No Safety Concerns Seen In Uniqure's Early-Stage Huntington's Gene Therapy Study

 

• Independent Data Safety Monitoring Board (DSMB) has reviewed the six-month safety data from the first two enrolled patients and 90-day safety data from the next two enrolled patients from Phase 1/2 trial evaluating Uniqure N.V.'s (NASDAQ: QURE) AMT-130 candidate in Huntington's disease.

• IDSB did not observe safety concerns to prevent further dosing, and the final six patients in the first cohort are now cleared for enrollment. Full enrollment in cohort one is expected to complete by mid-year 2021. The 26-subject trial explores the safety, tolerability, and efficacy signals in patients with early manifest Huntington's disease, a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities, and cognitive decline.

• The company looks forward to sharing initial biomarker and imaging data by the end of the year.

https://finance.yahoo.com/news/no-safety-concerns-observed-uniqures-145708473.html