Optogenetics against retinitis pigmentosa

Optogenetics-based therapy
will save people from blindness

RVC Press Service

The portfolio company of the RBV Capital venture fund, created with the participation of RVC capital, RetroSense Therapeutics has started clinical trials of a drug for partial restoration of vision in patients suffering from retinitis pigmentosa. A unique method of gene therapy based on optogenetics opens up new ways in the fight against previously incurable diseases.

Retinitis pigmentosa is an inherited incurable degenerative eye disease that causes severe visual impairment and blindness due to damage to the light–sensitive cells of the retina. Existing drugs only slow down the process of vision loss, but do not prevent it. Today, about 50,000 people in Russia and 100,000 people in the CIS suffer from retinitis pigmentosa.

"The successful administration of the drug RTS-001 to our first patient is of great importance for the development of the program and evaluation of the potential effectiveness of the use of optogenetic methods of gene therapy both in the case of retinitis pigmentosa and in other areas of ophthalmology. We hope that the drug will become a powerful tool for partial restoration of vision in cases that are considered incurable today," said Sean Ainsworth, CEO of RetroSense.

RetroSense Terapeutics is a private biopharmaceutical company developing a drug to improve the quality of life of people suffering from retinitis pigmentosa and age–related macular degeneration. In 2015, the RBV Capital venture fund, together with a pool of international investors BlueWater Angels, ExSight Capital and Santen Pharmaceutical Co., Ltd., one of the leading pharmaceutical companies in the field of ophthalmology, invested $6 million USD in the company RetroSense Terapeutics in the investment round. The total amount of investments received by the company in two rounds reached $13 million.

The beginning of clinical trials was the result of many years of research and collaboration between leading universities and specialists, including technology developers Professor Zhuo-Hua Pan and Professor Richard Masland. Optogenetics is used to impart photosensitivity to cells that previously did not possess it. The first phase of the safety and tolerability study of the drug RTS-001 involves four groups of volunteers who have almost completely lost their eyesight. The experiment is carried out in 2 stages. At the first stage, three dose levels of RTS-001 are administered to patients. At the second stage, the experiment is carried out with the highest dose and a clinical base is created for the development of a further program to study the effectiveness of the drug.

In 2014, the American Food and Drug Administration (FDA) granted RetroSense Therapeutics a special Orphan Drug designation status and permission to develop and conduct clinical trials of RTS-001 for the treatment of retinitis pigmentosa, from which about 100 thousand people suffer in the United States.

RetroSense Therapeutics is a private American biotechnology company that develops drugs to improve the quality of life based on gene therapy in order to restore the vision of patients suffering from pigmented rhinitis and age–related macular dystrophy. Currently, there are no registered drugs for the treatment of these diseases on the US market. The basis for the development is the unique research of scientists from Wayne State University and Massachusetts General Hospital, the rights to which are owned by RetroSense. RetroSense is managed by a team of managers with experience in product and technology development from invention to completion of clinical trials.

RBV Capital Venture Fund under the management of RusBio Ventures Management Company was formed at the end of 2014 by venture market professionals with successful experience of domestic and international transactions in the field of bio-medicine. The Fund became the first large independent investment partnership established in the Russian Federation. The anchor private investor of the Fund is the leading Russian private pharmaceutical company CJSC R-Pharm.

Portal "Eternal youth" http://vechnayamolodost.ru  24.03.2016