Breakthrough Prize Awards $3 Million Each to Six Scientists for Gene Therapies and KRAS Research

The 2026 Breakthrough Prize awards $3 million each to six scientists for transformative medical discoveries, totaling $18.75 million in recognition. Jean Bennett, Katherine High, and Albert Maguire share one prize for developing the first FDA-approved gene replacement therapy treating Leber congenital amaurosis, a rare inherited blindness affecting the RPE65 gene, with nearly all eligible US patients now treated and maintaining stable vision over a decade later. Stuart Orkin and Swee Lay Thein receive another prize for research identifying BCL11A as the master repressor of fetal hemoglobin, leading to Casgevy, the first CRISPR-based medicine approved for sickle cell disease and beta-thalassemia affecting millions worldwide. Rosa Rademakers and Bryan Traynor are honored for simultaneously discovering the C9orf72 mutation in 2011, explaining about a third of familial ALS and frontotemporal dementia cases in European populations. Additional prizes recognize mathematician Frank Merle for work on nonlinear evolution equations and muon g-2 collaborations spanning six decades at CERN, Brookhaven, and Fermilab.

Gene Therapy Breakthroughs for Inherited Blindness

• ▪In 2005, Katherine A. High invited Jean Bennett and Albert Maguire to collaborate on a human trial for the Leber congenital amaurosis gene therapy
• ▪Nearly all eligible Leber congenital amaurosis patients with RPE65 mutations in the United States have been treated with the gene therapy
• ▪Jean Bennett, Katherine A. High, and Albert Maguire share the 2026 Breakthrough Prize in Life Sciences for work leading to the first FDA-approved gene replacement therapy

Gene Editing Treatments for Blood Disorders

• ▪Stuart H. Orkin and Swee Lay Thein share the 2026 Breakthrough Prize in Life Sciences for research that transformed sickle cell disease and beta-thalassemia from incurable to treatable conditions through gene editing therapy
• ▪Swee Lay Thein mapped the trait of persistent fetal hemoglobin production to chromosome 2

Discovery of Genetic Cause of ALS and Frontotemporal Dementia

• ▪In 2011, Rosa Rademakers and Bryan Traynor simultaneously identified a mutation in the C9orf72 gene

Advances in Mathematics and Fundamental Physics

• ▪The Breakthrough Prize has conferred more than $340 million over 15 years
• ▪Each of the six 2026 Breakthrough Prizes carries an award of $3 million
• ▪Three muon g-2 collaborations at CERN, Brookhaven National Laboratory, and Fermilab received the 2026 Breakthrough Prize in Fundamental Physics
• ▪Frank Merle received the 2026 Breakthrough Prize in Mathematics for work advancing the understanding of nonlinear evolution equations
• ▪The Breakthrough Prize is in its 14th year as of 2026
• ▪The 2026 Breakthrough Prize total prize money is $18.75 million

Perspective of Breakthrough Prize founders

• ▪The 2026 Breakthrough Prize awards honor discoveries that have led to FDA-approved therapies and clinical treatments