Published on
Jun 30, 2025
Ultrarare Therapies Could Be Reality with Advances in Tech, Delivery and Regulation
In early May, a nine-month-old baby named KJ became the first patient to ever receive a custom-made gene therapy, a single-use CRISPR treatment for an ultrarare disease called CPS1 deficiency. That therapy, created a collaboration of a series of companies …
Source:
BioSpace
-
Pending
