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 organizations. 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 (Sichting Afweerstoornissen, SAS). 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 clinical 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.”