REGENXBIO Announces Encouraging Interim Data from AFFINITY DUCHENNE® Trial of RGX-202
REGENXBIO Inc., a leading clinical-stage biotechnology company focused on transforming the lives of patients with severe genetic disorders, recently announced new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202. This investigational gene therapy is being developed for the treatment of Duchenne muscular dystrophy (DMD), a rare and progressive genetic disorder.
Consistent and Robust Microdystrophin and Transduction Levels
The company reported that these new patients, who were aged 1 and 3 at the time of dosing, showed consistent and robust microdystrophin and transduction levels. The younger patient, who was aged 1 at dosing, exhibited microdystrophin expression levels at 116.8% compared to age-matched controls. The older patient, who was aged 3 at dosing, had an expression level of 122.3% compared to controls.
Encouraging Safety Profile
With a differentiated novel construct and proactive short course immune modulation regimen, RGX-202 continues to demonstrate an encouraging safety profile. No serious adverse events (SAEs) or adverse events of special interest (AESIs) have been reported in the trial to date.
Phase III Trial and BLA Submission
The Phase III portion of the AFFINITY DUCHENNE® trial is currently enrolling ambulatory patients aged 1 and above. The trial is on track for a Biologics License Application (BLA) submission mid-2026.
Implications for Patients and the World
For patients with DMD and their families, these promising results offer hope for a potential treatment option that could help manage the symptoms of this debilitating disease. The consistent and robust expression levels of microdystrophin, along with the encouraging safety profile, suggest that RGX-202 could be an effective therapy for DMD. This could lead to improved quality of life and potentially even a cure for some patients.
On a larger scale, the successful development of RGX-202 could have significant implications for the gene therapy industry as a whole. It could pave the way for the development of more effective gene therapies for other genetic disorders. Additionally, it could help to advance the field of gene therapy by demonstrating the potential for long-term efficacy and safety in large patient populations.
Conclusion
REGENXBIO’s announcement of new, positive interim data from the AFFINITY DUCHENNE® trial of RGX-202 is a significant step forward in the development of a potential treatment for Duchenne muscular dystrophy. The consistent and robust microdystrophin and transduction levels, along with the encouraging safety profile, suggest that RGX-202 could be an effective therapy for this debilitating disease. Furthermore, the successful development of RGX-202 could have far-reaching implications for the gene therapy industry and the treatment of other genetic disorders.
- REGENXBIO reports positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202
- Consistent and robust microdystrophin and transduction levels in patients aged 1 and 3 at dosing
- Encouraging safety profile with no SAEs or AESIs reported
- Phase III portion of the trial enrolling ambulatory patients aged 1 and above, on track for BLA submission mid-2026
- Implications for patients with DMD and the gene therapy industry as a whole
