LIFE-CHANGING INNOVATIONS

Targeting genetic diseases with no treatment options available today
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DISEASE AREAS

We believe that diseases caused by mutations of a single gene (monogenic indications) are a fundamental driver of therapeutic success in genetic diseases – and that this offers the greatest potential to provide life-changing treatments to patients.

We have a deep understanding of the processes that enable gene expression to optimise RNA therapies and their delivery systems. Our goal is to translate this expertise into potentially curative, life-changing therapeutic options.

There is an urgent need to create treatments for patients with rare genetic diseases – approximately 6,000 known rare diseases affect 400 million people worldwide.1

Our pipeline of RNA therapies

Our pipeline is initially focused on three core therapeutic areas – the ocular, central nervous and renal systems – although our drug design capability and proprietary drug delivery platform has broad potential to expand to numerous further indications.
ORGAN
PROGRAM & TARGET
STATUS
An icon of a white eye.
VP-001*
PRPF31
Retinitis Pigmentosa type 11
Clinical trials
DiscoveryPre-clinicalIND-enablingClinical trialsMarketed
An icon of a white eye.
PYC-001
OPA1
Autosomal Dominant Optic Atrophy
Clinical trials
DiscoveryPre-clinicalIND-enablingClinical trialsMarketed
An icon of two white kidneys connected by a large blood vessel.
PYC-003
PKD1
Autosomal Dominant Polycystic Kidney Disease
IND-enabling
DiscoveryPre-clinicalIND-enablingClinical trialsMarketed
A white icon of the brain from a birds eye view.
PYC-002
SHANK3
Phelan-McDermid Syndrome
Pre-clinical
DiscoveryPre-clinicalIND-enablingClinical trialsMarketed
PYC Therapeutics logo (white version)
Not disclosed
Discovery programs
Discovery
DiscoveryPre-clinicalIND-enablingClinical trialsMarketed
*PYC owns 96.2% of VP-001 (3.8% ownership by Lions Eye Institute, Australia).

Our disease areas

Explore the conditions for which we are developing life-changing therapeutics.
ocular system

Retinitis Pigmentosa type 11 (RP11)

RP11 is a devastating, progressive and blinding eye disease that begins in childhood, for which there are no available treatments. PYC has developed VP-001, a first-in-class and potentially disease-modifying drug that addresses the underlying genetic cause of RP11. VP-001 was the first treatment for RP11 to enter clinical trials and is currently progressing through mid-stage clinical studies.

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ocular system

Autosomal Dominant Optic Atrophy (ADOA)

ADOA is a debilitating, blinding eye disease caused by insufficient gene expression of the OPA1 gene in optic nerve cells of the eye. There are no current treatments available for ADOA patients. PYC has developed PYC-001, a potentially disease-modifying drug that is unique in its potential for full restoration of cell function. PYC-001 is currently in a Phase 1 clinical trial.

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renal system

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

ADPKD is a severe disease that causes cysts to form in a patient’s kidneys, which can ultimately lead to kidney failure, dialysis and transplantation. There are no available therapies for patients with ADPKD that address the underlying cause of the disease. PYC has developed PYC-003, a treatment that could arrest the disease course and enable regeneration and restoration of kidney function.

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central nervous system

Phelan-McDermid Syndrome (PMS)

PMS is a severe neurodevelopmental disease that can cause a wide range of medical, intellectual and behavioural challenges, including life-long intellectual and physical disability. There are no available treatments for patients with PMS. PYC has developed PYC-002, the first potentially disease-modifying therapeutic for PMS that treats the disease at the genetic level.

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References

  1. Haendel M, Vasilevsky N, Unni D et al. How many rare diseases are there? Nat Rev Drug Discov. 2020 Feb;19(2):77-8. doi: 10.1038/d41573-019-00180-y. ↩︎
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