Technologies

Aceso’s PTGR Platform: Restoring CFTR Function Through Innovative RNA-Based Therapeutics

A technology platform to restore CFTR function

Aceso Therapeutics’ proprietary PTGR (Post-Transcriptional Gene Restoration) platform, protected by three international patent families, is designed to restore gene expression through targeted modulation of microRNA–mRNA interactions, with the potential to deliver multiple first-in-class therapeutics across genetic, respiratory, and other diseases driven by these regulatory mechanisms.

ACESO’s technology platform is designed to restore CFTR function through scientifically grounded approaches that address expression, stability, and mutation-class biology.

01 — Who we are

Aceso, in one paragraph.

ACESO Therapeutics is a biotech company focused on developing differentiated therapeutic approaches for cystic fibrosis patients who remain underserved by current treatment options. Our work is grounded in scientific precision, translational research, and a belief that every patient deserves a path toward better breathing, stronger outcomes, and renewed possibility.

Translational research

Bench → bedside

Why Today's Therapies Fall Short

A breakthrough generation of CF therapy is still needed.

Untargeted mutations

Existing modulators fail to address rare and severe CFTR variants, leaving thousands without a viable therapy.

01

Partial responses

Many patients experience only modest improvement in lung function and quality of life on current treatments.

02

Unbearable costs

Lifetime treatment costs exceeding $300K per year per patient strain healthcare systems globally.

03

TECHNOLOGY PLATFORM

Scalable plateform technologies to improve patient care across multiple indication

A technology platform built around CFTR restoration

A scientifically grounded investment thesis, rooted in complementary mechanisms and validated mutation-class biology.

TECHNOLOGY 01

CFTR expression

Stabilize CRFTR mRNA Oligonucleotide based therapy designed to re-express functional CFTR in epithelial airway cells.

TECHNOLOGY 02

CFTR stability

Stabilization of CFTR protein at the apical membrane to extend residency and function.

TECHNOLOGY 03

Bitherapy potential

Combination strategies pairing expression and stability mechanisms for synergistic rescue.

TECHNOLOGY 04

Mutation-class expansion

Programs designed to extend CFTR rescue across all mutation classes and rare genotypes.

Other indications : COPD and more please contact us

CASE STUDIES

Selected collaborations

A look at how ACESO partners with academic, strategic, and research collaborators to advance science for underserved CF populations.

ACADEMIC · SINCE 2021

Advancing CFTR restoration through academic collaboration.

A focused research partnership exploring complementary mechanisms to restore CFTR function for underserved cystic fibrosis mutation classes.

STRATEGIC · SINCE 2022

Translating dual-therapy science into preclinical validation.

A development-focused collaboration supporting the progression of dual-therapy concepts toward structured preclinical evidence and program readiness.

RESEARCH · SINCE 2023

Building pathways for difficult-to-treat CF populations.

A scientific collaboration designed to identify mutation-class opportunities and accelerate therapeutic strategies for patients with limited options.

PLATFORM-LED. SCIENCE-GROUNDED. PATIENT-FOCUSED.

Technologies designed to move CFTR restoration forward.

Connect with ACESO to learn more about our technology platform, pipeline strategy, and commitment to advancing cystic fibrosis care.

Email

Headquarters

Montpellier, France