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Clinical Trials and Commercialisation Workshop 2024

Tuesday 22 October

Setting the stage for gene editing: clinical and scientific-regulatory considerations

Chairs: Martina Schuessler-Lenz, Alessandro Aiuti


Progress in human gene editing technologies have the potential to expand treatment options for patients with severe diseases and unmet medical need. The translation of research candidates to the clinic and the recent approval of the first gene-editing based therapy are a reflection of this rapidly developing field. While the development paths for gene therapies like CART-cells and recombinant AAV vectors have matured gene editing approaches present new challenges for all stakeholders, developers, regulators and as well as experts involved in health technology assessments.

In this workshop you will hear about gene editing related product characterization and control, non-clinical data requirements towards clinical trial authorization, patient selection in clinical trials as well as clinical evidence requirements for marketing authorization and patient access. The workshop will address regulatory, scientific and clinical aspects and provide a forum for exchange between the different stakeholders involved in the development of gene-editing based gene and cell therapies.

09:00-10:00 | Registration Foyer

10:00-11:35 | Meeting Room 2

Workshop 1


10:00-10:05: Martina Schüssler Lenz, PEI Langen, Alessandro Aiuti, SR-TIGET, Milan


10:05-10:20: Martina Schüssler Lenz, PEI Langen


10:20-10:45  CTC01: Franco Locatelli, Italy

Clinical experience with an ex-vivo gene editing product in patients with hemoglobinopathies

10:45-11:10  CTC02: Matthias Renner, PEI, Langen

Key aspects of manufacture and quality control

11:10-11:35 CTC03: Brigitte Anliker, PEI, Langen

Regulatory non-clinical aspects of gene-editing based products

11:35-11:45 | 
Coffee / Bio Break

11:45-13:00 | Meeting Room 2

Workshop 2


11:45-12:10 CTC04: Jan Müller-Berghaus, PEI Langen

Clinical aspects

12:10-12:35 CTC05: Odoardo Olimpieri, AIFA Rome

Patient access to gene and cell therapies – the Italian HTA perspective/experience

12:35-13:00: Roundtable and Q&A

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