PYC Doses First Patient in Clinical Trial of RNA Therapy for RP11 (PRPF31 Mutations)

PYC Therapeutics, an Australia-based developer of RNA therapies, has launched a Phase 1 clinical trial for its RNA therapy known as VP-001 for people with retinitis pigmentosa 11 (RP11) which is caused by mutations in the gene PRPF31. The 20-person clinical trial is taking place in the US. The first patient was dosed at the Retina Foundation of the Southwest in Dallas. Three doses of VP-001 are being evaluated. VP-001 is administered through an intravitreal injection. Investigators will be evaluating safety as well as a number of measures of retinal structure and visual function.

“We are delighted to initiate a clinical trial of the first therapy specifically for RP11 patients,” says Sri Mudumba, Ph.D., chief research and development officer at PYC. “We believe our innovative RNA therapy and delivery technologies are ideal for targeting inherited retinal diseases. These conditions are a critical unmet need.”

PYC has reported that approximately 1 in 100,000 people have RP11. That equates to nearly 80,000 people affected worldwide.

PYC’s emerging therapies are designed to modify RNA, the genetic messages that cells read to make the proteins which are critical to the health and function of all the cells in the body. By modifying RNA, protein expression can be boosted or reduced, depending on the therapeutic need.

In people with RP11, one copy of their PRPF31 gene is normal and producing a relatively normal level of protein while the other PRPF31 copy is mutated and not producing sufficient protein. The overall reduced level of PRPF31 protein for RP11 patients leads to retinal degeneration and vision loss.

Researchers from PYC found that by downregulating the activity of a different gene, CNOT3, they could boost PRPF31 protein expression. So, they developed VP-001, a tiny piece of synthetic genetic material designed to alter the RNA expressed by the gene CNOT3, thereby increasing PRPF31 protein expression.