Drug intelligence / Profile preview

LNP-168-ABE8e-HBG

Development stage
Preclinical
Lead developer
YolTech Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

**LNP-168-ABE8e-HBG** is an investigational, antibody-free bone-marrow-targeted lipid nanoparticle gene-editing therapy developed by YolTech. The LNP uses the ionizable lipid Lipid-168 to intravenously deliver mRNA encoding the adenine base editor ABE8e together with sgRNA-25 to hematopoietic stem and progenitor cells. The guide directs base editing of the HBG1 and HBG2 promoter region, increasing gamma-globin and fetal hemoglobin expression to restore globin-chain balance. It is being developed as a potential one-time in vivo treatment for transfusion-dependent beta-thalassemia and sickle cell disease without ex vivo cell collection, myeloablative conditioning, or hematopoietic stem-cell transplantation.

02

Targets

BCL11A (B-cell CLL/lymphoma 11A)Hemoglobin subunit gamma-1 and gamma-2 promoter regulatory DNA sequencesHemoglobin subunit gamma-1 promoterHemoglobin subunit gamma-2 promoter

Beyond the preview

Go deeper on LNP-168-ABE8e-HBG.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

  • Trial phase
  • Status
  • Readouts

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 LNP-168-ABE8e-HBG.

Gosset Free

Get started with Gosset.

Enter your corporate email.

Work email preferred.

Book a call