By Dr. Ineka Whiteman, BDSRA Foundation Head of Research & Medical Affairs
BDSRA Foundation would like to share the latest developments in REGENXBIO’s first-in-human gene therapy programs for the treatment of CLN2 Batten disease.
Earlier this month, REGENXBIO announced the first patient has been dosed in the Phase I/II ocular trial of RGX-381 at Great Ormond Street Hospital in the United Kingdom.
RGX-381 is an investigational one-time AAV gene therapy for the treatment of ocular manifestations of CLN2 disease, using the NAV® AAV9 vector to deliver the TPP1 gene directly to the retina. Initial data from this trial are expected to be shared in 2024.
In addition, REGENXBIO has an investigational gene therapy, known as RGX-181, that targets the brain/central nervous system. Physicians in Brazil continue to follow the child who was dosed with RGX-181 under a single patient investigator-initiated study in Brazil. We expect investigators to report initial interim data from this study, including 6-month results, at the upcoming SSIEM (Society for the Study of Inborn Errors of Metabolism) Annual Symposium later this month.
RGX-181 is an investigational one-time AAV therapeutic for the treatment of CLN2, using the NAV AAV9 vector to deliver the TPP1 gene directly to the CNS.
For further information see the full press release by clicking here or email Dr. Ineka Whiteman at research@bdsraaustralia.org.
