Here’s another breathtaking news for gene therapy and especially for Retinitis Pigmentosa treatment.
Astellas Pharma Inc. and CLINO Corporation announced that Astella and CLINO have entered into a license agreement for the development and commercialization of gene therapy to treat Retinitis Pigmentosa.
The gene therapy is Adeno-associated Virus-modified Volvox channelrhodopsin-1 (mVChR1). This therapy was invented by Professor Hiroshi Tomita. He is currently on the Faculty of Engineering in Iwate University. The therapy is an engineered photoreceptive ion channel with the ability to respond to broad wavelengths of lights, created by modifying channelrhodopsins found in green algae.
As stated in the news release:
Modified Volvox channelrhodopsin-1 (mVChR1), is an engineered photoreceptive ion channel with the ability to respond to broad wavelengths of lights, created by modifying channelrhodopsins*3) found in green algae. It has been shown that administration of AAV-mVChR1 in a rat model of retinitis pigmentosa results in expression of mVChR1 in retinal ganglion cells and recovery of light sensitivity of those rats, providing a possibility that AAV-mVChR1 may restore visual functions in patients with retinitis pigmentosa.
Kenji Yasukawa, Ph.D., Chief Strategy Officer, Astellas stated:
We are pleased to enter into this agreement with CLINO whose breakthrough technology has high potential to advance this innovative therapy. We expect to provide this innovative therapy to ophthalmic diseases with unmet medical needs through this collaboration to develop AAV-mVChR1, and to make further contributions to the treatment of patients.
The news release also provides with the terminologies used above:
a therapy to administer genes or cells introduced with genes into a patient’s body.
AAV-mVChR1 (Adeno-associated Virus-modified Volvox channelrhodopsin-1)
Adeno-associated virus vector carrying modified VChR1 gene.
A protein that permeates ions in and out of cells in response to photo stimulations.
Adeno-associated virus vector
A small non-pathogenic virus that packages a single-stranded DNA. In gene therapies, it is often used as a carrier (vector) to express therapeutic genes in human bodies