Homology Medicines Announces First Presentation of Data with HMI-203 In Vivo Gene Therapy Development Candidate for Hunter Syndrome
- IND-Enabling Studies Demonstrated Potential of a
- Data at WORLDSymposium™ Support Plans to Initiate a HMI-203 Phase 1/2 Clinical Trial
This Year -
“By leveraging our family of AAVHSC vectors to target both peripheral organs and the central and peripheral nervous systems in preclinical models of Hunter syndrome and MLD, debilitating diseases that have high unmet medical need, we also demonstrated the continued expansion of our CNS platform,” stated
In the poster titled, “HMI-203: Investigational Gene Therapy for Mucopolysaccharidosis Type II (MPS II), or Hunter Syndrome,” a single I.V. administration of HMI-203 in the adult murine model:
- Led to robust biodistribution and sustained human I2S (hI2S) enzyme expression, which resulted in:
- Significant reductions in key Hunter syndrome biomarkers of heparin sulfate glycosaminoglycans (GAGs) and lysosomal-associated membrane protein 1 (LAMP-1) in the brain, liver, heart, spleen, lung and kidneys compared with vehicle.
- Significant reductions in heparan sulfate GAGs in the cerebrospinal fluid (CSF) compared with vehicle.
- Ameliorated paw deformities, as shown by significant changes in measurements of ankle depth, paw width, paw depth and ankle width compared with vehicle.
- Led to uptake of hI2S from the serum of the HMI-203-treated model in human cell lines, demonstrating potential for cell cross-correction.
In an additional poster titled, “HMI-202: Gene Therapy Development Candidate for Metachromatic Leukodystrophy (MLD),” a single I.V. administration of HMI-202:
- Crossed the blood-brain-barrier and blood-nerve-barrier in the murine MLD disease model and in non-human primates (NHPs), with human ARSA (hARSA) detected in neuronal and glial cells.
- Showed durable hARSA activity in the central nervous system of the disease model, with distribution levels resembling those of Arsa in normal age-matched controls.
- Demonstrated significant changes in key MLD biomarkers of LAMP-1, glial fibrillary acidic protein (GFAP), MAL transcript and neuronal sulfatides in the disease model compared with vehicle, similar to age-matched wild type controls.
Homology’s e-poster presentations will take place on
About Mucopolysaccharidosis Type II (MPS II), Hunter Syndrome
MPS II, or Hunter syndrome, is a rare, X-linked lysosomal storage disorder caused by mutations in the iduronate-2-sulfatase (IDS) gene, which is responsible for producing the I2S enzyme that breaks down large sugar molecules, or cellular waste, called glycosaminoglycans (GAGs). Severe Hunter syndrome results in toxic lysosomal accumulation of GAGs that causes progressive debilitation and decline in intellectual function. Hunter syndrome occurs in approximately 1 in 100,000 to 1 in 170,000 males, and the severe form leads to life expectancy of 10 to 20 years.
About Metachromatic Leukodystrophy (MLD)
MLD is a rare lysosomal storage disorder caused by mutations in the ARSA gene. ARSA is responsible for the creation of the arylsulfatase A (ARSA) protein, which is required for the breakdown of cellular components. In MLD, these cellular components accumulate and destroy myelin-producing cells in the peripheral and central nervous system leading to progressive and serious neurological deterioration. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within five to ten years after onset.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; our plans to move forward into a Phase 1/2 clinical trial for HMI-203 this year; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain
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Source: Homology Medicines, Inc.