By Cyndi Root

RetroSense Therapeutics announced in a press release that the Food and Drug Administration (FDA) has granted Orphan Drug status to RST-001. The treatment for retinitis pigmentosa (RP) combines gene therapy and optogenetics. RetroSense developed the proprietary technology from research conducted at Wayne State University and Massachusetts General Hospital.

Sean Ainsworth, RetroSense CEO, said, We are hopeful that the benefits associated with Orphan Drug status will better enable us to advance RST-001 through development and ultimately into the marketplace where it may benefit many who are suffering from blindness due to retinitis pigmentosa.

Optogenetics

Retinitis pigmentosa causes the degeneration and loss of rod and cone photoreceptors in the retina, causing severe vision loss and blindness. Currently there are no FDA-approved drugs to treat RP. RetroSenses work in optogenetics involves making the retina more light sensitive, thereby improving vision. The company expects RST-100 to have broad applications and to be useful in heredity or acquired RP.

RST-001 uses a photosensitivity gene, channelrhodopsin-2, and creates new photosensors in the retinal cells. Channelrhodopsin-2 has been shown in numerous animal studies to restore light perception and vision, and in primate studies, the agent was well tolerated. RetroSense is using optogenetics and channelrhodopsin-2 in the pre-clinical stage and hopes to begin clinical trials soon.

Retinitis Pigmentosa and Gene Therapy

Astellas and Harvard recently announced a new partnership to use gene therapy in the study for retinitis pigmentosa. The collaboration is led by Constance L. Cepko, a professor of Genetics and Ophthalmology at Harvard. Using adeno-associated virus vectors (AAVV), the team will identify and characterize genes implicated in RP.

The UKs Telegraph reported early in 2014 that researchers at Oxford University have replaced a missing gene in the retina and reversed blindness. The results startled the investigators who did not expect to see such dramatic improvements. In the study on choroideremia, inherited blindness, scientists put the missing REP-1 protein back in the retina by inserting it into the DNA of a harmless virus and then injecting that DNA into the cells beneath the retina.

Since, a third of eye diseases are hereditary, the researchers are hopeful that the treatment is applicable to various eye diseases and conditions. The research team at Oxford is developing a Phase 2 trial on the investigational therapy.

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FDA Awards Orphan Status To RetroSense's RST-001 For Retinitis Pigmentosa