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Homology Medicines Announces Peer-Reviewed Publication Describing Molecular Characterization of Precise In Vivo Nuclease-Free Gene Editing with PKU Program
- Methods Showed Efficient On-Target Gene Integration with No Unintended DNA Modifications - BEDFORD, Mass., May 26, 2020 (GLOBE NEWSWIRE) -- Homology
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[{"type":"text","content":" - Methods Showed Efficient On-Target Gene Integration with No Unintended DNA Modifications -\n BEDFORD, Mass., May 26, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the peer-reviewed publication of methods used to evaluate the on-target efficiency and precision of Homology’s in vivo phenylketonuria (PKU) gene editing program. These quantitative molecular methods provide a framework to characterize homologous recombination-based, nuclease-free gene integration and evaluate whether any unintended on-target mutations or viral insertions occurred. “As we continue to develop our AAVHSC-based gene editing technology into new treatment options and potential cures for patients, we believe that it is important to employ quantitative molecular methods to characterize changes to the genome,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “We used multiple molecular methods to assess our gene editing technology, which demonstrated precise PAH gene integration into the target location without introducing any unintended mutations or viral components. We believe that the framework described herein represents a major advancement in the field of AAV-based gene editing and, going forward, should now enable the ability to make direct data comparisons across studies.” In the publication, one of Homology’s family of 15 adeno-associated viral vectors derived from human hematopoietic stem cells (AAVHSC15) was used to deliver the human PAH gene into the murine model with human hepatocytes. This construct is designed to leverage the body’s natural DNA repair process of homologous recombination, without requiring a nuclease to cut the DNA. Key findings in the publication include: Single I.V. administration of the AAVHSC15-based gene editing construct resulted in seamless integration of the human PAH gene in human hepatocytes in the murine model To determine whether gene integration occurred at the human PAH locus, the study employed a targeted integration (TI) PCR, and the TI amplicon was then purified and Sanger sequenced. The resulting data matched with reference, confirming PAH gene integration.Gene integration was precise without de novo mutations Next-generation sequence analysis spanning the full integration site revealed there were no unintended on-tar...