What Progeria Research Foundation does

The Progeria Research Foundation (PRF) is a 501(c)(3) non-profit organization founded in 1999 and headquartered in Peabody, Massachusetts. It is the only organization solely dedicated to discovering treatments and a cure for Hutchinson-Gilford Progeria Syndrome (HGPS), an ultra-rare, fatal rapid-aging disease that causes children to die of heart disease at an average age of 14.5 years without treatment. PRF serves three core constituencies: affected children and their families (the International Progeria Registry had sufficient enrollment to place 107 children from 42 countries into the lonafarnib trials), scientific researchers (supplied via the Cell and Tissue Bank, Medical Database, and research grants program), and donors/sponsors (supported through donations, recurring gifts, and signature events). Mission-adjacent work extends to aging-related disorders more broadly, particularly cardiovascular disease.

PRF's core technology portfolio spans an FDA-approved small molecule (lonafarnib, a farnesyltransferase inhibitor that became the first and only approved Progeria therapy in 2020), a clinical-stage small molecule (Progerinin, in trials with PRG Science & Technology), and an investigational gene therapy (SamPro-2). SamPro-2 is an AAV-delivered base editing therapy designed to permanently correct the single T-to-C mutation in the LMNA gene that produces toxic progerin; the base editing chemistry was developed by Dr. David Liu at the Broad Institute of MIT and Harvard, and PRF holds an exclusive relationship for Progeria application. Manufacturing for IND-enabling studies is being executed through a partnership with Forge Biologics announced in March 2026. Supporting research infrastructure includes the Cell and Tissue Bank (lymphoblast, fibroblast, immortalized, and iPSC lines), the International Progeria Registry, the Medical Database, the Diagnostic Testing Program, and the Research Grants Program.

PRF operates a non-commercial revenue model. Income is generated entirely through charitable donations (one-time gifts, Circle of Hope recurring giving, matching gifts, planned giving), fundraising events (the annual International Race for Research 5K/2-mile walk, the biennial Night of Wonder gala with corporate sponsorships ranging from $2,500 to $50,000, and the mid-year ONEpossible campaign), and research grants. Clinical trials and diagnostic testing are provided at no cost to families. Distribution of impact occurs through Boston Children's Hospital and Brigham and Women's Hospital (clinical trial sites), a multilingual ambassador program (covering 20+ languages), and direct provision of cell lines, tissue samples, and registry data to approved researchers worldwide. Leadership is anchored by co-founder and Medical Director Leslie Gordon, MD, PhD, and Senior Research Advisor Francis Collins, MD, PhD (former NIH Director).

Name

Progeria Research Foundation

Legal name

The Progeria Research Foundation, Inc.

Website

https://progeriaresearch.org

Company type

Private

Founded year

1999

Operating status

Operating

Headcount range

11–50 employees

Short description

The Progeria Research Foundation is a 501(c)(3) non-profit founded in 1999 solely dedicated to discovering treatments and a cure for Progeria. It runs clinical trials, the International Progeria Registry, a Cell and Tissue Bank, and is advancing the SamPro-2 AAV-based gene therapy toward clinical trials.

Ownership category

akta.pro rank

Product category

Rare Disease Research Foundation

NAICS

Voluntary Health Organizations (813212), Scientific Research and Development Services (5417)

SIC

Services-Health Services (8000), Services-Misc Health & Allied Services, Nec (8090)

akta.pro primary industry

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)

akta.pro secondary industries

Global Health Research, Clinical Trials & Product Development Partnerships (PDPs) (HLAJAOAM), Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

Keywords

• Rare disease research
• Gene therapy development
• Clinical trial management
• Patient registry services
• Biomedical research funding

Headquarters

HQ city

Maussane

HQ country

France

HQ region

Europe

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

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

Revenue model

1. Charitable Donations: The foundation relies primarily on donations from individuals, families, and supporters to fund research programs, clinical trials, and operational activities. Various giving options include one-time donations, monthly giving (Circle of Hope), matching gifts, and planned giving.
2. Fundraising Events: Annual events including the International Race for Research (5K run and 2-mile walk), Night of Wonder gala (biennial signature event), and ONEpossible mid-year campaigns generate revenue through registration fees, sponsorships, and auction items.
3. Research Grants: PRF funds research through grants awarded to scientific researchers studying Progeria, funded by donations and potentially government or institutional grants.

