LIFE-CHANGING RESEARCH

Translating scientific advances into meaningful treatments
An image of a woman wearing a white lab coat, clear goggles and blue gloves in a lab. Her hair is plaited and lying across her right shoulder. She is standing in front of a scientific machine and her right hand is resting on the machine while she smiles at the camera.
OUR CLINICAL TRIALS

We are committed to applying our breakthrough RNA technology to the development of life-changing therapies.

We are initially focused on three core therapeutic areas – the ocular, central nervous and renal systems – although our proprietary drug delivery platform has the potential to revolutionise treatment for many genetic diseases.

Patient trials are essential to the development of all novel therapeutics. At PYC, we conduct responsible patient trials to ensure that our treatments are safe and effective. These trials rely on patient volunteers and are crucial to the development of disease-modifying therapies for genetic diseases.

Current trials

An up-to-date overview of our current clinical trials.

Autosomal Dominant Polycystic Disease (ADPKD)

ADPKD Phase 1 study

Trial status: Enrolling

Location: Australia

Contact for participation: [email protected]

PYC is conducting a Phase 1 study to evaluate the safety, tolerability and pharmacokinetics of intravenously administered PYC-003 in healthy volunteers and ADPKD patients.

SRC: safety review committee.

This is a first-in-human multi-centre Phase 1a and Phase 1b, single ascending dose (SAD) interventional study of PYC-003 in healthy volunteers and patients with confirmed PKD1 mutation-associated Autosomal Dominant Polycystic Kidney Disease.

If you are interested in learning more about this clinical trial, visit clinicaltrials.gov or pkdstudy.org, or email [email protected].

Phelan-McDermid Syndrome (PMS)

Clinical trial information

PYC’s PMS program is expected to advance into clinical trials in 2026.

For more information please email [email protected].

Autosomal Dominant Optic Atrophy (ADOA)

ADOA Phase 1a study

Trial Status: Enrolling

Location: Australia

Contact for participation: [email protected]

PYC is conducting a Phase 1a open-label, single ascending dose study to evaluate the safety and tolerability of intravitreally administered PYC-001 in participants with confirmed OPA1 mutation-associated autosomal dominant optic atrophy.

SRC: safety review committee.

This is a first-in-human multi-centre, prospective, Phase 1a, single ascending dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.

If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for more information or email [email protected].

ADOA Phase 1b study

Trial Status: Not Yet Recruiting (starting Sep 2025)

Location: Australia and United Kingdom

Contact for participation: [email protected]

 PYC is conducting Phase 1b Open-Label, Randomized, Single Dose and Repeat Dose Study to Evaluate the Single and Repeat Dose Safety and Tolerability of Intravitreally Administered PYC-001 in Participants with Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (ADOA)

 Who may be eligible?

A patient may be able to participate in the clinical trial if they meet eligibility criteria including (but not limited to):

  • Adults at least 18 years of age
  • A recent (within 5 years) genetic diagnosis of OPA1 mutation associated ADOA
  • A BCVA of between ≤20/40 and ≥20/200

If you are interested in learning more about this clinical trial, visit clinicaltrials.gov  for more information or email [email protected].

Retinitis Pigmentosa type 11 (RP11)

Clinical trial information

PYC’s RP11 program have closed enrolment for the Natural History Study and Phase 1 clinical trials.

Future trials are being planned. For more information, please email [email protected].