Nava Therapeutics Launches with $89M to Advance Extrahepatic LNP Delivery for In Vivo CAR-T and Kidney Gene Editing

Jul 16 , 2026
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July 16, 2026 —

Nava Therapeutics has launched with $89 million in funding to advance a lipid nanoparticle platform designed to deliver RNA medicines beyond the liver, with initial programs focused on in vivo CAR-T and kidney-targeted genetic medicine.

The Cambridge- and Philadelphia-based company is developing lipid nanoparticles, or LNPs, intended to reach immune cells and kidney tissue rather than defaulting to liver uptake. The financing was backed by a group of investors including RA Capital Management, Leaps by Bayer, PureTech Health, a large U.S. healthcare-focused fund, and a sovereign wealth fund. Proceeds will support preclinical programs toward clinical trials, expansion of Nava’s lipid chemistry library, and operations across both sites.

Nava’s lead program, NT-001, is an anti-CD19 in vivo CAR-T candidate designed to generate CAR-T cells directly inside the body. Instead of extracting a patient’s T cells, modifying them at a specialized manufacturing facility, and reinfusing them weeks later, the approach uses an LNP to deliver mRNA encoding a humanized CD19 CAR directly to T cells in vivo.

This strategy could simplify the CAR-T treatment paradigm by eliminating patient-specific ex vivo manufacturing, viral vector production, cold-chain logistics, and lengthy turnaround times. Because the CAR is expressed transiently for days to weeks, the approach may also allow repeat or adjustable dosing without permanent genomic integration.

A central challenge for LNP-based medicines is liver tropism. After intravenous administration, many conventional LNPs adsorb serum apolipoproteins such as ApoE, which promotes recognition by LDL receptors on hepatocytes and drives liver uptake. Nava’s platform uses a novel “immunotropic” ionizable lipid designed to reduce ApoE adsorption and limit hepatic biodistribution through lipid chemistry alone.

For NT-001, Nava combines this passive liver de-targeting approach with CD8-specific targeting ligands to actively direct the particle toward CD8-positive T cells. The company describes this as a layered delivery strategy in which lipid chemistry addresses organ-level biodistribution and targeting ligands address cell-type specificity.

The platform builds on SENT-seq, a single-cell LNP screening technology developed in the laboratory of James Dahlman at Georgia Tech and Emory University. SENT-seq allows hundreds of barcoded LNP formulations to be screened simultaneously in vivo, measuring biodistribution, functional RNA delivery, and cellular response at single-cell resolution.

In non-human primate studies, NT-001 produced deep B-cell depletion in the spleen and lymph nodes, consistent with on-target CD19 CAR-T activity. PET imaging showed selective splenic and lymph node uptake with minimal off-target signal, while liver enzymes remained within the normal range. Cytokine elevations were transient and returned to baseline within 72 hours.

Beyond T-cell delivery, Nava is also advancing a kidney-targeted program. The kidney remains a difficult organ for LNP delivery because glomerular filtration and tubular architecture create size and charge barriers. Nava has presented preclinical data on kidney gene editing, suggesting the platform may support delivery of more complex RNA payloads such as guide RNA and nuclease-encoding mRNA.

Nava enters a highly competitive in vivo CAR-T field that has attracted major pharmaceutical investment. Companies pursuing LNP-mRNA or viral delivery approaches are seeking to create CAR-T cells directly inside patients, potentially reducing cost and improving access compared with conventional ex vivo CAR-T manufacturing.

However, the field still lacks public human proof-of-concept data for LNP-mRNA in vivo CAR-T. Nava’s next key milestones will include IND-enabling development, clinical translation of its liver de-targeting and T-cell targeting profile, and demonstration that its preclinical delivery advantages can translate safely and effectively in humans.

If successful, Nava’s platform could help expand RNA medicines into immune cell and kidney applications while supporting a new generation of off-the-shelf, in vivo cell therapy approaches.

Source:

https://www.techtimes.com/articles/320597/20260715/vivo-car-t-startup-nava-emerges-89m-solve-rna-deliverys-liver-problem.htm

About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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