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.
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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