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The Gene Therapy Area of Major Interest (DIM Gene Therapy) launches the first edition of Gene Therapy for Rare Monogenic Diseases, an international symposium dedicated to recent knowledge and progress in the field to ensure faster and more efficacious development of therapies for diseases with high unmet medical needs. With 200 intended key actors, this two-day event is the unique opportunity to discover the latest gene therapy trends and to see emerge high-potential collaborations.

Back to the first edition of the GTRD

For the first edition of Gene Therapy for Rare monogenic Diseases, the DIM Gene Therapy and the Imagine Institute brought together nearly 185 European participants around the challenges of clinical gene therapy.

Opened by Pr Arnold Munnich, president of the Imagine Institute and Mrs Zahia Zaidat, project manager in research at Paris Region, they reminded the implication of the Imagine Institute in this event and in the DIM Gene Therapy, and the desire to make the Paris Region, a pioneering territory in the field with a high potential for innovation.

The main European actors in gene therapy attended plenary sessions dedicated to the challenges of gene therapy, with the exceptional presence of Dr. Alessandra Biffi, co-director of gene therapy program at Boston Children’s cancer and blood disorders center, United States; Pr Amit Nathwani, Professor of haematology at University College of London, Pr Matthew Porteus, professor of paediatrics at Stanford Medicine. The 2 days event has been punctuated by poster sessions and presentations of the research projects supported by the DIM Gene Therapy.

From left to right: Mrs Zahia Zaidat, project manager in research at Paris Region ; Pr Matthew Porteus, professor of paediatrics at Stanford Medicine ; Pr. Marina Cavazanna, DIM Gene Therapy scientific coordinator ; poster sessions.

This event was made possible thanks to the support of the Ile-de-France Paris Region, initiator of the program DIM Gene Therapy which aims to promote the excellence and the influence of the territory and these teams on innovation.

We also thank all of our generous partners who contributed to the success of this event: Audentes Therapeutics, RegenxBio, Rocket Pharma, Spark Therapeutics, Bluebirdbio, Delphi Genetics, Vector Builder and Viralgen.

“Participating to this meeting was an amazing experience. It was one of those rare opportunities when you can meet high-level scientists and clinicians, listen to their talks, ask questions and discuss in an informal and friendly atmosphere. Great speakers, interactive audience, top science; I am looking forward to the next GTRD meeting!”, as testifies Fulvio Mavilio, professor of molecular biology, university of Modena and Reggio Emilia in Italy, and Scientific Officer & Senior Vice President at Audentes Therapeutics. Successful bet for the DIM Gene Therapy that delighted many participants whether they are academic researchers, clinicians, big pharma companies or Biotechs.

For its first successful edition, Gene Therapy for Rare monogenic Diseases has reinforced, in the light of personalized medicine, the grade of gene therapy at the heart of therapeutic innovation.

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Over 20 international experts from academia, industry and biotechs will address issues to discover the latest trends and approaches in the field of gene therapy, by sharing their expertise and knowledge.

Head of Human Lymphohematopoiesis Lab

Imagine Institute

Professor of Molecular Biology at University

University of Modena and Reggio Emilia

Professor of pediatrics – Stem cell transplantation

Stanford Medicine, Department of Pediatrics

Professor of Haematology and Director

Katharine Dormandy Heamophilia Centre and Thrombosis Unit, Royal Free Hospital

Professor of Ophthalmology, University Paris VII Diderot

Ophthalmological Foundation A. de Rothschild - Bichat Hospital

Director of the Center for Gene Therapy

The Research Institute at Nationwide Children's

Director of the Hematopoietic Innovative Therapies Division


Director Gene Therapy Program

Dana-Farber/Boston Children’s Cancer and Blood Disorders Center

Senior Vice President & CSO

RegenX bio

Pharmacology and toxicology lead

Spark Therapeutics

Associate Clinical Director, Clinical Research Development

Bluebird Bio

Researcher in Transfusion and Pathologies of the Red Blood Cell Unit

Henri-Mondor University Hospital

Head of Unit at Scientific Institute San Raffaele

Scientific Institute San Raffael

Head of Chromatin and gene regulation during development Lab

Imagine Institute

Group Leader Biotherapeutics departement

Vision Institute

Researcher in Immunology department

Pitié-Salpètrière Hospital

Senior scientist in Integrated Biology of the Red Cell Unit

National Institute of Blood Transfusion

Researcher in Hearing Genetic and physiology unit

Pasteur Institute

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