ARPA-H awards to advance personalized curative medicines for rare genetic diseases
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TL;DR
- ARPA-H awards up to $160 million to advance personalized curative medicines for rare genetic diseases
- Program performer teams, including GemmaBio and Profluent, will build toward the department's goal of accelerating the development and approval of rare disease therapies
GEMMABio supported by Profluent Bio will be developing an AI/ML-based platform for highly modular gene editors. Such gene editors promise ultimate scalability as opposed to current bespoke and artisanal editors. The team will be developing curative approaches for genetic diseases, including those that lead to extreme cholesterol levels due to inheritance.
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