MUCOPOLYSACCHARIDOSIS (MPS) TREATMENT MARKET ANALYSIS

Market News

Global Mucopolysaccharidosis (MPS) Treatment Market: Key Developments

In September 2022, ROBIO Inc., a medical device technology company developing diagnostic equipment for the oncology space, announced that the UK MHRA, Research Ethics Committee (REC), and Health Research Authority (HRA) approved AVROBIO's (Lentiviral-Based Gene Therapy) collaborators for the initiation of the Phase 1/2 clinical trial of investigational autologous hematopoietic stem cell (HSC) gene therapy in infants diagnosed with neuronopathic mucopolysaccharidosis or hunter disease.

In February 2022, Denali Therapeutics Inc., which is dedicated to defeating neurodegenerative diseases through rigorous therapeutic discovery and development, presented new longer-term data from an ongoing phase 1/2 clinical trial of DNL310, an investigational brain-penetrant enzyme replacement therapy intended to treat both the central nervous system and peripheral manifestations of MPS II (hunter syndrome), at the World Symposium.

In June 2022, Gilead Sciences, Inc.,  a research-based biopharmaceutical company focused on the discovery, development, and commercialization of innovative medicines, announced that more than 80 abstracts will be presented at the International Liver Congress (ILC) 2022, placed from June 22-26, 2022. Key oral presentations will include Week 48 primary endpoint data from the Pivotal Phase 3 program of Hepcludex (bulevirtide) evaluating its efficacy and safety for the treatment of hepatitis delta virus (HDV) and the impact of the treatment on patient-reported outcomes.