HMI-203 Leverages Ability to Cross Blood-Brain-Barrier Following I.V. Administration, and Preclinical Data ShowedImprovements in Key Disease Biomarkers
Program Expands Homology’s CNS Portfolio
“We are pleased to unveil our third gene therapy program, which leverages the ability of our AAVHSC vectors to cross the blood-brain-barrier and blood-nerve-barrier, as well as reach other peripheral organs involved in MPS II. Our in vivo approach broadens our CNS portfolio and differentiates Homology’s HMI-203 from other programs in development for Hunter syndrome,” stated
In preclinical studies, a single I.V. administration of HMI-203 resulted in systemic and CNS transduction and I2S expression, leading to a significant reduction in GAG levels in the brain, liver, heart, spleen, lung and kidney, compared to the vehicle-treated disease model. Prevention of phenotypic symptoms was also observed in the model. Secreted I2S collected from the in vivo preclinical model following HMI-203 administration was taken up by human cells in an additional in vitro experiment, which supports the potential of HMI-203 to result in cell cross-correction.
Homology plans to present data from the HMI-203 program at upcoming scientific conferences as it continues its IND-enabling studies.
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.
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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,including with respect to the ongoing IND-enabling studies for HMI-203 and related regulatory submissions and presentation of data 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; 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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