Catarina Silva, MSc,  —

Abeona Therapeutics has started dosing the second group in its ongoing Phase 1/2 clinical trial testing ABO-101, treating the first patient with Sanfilippo syndrome type B with a high dose of the investigational gene therapy. This follows completion of the first part of the trial (NCT03315182), in…

Abeona Therapeutics’ experimental gene therapy ABO-101 for people diagnosed with Sanfilippo syndrome type B has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). The company is currently enrolling eligible patients at sites across the U.S. and Spain for its Phase 1/2 trial. Fast Track designation is intended to…

Investigational enzyme replacement therapy BMN-250 successfully delivers the NAGLU enzyme — present in low levels in Sanfilippo type B patients — to key cells of the brain using different cellular pathways, a study has found. The study, “BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different…