Avexis has announced the imminent start of its first gene therapy trial for patients with spinal muscular atrophy (SMA) Type 2 in the United States.
After initiating a pivotal gene therapy trial in SMA Type 1 patients (STR1VE trial) last September and following encouraging preliminary results from its Phase I trial in SMA Type 1, Avexis has initiated a new gene therapy trial with its AVXS-101 product in SMA Type 2.
This Phase I trial, called STRONG, will take place in the United States. Twenty-seven participants who are able to sit but cannot stand or walk will be divided into two groups: a first group of participants under 2 years old (3 participants) treated with a first dose of AVXS-101 and a second group of 2- to 5-year-old participants (24 participants) treated with a second dose of AVXS-101.
In this trial, the AVXS-101 product will be administered intrathecally in the cerebrospinal fluid (lumbar puncture), in contrast to the STR1VE trial in which the product was administered intravenously.
Read the Press release «AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101 »
