Recombinant AAV

AAV is one of the most promising vectors for therapeutic gene delivery. It has its drawbacks, however, some of which can be bypassed by recombination of the virus.

Platform

AAV are adeno-associated viruses used in biotechnology for genome editing. AAV are a popular choice of viral vector for many applications, including gene therapies. Wild type AAV are well-studied reliable vectors and are the best candidates for gene therapies, especially in ophthalmology.

How we select the AAV serotype for our therapies

In vitro transduction
(including in the presence of antibodies)

Tropism testing in human retina explants
and pathological retina-like organoids

Biodistribution and in vivo tropism tests
on wild-type and disease model mice