Business
Homology Medicines Presents Details of Optimized HMI-103 Nuclease-Free Gene Editing Candidate Featuring Integrated Liver-Specific Promoter to Maximize Long-Term Expression
- Additional Data at ASGCT Annual Meeting Demonstrate Precision of HR-Based Approach with Genome-Wide Integration Assays Confirming On-Target Editing and No
About this update from Q32 Bio Inc.
[{"type":"text","content":"- Additional Data at ASGCT Annual Meeting Demonstrate Precision of HR-Based Approach with Genome-Wide Integration Assays Confirming On-Target Editing and No Off-Target Events - - New Data from GTx-mAb Platform Support Potential for Targeting Many Complement-Related Disorders - - Further Characterization of Homology’s Naturally Derived AAVHSC Capsids Highlight Their Broad Biodistribution and a Novel Discovery Revealed One Capsid with Low Tropism to the Liver, a Key Functional Benefit for Selecting New Disease Indications - - Homology Symposium to be Held Today, May 18, at 7:30 a.m. ET - BEDFORD, Mass., May 18, 2022 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, unveiled today the mechanism of action (MOA) and optimization of HMI-103, the nuclease-free gene editing candidate currently in a Phase 1 trial for phenylketonuria (PKU). HMI-103 utilizes the body’s natural DNA repair process of homologous recombination (HR) to insert a functional copy of the PAH gene into a specific region of the genome. It is designed to provide a permanent DNA correction for PKU by replacing at least one disease-causing allele with a normal gene sequence in edited cells. HMI-103 was optimized to integrate the PAH gene and a liver-specific promoter into the genome and to maximize enzyme expression in all transduced cells. “Our dedication to providing new solutions for patients living with PKU led us to develop an AAV-based nuclease-free gene editing vector with a new MOA,” said Albert Seymour, Ph.D., President and Chief Scientific Officer of Homology Medicines. “We believe an innovative approach that combines a permanent correction in the genome while providing expression in all cells that are transduced will be an important advance in the field of gene editing. Preclinical data from our optimized HMI-103 in PKU showed Phe normalization and durability of expression, which is the ultimate goal for treating younger patients whose livers are still growing and dividing.” Also at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, Homology presented data from its long-read, genome-wide integration assays, which confirmed the precision of HR-based integration of HMI-103, including no off-target editing in human hepatocytes in a humanized murine liver model. In addition, Homology shared new data from ...