A first-in-class AAV-gene therapy for the curative treatment of Friedreich’s ataxia

Summary

We have developed FRATAXAV, a novel patented drug designed for the curative treatment of Friedreich’s ataxia (FRDA), a rare neurodegenerative disease. This first-in-class gene therapy is based on recombinant adeno-associated virus vector (rAAV9).

Our asset is designed to supplement frataxin, a protein which is reduced in FDRA, to prevent, stop and reverse clinical symptoms.

FRATAXAV has shown efficacy in vitro and in vivo, and it is currently the only product in development showing relevant impact in functional and anatomical readouts in the central neurological and peripheral cardiac features of the disease.

Achievements

  • In 2015, we started the preclinical in vivo proof-of-concept
  • In July 2018, we established a spinoff of IGTP called Biointaxis S.L. to develop the product, create value in the early stages of product development and bring it to the clinical phase I/II in FRDA patients.
  • In 2019, we successfully completed a pre-clinical in vivo assay to demonstrate in a proof-of-concept the efficacy and safety of FRATAXAV, a gene therapy vector aimed to cure Friedreich's ataxia. The gene therapy vector has already been optimized for efficient neuronal transduction of the dorsal root ganglia in the spinal cord, the deep cerebellar nuclei and cardiomyocytes the primarily affected target tissues in FRDA.

Next Steps

With the help of CaixaImpulse Consolidate, we aim to optimize, validate and evaluate the final rAAV9-FXN clinical candidate in the reversal of ataxia and its associated neurological and cardiac dysfunctions in the acute animal model.

In addition, we aim to enter national phases at the end of the PCT procedure in 2020 and initiate the pre-clinical regulatory process. To this aim, we have identified the steps to request authorization to start the clinical phases with the ultimate objective of taking the asset to the market by 2030.

Team

Ph.D. Director of the Neurogenetics Unit. Head of the research group

Antoni Matilla Dueñas

Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol

Project leader

Ph.D. Co-Director of the Functional Biology and Experimental Therapeutics Laboratory

Ivelisse Sánchez

Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol

Researcher

Eudald Balagué Cabasés

Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol

Project manager

Daniel Cota González

Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol

Research assistant

Kerrie Adrian Campbell

Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol

Innovation and Technology Transfer Manager

Marc Cusachs

Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol

Head of Business Development

Luis Ruiz Ávila

Biointaxis S.L.

Partners

Validate-16
Consolidate-19

Scientific Area

Pharmacology

Business area

Therapeutic

Research center

Fundació Institut d'Investigació Germans Trias i Pujol