Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
HEAL-101
- Development stage
- Unknown
- 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-101 is an in vivo base editing therapeutic candidate developed by Cellectis for the treatment of severe hypertriglyceridemia (sHTG). It consists of a transcription activator-like effector (TALE) base editor specific to the human apolipoprotein C-III (APOC3) gene, termed APOC3 TALEB, formulated within lipid nanoparticles (LNPs) for delivery. By targeting and editing the APOC3 gene, HEAL-101 aims to permanently reduce APOC3 expression and lower plasma triglyceride levels, thereby mitigating the risk of acute pancreatitis and cardiovascular disease in patients with high-risk severe hypertriglyceridemia.
- Other names
- APOC3 TALEBAPOC-3 TALEBAPOC 3 TALEB
02
Targets
APOC3 (Apolipoprotein C-III)
Beyond the preview
Go deeper on HEAL-101.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on HEAL-101.