We will bring about a step change in gene therapy development.
We have a strong current pipeline with two programmes in development and a powerful discovery engine for future programmes – FunSel, our in-vivo screening platform.
We are challenging the status quo and are developing therapies that have the potential to stop or significantly slow down chronic kidney diseases.
Our pipeline has therapies in development for kidney diseases with high unmet need, encompassing rare monogenic diseases, as well as much more prevalent common forms of kidney disease. They both rapidly lead to end-stage renal disease, with devastating consequences to patients.
Our current pipeline is supplemented by FunSel, our “gene therapy search engine”.
This proprietary technology was developed by our co-founder Professor Mauro Giacca and allows us to discover new gene therapy candidates in a completely novel and functional way – regardless of causes of the disease.
FunSel contains a library of more than a thousand biological factors, encoded in AAV vectors, that can be screened for novel gene therapy candidates. It is a library that is unconstrained by genetics.
FunSel does not need to know which gene causes a disease. This will enable us to deliver the promise of gene therapy to non-monogenic disorders and treat a far broader patient population.
Going forward, FunSel can also be applied to a wide range of diseases, in the kidney and in other organs. FunSel provides us with multiple opportunities to add value through partnering and potential collaborations.
Library
Library of viral vectors encoding secreted proteins
Testing
Functional testing in animal models of disease
