Homology Medicines advances 1st gene editing development candidate to study

Homology  Medicines announced that it has nominated an in vivo gene editing development candidate and initiated IND-enabling studies for the treatment of pediatric patients with phenylketonuria. Homology also completed its internal GMP manufacturing facility with capabilities to support both gene editing and gene therapy programs. “Starting IND-enabling studies with an in vivo, nuclease-free gene editing candidate is an important milestone for Homology and achieves a key 2018 corporate goal,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Our dual platform has enabled us to progress specific genetic medicine programs for PKU that may address two distinct patient populations who require different therapeutic approaches based on human biology, with gene therapy for adults and gene editing for children. We look forward to continuing to move these one-time potentially curative treatments forward as rapidly as possible.”
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