Ultragenyx in-licenses rights to ABO-102 for Sanfilippo syndrome

18 May 2022

US ultra-rare disease specialist Ultragenyx Pharmaceutical (Nasdaq: RARE) and gene and cell therapy company Abeona Therapeutics (Nasdaq: ABEO) have announced an exclusive license agreement for AAV gene therapy ABO-102 (now UX111) for the treatment of Sanfilippo syndrome type A (MPS IIIA).

Under the terms of the agreement, Ultragenyx will assume responsibility for the ABO-102 program. Also, following regulatory approval, Abeona is 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.

Shares of both companies dipped on the news, with Abeona closing down 2.8% on Tuesday.

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