RGX-181 / REGENXBIO - LARVOL DELTA

REGENXBIO Announces Presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023 (PRNewswire) - "REGENXBIO...announced presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023 in Jerusalem, Israel (August 29 – September 1). Investigators will deliver encore presentations of interim clinical trial data from REGENXBIO's pipeline of one-time investigational AAV Therapeutics for progressive, neurodegenerative lysosomal storage disorders, as well as initial interim data from the single-patient, investigator-initiated study of RGX-181 for the treatment of CLN2 disease."

Clinical data • CNS Disorders • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

First in-human intracisternal dosing of RGX-181 (adeno-associated virus 9 / human tripeptidyl peptidase 1) for a 5-year-old child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): 6 month follow-up (SSIEM 2023) - "Treatment involves biweekly intracerebroventricular enzyme replacement therapy (ERT) with cerliponase alfa via an indwelling port...RGX-181 is a recombinant adeno-associated virus serotype 9 vector (AAV9) containing a human CLN2 expression cassette (AAV9.CB7.hCLN2) designed to induce sustained secretion of TPP1 enzyme in the CNS...Prednisone, tacrolimus, and sirolimus were administered per the protocol’s immunosuppressive regimen...Discussion/Conclusion First-in-human administration of RGX-181 has been well tolerated to date. Observation and data collection are ongoing."

Clinical • P1 data • CNS Disorders • Epilepsy • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • TPP1

First in-human intracisternal dosing of RGX-181 (adeno-associated virus 9 / human tripeptidyl peptidase 1) for a 5-year-old child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): 6 month follow-up (SSIEM 2023) - No abstract available

Clinical • P1 data • CNS Disorders • TPP1

First in-human intracisternal dosing of RGX-181 (adeno-associated virus 9 / human tripeptidyl peptidase 1) for a 5-year-old child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): 6 month follow-up (SSIEM 2023) - No abstract available

Clinical • P1 data • CNS Disorders • TPP1

First in-human intracisternal dosing of RGX-181 (adeno-associated virus 9 / human tripeptidyl peptidase 1) for a 5-year-old child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): 6 month follow-up (SSIEM 2023) - No abstract available

Clinical • P1 data • CNS Disorders • TPP1

First in-human intracisternal dosing of RGX-181 (adeno-associated virus 9 / human tripeptidyl peptidase 1) for a 5-year-old child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): 6 month follow-up (SSIEM 2023) - No abstract available

Clinical • P1 data • CNS Disorders • TPP1