Date: 2016-04-04
Type of information: Licensing agreement
Compound: human rhodopsin based optogenetic gene therapy
Company: Acucela (USA - MA) The University of Manchester (UK)
Therapeutic area: Ophtalmological diseases - Genetic diseases - Rare diseases
Type agreement: licensing
Action mechanism: gene therapy.
Disease: retinal degenerative diseases including retinitis pigmentosa .
Details: * On April 4, 2016, Acucela, a clinical-stage ophthalmology company that specializes in identifying and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases, and The University of Manchester announced an exclusive license agreement whereby Acucela will develop and commercialize UoM’s human rhodopsin based optogenetic gene therapy for the treatment of retinal degenerative disease, including retinitis pigmentosa . If approved, this genetic mutation-independent therapy, initially developed by Dr. Jasmina Cehajic-Kapetanovic and Professors Robert Lucas and Paul Bishop at the University of Manchester, will utilize a viral vector to transduce the ON-bipolar cells of the retina with human rhodopsin, a light sensitive protein normally expressed in the rod photoreceptors. Under the control of a cell specific promoter, this technology has been shown to be effective at restoring visual response in a mouse model of retinal degeneration. Additionally, rhodopsin’s biological mechanism imparts a relatively high level of light sensitivity for this type of therapy and as a human protein, may minimize any immunological impact. This optogenetic technology will be studied across a variety of genetic mutations in retinitis pigmentosa and related conditions, and as such may be useful as a “pan-RP” therapy that is mutation-independent. Acucela plans to ultimately evaluate the ability of this technology to restore some level of vision in patients who are legally blind. The agreement was negotiated on behalf of The University of Manchester by its technology transfer office, UMIP.
Financial terms:
Latest news:
