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Homology Medicines Announces Presentation of Data Supporting Clinical Programs in MPS II and PKU, including Nonclinical and Patient-Focused Research, at American Society of Human Genetics Meeting
- Oral Presentation on In Vivo Gene Therapy Product Candidate and Data on Gene Editing Candidate Paved Way for Initiation of juMPStart and pheEDIT Trials -
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[{"type":"text","content":"- Oral Presentation on In Vivo Gene Therapy Product Candidate and Data on Gene Editing Candidate Paved Way for Initiation of juMPStart and pheEDIT Trials - BEDFORD, Mass., Oct. 20, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today presentations of nonclinical data and patient and caregiver feedback that support the Company’s clinical programs for mucopolysaccharidosis type II (MPS II), or Hunter syndrome, and phenylketonuria (PKU). During the American Society of Human Genetics (ASHG) 2021 Virtual Meeting, an oral presentation included data from Homology’s IND-enabling studies with HMI-203, a one-time, in vivo investigational gene therapy in development for the treatment of MPS II in the recently initiated Phase 1 juMPStart clinical trial. Patient and caregiver feedback on unmet medical needs in MPS II that informed trial design were also presented. Additionally, Homology shared results from a nonclinical study that demonstrated the precision of its nuclease-free, homologous recombination-based, in vivo gene editing candidate for PKU as the Company starts its first gene editing trial. “Our presentations at ASHG show the holistic approach we have taken to prepare for our Hunter syndrome gene therapy and PKU gene editing trials, demonstrating our commitment to thoroughly evaluating the product candidates and the needs of the patient community ahead of clinical studies,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Armed with patient and caregiver feedback on the unmet medical needs that persist, we recently initiated our juMPStart trial to evaluate a single-dose of HMI-203 in adults with Hunter syndrome. Additionally, integration assays from our in vivo PKU gene editing program demonstrated no evidence of off-target integration in human hepatocytes in a xenograft murine model and support our Phase 1 trial.” In the oral presentation titled, “Long-Term Systemic Expression and Cross-Correction Ability of HMI-203: Investigational Gene Therapy Candidate for Mucopolysaccharidosis Type II or Hunter Syndrome,” data showed that a single I.V. dose of HMI-203 in the MPS II murine model resulted in the following, through 52 weeks (end of study): Systemic expression of vector genomes, transcripts and functional I2S enzyme;Secretion of active I2S into ...