With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...
Researchers have described the neurodegeneration that occurs in the nervous system of the bowel in Batten disease, a rare and fatal genetic condition. In their latest study, a team showed that gene ...
Mucopolysaccharidosis (MPS ... others face life-threatening complications early in life. Enzyme replacement therapy (ERT) has emerged as a key treatment option, aiming to supplement the deficient ...
Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the ...
GlobalData on MSN11d
Regenxbio and Nippon Shinyaku forge $810m gene therapy dealRegenxbio will receive $110m upfront from Nippon Shinyaku and is eligible to get up to $700m in development, regulatory, and ...
Five children under one year of age with the condition also known as mucopolysaccharidosis type II (MPS II) will be treated with autologous hematopoietic stem cell (HSC) gene therapy. The children ...
Regenxbio, a prolific developer of gene therapy technology, will hand Japan’s Nippon ... The other treatment, RGX-111, treats ...
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