Over the past several years, breakthroughs in gene therapy have led to treatments for rare diseases that were deadly just a decade ago. Take Zolgensma—in 2019, it was the first gene therapy approved by the FDA to treat spinal muscular atrophy, a rare genetic disease that affects the mobility of infants and children. But gene therapies have historically had two drawbacks: They are only used for rare diseases, and they carry a hefty price tag .
“We think this is going to be an extraordinarily important therapeutic class that will revolutionize the treatment of many diseases,” says Jim Momtazee, managing partner at Patient Square Capital. Kriya’s main focus is its uniquely designed Adeno-associated viruses—viruses that are harmless when they enter the body, but deliver instructions to cells that then pump out genes that are missing in some people with genetic diseases. Though the company still plans to develop treatments for rare diseases, what sets it apart is its focus on more common genetic diseases, like some forms of diabetes and obesity.
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Fuente: CNBC - 🏆 12. / 72 Leer más »