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Homology Medicines Announces Preclinical Gene Therapy Candidate For Genetic Storage Disorder

  • Homology Medicines Inc (NASDAQ: FIXX) announced the details of HMI-204, its optimized, in vivo, one-time gene therapy candidate for metachromatic leukodystrophy (MLD).

  • Following a single I.V. administration in the MLD animal model, the candidate crossed the blood-brain barrier to the central nervous system (CNS) and reached key peripheral organs involved in MLD.

  • Also Read: Homology Medicines Stock Plunges After FDA Clinical Hold On Gene Therapy Trial.

  • Data demonstrated biodistribution to brain regions and multiple cell types.

  • Homology is actively seeking a partner to advance this preclinical-stage candidate.

  • MLD is a rare genetic disorder that causes fatty substances (lipids) to build up in cells, particularly in the brain, spinal cord, and peripheral nerves.

  • The late infantile form of the disorder is estimated to affect 1 in 40,000 people and is fatal within 5-10 years after onset.

  • Price Action: FIXX shares closed at $2.85 on Thursday.

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