JR-479 / JCR Pharma - LARVOL DELTA

JCR Pharmaceuticals to Present at the 21st Annual WORLDSymposium 2025 (Businesswire) - "The first presentation will spotlight JR-479, an investigational BBB-penetrating β-Hexosaminidase A (rDNA origin) enzyme replacement therapy (ERT) that JCR is developing for the treatment of people with GM2 gangliosidoses....The second presentation will highlight JCR’s lead product candidate, JR-141 (pabinafusp alfa, intravenous infusion), a recombinant fusion protein consisting of a humanized anti-transferrin receptor antibody and iduronate-2-sulfatase for the treatment of people with mucopolysaccharidosis type II (MPS II, or Hunter syndrome)."

Clinical data • Preclinical • Hunter Syndrome • Lysosomal Storage Diseases