PYC Therapeutics

What PYC Therapeutics does

PYC Therapeutics is a publicly listed, clinical-stage biotechnology company (ASX: PYC / OTC: PYCXF) headquartered in Nedlands, Australia, that develops RNA-based oligonucleotide therapeutics for genetic diseases. The company's pipeline consists of four assets: PYC-003, the lead program targeting polycystic kidney disease (PKD) and currently in a Phase 1b Multiple Ascending Dose study with Phase 1a Single Ascending Dose results expected in H2 CY26; VP-001 and PYC-001, two ophthalmology drug candidates being evaluated in a Phase 1/2 trial for inherited retinal diseases with interim data presented at ARVO 2026; and a fourth pipeline asset referenced but not detailed in the company's Q1 2026 update. The underlying technology applies antisense/oligonucleotide chemistry to address genetically defined diseases with measurable molecular endpoints, such as urinary PC1 protein levels, total kidney volume on MRI, and estimated glomerular filtration rate for PKD.

PYC Therapeutics is pre-revenue and does not yet have commercial products; its primary patient segments are individuals with polycystic kidney disease and inherited retinal diseases, while broader rare-disease and developmental-disability patient communities are addressed through disease-awareness initiatives such as the Genetic Testing Action Day. Operations are funded through equity capital markets rather than product sales, with a recent AUD 600-650 million raise in Q1 2026 intended to fund all four pipeline assets through efficacy and safety readouts within the next 24 months and to position the lead program for a registrational combined Phase 2/3 trial and NDA filing. Future commercialization, if approved, is expected to follow a specialty rare-disease launch model via specialist physicians and academic medical centers, consistent with orphan-disease indications. The company has 51-100 employees, is led by CEO Rohan Hockings with Glenn Noronha as Chief Development Officer, and operates with no parent or controlling entity.

Name

PYC Therapeutics

Legal name

PYC Therapeutics Limited

Website

https://pyctx.com

Company type

Public

Founded year

2005

Operating status

Operating

Headcount range

51–100 employees

Short description

PYC Therapeutics is a clinical-stage Australian biotechnology company developing RNA-based oligonucleotide therapeutics for genetic diseases, with four pipeline assets including PYC-003 for polycystic kidney disease and two ophthalmology candidates for inherited retinal disease.

Ownership category

akta.pro rank

Headquarters

HQ city

Nedlands

HQ country

Australia

HQ region

Oceania

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Infrastructure

Revenue model

1. Capital markets funding (pre-revenue): Clinical-stage biotech with no commercial product revenue; operations are funded through equity capital raises, including a recent raise of AUD 600-650 million to strengthen the balance sheet and fund four pipeline assets through clinical milestones and efficacy/safety data readouts within the next 24 months.

Go-to-market motion3 records

Marketing channels4 records

Core offering

PYC Therapeutics is a clinical-stage biotechnology company that develops RNA-based oligonucleotide therapeutics for genetic diseases. The company is pioneering treatments using its proprietary RNA platform technology, with a pipeline targeting polycystic kidney disease (PKD) and inherited retinal diseases (IRDs).

Product overview

PYC Therapeutics is a clinical-stage biotechnology company developing a portfolio of RNA-based (oligonucleotide) therapeutics rather than a software product. The pipeline comprises four assets, three of which are named in the source material: PYC-003 (lead program targeting polycystic kidney disease, currently in a Phase 1b Multiple Ascending Dose study with Phase 1a Single Ascending Dose results expected in H2 CY26), VP-001 and PYC-001 (two ophthalmology drug candidates being evaluated in a Phase 1/2 trial with interim data presented at ARVO 2026), and a fourth undisclosed pipeline asset referenced in the Q1 2026 investor update. The portfolio is funded by an AUD 600-650 million capital raise intended to support clinical advancement with efficacy and safety data expected within 24 months.

Differentiator

Problem solved

Functional benefit

Brands

• PYC-003: Investigational RNA-based therapeutic for polycystic kidney disease (PKD), currently in Phase 1b Multiple Ascending Dose study.
• VP-001
• PYC-001

Products and services

• PYC-003
• VP-001
• PYC-001
• Undisclosed pipeline asset

Quantifiable outcome

• AUD 600-650 million capital raised in Q1 2026 to fund pipeline through upcoming data readouts
• +2 more outcomes

Segments3 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Two partnerships are on record, tiered minor.

