Regenxbio reports data from trial of RGX-181

REGENXBIO 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, or CLN2, disease, a form of Batten disease, were presented at the Society for the Study of Inborn Errors of Metabolism, or SSIEM, Annual Symposium in Jerusalem. 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. As of June 30 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, or ERT, infusions and an 86% reduction in seizure frequency through six months, leading to withdrawal of two anti-epileptic medications. Encouraging improvements in fine motor and expressive language skills were also observed.