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Treating Stargardt’s disease by removing lipofuscin

Katairo GmbH awarded orphan medicinal product designation by the EMA

What is Stargardt’s disease?

Stargardt’s disease is a genetic (hereditary) disorder of the eye that leads to progressive loss of sight. Stargardt’s disease is caused by abnormalities in a gene called ABCA4. The ABCA4gene is responsible for the production of a protein called ABCR that regulates the transport of substances in and out of photoreceptor outer segments. In patients with Stargardt’s disease, ABCR does not work properly. This causes lipofuscin deposits to build up inside the retinal pigment epithelium cells, which become damaged and eventually die. Stargardt’s disease is a long-term debilitating disease because the patient’s sight becomes progressively worse and eventually leads to blindness.

Presently, Stargardt’s disease affects approximately 1 to 1.3 in 10,000 people in the European Union (EU). This is equivalent to a total of between 51,000 and 67,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000.

At the time of submission of the application for orphan designation, no satisfactory methods were authorised in the EU for the treatment of Stargardt’s disease.

How is this medicine expected to work?

The substance used by Katairo is expected to be able to enter the cells of the retina, where it attaches to the abnormal deposits that damage the retina cells. Although the way it works is not disclosed in detail, the mechanism involves the break up and degradation of the deposits. The degraded deposits can then be expelled by the cell’s own natural mechanisms, reducing their build-up and therefore the damage to the cell.

What is the stage of development of this medicine?

The evaluation of the effects of Soraprazan in experimental models is ongoing. No clinical trials with soraprazan in patients with Stargardt’s disease have been started to date. The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) adopted a positive opinion on 9 October 2013 recommending the granting of this designation.

About Ulrich Schraermeyer

Prof. Dr. Ulrich Schraermeyer is basic scientist and head of the Section for experimental retinal surgery at the University Eye Hospital in Tübingen. Since 15 years he investigated pathomechanisms and therapeutic approaches around dry and wet AMD with different methods but always in combination with electron microscopy. Electron microscopy offers insights into many still unknown properties, mechanisms, functions and structures of eye tissues. Electron microscopy is a big tool that helps to understand molecular biological and physiological findings.

More information on the website of the Schraermeyer Lab at the Insititute for Ophthalmic Research

About Katairo

Katairo GmbH was founded by Prof. Dr. Ulrich Schraermeyer, Section Head at the University Eye Hospital in Tübingen, and three partners in 2011.

The company, based near Tübingen, Germany, enables the patenting and development of active substances for the effective treatment of lipofuscin associated diseases such as the dry form of age-related macular degeneration (AMD) and Stargardt’s Disease.