JR-446 is a blood-brain barrier-penetrating αN-acetylglucosaminidase enzyme replacement therapy for Sanfilippo Type B developed by JCR using its proprietary J-Brain Cargo® technology.
JCR and MEDIPAL shared the following in a public statement:
“Dosing the first patient in this Phase I/II clinical trial is an important milestone for the MPS IIIB community, as there is no approved treatment for this devastating and life limiting lysosomal storage disorder that impacts patients’ neurocognitive development, independence, and quality of life,” said Dr. Irene Chang, Principal Investigator, Associate Professor of Pediatrics, Section of Biochemical Genetics, Division of Medical Genetics, at the University of California, San Francisco (UCSF). “Patients and families affected by this condition continue to face significant unmet medical needs, and we are encouraged to take this next step in evaluating the potential of this investigational therapy. We are grateful to the patients, caregivers, and study teams whose commitment made this milestone possible, and we are hopeful it may bring meaningful improvements in the quality of life for individuals living with MPS IIIB and their families.”
Read more about JR-446, including information about the clinical trials, therapeutic method, and therapy history.