Astellas, Harvard Partner on Retinitis Pigmentosa Targets

Constance Cepko (Harvard Medical School)

14 October 2014. Astellas Pharma Inc. in Tokyo is collaborating with a genetics and ophthalmology lab at Harvard University to discover more about the onset of the eye disease retinitis pigmentosa and identify therapy targets. Financial details of the three-year deal with Harvard were not disclosed.

Retinitis pigmentosa is a family of inherited eye disorders that result in damage to the retina, specifically breakdown and failure of photoreceptor cells in the retina leading to progressive vision loss. The disease takes several forms, but it generally results in failure of photoreceptor cells detecting light and color, and helping eyes see in dim light. As a result, people with retinitis pigmentosa may experience symptoms such as night blindness and loss of peripheral vision. The organization Research to Prevent Blindness says some 100,000 people in the U.S. have retinitis pigmentosa.

The collaboration engages the lab of Constance Cepko, a professor of genetics and ophthalmology at Harvard Medical School. Cepko’s lab conducts research on the role of genetics in eye disorders, including work on genetic causes of photoreceptor cell loss and design of gene therapies to address these disorders. The lab is also studying use of benign viruses — those not causing disease — to trace nerve cell pathways in the eyes.

For this partnership, Cepko and colleagues will harness adeno-associated viruses, benign viruses for delivering gene therapies, to identify and verify genes associated with prolonging healthy vision among people inheriting retinitis pigmentosa. If the studies by Cepko’s lab lead to promising treatment options, Astellas will negotiate an exclusive license of the technologies for further development and commercialization.

The deal with Harvard is one of the first collaborations generated by Astellas’s Innovation Management division that started in October 2013. The division, says Astellas, establishes partnerships with labs outside the company to find opportunities in preclinical research.

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