Benzinga | Apr 28, 2021 08:16AM EDT

Homology Medicines Announces New Approach To 'Leverage AAVHSC Platform By Delivering One-Time In Vivo Gene Therapy To Produce Antibodies In Humanized Murine Model'

Homology Medicines, Inc. (NASDAQ:FIXX), a clinical-stage genetic medicines company, announced today a new approach to its AAVHSC platform that delivers one-time gene therapy to produce antibodies throughout the body. Preclinical data that demonstrated proof of principle for this GTx-mAb platform is the subject of an upcoming presentation, alongside other digital and oral presentations from the Company's in vivo gene therapy and gene editing programs, that will be featured at the virtual American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting May 11 - 13, 2021.

"We are excited to unveil our GTx-mAb platform and preclinical data targeting complement protein 5 with a one-time treatment," stated Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. "These data demonstrated that our AAVHSCs delivered vectors at a high efficiency to the liver and secrete antibodies throughout the body, which resulted in sustained expression levels consistent with C5 antibody therapeutics in a humanized murine model. Importantly, this proof of concept for our approach unlocks the potential for a one-time treatment that leverages the liver to produce fully functional antibodies and the expansion of our pipeline to address diseases with larger patient populations."

Albert Seymour, Ph.D., Chief Scientific Officer, added, "We also look forward to sharing new data across our genetic medicines pipeline programs, including PKU and Hunter syndrome, which support our plans to advance these two programs into the clinic this year. These data further demonstrated that our AAVHSCs efficiently transduced the liver and reached relevant tissues, including crossing the blood-brain and peripheral-nerve barriers with a single I.V. administration. Additionally, we plan to highlight advancements from our capsid characterizations and our internal GMP manufacturing process and platform, including an oral presentation showing the impact of novel formulations on capsid stability."

Homology management plans to host a conference call and webcast on May 13, 2021, at 8:15 a.m. ET to discuss the new GTx-mAb platform and ASGCT data highlighting the Company's human hematopoietic stem cell-derived adeno-associated virus vector (AAVHSC) platform and pipeline.

Homology's digital and oral presentations include: