In a major incidence across clinical laboratory services market, Audentes Therapeutics, Inc. one of the most renowned biotechnology companies, has scarcely declared that its latest gene therapy product, the AT342 has received the approval for its CTA (Clinical Trial Authorization) application from the Medicines and Healthcare Products Regulatory Agency (MHRA). Apparently, this gene therapy product candidate has been developed for the treatment of the Crigler-Najjar Syndrome by the San Francisco headquartered biotech giant, which specializes on the development and commercialization of gene therapy products for patients afflicted with life-threatening diseases.For the uninitiated, AT342 is essentially an AAV8 vector that contains a copy of the UGT1A1 gene, useful for the treatment of Crigler-Najjar Syndrome, that is a rather rarely occurring, monogenic disease, specifically defined by high levels of unconjugated bilirubin content in blood.
For the record, this approval marks a compelling significance in clinical laboratory services industry, given that this is the debut European CTA approval for the AT342 program. Reportedly, this approval can now enable Audentes to commence the process of enrollment at all the clinical sites across the UK for AT342’s Phase 1/2 clinical study – VALENS.
As per reports, VALENS is basically an open-label, multinational dose study that is conducted in order to evaluate the preliminary efficacy and the safety parameter of AT342 in 12 patients above the age of one, suffering from the Crigler-Najjar syndrome. The study has also been anticipated to include nine patients who have undergone treatment with AT342 and three delayed-treatment, concurrent control patients. Sources familiar with the matter claim that the main endpoints of the study are efficacy and safety.
Sources cite that apart from VALENS, AT342’s clinical development program is also inclusive of LUSTRO, a natural history run-in study, that has also made its presence felt across clinical laboratory services market. LUSTRO’s main objectives are to basically serve as a longitudinal baseline for VALENS, analyze the clinical condition of the patients suffering from the Crigler-Najjar Syndrome, and recognize subjects that may be potentially eligible for enrollment in the VALENS.