Homology Medicines Inc. (NASDAQ: FIXX) has announced various presentations on HMI-203 gene therapy products for Hunter syndrome (MPS II) treatment. HMI-203 is currently under evaluation in juMPStart, a first phase open-label dose-expansion clinical study, in Hunter syndrome patients.
Homology Medicines presents in MPS II therapy product
Major eligibility criteria and intended outcomes for the juMPStart study were highlighted during oral platform presentations, as well as findings from the HMI-203 IND-supporting investigations. Despite enzyme replacement therapy (ERT) being available, the company also shared patient, care provider, and key opinion leader (KOL) responses on the unmet medical needs for Hunter syndrome, as well as the possibilities for a one-time gene treatment in patients that might affect peripheral and central nervous system (CNS) manifestations.
Based on CNS transduction in animal models, the presentations at the 18th Annual WORLDSymposium Meeting provided data on the company’s AAVHSC platform and the prospect to treat other lysosomal storage diseases, including metachromatic leukodystrophy (MLD) (NHPs).
Interestingly, according to the presentations, the company’s gene therapy strategy for Hunter syndrome and other lysosomal storage illnesses has been developed to treat both the CNS and peripheral features of these multi-organ diseases.
HMI-203 has the potential of addressing peripheral and CNS challenges
The company’s Chief Scientific Officer, Albert Seymour, said, “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.”
New details regarding the juMPStart study, as well as information from IND-enabling studies, were presented in the platform presentation entitled, “Clinical Trial Design for HMI-203 Investigational Gene Therapy for Mucopolysaccharidosis Type II (MPS II) Informed by Cross-Correction Potential and Key Opinion Leader Input.” In addition, there was an accompanying presentation titled, “Summary of Nonclinical Data for a Gene Therapy Developmental Candidate HMI-203 for Mucopolysaccharidosis Type II, or Hunter Syndrome.”