Initial Clinical Data of First Pediatric CLN2 Patient Dosed with RGX-181 Presented at SSIEM Annual Symposium
Retrieved on:
Wednesday, August 30, 2023
Seizure, Society, Therapy, TPP1, Neurodegenerative disease, Kia Challenge, Gene expression, Accelerated approval (FDA), Language, AAV, Hunter syndrome, Batten disease, Death, ERT, Patient, Child, Hospital, Enzyme replacement therapy, Safety, CLN2, BLA, Report of the 1st Annual Symposium on Relaxed Improvisation, Peptide, Disease, Pharmaceutical industry, Metabolism
"CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.
Key Points:
- "CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.
- "We are encouraged by the initial results demonstrating that RGX-181 is well tolerated and dramatically reduced the number of seizures in the patient enrolled in this trial."
- "The remarkable decrease in seizures, encouraging safety results and reduction in ERT frequency highlight the potential of this gene therapy to provide a meaningful treatment option to the CLN2 patient community."
- Today, a physician investigator from the Hospital de Clinicas in Porto Alegre, Brazil reported initial results from a five-year-old child who received a one-time intracisternal dose of RGX-181.