Biotherapy Institute for Rare Diseases – BIRD

The Biotherapies Institute for Rare Diseases - BIRD - federates the expertise of four laboratories (I-Stem, Genethon, Myology Institute and Atlantic Biotherapy Institute - ABI) created and supported by the AFM-Telethon. World leaders in biotherapies for rare diseases, they share the common goal of accelerating the availability of innovative treatments for patients.


  • I-Stem, the Institute of stem cells for the treatment and study of monogenic diseases (Evry) is an international referral R&D center dedicated to the development of treatments based on pluripotent stem cells (iPS and ECs) applicable to rare genetic diseases. Its aim is to promote these approaches from their development to their transfer to clinical research. I-Stem has 80 employees.

  • Genethon’s (Evry) mission is to promote the development of innovative therapies based on gene therapy for rare diseases. It holds/bears a wide range of skills and tools necessary to convert the proofs of concept to validated treatments in patients. Several facilities are available in the fields of bioprocessing and applied vectorology, applied immunology, functional studies, production of clinical grade vectors and DNA and cell banks.

  • Atlantic Gene Therapies (Nantes) employs about 80 researchers involved in the development of gene therapy strategies, from the design of viral vectors up to phase I and II clinical trials for genetic retinal diseases, as well as neuromuscular and central nervous system diseases.  The UMR 703 PAnTher INRA, implanted in the National College of Veterinary Medicine in Nantes, has been working since over 15 years in gene therapy translational research. Animal models of pathologies are at the centre of the unit's integrated approach, from gene to animal. The unit is certified ISO 9001:2015 and develops gene therapyc approaches to fight genetic diseases affecting the nervous system (neurodegenerative diseases of lysosomial overload, motoneurons diseases...). The UMR 703 PAnTher includes a core facility, APEX, dedicated to veterinary pathologies and to fluorescence bio-imaging offering an integrated expertise in veterinary histopathology and in tissue and cell phenotyping, in a wide spectrum of animals.

  • The Institute of Myology (Pitié Salpêtrière Hospital, Paris) is a center for research, healthcare and education on the muscle and muscle diseases that participates today in more than 30 clinical studies. With 280 doctors, researchers, engineers, paramedics, doctoral and post-doctoral students and other experts, the Myology Institute brings together diverse and complementary skills from basic research to healthcare. It was founded by the AFM-Telethon in partnership with AP-HP, Inserm, Paris VI University and the CEA.


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