Site-specific conjugation onto capsid proteins enables altering of extracellular capsid sequestration, by blocking the binding of the AAV capsid to extracellular motifs.
Ligands are rationally designed to improve cell and tissue targeting, based on defined ligand-receptor interactions.
Site-specific conjugation of capsid proteins leads to improvement of intracellular AAV capsid trafficking and payload delivery to the nucleus.
Ligand conjugation reduces coAAV exposure to immune reaction and neutralizing antibodies.
Neurodegenerative disorders are a group of conditions characterized by progressive loss of structure or function of neurons, often leading to cognitive or motor impairment. These diseases, such as Alzheimer’s, Parkinson’s, and ALS, can arise from mutations that cause either a loss or gain of function in critical genes. Gene therapy offers a promising approach to target these underlying genetic causes. By delivering functional gene copies or silencing harmful ones, gene therapy can potentially restore normal cellular function.
Inherited retinal dystrophies are rare ophthalmic pathologies that can be divided into two groups:
Retinitis pigmentosa is the most common form of inherited retinal dystrophy representing 50% of all retinal dystrophies.
While multiple genes are implicated in each of these groups, within each patient or family, only one causative gene is involved.
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JOB OPENINGS
We are a talented, passionate group of colleagues with a desire to translate innovative science into novel gene therapies for patients with neurodegenerative and ocular diseases and beyond.
We are committed to building a vibrant team combining deep expertise in AAV vector engineering and genetic construct design, innovative and advanced therapeutic product development, and manufacturing.
We are looking for more talented individuals to join our team.
Headquarters
63bis avenue Ledru Rolin
75012 Paris – France
Labs
INSTITUT DU CERVEAU ET DE LA MOELLE EPINIÈRE – ICM
Hôpital Pitié-Salpêtrière
47 bd de l’Hôpital
75013 Paris