Go-to-market motion1 record

Distribution channels7 records

Marketing channels13 records

Core offering

PRF operates research, clinical, and patient-support programs aimed at discovering treatments and the cure for Hutchinson-Gilford Progeria Syndrome (HGPS) and its aging-related disorders. Its offering spans the FDA-approved treatment lonafarnib, the investigational AAV-based gene therapy SamPro-2, an International Progeria Registry, a Cell and Tissue Bank, diagnostic testing, clinical trial coordination, and research grants. Programs are delivered at no cost to participating patient families and are sustained through donations, grants, and fundraising events.

Product overview

The Progeria Research Foundation (PRF) is a non-profit organization solely dedicated to finding treatments and the cure for Progeria. PRF operates a portfolio of research programs and resources rather than a commercial product platform. The primary investigational therapy is SamPro-2, an AAV-based gene editing treatment using base editing technology to correct the genetic mutation causing Progeria, currently in development toward clinical trials in partnership with Forge Biologics. PRF also manages the FDA-approved treatment lonafarnib (Zokinvy), which has extended the lives of affected children. Supporting programs include the International Progeria Registry, Cell and Tissue Bank, Clinical Trials Program, Diagnostic Testing Program, and Medical Database, all designed to facilitate research toward curing Progeria and its aging-related disorders including heart disease.

Differentiator

Problem solved

Functional benefit

Products and services

• SamPro-2 Gene Therapy Investigational AAV-based gene therapy using base editing technology to permanently correct the single T-to-C DNA letter error in the LMNA gene that causes Progeria; designed for children with Hutchinson-Gilford Progeria Syndrome and being advanced toward clinical trials via a manufacturing partnership with Forge Biologics.
• Lonafarnib Treatment Program Coordination and ongoing clinical support for the FDA-approved farnesyltransferase inhibitor (FTI) treatment (marketed as Zokinvy) for Progeria; PRF's trials enrolled 107 children from 42 countries and demonstrated lifespan extension.
• International Progeria Registry Global patient registry connecting children with Progeria and their families to research, clinical trials, and support services; enables cohort tracking and clinical trial recruitment worldwide.
• Cell and Tissue Bank Repository of validated biological samples (lymphoblast, fibroblast, immortalized cell lines, and induced pluripotent stem cells) plus protocols distributed to approved researchers studying Progeria.
• Clinical Trials Program Coordination of clinical trials for Progeria treatments, including the historical lonafarnib trials, the ongoing Progerinin trial (with PRG Science & Technology), and upcoming SamPro-2 gene therapy trials at Boston Children's Hospital and Brigham and Women's Hospital.
• Diagnostic Testing Program Program that provides diagnostic testing services to confirm Progeria in suspected patients worldwide, enabling early identification and registry/clinical trial enrollment.
• Medical Database Comprehensive database of clinical and medical information collected from Progeria patients participating in PRF research programs and clinical trials, supporting research and care improvement.
• Research Grants Program Award program that funds scientific research on Progeria and related aging disorders, made possible by donations and grants.

Quantifiable outcome

• Average lifespan extended significantly with lonafarnib treatment
• +3 more outcomes

Named customers4 records

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Eight partnerships are on record, tiered core, secondary and minor.

