La voie vers un traitement | RarbmutationFR

La voie vers un traitement

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€

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