Prader-Willi Research Foundation of Australia

What Prader-Willi Research Foundation of Australia does

Prader-Willi Research Foundation of Australia (PWRFA) is a nonprofit charitable foundation founded in 2015 and based in Australia, with operations across VIC, NSW, QLD, NT, WA, ACT and TAS. Its mission is to improve clinical outcomes and treatments for people living with Prader-Willi syndrome (PWS) by funding cutting-edge genetic and clinical research at leading Australian institutions, including the Walter and Eliza Hall Institute of Medical Research (WEHI), Murdoch Children's Research Institute (MCRI), the Florey Institute of Neuroscience and Mental Health, the Garvan Institute, the University of Sydney, and La Trobe University. The foundation serves PWS-affected families, researchers, and clinicians, and aims to translate insights from rare-disease research into broader advances in obesity and mental health.

PWRFA's core scientific bets are SMCHD1-targeting gene activation therapy and the MAGEL2 gene cluster, with sustained funding supporting Professor Marnie Blewitt's laboratory at WEHI. This research program produced measurable partial restoration of gene expression in PWS mouse models (improvements in body weight, grip strength, motor coordination, and social behavior) and catalysed the 2026 spinout of Togglelux Therapeutics, a WEHI-derived company commercialising SMCHD1 as a therapeutic target. Additional research infrastructure includes Australia's first Chromosome 15 Biobank, established in 2020 with MCRI using a $65,500 Perpetual IMPACT Philanthropy grant, and a clinical-trials program that brought the TEMPO phase 3 Pitolisant trial to Australia in 2024 via Harmony Biosciences.

The foundation operates a small team of 7 employees under founder and CEO Kathlene Jones, governed by Chair Duncan McGauchie and a board including Susan Blair and Professor Ian Cooke. Its revenue model relies entirely on individual donations, philanthropic grants, and community fundraising events such as the annual Finding 15 National Fundraising Walk, MOVE campaigns, and Ironman fundraisers. Engagement with beneficiaries and donors is community-led, channelled through a newsletter, Facebook presence, a blog, and biennial research and family conferences (next iteration: 2026 in Brisbane). PWRFA also publishes a PWS Research Roadmap and operates the PWS Centre of Expertise, a clinical-care program intended to provide specialised, integrated care for PWS patients across Australia.

Name

Prader-Willi Research Foundation of Australia

Legal name

Prader-Willi Research Foundation of Australia

Website

https://praderwilli.org.au

Company type

Private

Founded year

2015

Operating status

Operating

Headcount range

1–10 employees

Short description

Prader-Willi Research Foundation of Australia is a nonprofit charitable foundation founded in 2015 that funds genetic and clinical research into Prader-Willi syndrome at leading Australian institutes, including WEHI, MCRI, Florey, the Garvan Institute, and the University of Sydney. It supports SMCHD1 and MAGEL2 gene-activation research, the Australian Chromosome 15 Biobank, and clinical trials, serving PWS-affected families, researchers, and clinicians across Australia.

Ownership category

akta.pro rank

Product category

Rare Disease Research

NAICS

Voluntary Health Organizations (813212), Grantmaking Foundations (813211), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Health & Medical Research Grantmaking Foundations (BPAGAKAL)

akta.pro secondary industry

Research & Science Grantmaking Foundations (BPAGAKAI)

Keywords

• Prader-Willi syndrome research
• Rare disease research
• Genetic disorder funding
• Medical research grants
• Gene activation therapy

Headquarters

HQ city

Melbourne

HQ country

Australia

HQ region

Oceania

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Others

Revenue model

1. Donations: Individual and family donations from the PWS community and supporters to fund research initiatives
2. Grants and Philanthropic Funding: Foundation receives grants from organizations such as Perpetual IMPACT Philanthropy ($65,500 grant for biobank establishment) and corporate sponsors like Westfield Southland ($10,000 Local Heroes grant)
3. Fundraising Events: Events including Finding 15 Walks, MOVE campaigns, Ironman fundraisers, and community fundraising activities

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

PWRFA is a nonprofit research foundation that funds and coordinates scientific research into Prader-Willi Syndrome, a rare genetic disorder. Its core offerings include funding gene activation therapy research (notably SMCHD1 targeting), operating the Australian Chromosome 15 Biobank, coordinating the PWS Centre of Expertise for clinical care, facilitating clinical trials such as the TEMPO trial, and running the annual Finding 15 fundraising walk and biennial Research and Family Conference.

