“The use of organoids in preclinical research has reached a tipping point, with U.S. FDA approval of the first drug to enter clinical trials on the basis of efficacy data derived only from these advanced cell models.”

The use of organoids in preclinical research has reached a tipping point, with U.S. FDA approval of the first drug to enter clinical trials on the basis of efficacy data derived only from these advanced cell models.

The FDA’s decision to allow an existing drug, for which there are safety data, to be repurposed on efficacy data generated in microphysiological systems (MPS), is both a breakthrough in validating the utility of these systems and an important advance in finding treatments for rare diseases.

The paper published in April, describes testing a murine antibody that inhibits C1s, a key protease in the classical complement pathway, in an MPS designed to mimic key features of chronic inflammatory demyelinating polyneuropathy (CIDP) (...)

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Organoids making their US clinical debut with CIDP drug

Robert T. Fremeau, Jr., Ph.D.

Founder, CSO

is an accomplished scientist and biotech entrepreneur with a track record of scientific innovation and program leadership at the intersection of target validation, translation, and clinical development.

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