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Homology Medicines Announces Presentations on HMI-203 Investigational Gene Therapy for Hunter Syndrome and Broad Applicability of AAVHSC Platform for Lysosomal Storage Disorders at the 18th Annual WORLDSymposium™ Meeting
- Data Supported juMPStart Trial Evaluating One-Time, Systemic Administration of HMI-203 in ERT-Treated Adults - - Details of HMI-203 Clinical Trial Design
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[{"type":"text","content":"- Data Supported juMPStart Trial Evaluating One-Time, Systemic Administration of HMI-203 in ERT-Treated Adults - - Details of HMI-203 Clinical Trial Design and Encouraging Preclinical Data Featured in Platform Presentations - BEDFORD, Mass., Feb. 10, 2022 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today multiple presentations on HMI-203 gene therapy candidate for the treatment of Hunter syndrome (MPS II), which is being evaluated in juMPStart, a Phase 1 open-label, dose-escalation clinical trial in adults with Hunter syndrome. In oral platform presentations, key eligibility criteria and planned endpoints for the juMPStart trial were discussed and data from the HMI-203 IND-enabling studies were presented. Homology also shared patient, caregiver and key opinion leader (KOL) feedback on the unmet medical need in Hunter syndrome, despite the availability of enzyme replacement therapy (ERT), and the potential for a one-time gene therapy for patients that could impact peripheral and central nervous system (CNS) manifestations. These presentations at the 18th Annual WORLDSymposium™ Meeting also included data on Homology’s AAVHSC platform and potential to treat additional lysosomal storage disorders, including metachromatic leukodystrophy (MLD), based on CNS transduction in non-human primates (NHPs). “These presentations highlight how Homology’s gene therapy approach to Hunter syndrome and other lysosomal storage diseases is developed to target both the peripheral as well as the CNS manifestations of these multi-organ disorders,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “We designed the juMPStart clinical trial with our one-time, systemic gene therapy candidate by incorporating data and feedback we generated from our IND-enabling studies, as well as patient, caregiver and physician feedback. We believe that HMI-203 has the potential to address the peripheral and CNS challenges of Hunter syndrome that are not addressed by standard of care ERT, and that a one-time I.V. administration would be a major advance for patients and their families.” In the platform presentation titled, “Clinical Trial Design for HMI-203 Investigational Gene Therapy for Mucopolysaccharidosis Type II (MPS II) Informed by Cross-Correction Potential and Key Opinion Leader In...