Product overview

Prader-Willi Research Foundation of Australia is a research foundation (not a product company) that operates a portfolio of research programs and initiatives to improve clinical outcomes and treatments for Prader-Willi Syndrome. Its key offerings include: the Vykat XR product; the Australian Chromosome 15 Biobank for storing biological samples; the PWS Centre of Expertise for clinical care; a Clinical Trials Program facilitating international trials in Australia; a Research Grants Program funding scientists; the Finding 15 Walk fundraising initiative; annual Research and Family Conferences; and the PWS Research Roadmap for strategic direction. The foundation's research also aims to inform understanding of obesity and mental health.

Differentiator

Problem solved

Functional benefit

Products and services

• Australian Chromosome 15 Biobank Australia's first purpose-built biobank storing tissue samples and clinical data for research into rare genetic diseases caused by changes to genes on Chromosome 15, including Prader-Willi Syndrome and Angelman Syndrome. Established in partnership with Murdoch Children's Research Institute (MCRI) and Royal Children's Hospital.
• PWS Centre of Expertise A specialized clinical center dedicated to providing expert care and coordination for people living with Prader-Willi Syndrome in Australia, providing national access to a PWS specialist doctor, an integrated care plan, and connecting doctors to international research.
• Clinical Trials Program Program facilitating clinical trials for Prader-Willi Syndrome treatments in Australia, including the TEMPO phase 3 trial of Pitolisant conducted by Harmony Biosciences (announced June 2024).
• Research Grants Program Grant program funding scientists and researchers working on Prader-Willi Syndrome projects, including gene activation therapy (SMCHD1 targeting), SNORD116 research, sleep-disordered breathing studies, exercise training, oxytocin and autonomic nervous system research, and chromosome 15 imprinting disorders research.
• Finding 15 National Fundraising Walk National fundraising walk campaign to raise awareness and funds for Prader-Willi Syndrome research, held annually across Australian states.
• Prader-Willi Research and Family Conference Biennial landmark conference bringing together researchers, scientists, clinicians, families, and the wider disability and rare disease community, featuring international plenary speakers, research symposiums, and dedicated family programming.

Quantifiable outcome

• Gene activation therapy research showed partial restoration of gene expression in PWS mouse models with measurable improvements in body weight, grip strength, motor coordination, and social behavior

Named customers1 record

Segments1 record

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Twelve partnerships are on record, tiered core and secondary.

