-rokl). In the first three patients, a single dose of imlifidase rapidly reduced IgG antibodies by over 95%, with anti-AAV antibodies falling below the threshold required for gene therapy access.
Twelve weeks after Elevidys administration, patients showed evidence of AAV-mediated micro-dystrophin expression – though at lower levels than previously observed in other trials. No new safety concerns were identified.
“This is the first clinical data to suggest that imlifidase may help patients with high anti-AAV antibodies become eligible for approved gene therapies,” said Renée Aguiar-Lucander, CEO of Hansa Biopharma. “We are encouraged by these early findings and look forward to additional data from other ongoing collaborations.”
DMD is a severe genetic disorder affecting one in 3,500–5,000 boys worldwide. About 14% of patients have pre-existing antibodies that block access to AAV-based gene therapies. Imlifidase, a novel IgG-cleaving enzyme, is being studied as a way to overcome this barrier and expand access to potentially transformative treatments.
Ella Day
4/8/25
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