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ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ:RGNX) announced Thursday it has completed enrollment of 30 participants in the pivotal portion of its AFFINITY DUCHENNE trial for RGX-202, an investigational gene therapy for Duchenne muscular dystrophy. The biotech company, currently valued at approximately $649 million, has seen its stock surge 66% year-to-date as investors react positively to its clinical progress. According to InvestingPro data, analysts anticipate significant sales growth for REGENXBIO in the current year.
The company also reported successful production of its first commercial-intended batches at its in-house Manufacturing Innovation Center, where it has capacity to produce up to 2,500 doses annually.
In the Phase I/II portion of the trial, participants receiving the pivotal dose showed microdystrophin expression levels ranging from 20% to 122%, exceeding baseline-matched external natural history controls on all functional measures. The therapy has been well tolerated with no serious adverse events reported as of May 7, 2025.
"The Duchenne community urgently needs new treatment options that provide durable, safe outcomes and can meaningfully change the course of this degenerative disease," said Curran Simpson, President and Chief Executive Officer of REGENXBIO, according to the press release.
The primary endpoint for the pivotal trial is the proportion of participants whose RGX-202 microdystrophin expression is at least 10% at Week 12. Secondary endpoints include changes in timed function tests for participants aged 4 years and older.
RGX-202 is manufactured using the company’s NAVXpress suspension-based process, which has consistently achieved product purity levels exceeding 80% full capsids. The company continues to enroll patients in a confirmatory trial.
REGENXBIO expects to report topline pivotal data in early Q2 2026 and submit a Biologics License Application (BLA) in mid-2026, with potential commercial launch in 2027.
The gene therapy features a differentiated microdystrophin construct that includes the C-Terminal domain, which the company notes is unique among approved or late-stage Duchenne gene therapies in development.
In other recent news, Regenxbio Inc. has announced the completion of enrollment for its pivotal ATMOSPHERE and ASCENT studies, which are evaluating surabgene lomparvovec for treating wet age-related macular degeneration (wet AMD) using subretinal delivery. These trials have collectively enrolled over 1,200 participants across more than 200 sites globally, marking the largest gene therapy program ever conducted. Topline data from these studies is expected in the fourth quarter of 2026. Meanwhile, Regenxbio’s clemidsogene lanparvovec (RGX-121) for Mucopolysaccharidosis II has shown positive 12-month data, maintaining an 82% median reduction in cerebrospinal fluid levels of heparan sulfate, consistent with previous results.
Following these developments, H.C. Wainwright reiterated a Buy rating with a $34.00 price target on Regenxbio, citing the company’s promising trial data. Raymond James also maintained an Outperform rating with a $29.00 price target, highlighting the consistency of the RGX-121 data with earlier results. In contrast, Goldman Sachs reiterated a Neutral rating with a $14.00 price target, despite acknowledging the positive clinical data for the Hunter syndrome treatment. These recent updates reflect ongoing interest and varied perspectives from analysts on Regenxbio’s gene therapy initiatives.
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