After pumping the brakes on their Angelman syndrome study, Ultragenyx and GeneTx are back in the race
Eleven months after high doses of Ultragenyx and GeneTx’s Angelman syndrome candidate temporarily caused some patients to lose their ability to walk, the FDA is allowing the partners to restart the trial — but with a much lower dose regimen.
Regulators have removed a clinical hold on Ultragenyx and GeneTx’s antisense oligonucleotide GTX-102, opening the door for more pediatric patients to enroll in a Phase I/II trial. The partners halted the trial in October after all five patients in the study experienced “lower extremity weakness” receiving the two highest doses during an escalation period. Two of them lost their ability to walk or bear weight.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.