Takeda has announced a Post-Trial Access program for patients currently enrolled in extension trials for TAK-609, a treatment for MPS II Hunter syndrome. This program will provide continued access for patients who have benefited from treatment on existing studies....
In our March edition we reported that Abeona Therapeutics had decided to end enrollment for their clinical trial for MPS IIIA children. We are now happy to share that Ultragenyx will be taking over the clinical trials for AAV gene therapy ABO-102 (now UX111). “Based...
We are excited to share the news that REGENXBIO Inc. has announced its intention to file a Biologics License Application (BLA) in 2024 using the FDA’s accelerated approval pathway for the treatment of Hunter Syndrome, Mucopolysaccharidosis Type II (MPS II). The...
Homology Medicines have received clearance from Health Canada to initiate the juMPStart trial for HMI-203, a one-time, in vivo gene therapy candidate for the treatment of adults with Hunter Syndrome (MPS II). If you are interested in knowing more, join Homology...
New data from the AAVance trial has revealed children with Sanfilippo syndrome type A, including those with severe disease, showed stable or continuously increasing cognitive, language, and motor functions two years after dosing with the investigational gene therapy...
Avrobio has received orphan drug designation for its gene therapy, AVR-RD-05, from the US Food and Drug Administration (FDA) to treat mucopolysaccharidosis type II or Hunter syndrome. and is expected to initiate a Phase I/II clinical trial for MPS II in partnership...
