What Fair Therapeutics does

Fair Therapeutics was a clinical-stage biopharmaceutical company founded in 2021 in Utrecht, the Netherlands, focused on developing treatments for people with cystic fibrosis (CF) who have rare or ultra-rare CFTR gene mutations and lack access to existing disease-modifying therapies. The company pursued a unified drug-plus-diagnostic strategy built around Diponecaftor, a CFTR triple modulator therapy combining a potentiator (Dirocaftor), corrector (Posenacaftor), and amplifier (Nesolicaftor) originally licensed from Proteostasis Therapeutics and subsequently held by HealthCare Royalty Partners. The companion diagnostic used intestinal organoids grown from patient rectal biopsies, with CFTR function quantified through the Forskolin-Induced Swelling (FIS) assay to pre-select patients most likely to respond to therapy.

The business model followed a traditional biopharma development path: the company sponsored the Phase 2b CHOICES trial (NCT06468527) across 15 clinical centers in Europe, ultimately enrolling 40 adult patients from 9-10 countries, with organoid screening data drawn from over 500 CF patients across 16 European countries through the HIT-CF Europe consortium supported by EU Horizon 2020 grant 755021. Final CHOICES results announced October 2025 demonstrated clinical effect proportional to in-vitro organoid response (FEV1 changes from -12% to +22 percentage points), validating organoid-based stratification. Commercial revenue was never generated because the company had not yet launched a product; planned monetization was through prescription drug sales to CF patients with rare and ultra-rare variants via specialty pharmacies and CF treatment centers.

On 28 May 2026, Fair Therapeutics announced wind-down of operations after failing to secure the financing required to continue modulator development, returning the CFTR modulator license to HealthCare Royalty Partners. The company was founded by Prof. Jeffrey Beekman (CSO, inventor of the organoid FIS assay and recipient of the 2019 Prix Galien Research Award) and Prof. Kors van der Ent (pediatric pulmonologist and HIT-CF coordinator). Operational leadership was led by CEO Ines Rodriguez Alvarez (appointed July 2022) and CBO Anne van Loon (appointed July 2024), with a total headcount of 1-10 employees.

Name

Fair Therapeutics

Legal name

Fair Therapeutics B.V.

Website

https://fairtherapeutics.com

Company type

Private

Founded year

2021

Operating status

Closed

Headcount range

1–10 employees

Short description

Fair Therapeutics was a clinical-stage biopharmaceutical company developing Diponecaftor, a CFTR triple modulator therapy paired with an organoid-based companion diagnostic, to deliver personalized treatment for cystic fibrosis patients with rare and ultra-rare mutations. The company ceased operations in May 2026.

Ownership category

akta.pro rank

Product category

Cystic Fibrosis Therapeutics

NAICS

Medical Laboratories (621511)

SIC

Pharmaceutical Preparations (2834), Services-Medical Laboratories (8071)

akta.pro primary industry

Pulmonary Hypertension & Rare Pulmonary Disease Therapies (HLAIAIAG)

akta.pro secondary industry

Cystic Fibrosis & Bronchiectasis Care (HLAKANAK)

Keywords

• Cystic fibrosis treatment
• CFTR modulator therapy
• Organoid companion diagnostics
• Rare disease therapeutics
• Personalized medicine

Headquarters

HQ city

Utrecht

HQ country

Netherlands

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. CFTR Modulator Therapy (Diponecaftor): Fair Therapeutics was developing a CFTR triple modulator therapy combined with a companion diagnostic for personalized CF treatment. The company was in clinical stage (Phase 2b) and had not yet commercialized products. Revenue would have been generated through prescription drug sales to CF patients with rare/ultra-rare variants once approved.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels1 record

Core offering

Fair Therapeutics was developing a CFTR triple modulator therapy (Diponecaftor, combining the potentiator Dirocaftor, corrector Posenacaftor, and amplifier Nesolicaftor) alongside an organoid-based companion diagnostic using the forskolin-induced swelling (FIS) assay. The combined offering aimed to deliver personalized cystic fibrosis treatment for patients with rare and ultra-rare CFTR gene mutations who lack access to existing disease-modifying therapies, by pre-selecting responders through patient-derived intestinal organoids.

Product overview

Fair Therapeutics is developing a unified therapeutic approach combining its core CFTR triple modulator therapy Diponecaftor (Dirocaftor/Posenacaftor/Nesolicaftor) with a companion diagnostic based on organoid technology. The Diponecaftor therapy repairs CFTR protein function through a potentiator, corrector, and amplifier mechanism, while the organoid-based FIS (forskolin-induced swelling) assay serves as a predictive diagnostic to select patients most likely to respond to treatment, enabling personalized medicine for cystic fibrosis patients with rare and ultra-rare mutations.

