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Homology Medicines Mulls Strategic Alternatives for Company, Gene Therapy Assets

NEW YORK – Homology Medicines, a biotechnology company developing therapies for rare diseases, on Thursday said it is evaluating strategic alternatives for the firm and its assets and will cease further development of its drug candidates.

Bedford, Massachusetts-based Homology's clinical-stage pipeline includes HMI-103, an in vivo gene-editing therapy that aims to replace the mutated PAH gene with a functional gene for phenylketonuria (PKU) patients, and HMI-203, a gene therapy for Hunter syndrome that is designed to deliver functional copies of the IDS gene that is mutated in the condition.

Also Thursday, the firm released initial data from a Phase I trial of HMI-103, which showed the drug was generally well tolerated and resulted in a reduction in plasma phenylalanine. Despite these results, it will not be developing this drug candidate or its other programs further. 

"We are pleased with the initial data from the first dose level in our PKU gene editing trial, which support dose-escalation," Homology President and CEO Albert Seymour said in a statement. However, "we believe the best path forward for our shareholders is to evaluate all strategic options for the company and our pipeline."

The company is considering strategic alternatives such as an acquisition, merger, sale, or other transaction and will reduce its workforce by 87 percent as it looks to reduce its operating costs. Seymour attributed the decision to the company's anticipated clinical development timelines and the broader financing environment.

Altogether, the firm said these measures are expected to extend its cash runway into 2026. It had about $150 million in cash, cash equivalents, and short-term investments as of March 31.

The company's other, preclinical stage drug development programs include gene therapy candidates for metachromatic leukodystrophy and paroxysmal nocturnal hemoglobinuria.