Weg zur Therapie

2013
2013

First patient with RARB Mutation identified

2015
2015

Research activities on RARB mutation started by the Michaud and Krezel Lab

2016
2016

Establishment of disease models (ongoing)

Already funded
2016

Patient registry and natural history studies (ongoing)

Costs: 95.000€
2020
2020

Determination of disease mechanisms (ongoing)

Already funded but additional resources required: 195.000€
2021
2021

Identification of biomarkers

2021

Gene therapy approach vs. small molecule approach via drug repurposing

Costs: 1.030.000€
2022
2022

Candidate Selection Point (CSP)

2022

Preclinical safety trials

Costs: 1.200.000€
2023
2023

Regulatory activities (Submission in USA and EU, IND / CTA)

Costs: 110.000€
2024
2024

First human in clinical trial (Phase 1)

Overall Costs: 2.630.000€