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SERP-01
- Development stage
- Phase 1
- Lead developer
- YolTech Therapeutics
- Modality
- Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
SERP-01 (also known as YOLT-202) is an investigational, in vivo adenine base-editing therapy designed to treat alpha-1 antitrypsin deficiency (AATD), specifically targeting patients with the severe PiZZ genotype. Delivered via a lipid nanoparticle (LNP) formulation, the therapy contains messenger RNA (mRNA) encoding a proprietary adenine base editor along with a guide RNA (gRNA) targeting hepatocytes. It is designed to precisely correct the disease-causing SERPINA1 E342K point mutation at its source, restoring the production of functional, structurally corrected M-AAT protein while reducing the accumulation of toxic, misfolded Z-AAT polymers. This dual mechanism has the potential to address both the progressive lung and liver manifestations of AATD with a single intravenous administration. Originally developed by YolTech Therapeutics, the global rights (excluding Greater China) were licensed to Serapha Bio, which is advancing the program into Phase 2/3 clinical trials.
- Other names
- LNP-encapsulated base editorlipid nanoparticle-encapsulated base editorYolBE
02
Targets
SERPINA1 (Alpha-1-antitrypsin)
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