Nonprofits launch ultra-rare immune disease gene therapy Waskyra at $3.25 million price

Two nonprofit organizations have received FDA approval for a gene therapy that targets an ultra-rare immune disorder.

The therapy, called Waskyra, is now being launched with a list price of $3.25 million per patient.

The steep price highlights the financial challenges of delivering gene therapies for rare diseases, while the nonprofits aim to meet an unmet medical need.