Frank Staal receives Rare Angel Award

Professor Frank Staal

Professor Frank Staal, reNEW Leiden PI, has received a prestigious Rare Angel Award for his ongoing research on stem cell based gene therapies for hereditary immune deficiencies. He received the award on Rare Disease Day, February 29th,  during a national event at the Royal Palace Soestdijk.

The Rare Angel Award is an initiative of the Dutch patient advocacy organization (VSOP). This organization is committed to help 1,2 million patients in the Netherlands that suffer 1 of 7000 rare diseases on behalf of over 100 smaller member organisations. Every year, people that have made a remarkable contribution to the lives of these patients receive a Rare Angel Award in three categories (media, patients/representatives and healthcare/research).

Staal was nominated for the award for his ongoing work on brining stem cell based gene therapies towards the clinic by the Dutch Foundation for Immune Deficiencies. Over the last years, his team has developed a gene therapy for patients without a functioning immune system suffering from the genetic immunodeficiency RAG1-SCID. In this therapy, the patient’s own stem cells are “repaired” in the lab and transplanted back into the patient. The therapy has now reached a phase I/II clnical trial in collaboration with Murdoch Children’s Research Institute, reNEW Melbourne.

“I am profoundly honored, grateful and deeply humbled to have been given the 2024 Rare Angel Award. This recognition holds immense significance as it represents the collective voice of patients,” says Staal. “This award is testament to the shared dedication and efforts of all my collaborators and funders towards advancing gene therapy for rare diseases. I remain committed to our ongoing mission to improve the lives of those affected by rare diseases.”

Within reNEW, Staal aims to develop stem cell based gene therapies for a plethora of other immunodeficiencies based on the principle used in his ongoing trial. The research should lead to curative treatments for a larger amount of patients suffering from rare genetic immunodeficiencies. Read more about Staal’s research here: Stem cell research for blood and immune system diseases – reNEW

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