A New Frontier in Cystic Fibrosis
Aceso is committed to transform cystic fibrosis (in French: mucoviscidose) care beyond current therapeutic limitations.
Cystic Fibrosis (CF) — Daily burden
For people with cystic fibrosis, breathing is a daily challenge. This genetic condition, caused by mutations in the CFTR gene, primarily affects the lungs and digestive organs. It leads to the buildup of thick mucus, resulting in chronic infections, inflammation, daily treatments, and a reduced life expectancy.
Progress has been made — but remains limited.
Stage
IND-enabling studies
IP
Composition & method
A life-shortening autosomal recessive genetic disease caused by variants in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, with F508del being the most common mutation.
International disparities in diagnosis and treatment access for CF – Guo et al. (2024) – Pediatric Pulmonology published by Wiley Periodicals LLC.
New drugs, new challenges in cystic fibrosis care. Eur. Respir Rev 2024; 33: 240045 [DOI: 10.1183/16000617.0045-2024]
Backed by world-class scientific & financial partners
The Challenge
While CFTR modulators have transformed outcomes for many, thousands of patients remain without effective options — constrained by genotype, partial efficacy, or prohibitive cost.
162,000+ People living with cystic fibrosis worldwide
A devastating, life-shortening genetic disease.
20% Patients with no approved modulator therapy
Severe and rare CFTR mutations remain underserved.
Partial Response in many patients on existing therapies
Significant unmet clinical need for next-generation correctors.
Why Today's Therapies Fall Short
Existing modulators fail to address rare and severe CFTR variants, leaving thousands without a viable therapy.
Many patients experience only modest improvement in lung function and quality of life on current treatments.
Lifetime treatment costs exceeding $300K per year per patient strain healthcare systems globally.
Lead Program
Inhaled antisense oligonucleotide (ASO) designed for CF patients with F508del.
Molecules and formulation protected with robust IP strategy.
ACT-101 stabilizes CFTR mRNA expression to increase CFTR protein levels, offering a potential inhaled therapy alone or in combination with current treatments.
in vivo biodistribution and repeat-dose studies up to MTD show a promising safety profile.
Development Stage
Discovery
Preclinical
CTA
Phase I
Over €1.5M already invested to advance the lead candidate to TRL6
Backed by recognized scientific & financial partners
Pipeline
Active programs
Filter by indication
Clinical development stages
| Indication | Project | Mechanism | PoC | Preclinical | Phase 1/2 | Phase 2/3 | Phase 4 | Target year | Status |
|---|---|---|---|---|---|---|---|---|---|
| CF — Class II | ACT-101Lead program | Expression mRNA | Efficacy in 2031 | Ongoing — CTA 2027* | |||||
ACT-201 | Stability CFTR | To define* | PoC done | ||||||
ACT-301 | Bitherapy ACT-101/ACT-201 | To define* | PoC done | ||||||
| CF — Class I | ACT-401 | Splicing defect | n.a | PoC done | |||||
ACT-102 | Non-sense | n.a | PoC done | ||||||
| CPP | WRAP-1 | CF TSB carrier | To define* | PoC done | |||||
| COPD | ACT-105 | Inflammation | n.a | PoC initiated |
Clinical development stages
| Indication | Project | Mechanism | PoC | Preclinical | Phase 1/2 | Phase 2/3 | Phase 4 | Target year | Status |
|---|---|---|---|---|---|---|---|---|---|
| CF — Class II | ACT-101Lead program | Expression mRNA | Efficacy in 2031 | Ongoing — CTA 2027* | |||||
ACT-201 | Stability CFTR | To define* | PoC done | ||||||
ACT-301 | Bitherapy ACT-101/ACT-201 | To define* | PoC done | ||||||
| CF — Class I | ACT-401 | Splicing defect | n.a | PoC done | |||||
ACT-102 | Non-sense | n.a | PoC done |
Clinical development stages
| Indication | Project | Mechanism | PoC | Preclinical | Phase 1/2 | Phase 2/3 | Phase 4 | Target year | Status |
|---|---|---|---|---|---|---|---|---|---|
| CPP | WRAP-1 | CF TSB carrier | To define* | PoC done |
Clinical development stages
| Indication | Project | Mechanism | PoC | Preclinical | Phase 1/2 | Phase 2/3 | Phase 4 | Target year | Status |
|---|---|---|---|---|---|---|---|---|---|
| COPD | ACT-105 | Inflammation | n.a | PoC initiated |
Clinical development stages
| Indication | Project | Mechanism | PoC | Preclinical | Phase 1/2 | Phase 2/3 | Phase 4 | Target year | Status |
