République Française Inserm
Institut thématique Technologies pour la Santé

Partner 5 – Node 5 – Biotherapies Institute for Rare Diseases (B.I.R.D.)

BIRD brings together AFM’s (French Muscular Dystrophy Association) major research partners funded by the Telethon. BIRD includes I-Stem and Genethon (both located in Evry), ABI (Nantes) and the Institute of Myology (La Pitié-Salpêtrière Hospital, Paris). The Institute for Stem cell Therapy and Exploration of Monogenic diseases (I-Stem – AERES mark A; Head: Marc Peschanski) is a R&D centre, for the development of treatments based on the potential offered by stem cells and their applicability to rare diseases of genetic origin. Its activity extends from basic biological research and pathological mechanisms up to the transfer of new therapies. Atlantic Biotherapy Institute (ABI – AERES mark A; Head: Philippe Moullier) is located on the Nantes campus. It gathers more than 80 researchers involved in gene therapy from the design of recombinant viral vectors to Phase I/II clinical trials for rare diseases. It has developed a unique expertise in assessing gene transfer in the central nervous system using AAV vectors in various applications and clinical indications. Genethon (Scientific director: Philippe Moullier, Bioproduction director: Mehdi Gasmi) is a non profit organisation and its mission is to promote the development of innovative therapies based on gene therapy, for rare diseases. Genethon has wide range of skills and tools necessary to convert proofs of concept into treatments validated in patients. Several core facilities are available in the field of bioprocesses & applied vectorology, applied immunology, functional studies, clinical-grade vector production and DNA and cell banking. The Institute of Myology’s (Head: Dr Thomas Voit) mission is to be a centre of reference, where specialised consultations, fundamental and clinical research teams and teaching on muscle and muscle diseases would all be grouped together in a public hospital. This institute was founded through AFM’s partnerships with the AP-HP, INSERM, Paris (VI) University and the CEA.
BIRD aims to break new grounds in the understanding and treatment of rare genetic diseases. Within NEURATRIS, I-Stem, ABI, Genethon and the Institute of Myology will make their neuroscience-related research teams and platforms available to promote cell, gene or drug-based therapy in humans for neurological disorders.

Existing Platform(s)

BIRD/I-Stem core facilities :

  • Human pluripotent stem cells derivation and training facility (Atelier iPS): Long-term (3 months) hands-on training in reprogrammation, maintenance and differentiation of human pluripotent stem cells. So far 15 research groups have been hosted and trained over the past two years.
  • High throughput and high content screening platforms: A HTS platform with direct access to a small chemical compounds library (11,000 chemical compounds). A HCS platform with automated imaging, cellular and molecular analysis.
  • Cell factory and human stem cell bank:  A stem-cell banking and production automated platform for human pluripotent stem cell lines, with particular focus on monogenic diseases-specific resources.

BIRD/ABI core facilities :

  • Research vector core: Production of >200 lots/year distributed across Europe to academic and private entities
  • BL2/BL3 large animal facility: with surgical rooms, hosting rare genetic diseases in canine and feline spontaneous models (MPS 1, MPS 7, SMA, RPE65-/-, RPGRIP-/-, PDE6ß-/-, GRMD) unique in Europe
  • EMEA-compliant pathology core: specialized in genetically-modified tissues obtained from large animal models
  • GMP compliant cell and gene therapy core: to allow cell manipulations and viral vector manufacturing for Phase I/II cell and gene therapy trials.

BIRD/Genethon core facilities :

  • Bioprocesses & Applied vectorology: Development of large-scale production and purification methods in accordance with GMP of viral vector for gene therapies (AAV and lentiviral vectors)
  • Biomarkers: Identification of biomarkers that can be used to assess patients' response to gene therapy and new diagnostic (e.g Duchenne muscular dystrophy)
  • Applied Immunology: Assessment and monitoring of immune responses to gene therapy products (experimental models and immune assays in vitro, in vivo and in patients)
  • Functional Studies, Imaging and Histology: Assessment of functional efficacy of gene therapy strategies in animal model of pathologies of interest (functional and histological analyses)
  • Genethon-Bioproduction: The largest clinical-grade vector production core in the world that complies with GMP and has been already qualified in part as an ETGC (Gene and Cell Therapy) establishment by the AFSSAPS.
  • DNA and Cell Bank: Europe’s leading bank for genetic diseases, high quality cell and human tissue products resource

BIRD/Institute of Myology :

  • UMR 787 - Group Myology
  • Clinical Unit of Neuromuscular Pathology
  • Laboratory of Neuromuscular Physiology
  • Nuclear Magnetic Resonance Laboratory (NMR)
  • UMR 974 - Therapies of the disorders of striated muscle
  • Neuromuscular Morphology Unit

Platform(s) equipments

BIRD/I-Stem :

  • HTS/HCS Platforms :
    • Biocel 1800 automat (Agilent), HTS capacity over 70,000 parallel wells per run
    • Benchcel automat (Agilent), HTS capacity over 3,000 parallel wells per run
    • Bravo (Agilent), HCS capacity over 2,000 parallel wells per run
    • Arrayscan automat (Cellomics): imaging throughput: 50 plates per run.
  • Cell Factory :
    • CompacT SelecT automat (TAP), culture capacity 130x175cm²-Flasks per run)
    • LEAP (Cyntellect), a cell processing workstation
    • Metafer karyotyping robot (Metasystems), throughput: 80 slides per run
    • BIRD/ ABI: iCellis 1000, pre industrial scale bioreactor for adherent viral vector packaging cell lines
  • Three independent class B GMP suites allowing cell and viral vector manufacturing for subsequent in vitro transduction
  • Complete set of equipment for retinal, intrathecal, brain injections and clinical follow up in feline and canine models
  • Dedicated pathology core with full equipment for regulatory compliant analysis and reports

BIRD/Genethon :

2,500 m² of GMP compliant manufacturing and quality control space including a BL3 classified suite.

  • Four 200-liter bioreactor systems for AAV vectors (20 clinical batches a year)
  • 100-liter production system for lentivirus vectors
  • DNA and cell bank: 13 liquid nitrogen tank (660L), automated DNA extraction system (Autogene) for processing and storage of biological samples from 50392 individuals including 17063 individuals related to neurological disorders.

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