PROT-001: Our Lead Program

Targeting AL Amyloidosis

PROT-001 is our most advanced program, focused on AL (light chain) amyloidosis—a severe and often fatal disease driven by the misfolding of immunoglobulin light chain proteins. These misfolded proteins aggregate into amyloid deposits that accumulate in vital organs, leading to progressive organ dysfunction and death.

Mechanism of Action: PROT-001 is a small molecule pharmacological chaperone designed to bind and stabilize light chain proteins, preventing their misfolding and downstream toxic aggregation at the source.

Clinical Progress: Following successful completion of Phase 1 clinical studies in late 2025, PROT-001 is advancing into pivotal clinical trials in 2026.