Children with Hunter syndrome have a missing gene ... Following successful engraftment of modified HSCs in the bone marrow, these cells start to produce daughter blood cells which contain the IDS gene ...

or Hunter syndrome, where the underlying issue is at the genetic level, cell therapies cannot offer the same hope for treatment. Exosomes, on the other hand, are acellular vesicles secreted by ...

The Hunter syndrome program was developed by Brian Bigger, a professor of cell and gene therapy at The University of Manchester. Professor Bigger has published preclinical data demonstrating that the ...

Feb. 06, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced the primary analysis of the Phase 1/2 study in 47 participants with Hunter syndrome (MPS II) in the 24-week ...

Regenxbio closes strategic partnership with Nippon Shinyaku for MPS diseases: Rockville, Maryland Wednesday, March 5, 2025, 14:00 Hrs [IST] Regenxbio Inc., a leading clinical-stag ...

GC Biopharma announced on the 5th that the Ministry of Food and Drug Safety has designated the treatment for severe Hunter syndrome, 'Hunterase ICV (intracerebroventricular),' as an orphan drug.