Gene Therapy

Gene Therapy

2021: TMA launches a holistic and multidisciplinary Gene therapy educational initiative

Gene Therapy is a fast growing field with therapeutic options already available for clinicians and patients. A review of ongoing clinical trials shows the extreme dynamism of clinical research. Around 200 clinical trials are ongoing in almost 80 diseases. The therapeutic applications encompass almost all medical fields. Gene therapy has the potential to revolutionize different cancer therapies. The correction of severe metabolic or hematologic inherited diseases are offering new hopes to help patients suffering from debilitating diseases. In Onco-hematology the CAR-T cells are also bringing new hopes and several approaches have been implemented in clinical practice. In Duchenne muscular dystrophy, gene editing by Cas9 and gRNAs for exon snipping may offer a path to future development of a clinically applicable therapeutic option. Cardiology, Dermatology, Neurology, Ophthalmology are also explored, hoping to bring innovative solutions for patient care.

TMA will soon launch an educational initiative with a series of digital conferences featuring renowned international speakers aiming at regular updates in gene therapy. With a translational and multidisciplinary approach, healthcare professionals will be able to better understand the multiple potential applications of gene therapy, for now but also in the near future. The Gene therapy updates program will run on a yearly basis and will be repeated in the following years to allow the international medical community to keep abreast with this innovative and fascinating field.


  • Ginn S, Amaya A. Gene therapy clinical trials worldwide to 2017: An update. J Gene Med 2018 May;20(5):e3015.
  • Ross M, Ofri R (2021) The future of retinal gene therapy: evolving from subretinal to intravitreal vector delivery. Neural Regen Res 16(9):1751-1759 
  • Bashir Yahya E, Alqadhi AM. Recent trends in cancer therapy: A review on the current state of gene delivery, Life Sciences, Volume 269, 2021
  • Kupatt, C., Windisch, A., Moretti, A. et al. Genome editing for Duchenne muscular dystrophy: a glimpse of the future?. Gene Ther (2021).