Sangrail Biologics, a clinical-stage gene therapy company focused on rare pediatric genetic diseases, announced the launch of the company on May 5, 2026, and announced its lead clinical asset, SNG-101 (formerly known as ABO-101 under Abeona Therapeutics), which is describes as “a potential first-in-class AAV gene therapy designed to treat the underlying cause of disease” in patients with Sanfilippo Syndrome Type B (MPS IIIB).
Cure Sanfilippo Foundation was proud to be early supporters of this program, when it was run by Abeona and Nationwide Children’s Hospital.
The new company Sangrail Biologics is led by the former CEO of Abeona (and subsequently Forge Biologics), so brings significant history and experience with Sanfilippo syndrome and gene therapy drug production.
While managed by Abeona, 14 children were treated with this intravenous AAV9 gene therapy across 3 dose levels.
Like other enzyme replacement and gene therapies in Sanfilippo to date, the most robust effects on neurological development occur the earlier that treatment is given. Importantly, meaningful benefit has also been reported by families even when treatment was initiated after Sanfilippo cognitive symptoms were evident.
In related news, Ultragenyx has advanced the intravenous AAV9 Type A gene therapy program (also previously with Abeona), and that is under FDA review for accelerated approval with a decision date by or before mid-September 2026. We hope this very similar Type B gene therapy program can follow with a streamlined path to accelerated approval.
“We look forward to hearing and learning more about next steps and more timelines as this program moves forward,” said Cure Sanfilippo Foundation President & Co-Founder Glenn O’Neill. “In discussing with the company, they will know more after engaging with the FDA in second half 2026.”