Genethon and Ampersand Biomedicines have announced an exclusive strategic collaboration aimed at developing a next generation of Adeno-Associated Virus (AAV) capsids engineered to improve tissue-specific targeting in gene therapies.

The partnership combines Genethon’s expertise in gene therapy development with Ampersand Biomedicines’ precision medicine and computational drug-design capabilities to address one of the major challenges in gene therapy—delivering treatments more accurately to diseased tissues while reducing unwanted exposure elsewhere in the body.

AAV vectors are widely used in gene therapy because of their effectiveness in delivering genetic material into cells. However, naturally occurring AAV capsids often show a strong preference for the liver, making it difficult to efficiently target other tissues without using higher vector doses. Elevated dosing requirements can affect treatment safety, tolerability and overall cost.

Under the collaboration, the two organisations will jointly design, evaluate and advance engineered AAV vectors intended to improve tissue precision and therapeutic performance.

The effort seeks to improve treatment outcomes by directing gene therapies more selectively toward intended tissues, potentially enabling lower doses, reducing systemic exposure and minimising immune-related complications.

Frédéric Revah, Chief Executive Officer of Genethon, said precision targeting remains critical to advancing gene therapy performance and patient safety.

He noted that combining Ampersand’s artificial intelligence-driven drug design technologies with Genethon’s expertise in gene therapy development could open new opportunities for future AAV-based therapeutic approaches.

Jason Gardner, Chief Executive Officer and President of Ampersand Biomedicines, said the company’s broader mission is to improve therapeutic precision by ensuring medicines act specifically where needed.

He added that integrating Ampersand’s targeting technologies with Genethon’s AAV engineering expertise could contribute to safer and more effective gene therapy solutions.

The partnership will leverage Ampersand’s proprietary Address, Navigate, Determine (AND) Platform, a computational drug development system designed to identify highly specific biological targets for therapeutic delivery.

Central to the platform is the company’s proprietary Address Map, which identifies tissue-specific biological targets intended to improve localisation of therapies.

As part of the collaboration, Ampersand will contribute tissue-specific targeting ligands, while Genethon will integrate these components into AAV capsids to enhance vector precision.

The initial focus area will be skeletal muscle applications, an area where Genethon has extensive scientific expertise. The companies also plan to explore expansion into additional organs and tissue targets over time.

The collaboration aims to advance safer, more precise and potentially more effective AAV-based gene therapies for patients with serious and rare diseases.

Genethon, a non-profit research organisation established by AFM-Téléthon, specialises in gene therapies for rare genetic diseases. The organisation has contributed to gene therapy innovations including treatments for spinal muscular atrophy and currently supports multiple clinical-stage programmes targeting disorders affecting the liver, immune system, blood, muscles and eyes.

Ampersand Biomedicines develops precision biologic therapies using computational approaches designed to improve how medicines are delivered within the body. Its therapeutic strategy combines disease-modifying agents with tissue-specific localisation technologies intended to maximise therapeutic benefit while limiting off-target effects.

Through the partnership, both organisations aim to help shape a new generation of precision gene therapies capable of improving outcomes for patients with rare and difficult-to-treat diseases.