REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they’re developing, RGX-121. RGX-121 is a gene therapy for children with the rare disease Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. MPS II is a rare, progressive disorder, caused by a genetic mutation. To learn more about MPS II, click here.
Researchers hope to develop a therapy that involves only one dose, and can stop neurocognitive decline in patients with MPS II. The company expects to start enrolling patients in the trial in the first half of next year.
Read about it on Globe Newswire.
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