NEWS BLOG

Priora is an early-stage company building a delivery route for therapies to the brain, using engineered immune cells to reach specific cell subsets non-invasively. The team of ~15 researchers is based in London and San Francisco. Open roles, all London and full-time: VP of Translational Research; Senior Scientist, In Vivo Neuroscience; Senior Computational Scientist; Research Scientist, In Vitro; and Scientist, Mouse Models. Full details and application links for each role ar

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This new award has been created to recognise individuals for their outstanding contribution to ESGCT's mission to advance knowledge.

The 9th ESGCT Spring School took place in Coimbra, Portugal from 14-17 April 2026. The Spring School was jointly organised with the the Gene Therapy Center of Excellence Portugal (GeneT). Spring School attendees can log in to their registration portal to access their registration details, event resources and certificates of attendance via the link below.

Join us for the 33rd ESGCT Annual Congress, which takes place in collaboration with DG-GT from 27-30 October 2026 at the Congress Center Hamburg, Germany.

View Claire's biography below 1. What advice do you wish you had received at both the start and the conclusion of your PhD journey? Be open to change — your original plan might evolve, but the learning along the way is just as valuable. Expect setbacks — it’s is a safe space to make mistakes, so treat them as part of the process, not as failures. The experience is as much about training and developing your skills as it is about producing results. Start writing up before

Foundation For Rare Diseases (Fondation Maladies Rares, France) organises an online MOOC on gene and gene-based cell therapies for rare diseases . The course features contributions from leading U.S. and European researchers — including Alain Fischer, Heather Gray Edwards, Alessio Cantore, Zoltan Ivics, Sylvie Odent, Salima Hacein-Bey-Abina, Manuel Gonçalves, Marina Cavazzana, Allison Bradbury, Anne Galy, Chantal Pichon, Claire Booth, Jeffrey Medin , among others — as well a

ESGCT are greatly saddened by the loss of Professor Claudio Bordignon, a pioneer in the gene therapy field, and the first President of the ESGCT.

Last Thursday, the FDA approved the LAD gene therapy developed by Juan A. Bueren and Elena Almarza, in collaboration with the Adrian Thrasher team. This treatment was licensed to Rocket Pharma, who developed the clinical trial and presented it to the FDA. We believe this is terrific news for the gene therapy field and for ESGCT, as all these advances have come from ESGCT members, and the society has played a fundamental role in favouring this collaboration and in the developm

Hosted as part of the AROUND THE WORLD SERIES in collaboration with the Coalition of International Gene Therapy Societies.

The Early Career Investigator Award recognises contributions made by ECRs.

This award is given each year to an individual or a team who has made a significant impact in the field of gene and cell therapy.

The Founders Award is given to an ESGCT senior scientist to celebrate key discoveries in gene and cell therapies.

The Early Career Investigator Award recognises contributions made by ECRs.

Gene and cell based approaches in neuromuscular disease Nantes, 14-16 January 2026 ESGCT is committed to the training and support of students and early career researchers, and the ESGCT Academy is a part of this remit. Each Academy is dedicated to a distinct topic and students undertaking a related PhD project are eligible to attend. The 2026 Academy focused on the development of gene and cell-based approaches in neuromuscular disease. The workshop provided the opportunity t

The ESGCT would like to congratulate Fondazione Telethon and HSR-TIGET on the milestone achievement of the FDA approval of WAS ex vivo gene therapy. Please see below their statement: We are honoured to share a moment of profound significance for us, for patients, families, and the scientific community. The U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra , the first gene therapy for the treatment of Wiskott-Aldrich

The 32nd ESGCT Annual Congress took place in collaboration with SETGYC from 7-10 October 2025 at Fibes in Seville. Congress delegates can log in to their online registration portal to access the Congress abstract book, online programme, and Certificate of Attendance. Login here > Abstracts published by Human Gene Therapy will be available soon. Event Statistics (Click to enlarge) We are very grateful for the support of all our sponsors and partners.

Outstanding Achievement Award Juan Bueren, Ciemat, Madrid Professor Juan A. Bueren is a leading global expert in gene therapy and hematopoietic stem cell research, with pioneering contributions to treatments for rare diseases such as Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency (LAD). He developed the first successful gene therapy for FA without prior conditioning—avoiding the toxicity of traditional transplantation—now under EMA and FDA review. He also led preclinic