It remains early days but Ultragenyx Pharmaceutical Inc. likes its chances to succeed in the rare neuro-genetic disorder Angelman syndrome where Ovid Therapeutics, Inc. notably flamed out in 2020. Ultragenyx unveiled interim Phase I/II data showing safety and indications of clinical activity on 18 July for GTX-102, a candidate it is developing with GeneTx Biotherapeutics LLC, and now the company has exercised an option to acquire its collaborator.
With Hopeful Interim Data, Ultragenyx Bets On GeneTx, Angelman Syndrome Candidate
Ultragenyx is buying its Angelman syndrome partner GeneTx for $75m on the heels of an interim Phase I/II readout that shows signs of clinical activity and appears to resolve earlier safety worries.
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