REGENXBIO’s MPS II Treatment Data to be Presented at WORLDSymposium™ 2025
REGENXBIO Inc., a leading clinical-stage biotechnology company focused on developing and commercializing gene therapy products for patients with rare genetic diseases, recently made an exciting announcement. The company will be sharing data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, at the 21st Annual WORLDSymposium™ 2025.
About MPS II
MPS II is a lysosomal storage disorder caused by the deficiency of the enzyme iduronate-2-sulfatase. This deficiency results in the accumulation of glycosaminoglycans (GAGs) in various body tissues and organs, leading to progressive and debilitating symptoms. Symptoms of MPS II include coarse facial features, developmental delays, short stature, joint stiffness, and heart valve problems, among others.
REGENXBIO’s RGX-121 for MPS II Treatment
RGX-121 is an investigational, one-time gene therapy designed to address the underlying cause of MPS II by delivering a functional copy of the iduronate-2-sulfatase gene to the liver. The therapy aims to allow the body to produce the missing enzyme and reduce the accumulation of GAGs.
Data to be Presented at WORLDSymposium™ 2025
The data to be presented at the WORLDSymposium™ 2025 includes results from the ongoing Phase 1/2 trial of RGX-121, which includes both pediatric and adult patients. The trial is evaluating the safety, tolerability, and efficacy of RGX-121 in treating MPS II. Preliminary data from the trial has shown promising results, with some patients demonstrating significant reductions in GAG accumulation and improvements in symptoms.
Impact on Individuals with MPS II
For individuals living with MPS II, the potential impact of RGX-121 could be life-changing. Currently, there is no cure for MPS II, and treatment options are limited to symptomatic management. The possibility of a one-time gene therapy that could potentially alleviate symptoms and improve quality of life is a significant development.
Impact on the World
Beyond the individual level, the potential impact of RGX-121 on the world could be substantial. MPS II is a rare but debilitating disease, and there are currently no approved treatments for it in the United States. The successful development and approval of RGX-121 could open the door for the treatment of other lysosomal storage disorders and potentially revolutionize the way we approach genetic diseases.
Conclusion
The announcement of REGENXBIO’s data presentation at WORLDSymposium™ 2025 marks an exciting step forward in the development of a potential treatment for MPS II. With promising preliminary data and the potential to significantly improve the lives of individuals with this debilitating disease, the impact of RGX-121 could be far-reaching. As we await the presentation of the full data, the hope for a potential cure for MPS II and other lysosomal storage disorders continues to grow.
- REGENXBIO to present data on RGX-121, a gene therapy for MPS II, at WORLDSymposium™ 2025
- MPS II is a rare, debilitating disease caused by the deficiency of iduronate-2-sulfatase
- RGX-121 aims to deliver a functional copy of the iduronate-2-sulfatase gene to the liver
- Preliminary data shows promising results in reducing GAG accumulation and improving symptoms
- Potential impact on individuals with MPS II: life-changing improvement in symptoms and quality of life
- Potential impact on the world: opens the door for the treatment of other lysosomal storage disorders and could revolutionize the approach to genetic diseases
