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Homology Medicines Announces Presentations Across Gene Therapy and Gene Editing Programs, including GTx-mAb, at European Society of Gene & Cell Therapy Meeting
- Data Support PKU and Hunter Syndrome Clinical Trials and PNH Program - BEDFORD, Mass., Oct. 21, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq:
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[{"type":"text","content":"- Data Support PKU and Hunter Syndrome Clinical Trials and PNH Program - BEDFORD, Mass., Oct. 21, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today four presentations of preclinical data spanning its clinical-stage gene therapy program for mucopolysaccharidosis type II (MPS II, or Hunter syndrome), clinical-stage gene editing program for phenylketonuria (PKU), GTx-mAb program for paroxysmal nocturnal hemoglobinuria (PNH) and assays to evaluate levels of pre-existing antibodies to the Company’s adeno-associated viral vectors (AAVHSCs) during the 2021 European Society for Gene & Cell Therapy Virtual Conference (ESGCT). “This week’s data presentations showed the breadth of preclinical work undertaken to support our three clinical trials, and more recent development efforts to expand our gene therapy platform to deliver and generate antibodies in the liver,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “We shared additional preclinical data from studies of our single-dose, I.V. gene therapy candidate for MPS II, and showcased gene editing data in the PKU and humanized murine models, including on- and off-target assessment confirming the precision of our nuclease-free in vivo gene editing. Encouraging data from our GTx-mAb platform showed a single dose resulted in sustained and robust expression of full-length antibodies from the liver consistent with anti-C5 therapeutics. Further, we presented details of the methods of our neutralizing antibody assays used in our clinical trial screening.” Building on this week’s Phase 1 trial initiation for HMI-203 and supportive data presented at ASHG, Homology featured central nervous system (CNS) data in the presentation titled, “Blood-Brain-Barrier Crossing Leads to Long-Term Efficacy in the CNS of HMI-203: Gene Therapy Development Candidate for Mucopolysaccharidosis Type II (MPS II), or Hunter Syndrome,” which showed a single I.V. dose of HMI-203 in the MPS II murine model: Crossed the blood-brain-barrier and blood-cerebrospinal-fluid-barrier and led to widespread brain transduction;Led to dose-dependent transduction, expression and I2S activity, as well as dose- and time-dependent glycosaminoglycan-heparin sulfate (GAG-HS) and LAMP1 reductions in the brain;Achieved brain I2S activity levels comparable ...