Business
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 AAVHSC Administration and Improved Gene Expression In
About this update from Q32 Bio Inc.
[{"type":"text","content":"Preclinical Studies Demonstrated a Targeted Immunosuppression Approach Led to Reduced Immune Response to AAVHSC Administration and Improved Gene Expression In Vivo Gene Therapy Candidate HMI-204 for MLD Showed Robust CNS Distribution and Expression with Improved Packaging Productivity BEDFORD, Mass., Feb. 22, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of preclinical data that supports the targeted, prophylactic immunosuppression regimen in the ongoing pheEDIT gene editing clinical trial in adults with phenylketonuria (PKU) and juMPStart gene therapy trial in adults with Hunter syndrome (MPS II). Homology also shared additional details of the optimized, in vivo gene therapy candidate HMI-204 for metachromatic leukodystrophy (MLD) during the 19th Annual WORLDSymposium™ Meeting. “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 Albert Seymour, Ph.D., President and Chief Executive Officer of Homology Medicines. “In this study, we evaluated multiple regimens, including the combination of a T-cell inhibitor and steroid, and reported that the combination was the most effective in reducing B- and T-cell activity, reducing neutralizing antibody formation and improving gene expression. This targeted, prophylactic approach is part of our ongoing pheEDIT and juMPStart trials, from which we expect to share initial clinical data mid-year and in the second half of the year, respectively.” Dr. Seymour continued, “Also at WORLD, we presented the details of our optimized gene therapy development candidate for MLD, which showed the ability to cross the blood-brain-barrier following a single I.V. administration in the murine disease model and resulted in levels of enzyme activity predicted to lead to efficacy in vivo. The data also included HMI-204’s optimized tissue expression profile and improvements in productivity packaging. We continue to seek a partner for this candidate, which is ready to enter IND-enabling studies.” Details of Homology’s Presentations at WORLDSymposium™In the poster titled, “Tacrolimus Administration in Combination with Dexamethasone Reduces Neutralizing Antibody For...