Platymonas subcordiformis Calcium-transmitting Channelrhodopsin 2.0 (PsCatCh2.0) (PsCatCh2.0)

PsCatCh2.0 is an engineered, light-sensitive cation channel derived from the green alga Platymonas subcordiformis, optimized for high light sensitivity and rapid kinetics (Bionic Sight, 2021). It is the therapeutic transgene utilized in optogenetic gene therapy, such as BS01, aimed at restoring vision in patients with advanced retinal degenerative diseases like retinitis pigmentosa (ClinicalTrials.gov, NCT04278131). When expressed in retinal ganglion cells (RGCs) via an adeno-associated virus (AAV) vector, PsCatCh2.0 allows these cells to bypass the degenerate photoreceptor layer and respond directly to light (Nirenberg & Pandarinath, 2012). Upon activation by specific wavelengths, the channel facilitates the influx of cations, primarily sodium and calcium, leading to neuronal depolarization and the transmission of visual information to the brain (Mager et al., 2018). This approach is intended to work in conjunction with an external wearable device that captures visual scenes and projects optimized light patterns onto the retina to provide high-resolution visual restoration. The high sensitivity of PsCatCh2.0 is a critical feature, as it enables the restoration of visual function under ambient light conditions or with lower-intensity stimulation from external devices.

Other names

PsCatChPsChR variantBionic Sight optogenetic transgeneAAV2-PsCatCh2.0

Light-activated cation channel that induces depolarization in retinal ganglion cells to restore visual signaling.