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PROT-001

Development stage: Phase 1
Lead developer: Protego Biopharma
Modality: Small Molecules
Administration: Oral

Overview

PROT-001 is an orally bioavailable small molecule kinetic stabilizer of lambda light chains (λLCs). It is being developed by Protego Biopharma for the treatment of diseases associated with misfolded or unstable lambda light chains, such as systemic amyloidosis. The drug works by stabilizing these proteins, potentially preventing their aggregation and deposition in tissues. As of June 2025, PROT-001 is undergoing Phase 1 clinical trials to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy adult participants[1][2][4].

Targets

Cλ (Immunoglobulin lambda light chain constant region)

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