“Advancement in Enzyme Replacement and Gene Therapy Technologies”
- A significant and accelerating trend in the global mucopolysaccharidosis (MPS) market is the development and integration of next-generation enzyme replacement therapies (ERTs) and gene therapy solutions. These novel approaches aim to address the root cause of MPS, offering improved efficacy, longer-lasting benefits, and reduced treatment burden
- For instance, therapies such as Aldurazyme and Elaprase have set the foundation for ERTs, while clinical-stage gene therapies such as RGX-111 and ABO-102 are demonstrating promise in treating MPS subtypes with potential for durable, one-time treatments
- Gene therapy platforms targeting MPS I and MPS IIIA are now leveraging adeno-associated viral (AAV) vectors to enable targeted delivery of functional genes to central nervous system tissues, aiming to overcome the blood-brain barrier—a long-standing challenge in MPS treatment
- The use of advanced technologies in these therapies not only improves the bioavailability and half-life of enzymes but also expands treatment access to neurological symptoms, which were previously less responsive to conventional ERTs
- This evolution in treatment methodology is reshaping patient care, offering better disease management and potential long-term remission, particularly in pediatric cases.
- Companies such as Sangamo Therapeutics, REGENXBIO, and Lysogene are actively investing in these advanced therapeutics, indicating strong momentum in R&D efforts and partnerships. The ongoing clinical success and regulatory support for such innovations are driving robust growth in the MPS treatment landscape across global healthcare markets
