Homology Medicines Announces Optimized, In Vivo Gene Therapy Candidate for the Treatment of Metachromatic Leukodystrophy
- Candidate Showed Ability to Target Central Nervous System and Peripheral Organs
Treatments and Product Candidates -
- Data Demonstrated Biodistribution to Brain Regions and Multiple Cell Types -
- Optimizations Included Significant Improvements in Expression,
- Homology Seeks Partner to Advance Development Candidate -
“Efforts to enhance our original MLD candidate led to our optimized candidate, which has a better therapeutic profile with respect to expression and packaging, while retaining its key differentiator of addressing the CNS and peripheral organ manifestations of the disease with a single I.V. administration,” said
In the murine model of MLD, a single I.V. administration of the optimized gene therapy candidate, which uses one of Homology’s proprietary AAVHSC capsids, resulted in:
- Broad biodistribution to peripheral organs and the CNS;
- Expression of human ARSA (hARSA) levels in multiple brain regions and cell types, which were well-above the minimum levels of enzyme needed to correct the MLD disease phenotype*;
- hARSA activity levels in the brain that are predictive of functional improvements; and
- hARSA activity in the serum.
Additionally, optimizations led to significant improvements in vector yield and superior packaging for the candidate.
About Metachromatic Leukodystrophy (MLD)
MLD is a rare lysosomal storage disorder primarily caused by a mutation in the ARSA gene. ARSA is responsible for the creation of the arylsulfatase A (ARSA) protein, which is required for the breakdown of sulfatides in cells. In MLD, sulfatides accumulate and destroy myelin-producing cells in the peripheral and central nervous systems leading to progressive and serious neurological deterioration. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within 5-10 years after onset.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our plans and timing for the release of additional preclinical and clinical data; our expectations surrounding the potential, safety, and efficacy of our product candidates; the potential of our gene therapy and gene editing platforms; our plans to engage in future collaborations and strategic partnerships; and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties, including for the manufacture of materials for our research programs, preclinical and clinical studies; failure to obtain
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Source: Homology Medicines, Inc.