|Platforms||ATMP & Biological, Biomarkers|
|Tags||Biotherapies, Gene transfer, Pluripotent Stem cell (iPS and ESC)|
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.
2 rue Henri Desbruères
91100 Corbeil- Essonnes
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.
1 bis rue de l’Internationale
91 000 EVRY
Atlantic Biotherapy Institute
ABI – Atlantic Biotherapy Institute (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. ABI acquired a unique expertise in gene transfer for the central nervous system using AAV vectors in multiple applications and clinical indications. It is at the origin of the first French gene therapy trial on humans for Leber amaurosis, a rare genetic disorder of vision, which was launched in October 2011 at the Nantes University Hospital.
8 quai Moncousu
BP 70721, 44000 Nantes
The Institute of Myology
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.
Hôpital Pitié Salpêtrière
105 bd de l’Hôpital
75013 PARIS – France
This institute is part of NeurATRIS, the French part of EATRIS.