Good News for the Bubble Baby Disease Treatment Backed by Millions from California Taxpayers

^{Photo: CIRM}

Twenty children seeking treatment for a rare affliction called the “bubble baby disease” today have some big-time, good news concerning a life-saving genetic therapy that they were once denied as the result of a tangled affair involving private profit and the public funding of cutting-edge scientific research.  

After being halted nearly two years ago, a California state-financed clinical trial that can help the children is now solidly on its way to a new beginning at UCLA. Treatments of the children could begin as early as June. 

The California stem cell agency is expected to provide $5.8 million to resume the trial at UCLA. Millions more are likely to be forthcoming from the agency, officially known as the California Institute for Regenerative Medicine (CIRM).   

Despite the success of the trial over the last several years -- “100 percent overall survival” of 50 patients -- it was abandoned in 2020 by Orchard Therapeutics, PLC. Instead, Orchard turned towards research that it deemed would be more profitable.  At that time, Orchard had exclusive rights to the therapy, which was...