• Forge BiologicscoreStrategic or Co-development Partner · 3 March 2026Manufacturing partnership to develop and produce SamPro-2, PRF's investigational AAV-based gene therapy for Progeria. Forge Biologics provides process development, cGMP manufacturing, and regulatory consultation services to support IND-enabling studies for the therapy. The partnership combines PRF's decades of Progeria research with Forge's integrated gene therapy manufacturing capabilities as part of PRF's Path to Cure Progeria initiative.
• PRG Science & TechnologycoreStrategic or Co-development PartnerTrial sponsor for the Progerinin clinical trial. Korean-based biotech company collaborating with PRF on clinical development of Progerinin therapy for Progeria.
• Boston Children's HospitalcoreStrategic or Co-development PartnerPrimary clinical trial site for Progeria treatments including lonafarnib trials and Progerinin clinical trial. Families from around the world travel to Boston for week-long testing visits.
• Brigham and Women's HospitalsecondaryStrategic or Co-development PartnerClinical trial site conducting some trial testing for Progeria treatments in collaboration with Boston Children's Hospital.
• Dr. David Liu (MIT/Broad Institute/Harvard)coreStrategic or Co-development PartnerCollaboration with the Richard Merkin Professor and Director at the Merkin Institute for Transformative Technologies in Healthcare at Broad Institute of MIT and Harvard. Dr. Liu is the inventor of base editing technology and co-developer of SamPro-2 gene therapy for Progeria.
• National Human Genome Research Institute (NHGRI)secondaryStrategic or Co-development PartnerCollaboration with researchers including Dr. Michael Erdos (Associate Investigator, Molecular Genetics Section) advancing Progeria research.
• National Institutes of Health (NIH)secondaryStrategic or Co-development PartnerLong-standing partnership with NIH. Dr. Francis Collins, former NIH Director and leader of the Human Genome Project, serves as PRF's Senior Research Advisor and has been a longtime Progeria research partner.
• Massport (Boston Logan Airport)minorImplementation/ SI/ Consulting PartnerMeet and Greet program partnership to assist families arriving at Boston Logan Airport, helping them navigate to ground transportation for clinical visits.

Scale indicators6 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Cystic Fibrosis Foundation: Disease-specific nonprofit that funded and advanced transformative therapies (Kalydeco, Trikafta) through venture philanthropy and pharma partnerships. Closely mirrors PRF's model of patient-centered foundation driving drug development via research grants, clinical trial networks, and pharma partnerships.
• Cure SMA: Rare pediatric disease nonprofit that played a central role in developing and funding access to Spinraza, the first FDA-approved SMA treatment. Highly comparable to PRF in mission, organizational scale, and pipeline strategy.
• Parent Project Muscular Dystrophy: Patient-founded nonprofit focused exclusively on Duchenne muscular dystrophy, with active drug development pipeline and clinical trial coordination. Shares PRF's family-driven origin story, single-disease focus, and translational research orientation.
• Associazione Italiana Progeria Sammy Basso (AIPro.Sa.B.): Italian sister organization co-founded by the late Sammy Basso, a longtime PRF ambassador. Directly comparable as the only other Progeria-specific patient organization, with overlapping research and family-support mission.

Broad incumbents

• National Organization for Rare Disorders (NORD): Umbrella advocacy organization for all rare diseases in the US. Broader in scope than PRF but operates overlapping research grant programs, patient registry initiatives, and rare disease policy advocacy.
• EveryLife Foundation for Rare Diseases: Rare disease policy and advocacy foundation focused on accelerating biotech innovation for rare disorders. Comparable as a nonprofit enabler of rare disease drug development but operating at the policy/ecosystem level rather than single-disease.
• Muscular Dystrophy Association: Large-scale disease-focused nonprofit funding research across neuromuscular diseases. Shares PRF's research-grant, clinical-care, and family-support model but at substantially larger scale and broader disease scope.

Emerging players

• Beam Therapeutics: Clinical-stage biotech founded by David Liu to commercialize base editing technology — the same platform underlying PRF's SamPro-2. Indirect strategic peer as the technology originator that PRF depends on for translational know-how and IP.
• Prime Medicine: Gene editing company pursuing prime editing for rare genetic diseases. Comparable in targeting single-letter genetic corrections and rare disease focus, representing adjacent technological competition for PRF's therapeutic modality.

Others

• Eiger BioPharmaceuticals (now Sentynl Therapeutics): Commercial sponsor of lonafarnib (marketed as Zokinvy) for Progeria. Connected to PRF through the FDA-approved therapy whose development PRF led; relevant as the commercial partner on PRF's most successful asset to date.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks6 records

Key highlights6 records

Customer concentration

• YouTubeyoutube.com/user/progeria123
• Facebookfacebook.com/ProgeriaResearch
• LinkedInlinkedin.com/company/the-progeria-research-foundation
• Instagraminstagram.com/progeriaresearch
• TikToktiktok.com/@progeriaresearch
• Xx.com/Progeria

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Progeria Research Foundation