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UltraGenyx acquires global rights to AAV gene therapy ABO 102 for Sanfilippo syndrome Type A (MPS IIIA) from Abeona Therapeutics.

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Published:22nd May 2022

Ultragenyx Pharmaceutical Inc. and Abeona Therapeutics Inc. 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 and in return Abeona is eligible to receive tiered royalties of up to 10% on net sales and commercial milestone payments following regulatory approval.

Abeona has completed a successful Type B meeting with the FDA regarding the pivotal Transpher A trial to support filing and approval for ABO-102 for the treatment of patients with MPS IIIA. Interim results from the Transpher A trial presented in an encore presentation at the American Society of Gene & Cell Therapy (ASGCT) y demonstrate that neurocognitive development was preserved in children treated before 2 years old or with a development quotient (DQ) > 60 (n=10) within normal range of a non-afflicted child after treatment with ABO-102. The interim results also showed continued or stabilized cognitive function and behavioral progress using standard developmental assessments.

Condition: MPS IIIA/Sanfilippo Syndrome
Type: drug

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