Regenxbio has reported positive interim data from the Phase I/II portion of its Affinity Duchenne study (NCT05693142), which is examining its investigational gene therapy RGX-202.
The therapy is designed to treat Duchenne muscular dystrophy (DMD) and was able to induce a 122.3% microdystrophin expression in one child aged three.
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RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients.
DMD is a form of muscular dystrophy typically associated with the inability to produce the protein known as dystrophin, it is this protein that connects the cytoskeleton of muscle fibres to cells, forming a key part of muscle development.
According to Regenxbio, children under four years old currently don’t have access to any gene therapy intended to treat the condition that is likely to manifest in the early stages of life.
The news was reported as part of the Muscular Dystrophy Association (MDA) 2025, taking place from 16 to 19 March in Dallas, Texas.
Regenxbio chief medical officer Steve Pakola said: “RGX-202 is the only next-generation gene therapy for Duchenne in a pivotal phase trial. The new data from the age 1-3 cohort builds on the favourable safety and efficacy profile seen in ages 4 and older and reinforces the potential of RGX-202 to serve a wide age range of patients.
“The consistent, robust microdystrophin levels seen across the age range and the functional improvements previously reported support RGX-202’s potential to alter the course of this devastating disease. We look forward to sharing additional Phase I/II functional data in the first half of 2025.”
GlobalData’s Pharmaceutical Intelligence Center estimates that RGX-202 is likely to bring in $25m for Regenxbio by the end of 2027 if it can make it to market. That figure is predicted to rise to $179m by the end of 2030. Overall, the DMD market is expected to generate revenues of $4.2bn globally by the of 2025, with that figure predicted to also rise to more than $9.6bn by the end of 2030.
GlobalData is the parent company of Clinical Trials Arena.
Elsewhere at the MDA annual meeting, Dyne Therapeutics’ therapy DYNE-251 has seen “unprecedented and sustained” functional improvement in a Phase I/II trial. The following day at the conference saw results from Avidity Biosciences’ trial of del-zota saw it able to increase dystrophin expression in patients living with the exon 44 skipping variant of the disease.
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