General objectives:

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• To bring together in one room researchers from all over the world working on Wiskott-Aldrich Syndrome (WAS)

• To exchange knowledge on novel findings in basic biology, experimental therapeutics and advances in clinical care of patients affected by WAS

• To bridge the gaps between basic biology research and translational application

• To encourage new collaborations between established experts and to attract more junior researchers to the field, ultimately leading to advancement in care

 

Main outcomes.

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The symposium’s agenda was arranged in three main sections: basic biology, gene therapy and clinical research. Each part was introduced by a keynote lecture given by a renowned expert in the respective field, followed by scientific presentations of submitted abstracts.

The basic biology section was headed by Prof. Anna Villa from Milano presenting on the biology of platelet defects in WAS, followed by presentations focused on understanding the biology of WAS protein and finding potential novel strategies to stabilize the defective WAS protein, with the ultimate goal of disease amelioration.

The second part was highlighted by the presentation of Prof. David Rawlings from Seattle on immune tolerance in WAS and the development of stem cell gene addition therapies. The scientific abstracts presented in this session focused on the various pre-clinical approaches and clinical studies of stem cell gene therapy for WAS, before it was concluded by a panel discussion on how to make these novel treatments accessible for patients.

In the third part focused on clinical advancements Prof. Michael Albert from Munich summarized the journey of hematopoietic stem cell transplantation from a highly experimental treatment 50 years ago to a standard of care for the majority of WAS patients in 2022. The clinical abstracts addressed the challenges of HSCT in restricted resource countries, the treatment of autoinflammatory complications, and the presentation of female WAS mutation carriers.

In summary, the symposium summarized in a unique way the advancement of knowledge on WAS that was accumulated over the last few years. Especially in light of the COVID pandemic the personal interaction was overdue and extremely helpful to foster new research collaborations in order to further advance the field.