Drug intelligence / Profile preview

EMD-301

Development stage: Preclinical
Lead developer: EmendoBio
Modality: Small Molecules, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Parenteral (likely Intravenous Or Similar Injection)

Overview

EMD-301 is a CRISPR-based, non-viral gene editing therapy in preclinical development by Emendo Biotherapeutics (a subsidiary of AnGes). It is designed as a "one and done" gene editing treatment for familial hypercholesterolemia and other hypercholesterolemia-related disorders. The therapy acts as a gene modulator, aiming to correct or modify disease-causing genetic mutations associated with elevated cholesterol levels. Its primary modality is in vivo gene editing using CRISPR technology[1][2][3][4].

Other names: EMD 301EMD301EMD-301

Targets

ANGPTL3 (Angiopoietin-like protein 3)

Beyond the preview

Go deeper on EMD-301.

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 EMD-301.

Explore the full profile