Release Details
Homology Medicines Presents Preclinical Data Supporting Immunosuppression Regimen in Ongoing PKU and Hunter Syndrome Clinical Trials, and Details Optimized MLD Gene Therapy Candidate at the 19th Annual WORLDSymposium™ Meeting
Preclinical Studies Demonstrated a Targeted Immunosuppression Approach Led to Reduced Immune Response to
In
“We are pleased to share our work outlining the impact immunosuppression regimens had on outcomes following AAVHSC dosing in NHPs, as it contributes to a key area of research in the gene therapy and gene editing field,” stated
Details of Homology’s Presentations at WORLDSymposium™
In the poster titled, “Tacrolimus Administration in Combination with Dexamethasone Reduces Neutralizing Antibody Formation Against AAV Vector and Increases Transgene Expression in Cynomolgus Macaques,” data showed that administration of the T-cell inhibitor tacrolimus with dexamethasone in AAVHSC-PAH-treated non-human primates (NHPs) resulted in:
- Lowered secretion of inflammatory cytokines and activation state of CD8+ T cells compared to the no immunosuppression (IS) group;
- Reduced AAVHSC neutralizing antibody (nAb) formation compared to NHPs treated with each agent alone and by 4.8-fold compared to the no
IS group; and - Increased PAH gene expression, as measured by mRNA, compared to each agent alone and by two-fold compared to the no
IS group.
In the poster titled, “Gene Therapy for Metachromatic Leukodystrophy: Lead Candidate Optimization,” a single I.V. administration of optimized HMI-204 in the murine model of MLD showed:
- Systemic and central nervous system (CNS) biodistribution, including robust expression in the CNS;
- Human ARSA cellular expression patterns in the brain that were nearly identical to that of murine Arsa distribution in wildtype age-matched controls; and
- Sustained levels of ARSA activity reaching normal adult ARSA brain activity levels.
Additionally, packaging productivity of HMI-204 demonstrated a substantial improvement in vector genome yields, compared to the earlier MLD gene therapy candidate.
For more information, please visit the Publications and Presentations page on Homology’s website.
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.
About Homology Medicines, Inc.
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This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. 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 to engage in future collaborations and strategic partnerships; our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; the potential of our gene therapy and gene editing platforms, including our GTx-mAb platform; our plans and timing for the release of additional preclinical and clinical data; our plans to progress our pipeline of genetic medicine candidates and the anticipated timing for these milestones; and our position as a leader in the development of genetic medicines. The words “believe,” “may,” “will,” “estimate,” “potential,” “continue,” “anticipate,” “intend,” “expect,” “could,” “would,” “project,” “plan,” “target,” and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions. 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: 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.