- 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.
