Industry News: SIRION Biotech and Acucela Set Sights on Gene Delivery Tools for Ocular Gene Therapy

New partnership aims to tackle Retinitis pigmentosa with adeno-viral gene therapy

22 Jan 2018

SIRION Biotech GmbH, a world leader in virus-based technology innovation and gene delivery expertise, has announced that it signed a two-year development agreement with Acucela Inc. Acucela is a clinical-stage ophthalmology company and a wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. They are also committed to establishing optimized AAV vectors for clinical applications in ocular gene therapy.

With this cooperative effort, Acucela bolsters its move into the fast-growing gene therapy market to find a genetic cure for retinitis pigmentosa (RP). RP is the most common form of inherited retinal degeneration. Onset is usually in childhood and progresses to complete blindness by around age 40. It is estimated that approximately 1 in 4000 people are affected in their lifetime, and approximately 1.5 million people worldwide are affected by the disease. RP in fact describes a family of slowly progressing diseases that are caused by a variety of autosomal dominant, autosomal recessive and x-linked mutations that lead to photoreceptor degeneration. It is a complex disease with over 100 pathogenic mutations identified. 

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To date, no pharmacological treatment is available. Recombinant adeno associated virus (rAAV) vectors are considered the best and most 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 10 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. World renowned 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. Additionally, Dr. Ryo Kubota, MD, PhD, Chairman, President and CEO of Acucela stated, “We are pleased to advance our optogenetic gene therapy program in partnership with SIRION Biotech to bring a new therapeutic approach to RP patients suffering from this devastating disease.”

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