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Josh Mandel-Brehm

President and Chief Executive Officer, CAMP4 Therapeutics

Appears in 1 story

Notable Quotes

"For patients living with SYNGAP1-related disorder, this clearance means a potentially disease-modifying therapy is now one step closer to moving into human studies."

Stories

First treatment for SYNGAP1 genetic disorder enters human testing

New Capabilities

Leading CMP-002 into first-in-human testing

About 700 people worldwide have a confirmed diagnosis of SYNGAP1-related disorder. It causes intellectual disability and seizures, and no drug has ever been approved to treat it. On July 27, 2026, that changed at the edges: Australia's drug regulator cleared the first medicine designed to fix the disorder's root cause to be tested in people.

Updated Yesterday