AON based therapy for Startgardt disease

Astherna is a Nijmegen-based start-up company and spin-off of the Radboudumc that is developing a treatment for Stargardt disease (STGD1). Astherna was founded by the inventor, Prof. Rob Collin and key opinion leader, Prof. Carel Hoyng together with Radboudumc Holding BV.

STGD1 is an inherited retinal disease that has a global prevalence of 1:10,000 individuals. Patients with STGD1 have progressive vision loss, often leading to legal blindness. STGD1 is caused by bi-allelic mutations in the ABCA4 gene. There is no treatment available, illustrating the unmet medical need.

The proposition of Astherna is based on antisense oligonucleotides (AONs), small synthetic RNA molecules that, due to their specificity and easy design, have a high therapeutic potential. Astherna holds an exclusive license for four patents, employing AONs for various ABCA4 mutations. Proof-of-concept for these AONs has been demonstrated in relevant patient-derived cellular models. Furthermore, this approach has recently been successfully employed for two other subtypes of retinal disease in humans.

The calculated market value of our proposition exceeds 100 million Euro per year, based on the number of Caucasian STGD1 patients available for our therapeutic solution, an intraocular injection two-to-four times per year, and a market penetration of 25%. In addition, in collaboration with Radboudumc, AONs for other indications are expected to be patented and brought to clinical development by Astherna.

Currently, Astherna is raising seed funding of 1M€. This seed funding will be utilized to hire an experienced management team and experts in the field to complete our team, and to cover the pre-clinical development until final formulation of our lead molecule and starting the toxicology study.

Rob Collin
Carel Hoyng
Corné van den Kieboom
Startup activities

Venture Challenge Summer 2021

Rob Collin