Deal struck to develop retinitis pigmentosa gene therapy

Astellas and CLINO Corporation signed an agreement to develop and sell an adeno-associated virus (AAV) gene therapy to treat retinitis pigmentosa.

The 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 found in green algae.

Preliminary studies in rats showed recovery of light sensitivity in retinal ganglion cells, suggesting that AAV-mVChR1 may restore visual function in patients with retinitis pigmentosa. Professor Hiroshi Tomita, along with his colleagues at Iwate University in Japan, invented the mVChR1.

“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. We will continue to invest management resources in this field,” said Kenji Yasukawa, PhD, chief strategy officer at Astellas.