- In April 2023, REGENXBIO Inc. announced promising interim data from its Phase I/II clinical trial evaluating RGX-111, an investigational gene therapy for Mucopolysaccharidosis Type I (MPS I). The therapy utilizes AAV9 vectors to deliver functional copies of the IDUA gene to the central nervous system, addressing neurological symptoms. This development reflects a growing shift toward durable, one-time treatments in the MPS space, offering new hope for patients with severe forms of the disorder
- In March 2023, Sangamo Therapeutics, Inc. advanced its investigational gene therapy ST-920 into clinical trials targeting MPS II (Hunter Syndrome). This AAV-based therapy aims to achieve sustained enzyme expression through liver-targeted gene transfer. The trial progression emphasizes Sangamo’s commitment to expanding the therapeutic frontier for lysosomal storage diseases using in vivo genome editing technologies
- In February 2023, Ultragenyx Pharmaceutical Inc. initiated the Phase I/II study of UX111, a gene therapy developed in partnership with Abeona Therapeutics for MPS IIIA (Sanfilippo Syndrome). UX111 is designed to address the underlying genetic cause by enabling long-term enzyme expression in the brain. This move reinforces Ultragenyx’s strategic focus on CNS-targeted therapies and innovation in rare disease treatment
- In January 2023, BioMarin Pharmaceutical Inc. reported long-term outcomes data for Aldurazyme (laronidase), demonstrating sustained benefits in MPS I patients over a 10-year period. The findings underscore the long-term value of enzyme replacement therapy in managing MPS symptoms and improving quality of life, solidifying BioMarin’s position as a pioneer in the MPS treatment landscape
- In January 2023, Orchard Therapeutics plc expanded its clinical development pipeline with investigational hematopoietic stem cell gene therapies for MPS disorders, including preclinical programs targeting MPS IIIA and IIIB. By leveraging its ex vivo gene therapy platform, Orchard is aiming to provide curative potential for severe, neurodegenerative forms of MPS, reflecting the industry's push toward transformative genetic treatments in rare disease therapy
