Changing the lives of patients with inherited diseases

Our Pipeline

Program overview

Indication and stage of development

Organ

Program

Target

Discovery

Lead selection

IND-enabling

Clinical

Eye

VP-001

PRPF31

Retinitis Pigmentosa Type 11

Retinitis Pigmentosa

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PYC-001

OPA1

Autosomal Dominant Optic Atrophy

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CNS

PYC-003

SHANK3

Phelan-McDermid Syndrome

Other

Multiple

Undisclosed

Discovery Pipeline

PYC has 100% ownership of PYC-001 and 93.5% ownership of VP-001 (6.5% ownership by Lions Eye Institute, Australia)

Retinitis Pigmentosa Type 11

Retinitis Pigmentosa (RP) is a genetic eye condition that causes cells in the light-sensitive retina, located at the back of the eye, to degenerate slowly and progressively.

RP causes progressive loss of night and peripheral vision and often leads to legal and sometimes complete blindness.

VP-001 to treat RP-11

Retinitis pigmentosa 11 (RP11) is a dominantly inherited retinal degenerative disease caused by mutations in the PRPF31 (pre-mRNA processing factor 31) gene. PRPF31 mutations cause retinal disease by haploinsufficiency, a disease mechanism that occurs when one copy of a gene is inactivated or deleted, and the remaining functional gene does not produce sufficient protein to preserve normal function. There is no treatment available to patients living with RP11.

The strategy of our lead drug candidate, VP-001 is to overcome the haploinsufficiency that causes RP11 by moderating CNOT3, an “off-switch” (negative regulator) of PRPF31, and thereby increase functional PRPF31 protein from the remaining healthy gene.

Autosomal Dominant Optic Atrophy (ADOA)

Autosomal Dominant Optic Atrophy (ADOA) is an inherited optic nerve disorder which causes blindness. It is characterized by degeneration of the optic nerves and typically starts during the first decade of life. Affected people usually develop moderate visual loss and color vision defects. The severity varies, and visual acuity can range from normal to legal blindness.

ADOA is caused by mutations in the OPA1 gene. The syndrome is inherited in an autosomal dominant manner. There is currently no way to prevent or cure ADOA.

PYC-001 to treat ADOA

Our second lead drug candidate, PYC-001, works by correcting a protein deficiency in cells which are affected by the OPA1 type of ADOA. PYC-001 will use the same proprietary delivery method (i.e. the same CPP) as VP-001 as this CPP can deliver the ‘cargo’ (i.e. PMO) to the retina.

Future Targets

PYC considers treatment across all levels (prevention, halting progression, and regeneration) and understands the strategic advantages of PPMO, and identifies and prioritizes high value targets.

PYC’s objectives for its future targets are two-fold:

To harness the strategic advantages of PPMO to treat retinal and CNS disease.
To advance additional therapeutic modalities and approaches in areas of unmet need.

PYC’s Pipeline Team is currently investigating a number of new ocular and central nervous system targets.

Why RNA therapeutics?

An important advantage of RNA therapy is that it can be used to deliver drugs to previously “undruggable” targets using small molecule-based medicines. For many years, pharmaceutical companies have relied on small-molecule therapeutics to generate drugs, however, their potential is limited as they rely on the “druggability”of the target.

It is estimated that out of the ~20,000 human proteins, only ~3,000 are druggable. As of 2017, approved drugs targeted only 667 human proteins. This not only indicates that more drugs can be developed to reach the 3,000 druggable targets, but also highlights that the vast majority of the 20,000 human proteins remain undruggable. To tap into this unexplored potential, we need to look beyond small-molecule drugs.

Compared to small molecules, however, general RNA drugs are much bigger in size and have a high electric charge. This makes the intracellular delivery of RNA molecules in their native forms across cell membranes quite difficult. Delivering large drugs inside cells has remained the single biggest challenge preventing RNA therapeutics and precision medicine from realizing their full potential.

This is where our unique point of difference comes in. We are developing RNA drugs that can be delivered into the target cell with our proprietary CPP technology, giving us a clear strategic advantage in the RNA therapeutics sector.