REGENXBIO to Present New Data for Duchenne Muscular Dystrophy at 2025 MDA Conference

REGENXBIO Unveils New Findings at the 2025 MDA Conference

REGENXBIO Inc., a leader in gene therapy, has recently announced that it will present interim biomarker data from its Phase I/II AFFINITY DUCHENNE® trial involving RGX-202 at the upcoming 2025 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. This key event is scheduled to occur from March 16 to March 19, 2025, in Dallas, Texas, and promises to showcase the groundbreaking developments in the treatment of Duchenne muscular dystrophy (DMD).

The spotlight will be on two notable presentations during the conference. The first, titled "RGX-202, an investigational gene therapy for the treatment of Duchenne Muscular Dystrophy", is scheduled for Wednesday, March 19, 2025. Dr. Carolina Tesi Rocha, a Clinical Professor of Neurology at Stanford School of Medicine, will lead this presentation at 8:15 a.m. CT. Attendees can expect detailed insights into the promising clinical data surrounding RGX-202.

Following this, the second presentation, "Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain", will highlight pre-clinical research led by Dr. Steven Foltz, a Senior Scientist at REGENXBIO. In this session, findings will be discussed that underscore the potential improvements in therapeutic outcomes associated with a modified microdystrophin.

Both presentations emphasize REGENXBIO's commitment to advancing innovative therapies for those impacted by DMD, a serious genetic condition characterized by progressive muscle degeneration. The company has a robust pipeline of AAV Therapeutics targeting various diseases, and RGX-202 is a significant part of their efforts in addressing the challenges posed by rare and serious genetic disorders.

As a pioneer in AAV-based gene therapy since its inception in 2009, REGENXBIO continues to improve lives through its transformative approaches to treatment. The company has successfully treated thousands of patients with its AAV Therapeutic platform, distinguishing itself in the biotechnology landscape. As innovations in gene therapy broaden, the potential for changing the paradigm of care for patients with DMD and other genetic conditions becomes increasingly feasible.

The findings and details shared during the conference will be made available on REGENXBIO's website, ensuring that the scientific community and public have access to cutting-edge information that may shape future therapeutic strategies.

For those in industry and science, these presentations at the MDA Conference represent not just a discussion of data, but also a call to action for further research, awareness, and investment in crucial treatments for muscular dystrophy that could redefine patient outcomes.

Conclusion

As the 2025 MDA Clinical and Scientific Conference approaches, excitement builds around what could be pivotal announcements in the realm of gene-therapy and DMD treatment. Join us in following REGENXBIO’s significant contributions and their potential impact on the lives of many suffering from this challenging condition.