• Start Genetic (Genetic Testing Action Day)minorGTM or Marketing Partner · 25 July 2026PYC Therapeutics is a supporting sponsor of Start Genetic's second annual Genetic Testing Action Day, scheduled for July 25, 2026. The campaign aims to raise awareness about the role of genetic testing in helping patients with rare diseases, developmental disabilities, and other conditions obtain diagnoses. A cited Medicaid claims analysis found only 16.9% of children with autism receive genetic testing despite longstanding clinical recommendations. The initiative is operated by nonprofit CureSHANK and is supported by lead sponsor Illumina along with supporting sponsors PYC Therapeutics, Neuren Pharmaceuticals, GRIN Therapeutics, Jaguar Gene Therapy, and MyOme.
• CureSHANKminorGTM or Marketing Partner · 25 July 2026CureSHANK is the nonprofit organization that operates the Genetic Testing Action Day initiative, which PYC Therapeutics supports as a sponsor alongside other biotech and pharma companies. CureSHANK is the parent initiative for the awareness campaign launched in 2025 and now grown into an international effort.

Scale indicators4 records

Recent moves5 records

Expansion highlights4 records

Direct peers

• ProQR Therapeutics: Clinical-stage RNA therapeutics company developing Axiomer RNA-editing and antisense oligonucleotide programs targeting inherited retinal diseases and other rare genetic disorders. Highly comparable to PYC on modality, indication overlap (ophthalmology), and small-cap clinical-stage profile.
• Regulus Therapeutics: Small-cap clinical-stage biotech developing microRNA-targeted oligonucleotide therapeutics for rare renal and metabolic diseases. Closely comparable to PYC on modality, stage, and kidney-disease focus.
• Wave Life Sciences: Oligonucleotide therapeutics developer with PRISM platform pursuing rare neurological and ophthalmologic genetic diseases. Comparable to PYC on modality, rare-disease focus, and clinical-stage scale.
• Stoke Therapeutics: Clinical-stage antisense oligonucleotide developer focused on severe genetic diseases including inherited ophthalmology indications. Directly comparable to PYC on modality, rare-disease pipeline, and clinical-stage status.
• Ionis Pharmaceuticals: The leading antisense oligonucleotide platform company, with a broad pipeline across rare and genetic diseases including ophthalmology and kidney indications. Directly comparable to PYC on modality (oligonucleotide chemistry), target patient population (rare/genetic disease), and clinical-stage development model.

Broad incumbents

• Sarepta Therapeutics: Genetic medicine company with approved and clinical-stage oligonucleotide and gene-therapy assets for rare neuromuscular and ophthalmology indications. Comparable on oligonucleotide modality and rare-disease commercial model, though larger and commercially active.
• Alnylam Pharmaceuticals: Pioneering RNA interference therapeutics company with a broad, late-stage and commercial portfolio across rare genetic diseases. Comparable on RNA-based therapeutic modality and rare-disease focus, though significantly larger and more commercialised than PYC.
• BioMarin Pharmaceutical: Established rare-disease biotech with a commercial portfolio across genetic and orphan indications. Comparable on rare-disease focus and oligonucleotide/RNA-adjacent modality, though significantly more mature commercially.

Regional players

• Neuren Pharmaceuticals: ASX-listed rare-disease biotech developing therapeutics for neurodevelopmental disorders. Comparable on rare-disease focus, clinical-stage model, and Australian public-market listing; differs in indication area but co-markets the Genetic Testing Action Day with PYC.

Emerging players

• BridgeBio Pharma: Clinical-stage rare-disease biotech with multiple genetic-medicine programs including in renal indications. Comparable on rare genetic-disease focus and small-cap clinical-stage operating model.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat3 records

Key risks4 records

Key highlights6 records

Customer concentration

• Xx.com/PycTherapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Funding overview

Funding rounds1 record

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about PYC Therapeutics