Drug intelligence / Profile preview

F48 LNP

Development stage
Preclinical
Lead developer
Johns Hopkins University
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Recombinant Proteins and Enzymes, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

**F48 LNP** is an experimental lipid nanoparticle formulation developed at **Johns Hopkins University** for systemic intracellular delivery of gene-editing proteins to T cells. In the reported mouse studies, F48 LNP encapsulated CRISPR-Cas9 ribonucleoprotein containing a single-guide RNA directed against **PD-1**, producing preferential gene editing in splenic T cells after intravenous administration. PD-1 disruption reduced checkpoint expression in T cells and was evaluated as an immune-checkpoint-blockade strategy in a B16-OVA melanoma model. F48 is a research-stage delivery formulation rather than an approved drug; the precise lipid composition was not disclosed in the available abstract.

02

Targets

CCR5 (C-C chemokine receptor type 5)PDCD1 (Programmed cell death protein 1 receptor)

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