Business
Homology Medicines Announces Presentations on its Expanding Genetic Medicines Platform and Internal GMP Manufacturing at the American Society of Gene & Cell Therapy Annual Meeting
- Data From New GTx-mAb Platform Demonstrated Proof of Principle for AAVHSCs to Deliver One-Time In Vivo Gene Therapy to Produce Antibodies in Humanized
About this update from Q32 Bio Inc.
[{"type":"text","content":"- Data From New GTx-mAb Platform Demonstrated Proof of Principle for AAVHSCs to Deliver One-Time In Vivo Gene Therapy to Produce Antibodies in Humanized Murine Model - - Additional Data for HMI-203 Gene Therapy in Hunter Syndrome and HMI-103 Gene Editing in PKU Support Planned Phase 1/2 Clinical Trial Initiations by End of 2021 - - On Track to Report Initial Phase 2 Data From pheNIX PKU Clinical Trial by Year End - - Conference Call / Webcast Today, May 13 at 8:15 a.m. ET - BEDFORD, Mass., May 13, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of data in multiple disease areas supporting the Company’s human hematopoietic stem cell-derived adeno-associated virus vector (AAVHSC) platform. Among Homology’s presentations are data from its new GTx-mAb platform targeting complement protein 5 (C5), which showed proof of principle in paroxysmal nocturnal hemoglobinuria (PNH). The Company also presented data from its gene therapy program for MPS II (Hunter syndrome) and gene editing program for phenylketonuria (PKU), both of which are on track to enter the clinic this year. Presentations also focused on the Company’s internal commercial GMP manufacturing process and platform. These data were featured at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting. “Our new GTx-mAb platform is designed to leverage our AAVHSCs to deliver one-time in vivo gene therapy to produce antibodies from the liver and secrete them throughout the body,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “These data demonstrated once again that our vectors are highly efficient in targeting the liver. With this approach, we showed our vectors enabled the liver to produce antibodies that resulted in sustained expression levels in the serum of a humanized murine model that inhibited red blood cell destruction using an ex vivo assay. These PNH data are exciting, given the high unmet need, and underscore the potential to address other complement-related diseases, as well as diseases with larger patient populations. We are on track to name our first development candidate from the GTx-mAb platform in PNH this year.” Dr. Seymour continued, “Our multiple presentations at ASGCT highlight the maturation of our pipeline, which is expected to yield...