Arthur Tzianabos, Homology Medicines CEO

Ho­mol­o­gy cleared to re­sume gene ther­a­py tri­al for rare meta­bol­ic dis­ease af­ter FDA lifts hold

A few months af­ter get­ting the stop sign from the FDA, Ho­mol­o­gy Med­i­cines is now able to restart its Phase II tri­al of a gene ther­a­py for the rare meta­bol­ic dis­ease phenylke­tonuria, or PKU.

The FDA had shown con­cerns that Ho­mol­o­gy’s gene ther­a­py, known as HMI-102, might lead to liv­er dam­age is­sues. In an­nounc­ing the hold had been lift­ed, Ho­mol­o­gy said there were no hos­pi­tal­iza­tions re­quired per­tain­ing to the el­e­vat­ed liv­er func­tion tests seen in the tri­al of adults.

Endpoints News

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.