Homology Medicines Announces Preclinical Gene Therapy Candidate For Genetic Storage Disorder

Homology Medicines Announces Preclinical Gene Therapy Candidate For Genetic Storage Disorder
  • Homology Medicines Inc 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.
Posted In: Briefsgene therapyPreclinical PhaseBiotechNewsPenny StocksHealth CareGeneral