ERN-EuroBloodNet and the EDITSCD Consortium are launching a series of webinars entitled EDITSCD & ERN-EuroBloodNet: Focus on Genetic Therapy for People Living with SCD, aimed at providing an overview of gene therapy and genome editing approaches for Sickle Cell Disease. This educational program is developed in partnership with the EDITSCD project, led by Annarita Miccio (Imagine Institute of Genetic Diseases), and benefits from the contributions of members of the EDITSCD steering committee and SCD patient representatives: Miriam Santos Freire (APPDH Associação Portuguesa de Pais e Doentes com Hemoglobinopatias, Dianaba Ba and David Issom (Association Suisse Drépano), the three of them are part of the European Sickle Cell Federation).
The sessions will be led by renowned experts in the field and are designed to offer both fundamental knowledge and advanced perspectives, from disease mechanisms to clinical translation, to audience. Topics will cover lentiviral gene therapy, CRISPR/Cas9 tools and safety, as well as regulatory aspects and clinical trials.
This program consists of a cycle of 7 webinars provided by key experts speakers on gene therapy and genome editing approaches for sickle cell disease.
It will be conducted on the Zoom Platform, using AI translation by live, starting on the 26th of September 2025 and finishing on the 2nd of October 2026.
It will be held every two months at 5 pm (CET), on different days of the week. Please check the program below for specific dates.
Each webinar is a live session of 1 hour, with 20-30min of lecture and closing with a Q&A session, where interaction between speakers and the public will be encouraged.
Program:
- Session 1: Introduction to Sickle Cell Disease (SCD) and Gene Therapy
Date: 26 September 2025
Speaker: Annarita Miccio (Imagine Institute des Maladies Génétiques) and Prof. Michaela Semeraro (INSERM CIC1419 and AP-HP.Centre - Université de Paris-Cité)
- Session 2: Basics of Gene Therapy with Lentiviral Vectors
Date: 26 November 2025
Speakers: Annarita Miccio (Imagine Institute des Maladies Génétiques), Giuliana Ferrari (IRCCS Ospedale San Raffaele), and Prof. Michaela Semeraro (INSERM CIC1419 and AP-HP.Centre - Université de Paris-Cité)
- Session 3: Genome Editing: CRISPR/Cas9 and SCD - How It Works and Its Uses
Date: 16 February 2026
Speaker: Annarita Miccio (Imagine Institute des Maladies Génétiques) & Claudio Mussolino (Universitaetsklinikum Freiburg) & Mario Amendola (Institut National de la Santé et de la Recherche Médicale - INSERM)
- Session 4: Genome Editing: CRISPR/Cas9 Advanced Tools and SCD - Using New Methods
Date: 15 April 2026
Speakers: Annarita Miccio (Imagine Institute des Maladies Génétiques) & Marcello Maresca (AstraZeneca AB)
- Session 5: Safety of CRISPR/Cas9
Date: 3 June 2026
Speakers: Ayal Hendel (Bar-Ilan University) & Toni Cathomen (Universitaetsklinikum Freiburg)
- Session 6: Future Developments and CRISPR/Cas9 for SCD
Date: 17 July 2026
Speaker: Annarita Miccio (Imagine Institute des Maladies Génétiques)
- Session 7: Regulatory Path to the Clinic
Date: 2 October 2026
Speaker: David Morrow (EATRIS)
