Ultragenyx acquires option to buy gene therapy firm

16 August 2019

Ultra-rare disease specialist Ultragenyx Pharmaceutical (Nasdaq: RARE) has inked a deal with GeneTx Biotherapeutics in relation to the antisense oligonucleotide GTX-102.

The firms have agreed to collaborate on the development of GTX-102, which is designed to suppress the expression of the UBE3A-AS gene, as a possible treatment for Angelman syndrome.

Ultragenyx will make an upfront payment of $20 million for an exclusive option to acquire GeneTx, any time prior to 30 days after the Food and Drug Administration accepts an investigational new drug (IND) for GTX-102. The option period can be extended for an additional $25 million.

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