Winners of the 2023 "Innovative Therapeutic Proofs of Concept in Rare Diseases" Call for Projects

December 20, 2023 | News

The winners of the AFM-Téléthon and Fondation Maladies Rares call for projects entitled "Innovative Proof of Concept Therapeutics for Rare Diseases" have been announced!

This call for projects, designed to promote translational research within the Filières de Santé Maladies Rares, resulted in the selection of innovative projects with a high level of rigor.

Background to the Call for Projects

With over 7,000 rare diseases affecting 3 million people in France, 75% of whom are children, innovative solutions are needed. With no specific treatment available in 95% of cases, the need to develop innovative therapeutic approaches is urgent. With this in mind, the AFM-Téléthon and the Fondation Maladies Rares have joined forces to launch this call for projects.

Winners and their projects

  • Dr. Fabrice Antigny / RESPIFIL
    • Affiliation: INSERM UMR S999, Pulmonary Hypertension: Physiopathology and Therapeutic Innovation, Le Plessis Robinson
    • Project: "RESPIFIL - Orai1 Ca2+ channel: a new therapeutic target in PAH"
      Dr. Fabrice Antigny is exploring the Orai1 Ca2+ channel as a promising therapeutic target for pulmonary hypertension.
  • Dr. Bénédicte Chazaud / FILNEMUS
    • Affiliation: Univ Claude Bernard Lyon, INSERM, CNRS, Institut NeuroMyogene, Lyon
    • Project: "Promoting regenerative inflammation to improve muscle homeostasis in Duchenne Muscular Dystrophy".
    • Dr. Bénédicte Chazaud tackles Duchenne muscular dystrophy by promoting regenerative inflammation to improve muscle homeostasis.
  • Dr. Gilles Laverny / ORKID & OSCAR
    • Affiliation: Institute of Genetics and of Molecular and Cellular Biology (IGBMC), CNRS UMR 7104, INSERM U1258, University of Strasbourg
    • Project: "Consolidation of an innovative strategy for the treatment of calcitriol-mediated hypercalcemia".
    • Dr. Gilles Laverny consolidates an innovative strategy for treating calcitriol-mediated hypercalcemia.
  • Dr. Peter Lenting / MHEMO
    • Affiliation: INSERM, U1176, Hemostasis - Inflammation - Thrombosis, Development of new therapies, Kremlin Bicetre
    • Project: "Inducing albumin-mediated recycling of endogenous proteins: a novel strategy to treat quantitative deficiencies of plasma proteins".
    • Dr. Peter Lenting is developing a novel strategy to treat quantitative plasma protein deficiencies by inducing albumin-mediated recycling.
  • Dr. Laurent Schaeffer / FILNEMUS
    • Affiliation: Université Lyon1, Department Faculty of medicine, Laboratory Pathophysiology and Genetics of Neuron and Muscle, Lyon
    • Project: "HDAC6 inhibitors to treat Duchenne Muscular Dystrophy".
    • Dr. Laurent Schaeffer explores HDAC6 inhibitors to treat Duchenne muscular dystrophy.

Impact and Congratulations

These projects, funded to the tune of 200,000 euros, represent significant advances in research into rare diseases. 

Congratulations to the winners, who have made an outstanding contribution to the fight against rare diseases, embodying the ongoing commitment of the AFM-Téléthon and the Fondation Maladies Rares to medical research and the well-being of patients.