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Initial Clinical Data of First Pediatric CLN2 Patient Dosed with RGX-181 Presented at SSIEM Annual Symposium
Six-month data from single-patient, investigator-initiated trial showed that one-time administration of RGX-181 was well tolerated, achieved sustained gene
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[{"type":"text","content":"Six-month data from single-patient, investigator-initiated trial showed that one-time administration of RGX-181 was well tolerated, achieved sustained gene expression and demonstrated clinically meaningful improvements across multiple measures including reduced (86%) seizure frequency.Investigators observed encouraging neurodevelopmental skill acquisition at 6 monthsROCKVILLE, Md., Aug. 30, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that initial interim data from a first-in-human single-patient, investigator-initiated trial of RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem.\n\"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. Symptoms include seizures; loss of motor, language, and cognitive skills; vision loss and premature death; and existing treatments do not stop or reverse most manifestations of the disease,\" said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. \"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.\"\n\"As someone who treats patients with this devastating disease, I see the limitations of the current standard of care,\" said Carolina Fischinger de Souza, M.D., Ph.D., Hospital de Clínicas de Porto Alegre, Brazil and investigator of 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.\"\nData Summary and Safety Data\nToday, 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. Time of post-administration follow up was six months.\nAs of June 30, 2023, RGX-181 was well tolerated with no serious adverse events. Key efficacy measures demonstrated sustained levels of TPP1 along with increased intervals between enzyme replacement therapy...