Novel precision therapies are transforming the treatment of rare diseases. In autoimmunity, engineered cell therapies such as CAR T and CAAR T are delivering targeted immune reset. In metabolic disorders, gene therapy and genome editing are showing the potential for durable cures.
In this report, we explore recent breakthroughs, from early trial results in autoimmune conditions to genome editing successes in inborn errors of metabolism, and examine the challenges of bringing these therapies into real-world practice.
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