LOS ANGELES, April 19 — Malaysian physician-scientist Dr Swee Lay Thein and American researcher Dr Stuart H. Orkin have won the US$3 million (RM11.86 million) Breakthrough Prize in Life Sciences for research that helped create the first approved CRISPR gene-editing therapy.

According to a report in LiveScience, the pair were honoured for work that led to Casgevy, a treatment seen as the first functional cure for sickle cell disease and severe beta-thalassaemia.

The breakthrough works by switching fetal haemoglobin back on — a natural form of haemoglobin used before birth that does not trigger the dangerous cell damage linked to the disorders.

Their discovery centred on blocking BCL11A, a gene regulator that normally shuts fetal haemoglobin off as children grow.

According to its website, the Breakthrough Prize describes itself as the world’s largest science prize, and this year’s Los Angeles ceremony drew celebrities including Anne Hathaway, Margot Robbie, Robert Downey Jr. and Michelle Yeoh.

“I feel extremely honored, overwhelmed and humbled,” Thein reportedly said after receiving the award in Los Angeles.

Thein was born in Kuantan and studied medicine at the University of Malaya before building her research career in the United Kingdom and United States

Casgevy has significantly improved outcomes for some patients, reducing severe pain crises and the need for regular blood transfusions.

However, the treatment remains costly, physically demanding, and can take up to a year to complete.

Researchers are now racing to develop simpler gene-editing injections that could make such cures accessible to far more patients worldwide.