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HEAL-201
- Development stage
- Preclinical
- Lead developer
- Cellectis
- Modality
- Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
HEAL-201 is an in vivo epigenetic editing therapy being developed by Cellectis for the treatment of severe hypercholesterolemia. Delivered via lipid nanoparticles (LNPs), HEAL-201 consists of mRNA encoding a transcription activator-like effector (TALE) epigenetic modulator (PCSK9 TALEM) designed to target the promoter of the human *PCSK9* gene. By inducing stable epigenetic silencing of *PCSK9*, the therapy aims to provide a long-lasting reduction in PCSK9 protein levels and lower circulating low-density lipoprotein cholesterol (LDL-C). Preclinical studies in liver-humanized murine models have demonstrated high on-target epigenome editing and substantial reductions in PCSK9. Cellectis plans to initiate Phase 1 investigator-initiated trials in China, with preliminary clinical data expected in the first half of 2028.
- Other names
- PCSK9 TALEMPCSK-9 TALEMPCSK 9 TALEM
02
Targets
PCSK9 (Proprotein convertase subtilisin/kexin type 9)
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