Agreement formed for development of AAV vectors for ocular gene therapy

Posted on: Tuesday 9 January 2018

Sirion Biotech GmbH, a virus-based technology and gene delivery company, announced that it has signed a two-year development agreement with Acucela, a clinical-stage ophthalmology company.

Recombinant adeno associated virus (rAAV) vectors are considered a promising gene delivery system for therapeutic applications and have been shown in preclinical experiments to successfully deliver human rhodopsin (hRho) to the retina. Acucela, together with Sirion and an academic partner consortium, aims to develop the next generation of rAAV vectors. The goal is to secure new and modified AAV capsids that will ensure the therapeutic viral particles exhibit a safe product profile with improved specificity for therapeutic protein delivery over and above wild type vectors, to effectively restore light sensitivity to patients.

SIRION relies on ten years of experience in viral vector engineering and leverages close ties to leading academics in Europe that will play a pivotal role in this project. Experts in AAV biology, Prof. Grimm from the Unversitätsklinikum Heidelberg and Prof. Büning of the Medizinische Hochschule Hannover, as well as PD Dr.Michalakis from the Ludwig-Maximilians-Universität München, will contribute their individual expertise to this program.

“The academic acumen of our partners, together with our strong viral vector specialization and experience, will empower our client to enter clinical trials with an efficient, safe and scalable product. For us it is the chance to participate in a fundamental step for gene therapies that can help millions of patients worldwide win back their eyesight. This is what we started SIRION Biotech for,” said Dr. Christian Thirion, founder and CEO of the company.