ADOA

PYC-001 is in Phase 1/2 clinical trials
Autosomal Dominant Optic Atrophy (ADOA) is a debilitating, blinding eye disease caused by insufficient gene expression of the OPA1 gene in optic nerve cells of the eye.
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ADOA

PYC-001 is in Phase 1/2 clinical trials
Patients experience progressive and irreversible vision loss, with many experiencing their first symptoms of vision loss before 10 years of age. There are no current treatments available for ADOA patients.
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ADOA

PYC-001 is in Phase 1/2 clinical trials
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 clinical studies in ADOA patients and is the first precision therapy to be dosed in patients with ADOA.
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The human impact of ADOA

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10 years old

Patients are usually diagnosed in early childhood.

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30 years old

At first, patients lose their central vision and then their peripheral vision.

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50 years old

Patients are often declared legally blind at around 50 years of age, which significantly impacts their quality of life.

ADOA at a glance

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An urgent need to create treatments

ADOA affects 1 in every 35,000 people.1,2

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ADOA is a monogenic disease

ADOA is caused by a mutation in one of the patient’s two copies of the OPA1 gene in the retinal ganglion cells (RGCs) that form the optic nerve of the eye.

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The median age of ADOA onset is 7 years of age

80% of patients experience symptoms before 10 years of age.1

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Loss of central vision

Patients with ADOA initially experience central vision loss and then peripheral vision loss.

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

ADOA patients have a mutation in one copy of the OPA1 gene – this leads to their retinal cells producing half as much OPA1 protein as is required for normal function. PYC-001 seeks to restore the production of OPA1 protein in patients, therefore rescuing function of retinal cells and thus stopping disease progression.

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Encouraging results

PYC’s therapy, PYC-001, seeks to increase OPA1 protein expression in the RGCs to restore cell function and is currently in a Phase 1 clinical trial.

Mechanism of action of PYC-001 for ADOA

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

References

  1. Yu-Wai-Man P, Griffiths PG, Burke A et al. The prevalence and natural history of dominant optic atrophy due to OPA1 mutations. Ophthalmology. 2010 Aug;117(8):1538-46, 1546.e1. doi: 10.1016/j.ophtha.2009.12.038. ↩︎
  2. Amati-Bonneau P, Milea D, Bonneau D et al. OPA1-associated disorders: phenotypes and pathophysiology. Int J Biochem Cell Biol. 2009 Oct;41(10):1855-65. doi: 10.1016/j.biocel.2009.04.012. ↩︎