• Coave’s proprietary ALIGATER platform enables specific retargeting of AAV vectors to human receptors via a one-step chemical conjugation of peptide-based ligands at the surface of the capsid, bypassing the need for prior capsid modification

• ALIGATER offers unprecedent control over AAV and receptor interaction affinity, enhancing vector specificity and efficacy for systemic applications

Paris, France, October 22, 2024 – Coave Therapeutics (‘Coave’), a genetic medicine company developing novel delivery vectors for the treatment of eye and neurodegenerative diseases, today announced the presentation of new data in a Late-Breaking Abstract at the 2024 European Society of Gene and Cell Therapy (ESGCT) 31^st Annual Congress, held in Rome, Italy. The data highlight the potential of Coave’s proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform to enable the creation of peptide conjugated AAV (coAAV) vectors that precisely target human receptors.

Current gene therapy vectors often require high doses, leading to safety concerns due to low specificity, as well as elevated and increased manufacturing costs. Recently, several techniques have been developed to enhance vector tropism, such as genetically inserting peptides on the AAV capsid to modify their interactions with known cell receptors. While this technique is effective, it is limited on manufacturability and modularity, with constraints such as the size and presentation of the peptide.

Coave’s ALIGATER platform enables the direct conjugation of peptide ligands to the surface of AAV capsids, through a streamlined, one-step chemical process. This innovation allows for the fine-tuning of peptide density and presentation on the capsid surface, optimizing receptor targeting without the need for capsid modification or changes in the existing CMC processes.

The ALIGATER platform simplifies the engineering of both wild-type and de-targeted AAVs, offering flexibility in targeting specific human receptors, such as integrins expressed at the surface of muscle cells, or the transferrin receptor 1 (TfR1) to cross the blood-brain barrier.

Dr. Lolita Petit, Coave’s Chief Scientific Officer, commented: “Our ALIGATER platform represents a game changer in AAV vector engineering. For decades, the gene therapy field has sought ways to rationally engineer AAV vectors towards specific receptors, to ensure increased specificity, safety and transability. ALIGATER provides unmatched flexibility, while maintaining CMC processes intact. Our exciting new data showcase the platform’s potential to significantly improve the efficacy and safety of gene medicine therapies.”

Poster presentation details are as follows:

• Title: The Advanced Ligand Conjugation (ALIGATER™) platform chemically redirects AAV and LNP vectors towards specific receptors via peptide-mediated targeting
• Date & Time: Wednesday October 23; 13.30-15.00 CEST
• Poster ID: #P0074
• Presenter: Julien Spatazza, Senior Director of Discovery & Preclinical Development at Coave Therapeutics

The abstract is available to registered delegates at ESGCT Congress.

At Coave Therapeutics, we are leading the transition of genetic medicine from rare to prevalent conditions, starting with neurodegenerative and eye diseases. Our proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform introduces chemical modifications onto AAV capsids or Lipid Nanoparticles (LNPs) to overcome the limitations of current vectors on efficacy, safety, and manufacturability. 

With low doses and optimized routes of administration, our conjugated vectors have demonstrated markedly improved transduction and biodistribution across different species. Our diverse pipeline of novel genetic medicines can potentially transform the lives of people afflicted by rare and prevalent diseases – including genetically and non-genetically defined indications. 

Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information about the science, pipeline, and people, please visit https://coavetx.com/ and follow us on LinkedIn.