17 September 2018 / Paris, France
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17 September 2018
Paris, France
Gene Therapy Partnering Day


8:30-8:55  REGISTRATION AND COFFEE

MORNING SESSIONS

9:00   INTRODUCTION

Presentation of the DIM “Thérapie génique” initiative by Marina Cavazzana

Medicen Paris Region initiatives to support gene therapy development

9:10   Challenges in the field of Gene Therapy

  1. Technology tranfer:  Federico Mingozzi - Chief Scientific Officer, Spark Therapeutics 
  2.  "T-cell precursors to treat immune deficiencies" - Isabelle André-Schmutz - Group leader of Human Lymphohematopoiesis Lab, Imagine Institute
  3. Regulations- Nathalie Morgensztejn, Head of Department of Virology and Gene Therapy, ANSM
  4. “Reimbursement considerations for Gene Therapies”- Panos Kefalas - Head of Health Economics & Market Access, Cell and Gene Therapy Catapult

10:10    Pitch of research projects 

  1. "Dual AAV-mediated gene therapy cures deafness in a DFNB9 mouse model" - Said Safieddine - Researcher in Genetics and physiology of hearing lab, Pasteur Institute
  2. "Gene therapy approach to treat mitochondrial diseases caused by defects in mitochondrial gene expression" - Benedetta Ruzzenente -  Post-doctoral fellow, Genetics of mitochondrial diseases Lab, Imagine Institute
  3.  "Gene therapy for MSUD" - Manuel Schiff- Doctor, Reference centre for inherited metabolic diseases, Robert Debré Hospital and Imagine Institute
  4. "Development of an in vitro model for cerebral vasculopathy monitoring in gene therapy of sickle cell disease" - Kim-Anh Nguyen-Peyre - Postdoctoral fellow Transfusion and Pathologies of the Red Blood Cell, EFS
  5. "Gene therapy for sickle cell disease: new solutions explored" - Anne Galy - Director of integrated genetic approaches in therapeutic discovery for rare diseases, Généthon 
  6. "Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus" - Annarita Miccio - Group leader of Chromatin and gene regulation during development lab, Imagine Institute 
  7. "AAV vector platform for R&D purposes" - Marcelo Simon Sola - Engineer, AAV platform, Imagine Institute
  8. "Gene Therapy in haemophilia A : a hope for children too" - Annie Harroche- PH, Laboratory of biological hematology, Necker Enfants-Malades Hospital 

11:30    COFFEE BREAK

11:45   "Overview of gene therapy development by Pfizer" - Donna Armentano, Executive Director, Global Head GTx, External Science and Innovation at Pfizer, Inc.

12:00  Innovative technological solutions 

  1. Eukarÿs - Philippe Jais, CEO & CSO 
  2. Flash Therapeutics - Pascale Bouillé, President & founder 
  3. BrainVectis - Sandro Alves , Director of Preclinical Research 
  4. AmplyCell - Bastien Horrion, CSO 
  5. 20Med Therapeutics -  Lodder Michiel, CEO
  6. Amarna Therapeutics - Peter de Haan, CSO
  7. Astraveus - Jérémie Laurent , CTO and Founder


13: 00   LUNCH BREAK

AFTERNOON SESSION

14: 00   One-to-one partnering meetings (20 minutes/meeting)

Connect with the main European players in the field of gene therapy to build future collaborations

 

17: 00   END OF THE EVENT

Registration

Closed since 31 July 2018

Location

Imagine - Institut des Maladies Génétiques
Boulevard du Montparnasse 24
Paris, France

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