• Togglelux TherapeuticscoreStrategic or Co-development PartnerSpinout company from WEHI dedicated to targeting SMCHD1 as a treatment for PWS. Founded based on sustained investment in Prof Marnie Blewitt's research work funded by PWRFA. Represents direct translation of research into commercial drug development.
• Walter and Eliza Hall Institute of Medical Research (WEHI)coreStrategic or Co-development PartnerHome to Prof Marnie Blewitt's laboratory conducting gene activation research. Prof Blewitt's team has grown to four full-time and three part-time scientists working on SMCHD1 targeting for PWS treatment. Research has led to Togglelux Therapeutics spinout.
• Murdoch Children's Research Institute (MCRI)coreStrategic or Co-development PartnerPartner in Australian Chromosome 15 Biobank with A/Prof David Godler and Prof David Amor. Conducts research on genetic changes in PWS, newborn screening approaches, and health economics of PWS in Australia.
• Foundation for Prader-Willi Research (FPWR)coreStrategic or Co-development PartnerInternational partnership with US-based FPWR for joint research projects including exercise training studies with La Trobe University and gene activation research with WEHI.
• The Florey Institute of Neuroscience and Mental HealthcoreStrategic or Co-development PartnerResearch partner led by Prof Anthony Hannan focusing on understanding how genes and environment contribute to brain function in PWS. Prof Hannan serves on PWRFA's scientific advisory panel.
• University of SydneysecondaryStrategic or Co-development PartnerPartners with Brain and Mind Centre for oxytocin and autonomic nervous system research in PWS, led by Prof Stewart Einfeld and Dr Lauren Rice.
• The Garvan Institute for Medical ResearchsecondaryStrategic or Co-development PartnerOne of the research institutions where PWRFA-funded scientists conduct PWS research.
• La Trobe UniversitysecondaryStrategic or Co-development PartnerJoint funding with FPWR for exercise training research in PWS led by Prof Nora Shields, evaluating effects of exercise programs on daily task completion and cost-effectiveness.
• PWSA (Australian parents' support organisation)coreStrategic or Co-development PartnerPartnership for 2024 Asia Pacific PWS Conference, bringing together families, researchers, and clinicians across the Asia Pacific region.
• PWS New Zealand, PWS Thailand, PWS MalaysiasecondaryStrategic or Co-development PartnerInternational partnerships for 2024 Asia Pacific PWS Conference collaboration.
• University of Kansas Medical CentersecondaryStrategic or Co-development PartnerInternational research collaboration with Prof Merlin Butler and Dr Olivia Veatch for chromosome 15 imprinting disorders research.
• Brighton Hall InstitutesecondaryStrategic or Co-development PartnerListed as one of the research institutions where PWRFA-funded scientists work on PWS research.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Rett Syndrome Research Trust: US-focused nonprofit aggressively funding cure-oriented Rett syndrome research, including gene therapy approaches. A larger, more mature model of disease-specific research funding that PWRFA benchmarks against.
• National Organization for Rare Disorders (NORD): US umbrella advocacy and research-funding body for rare diseases including PWS. Operates at much greater scale than PWRFA but shares the model of grantmaking and patient support for rare disease communities.
• Children's Tumor Foundation: US nonprofit funding neurofibromatosis research via grants, clinical trials, and a research roadmap. Comparable to PWRFA in operating a structured research-funding roadmap and conference-based community engagement.
• Cure SMA: Large US nonprofit that helped drive the first approved genetic treatments for spinal muscular atrophy. A benchmark for how disease-focused foundations can translate donations into approved therapies, applicable to PWRFA's PWS gene-activation mission.

Direct peers

• International Prader-Willi Syndrome Organisation (IPWSO): Global umbrella body supporting PWS associations in 40+ countries. Shares PWRFA's mission of international collaboration, conferences, and research awareness for Prader-Willi syndrome.
• Foundation for Prader-Willi Research (FPWR): US-based nonprofit dedicated to funding PWS research and supporting therapeutics development. Directly comparable to PWRFA in mission (PWS research funding) and model (grants, clinical trials, family support) and is already a partner.
• Prader-Willi Syndrome Association (PWSA USA): The longest-standing US patient advocacy and support organization for PWS families. Comparable to PWRFA in serving the PWS community with research, family services, and advocacy, though US-focused.

Emerging players

• Angelman Syndrome Foundation: US nonprofit funding Angelman syndrome research, which shares Chromosome 15 biology with PWS and is served by the same Australian Chromosome 15 Biobank. Directly comparable Chromosome 15 grantmaking and patient-impact model.

Regional players

• Prader-Willi Syndrome Association UK (PWSA UK): UK charity supporting people with PWS and funding research. Operates a similar research-and-support model to PWRFA in a different geography and would be a natural comparator for European reach.
• Prader-Willi Research Foundation (PWRF New Zealand): New Zealand PWS research-focused organization already collaborating with PWRFA on the Asia Pacific PWS Conference. Highly comparable peer in mission, scale, and regional context.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks5 records

Key highlights7 records

Customer concentration

• Facebookfacebook.com/PWRFA
• LinkedInlinkedin.com/company/prader-willi-research-foundation-of-australia

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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