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Rare Diseases Business Strategies M & A

With Hopeful Interim Data, Ultragenyx Bets On GeneTx, Angelman Syndrome Candidate

  • Analysis

Executive Summary

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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This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

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