Differentiator

Problem solved

Functional benefit

Products and services

• Diponecaftor (Dirocaftor/Posenacaftor/Nesolicaftor) A CFTR triple modulator therapy consisting of a potentiator (Dirocaftor), corrector (Posenacaftor), and amplifier (Nesolicaftor) that repairs CFTR protein function, restores its 3D shape, and enhances production of mutant CFTR to treat cystic fibrosis in patients with rare and ultra-rare variants.
• Organoid-Based Companion Diagnostic (FIS Assay) A personalized diagnostic tool using laboratory-grown intestinal organoids from patient rectal biopsies to predict clinical response to CFTR modulators via the forskolin-induced swelling (FIS) assay, enabling patient stratification and selection for CFTR modulator clinical trials and treatment.

Quantifiable outcome

• Changes in FEV1 ranged from -12% to +22% percentage points with concordant changes in sweat chloride levels in placebo-controlled study
• +2 more outcomes

Segments3 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Five partnerships are on record, tiered core and minor.

• HIT-CF ConsortiumcoreStrategic or Co-development PartnerThe HIT-CF Europe consortium is a collaborative effort supported by the EU Horizon 2020 initiative consisting of academic research institutions, not-for-profit organizations, CROs, biopharma companies developing CF therapies, and supporting service providers. The consortium conducted the CHOICES Phase 2b clinical trial and earlier HIT-CF Organoid Study involving over 500 patients from 16 European countries. Partners include: University Medical Center Utrecht (coordinator), KU Leuven (Belgium), Faculdade de Ciências da Universidade de Lisboa (Portugal), European Cystic Fibrosis Society (Denmark), CF Europe, HUB Organoids (Utrecht), Julius Clinical (Utrecht), and Patergus-Biotechsubsidy (Belgium).
• University Medical Center UtrechtcoreStrategic or Co-development PartnerCoordinator of the HIT-CF project. Led the CHOICES Phase 2b study. Co-founded by Prof. Jeffrey Beekman (inventor of organoid assay technology) and Prof. Kors van der Ent (coordinator of HIT-CF project and Chair of CF-group within European Reference Network for rare lung diseases).
• HUB OrganoidscoreTechnology or IntegrationPartner in the HIT-CF consortium specializing in organoid technology for CF patient stratification and drug response prediction.
• CF EuropecoreStrategic or Co-development PartnerFederation of national and regional patient associations of people with CF in Europe. Actively involved in the HIT-CF project and supports the goal to realize treatment for patients with ultra-rare mutations and in underserved areas.
• Julius ClinicalminorImplementation/ SI/ Consulting PartnerContract research organization (CRO) partner in the HIT-CF consortium providing clinical trial support services.

Scale indicators5 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Eloxx Pharmaceuticals: Previously developed ELX-02, a readthrough agent for nonsense-mutation CF. Direct peer in the ultra-rare CF niche; the company substantially wound down in 2023, illustrating the commercial difficulty of ultra-rare CF programs.
• Enterprise Therapeutics: UK-based clinical-stage biotech developing novel modulators of ion channel function (including ENaC and TMEM16A) for CF. Comparable as a CF-focused small-molecule developer targeting patients who do not benefit from existing CFTR modulators.
• Proteostasis Therapeutics: Original developer of the Dirocaftor/Posenacaftor/Nesolicaftor triple modulator that Fair Therapeutics licensed and developed. Direct peer on the same asset and same indication, though the company itself wound down following the 2020 merger with Yumanity.

Broad incumbents

• Galapagos NV: Belgian-Dutch biotech that, together with AbbVie, previously developed CFTR modulators (GLPG1837/GLPG2222) before exiting the CF program. Comparable as a European biopharma that pursued and ultimately discontinued a CFTR-modulator strategy.
• Vertex Pharmaceuticals: Vertex is the dominant CF franchise owner (Trikafta/Kaftrio, Kalydeco) and the benchmark for CFTR modulator development. It is highly comparable as the commercial benchmark and likely strategic acquirer for any ultra-rare CFTR assets.

Others

• HUB Organoids: Utrecht-based organoid technology company and HIT-CF consortium partner responsible for establishing patient-derived organoid models used in Fair Therapeutics' stratification platform. Comparable as the underlying technology provider whose IP underpins the diagnostic.

Emerging players

• SpliSense: Israeli biotech developing antisense oligonucleotide therapies for specific CFTR mutations (including 3849+10kb C->T). Comparable as an ultra-rare, mutation-specific CF therapeutic developer.
• Spirovant Sciences: Gene therapy company developing adeno-associated virus (AAV) and lentiviral therapies for cystic fibrosis. Comparable as a CF-focused emerging player pursuing mutation-agnostic treatment for non-responsive CF populations.
• 4D Molecular Therapeutics: Clinical-stage gene therapy company developing an aerosolized CFTR gene therapy for cystic fibrosis lung disease. Comparable as a next-generation, mutation-agnostic CF therapeutic addressing patients underserved by small-molecule modulators.
• ReCode Therapeutics: Clinical-stage company developing mRNA and gene correction therapies for genetic respiratory diseases including cystic fibrosis. Comparable as a mutation-agnostic, nucleic-acid-based approach to treating CFTR-deficient patients.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat5 records

Key risks5 records

Key highlights5 records

Customer concentration

• LinkedInlinkedin.com/company/fairtx

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds

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 Fair Therapeutics