RP11

VP-001 is in Phase 1/2 clinical trials
Retinitis Pigmentosa type 11 (RP11) is a devastating, progressive and blinding eye disease caused by under-expression of the PRPF31 gene.
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RP11

VP-001 is in Phase 1/2 clinical trials
Patients experience vision changes from childhood, leading to legal blindness by 40–50 years of age. Disease management consists of visual aids and genetic counselling, but there are currently no available treatments for RP11 that treat the course of the disease.
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RP11

VP-001 is in Phase 1/2 clinical trials
PYC has developed VP-001, a drug candidate that seeks to address the underlying genetic cause of RP11. VP-001 is the first drug candidate to enter clinical trials for RP11 and is currently in a mid-stage clinical study in RP11 patients.
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The human impact of RP11

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6 years old

At first, a child is noticed to be clumsy, especially at night.

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26 years old

Over time, patients experience night blindness and increasing tunnel vision as their peripheral vision begins to decline.

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46 years old

Patients are diagnosed with legal blindness around 40–50 years of age, which significantly impacts their quality of life.

RP11 at a glance

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An urgent need to create treatments

RP11 affects 1 in every 100,000 people.1,2

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RP11 is a monogenic disease

RP11 is caused by a mutation in one of the patient’s two copies of the PRPF31 gene; this mutation causes insufficient production of the PRPF31 protein, which is required for normal retinal function.

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Loss of central vision and legal blindness

Patients with RP11 initially experience night blindness, followed by tunnel vision and ultimately loss of central vision and legal blindness.

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PYC's drug candidate, VP-001, seeks to stop disease progression

RP11 patients have a mutation in one copy of the PRPF31 gene – this leads to their retinal cells producing half as much PRPF31 protein as is required for normal function. VP-001 seeks to restore the production of PRPF31 protein in patients, therefore rescuing function of retinal cells and thus stopping disease progression.

Mechanism of action of VP-001 for RP11

This video provides an overview of the RP11 disease and the mechanism of action for PYC's candidate VP-001.

References

  1. Daiger P, Bowne SJ, Sullivan LS. Genes and Mutations Causing Autosomal Dominant Retinitis Pigmentosa. Cold Spring Harb Perspect Med. 2014 Oct;5(10):a017129. doi: 10.1101/cshperspect.a017129. ↩︎
  2. Sullivan LS, Bowne SJ, Seaman CR et al. Genomic rearrangements of the PRPF31 gene account for 2.5% of autosomal dominant retinitis pigmentosa. Invest Ophthalmol Vis Sci. 2006 Oct;47(10):4579–88. doi: 10.1167/iovs.06-0440. ↩︎