Homology Medicines, a company using proprietary gene editing and gene therapy platform announced the presentation of preclinical data supporting its investigational gene therapy programs for the treatment of metachromatic leukodystrophy (MLD) and phenylketonuria (PKU) at the American Society of Human Genetics (ASHG) 2019 Meeting.
The presented preclinical data showed the results from studies conducted in the murine model and non-human primates. The results demonstrated the potential of HMI-202 gene therapy in crossing the blood-brain-barrier and the blood-nerve-barrier. In addition, the results also indicated the increased levels of arylsulfatase a (ARSA) protein to therapeutic levels.
The results show positive signs for the future development of gene therapies in patients with genetic diseases and metabolic disorders. The pipeline already has several candidates in development for these therapeutic areas. Here is a snapshot of the gene therapies pipeline.
The overall gene therapy market is expected to reach close to $12 billion by 2030. More such positive results are expected to drive the growth in this innovative space.