Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines: deploying genetic medicine for rare disease.
THRIVE is an ARPA-H program focused on developing precision genetic medicines for rare genetic diseases. It supports integrated platforms intended to generate treatments that address disease at the genetic level, with the goal of making personalized therapies more affordable and scalable. The program also aims to use existing regional treatment centers and virtual clinics so patients can receive care closer to home. Its awardee teams are pursuing different disease areas and technical approaches, including gene-editing platforms and clinical-trial models that can accommodate treatments for multiple diseases.
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