Money will be leveraged to characterize a lead compound and to identify biomarkers that can aid clinical testing.
Repligen received $1.125 million in research grants to support the development of new treatments for Friedreich’s ataxia. Funding will support preclinical activities until a compound is selected for clinical trials. Repligen will also use the money to develop biomarkers related to these drug candidates, which will be used to augment clinical development.
Repligen obtained $1 million from the Muscular Dystrophy Association (MDA) and $125,000 from the Friedreich’s Ataxia Research Foundation (FARA) and the National Ataxia Foundation (NAF).
The MDA grant will partially support the characterization of the lead compounds, which have already been identified. This work includes the evaluation of compounds in preclinical models of disease and the completion of the pharmacology and toxicology.
The FARA and NAF grant will support the development of new methods to quantitate the impact of drug treatment on frataxin gene expression and frataxin protein in preclinical models and in patients.
