Conjugation of universal, functionally de-targeted OneAAVTM capsid provides on-demand specificity and scalability to address key challenges associated with systemic gene therapy
OneAAVTM demonstrated to enable efficient brain or muscle transduction when conjugated with tissue-targeting ligands, with up to 1000x lower liver transduction in vivo
PARIS, France – 28 April 2026: Coave Therapeutics (“Coave”), a company redefining targeted gene therapy with a best-in-class pipeline powered by ligand-conjugated vectors, today announces it will deliver two oral presentations and exhibit two posters at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting being held 11-15 May 2026 in Boston, including groundbreaking data on its OneAAVTM universal capsid.
The data will demonstrate for the first time how OneAAVTM, Coave’s family of universal, functionally de-targeted AAV capsid templates, decouple tissue targeting from capsid design and serotype selection, addressing key challenges associated with systemic gene therapy delivery. OneAAVTM is based on AAV serotypes with very low seroprevalence in humans, displays no intrinsic tropism and no detectable transgene expression in vivo, providing a neutral backbone where biological targeting is fully decoupled from the viral serotype and instead driven by externally conjugated ligands.
Utilising Coave’s unique technology, conjugating validated tissue-targeting ligands onto the surface of OneAAVTM reintroduces targeting in a fully modular and programmable manner, enabling on-demand control of tissue specificity. This represents a fundamental shift from traditional AAV engineering, where tropism is intrinsically encoded in the capsid, to a decoupled system in which targeting, de-targeting and immune evasion can be independently tuned. This approach has the potential to address the three critical pillars of intravenously administered gene therapy which existing technologies have failed to address simultaneously: precise tissue targeting, unprecedented liver de-targeting, and immune evasion.
Coave will present in vivo mouse model data that shows conjugation of OneAAVTM with brain-specific ligands enabled efficient blood-brain barrier crossing, robust neuron-specific brain transduction, with up to 1000x lower liver transduction compared to a benchmark vector. Conjugation of muscle-targeting ligands to OneAAVTM enabled transgene expression restricted to skeletal muscle, highlighting OneAAVTM as a plug-and-play platform to vector design that can substantially reduce the time and complexity of bringing new gene therapies to patients while improving precision and safety.
Rodolphe Clerval, Chief Executive Officer of Coave Therapeutics, said: “We are excited to present these data at ASGCT, which we believe represent a significant step forward for intravenous gene therapy. By combining a universal, inert capsid with our unique ligand conjugation technology, we can achieve modular, highly precise tissue delivery while dramatically reducing off-target expression, including in the liver, greatly enhancing its safety profile compared to traditional vectors. This addresses some of the field’s most difficult challenges, paving the way for a third generation of gene therapy vectors.”
Coave’s second oral presentation and two poster presentations at the meeting, will further showcase the potential and breadth of its ligand-conjugation technology to enhance vectors for targeted gene therapy, its first-in-class suprachoroidal vector coAAV-SCS, its own lead gene therapy program CoTx-101 for retinal vascular diseases, and a TfR1-targeted AAV for central nervous system (CNS) applications.
Presentation details below:
- Oral presentations at ASGCT Annual Meeting, Boston, USA:
Presentation title: OneAAV capsid: A conjugation-enabled universal AAV capsid for receptor-driven tissue targeting
Date and time: Thursday 14 May, 11:45 AM EDT
Session title: AAV capsid engineering III
Presentation ID: 318
Presenter: Adrien Savy, AAV Principal Scientist
Presentation title: A novel suprachoroidal AAV vector engineered with ALIGATER achieves unprecedent posterior segment targeting across multiple NHP studies
Date and time: Thursday 14 May, 9:00 AM EDT
Session title: Novel advanced therapies for ophthalmic and auditory applications
Presentation ID: 423
Presenter: Julien Spatazza, Sr. Director, Discovery & Preclinical Research
- Poster presentations at ASGCT Annual Meeting, Boston, USA:
Poster title: CoTx-101: a next-generation dual-pathway AAV gene therapy for retinal vascular diseases enabled by a validated suprachoroidal vector
Date and time: Wednesday 13 May, 5:00 – 6:30 PM EDT
Session number: ASGCT159
Presentation ID: 2434
Presenter: Brahim Belbellaa, Senior Director, Preclinical Ophthalmology
Poster title: Ligand conjugation enables cross-species TfR1 targeting for AAV CNS delivery
Date and time: Thursday 14 May , 5:00 – 6:30 PM EDT
Session number: ASGCT1243
Presentation ID: 3026
Presenter: Lavaniya Kunalingam, Principal Scientist
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About Coave Therapeutics
Starting with ophthalmology, Coave Therapeutics is redefining targeted gene therapy by solving its most critical challenge: delivery. The Company is pioneering first-in-class ligand-conjugated AAVs, enabling precision vectors that are highly tissue specific, precisely delivered and safer than traditional approaches.
Through this targeted gene therapy approach, Coave has created the first suprachoroidal vector, which has the potential to transform the treatment of retinal vascular diseases such as neovascular age-related macular degeneration (nAMD/wAMD) and diabetic macular edema (DME). Coave is advancing its lead program, CoTx-101, towards clinical development, with the goal of delivering a best-in-class, durable and convenient treatment that frees patients from the burden of frequent injections while providing durable vision gains.
Coave’s plug-and-play technology can be applied to any vector or payload, enabling exploration of prevalent indications previously considered unthinkable with traditional gene therapy approaches.
Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information please visit www.coavetx.com or follow us on LinkedIn.
For more information about Coave Therapeutics, please contact:
Coave Therapeutics
contact@coavetx.com
ICR Healthcare
Amber Fennell, Stephanie Cuthbert
coavetx@icrhealthcare.com
