21.12.2022 13:49:54
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Regenxbio To Start Phase I/II Study Of RGX-381 In H1 2023
(RTTNews) - Regenxbio Inc. (RGNX) Wednesday updated developmental progress of RGX-181 and RGX-381, the company's one-time gene therapy candidates for the treatment of different manifestations in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
In Brazil, first child patient with CLN2 disease was dosed under a single-patient investigator-initiated study of RGX-181. The drug was well tolerated and no drug-related SAEs were reported as of December 20, the company said.
Regenxbio also said that a clinical trial application (CTA) has been cleared by the UK Health Authority to initiate Phase I/II study of RGX-381 for the treatment of ocular manifestations of CLN2 disease.
The company plans to start Phase I/II study of RGX-381 in the first half of 2023.
RGX-181 and RGX-381 have received Orphan Drug and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration, as well as ATMP classification from the European Medicines Agency.
Neuronal ceroid lipofuscinosis (NCL) are a group of genetically mediated neurodegenerative disorders affecting children and young adults.
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