Safety scare cleared and early data in hand, Ultragenyx dishes out $75M to buy rare disease partner
It had its ups and downs, but Ultragenyx’s three-year-old partnership with an Angelman syndrome-focused biotech has culminated in a buyout.
With $75 million upfront, the rare disease specialist is exercising the option it lined up back in 2019 to acquire GeneTx on the back of what it called encouraging interim data from a Phase I/II dose escalation study showing “early clinical activity across multiple domains and multiple measurements without any evidence of drug-related safety issues.” The companies also got cleared to start testing higher doses of GTX-102 in search of an optimal dosing regimen — which Ultragenyx believes could lead to even better effects.
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