Ultragenyx updates on UX111 AAV gene therapy

California, USA-based rare disease company Ultragenyx Pharmaceutical (Nasdaq: RARE) yesterday released new data demonstrating treatment with UX111 (ABO-102) AAV gene therapy led to a statistically-significant improvement in the Bayley-III raw scores for the subdomains of cognition, receptive communication and expressive communication in patients with Sanfilippo syndrome type A (MPS IIIA) compared to natural history data from untreated patients.

These clinical endpoints were correlated with substantial and sustained reduction in levels of heparan sulfate (HS) in cerebrospinal fluid (CSF), the company noted.

Ultragenyx, whose shares closed up 2.7% at $45.37 following the announcement, gained rights to develop ABO-102 in 2022, under a deal with Abeona Therapeutics (Nasdaq: ABEO), which became eligible to receive tiered royalties from mid-single-digit up to 10% on net sales and up to $30.0 million in commercial milestone payments.