|---|---|---|---|---|---|---|---|---|---|
| CF — Class II | ACT-101Lead program | Expression mRNA | Efficacy in 2031 | Ongoing — CTA 2027* | |||||
ACT-201 | Stability CFTR | To define* | PoC done | ||||||
ACT-301 | Bitherapy ACT-101/ACT-201 | To define* | PoC done | ||||||
| CF — Class I | ACT-401 | Splicing defect | n.a | PoC done | |||||
ACT-102 | Non-sense | n.a | PoC done | ||||||
| CPP | WRAP-1 | CF TSB carrier | To define* | PoC done | |||||
| COPD | ACT-105 | Inflammation | n.a | PoC initiated |
Clinical development stages
| Indication | Project | Mechanism | PoC | Preclinical | Phase 1/2 | Phase 2/3 | Phase 4 | Target year | Status |
|---|---|---|---|---|---|---|---|---|---|
| CF — Class II | ACT-101Lead program | Expression mRNA | Efficacy in 2031 | Ongoing — CTA 2027* | |||||
ACT-201 | Stability CFTR | To define* | PoC done | ||||||
ACT-301 | Bitherapy ACT-101/ACT-201 | To define* | PoC done | ||||||
| CF — Class I | ACT-401 | Splicing defect | n.a | PoC done | |||||
ACT-102 | Non-sense | n.a | PoC done |
Clinical development stages
| Indication | Project | Mechanism | PoC | Preclinical | Phase 1/2 | Phase 2/3 | Phase 4 | Target year | Status |
|---|---|---|---|---|---|---|---|---|---|
| CF — Class I | ACT-102 | Non-sense | n.a | PoC initiated | |||||
ACT-401 | Splicing defect | n.a | PoC done |
Clinical development stages
| Indication | Project | Mechanism | PoC | Preclinical | Phase 1/2 | Phase 2/3 | Phase 4 | Target year | Status |
|---|---|---|---|---|---|---|---|---|---|
| CPP | WRAP-1 | CF TSB carrier | To define* | PoC done |
Legend
In progress
In progress
Lead program
Why Invest
01 — Market
A multi-billion dollar global CF market with a large segment still without effective therapy.
Addressable market
Robust preclinical evidence of CFTR rescue in patient-derived models supports clinical promise.
Orphan-drug positioning and breakthrough potential support an expedited path to patients.
About ACESO
Named after the Greek goddess of the healing process, our company embodies a singular commitment: translating breakthrough chemistry into meaningful, lasting clinical outcomes — and durable value for patients, partners, and investors alike.
Our mission
— ACESO Founding Team
Leadership
MBA
Founder of Aceso Therapeutics, with over 25 years of international leadership experience in healthcare, diagnostics, life sciences, and chemistry. An ESSEC and IIMA graduate, he has led growth and transformation at global companies. Thomas defines the company’s vision and strategy, driven by a passion for improving patients’ lives through innovation and by the values of sports, family, and personal well-being.
PHD
Co-founder, Magali brings over 20 years of experience in rare disease genetics, particularly cystic fibrosis. A recognized scientist with a PhD in Toxicogenomics and author of 33 scientific publications, her career combines academic research, clinical collaboration, and expertise in molecular diagnostics. She leads the company’s scientific vision and research strategy, dedicated to advancing innovative solutions for patients with rare diseases (Cystic Fibrosis).
PHD
Co-founder, Prisca is a trained biochemist who obtained her PhD in 2004 from the Freie Universität Berlin (Germany), focusing on peptide-based inhibitors of protein–protein interactions. She completed a postdoctoral fellowship in peptide vectors for therapeutic delivery at the University of Montpellier before joining the CNRS as a research scientist. Her expertise is supported by 57 publications, 5 book chapters, and 4 patents, with additional experience in myocardial infarction (MI) biotech. Prisca leads the company’s technological strategy and innovation, dedicated to developing advanced therapeutic solutions.
PHD
Senior scientist and biotech executive with a PhD in Molecular Biology and extensive experience in translational research and preclinical development. He has led multidisciplinary projects across academic, clinical, and biotech environments, advancing innovative therapeutic programs from research to application. Sébastien brings strong expertise in project leadership and scientific development within the life sciences sector, and leads the ACT-101 program through its translational and preclinical stages
MD, PhD
MD
Latest
Get in touch
Whether you are an investor, strategic partner, or member of the scientific community — we would be glad to talk.
Headquarters
