Flipping the switch on RP: SparingVision doses patients in their first in-human clinical trial for SPVN20
SparingVision has begun its first human trial of SPVN20, a gene-agnostic therapy designed to restore vision in people with advanced Retinitis Pigmentosa. To read more about this research piece, please see below.
SparingVision have announced this week that they have dosed the first human patients in their phase 1 clinical trial for SPVN20. The company, based in Paris, have begun trials in Belgium, and look to expand their recruitment efforts now in both France and Ireland. SPVN20 is a gene-agnostic treatment in development for advanced Retinitis Pigmentosa (RP). This means that it could potentially work for all types of RP, regardless of the genetic variant.
RP is one of the most common types of inherited retinal disease (IRD), associated with faults in over 100 genes, affecting around 25,000 people in the UK. RP causes progressive vision loss, starting peripherally, due to the loss of light-sensing photoreceptor cells in the retina. There are two types of photoreceptors, cones and rods, which differ in structure, function and location. Rods are located in the periphery of the retina and are responsible for night vision and peripheral awareness. Cones are located in the centre of the retina and give you sharp vision and colour detection in good lighting. To learn more, please watch this short animation video on Inherited Retinal Disease.
How does SPVN20 work:
SPVN20 is an adeno-associated viral vector (AAV) gene therapy, meaning that a small virus, that doesn’t cause illness in humans, is used as a delivery system to introduce or alter genetic material within cells. It is designed to target dormant (non-functioning) cones in individuals with RP, and as such improve visual acuity and colour detection. These dormant cone cells remain alive, despite having lost their light-sensing abilities as a result of the disease.
The AAV will penetrate the dormant cone cells, delivering a gene known as a G protein-gated inwardly rectifying potassium (GIRK) channel. This protein enables a biochemical process to take place, creating a new channel or pathway for converting light into electrical signals for the brain. Due to this mechanism, this therapy is designed to work for those with advanced stages of RP who still have dormant cone photoreceptors, with researchers believing that it won’t stop retinal degeneration from taking place. SparingVision also have another treatment in development, SPVN06, aimed at slowing disease progression in people with rod-cone dystrophy, of which RP is a type. SPVN06 provides copies of a gene that aids survival of the cone photoreceptors responsible for central vision. SparingVision are looking at combining SPVN20 and SPVN06 into one therapy in the future to potentially treat various disease stages.
How is it delivered:
SPVN20 is administered via a one-time intravitreal injection (an injection into the jelly-like substance of the eye). Although an intravitreal injection sounds scary, the eye is numbed with anaesthetic drops before the procedure takes place and the injection part only lasts a few seconds. This means that the main sensation is a brief feeling of pressure and discomfort rather than pain. It is common to feel some soreness and irritation as the anaesthetic wears off, but this shouldn’t last longer than a few days.
What happens in the trial:
The phase 1 trial, known as NYRVANA looks to evaluate the safety, tolerability and efficacy of SPVN20 over 6 months, with a long-term follow up of 5 years. It is an open-label, dose escalation trial of a single intravitreal injection of SPVN20. With the trials beginning in Belgium, SparingVision look to expand recruitment in France and Ireland. The trial is open to those 18+, with advanced RP (no light perception) who retain dormant cone photoreceptors.
To read more about the trial itself, please visit: NYRVANA, or to read more about SparingVision please visit: Home – SparingVision