Homology Medicines Announces Optimized, In Vivo Gene Therapy Candidate for the Treatment of Metachromatic Leukodystrophy

– Candidate Showed Ability to Target Central Nervous System and Peripheral Organs
Following a Single I.V. Administration in MLD Model, a Key Differentiator from Available
Treatments and Product Candidates –

       – Data Demonstrated Biodistribution to Brain Regions and Multiple Cell Types –

       – Optimizations Included Significant Improvements in Expression, Productivity and Packaging –

– Homology Seeks Partner to Advance Development Candidate –

Excerpt from the Press Release:

BEDFORD, Mass., Aug. 11, 2022 (GLOBE NEWSWIRE) — Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the details of HMI-204, its optimized, in vivo, one-time gene therapy product candidate for the treatment of metachromatic leukodystrophy (MLD). Following a single I.V. administration in the MLD murine model, the candidate crossed the blood-brain-barrier to the central nervous system (CNS) and reached key peripheral organs involved in MLD. As a rare and often fatal genetic disorder, efforts are underway in the U.S. and globally to implement prospective newborn screening for MLD. Homology is actively seeking a partner to advance this preclinical-stage candidate.

“Efforts to enhance our original MLD candidate led to our optimized candidate, which has a better therapeutic profile with respect to expression and packaging, while retaining its key differentiator of addressing the CNS and peripheral organ manifestations of the disease with a single I.V. administration,” said Albert Seymour, Ph.D., President and Chief Scientific Officer of Homology Medicines. “In addition to our understanding of MLD disease biology and our team’s prior experience in developing potential treatments for this disorder, we were able to apply our expertise in vector design and manufacturing to optimize our candidate. We believe that the preclinical data demonstrate its potential to make a meaningful difference for patients whose current treatment options are limited to ex vivo approaches that include difficult pre-conditioning regimens. We look forward to sharing data from this development candidate at future meetings.”

In the murine model of MLD, a single I.V. administration of the optimized gene therapy candidate, which uses one of Homology’s proprietary AAVHSC capsids, resulted in:

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