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. 2024 Sep 10;8:100283. doi: 10.1016/j.ijpx.2024.100283

Table 3.

Summary of ongoing or completed clinical trials utilizing LNPs. Formulation information is provided anywhere possible. Additional sources used in the construction of this table: https://clinicaltrials.gov; https://classic.clinicaltrials.gov; https://www.cdek.liu.edu. N/A: Formulation information not found, or any information identified were not peer-reviewed and not included.

Nuclei Acid Candidate Clinical Trial study ID (NCT) Phase Formulation/Description Lipid Composition Disease/Conditions Route of Administration Ref. Sponsor Status
mRNA-LNP
Cancer
mRNA-2752/ Durvalumab NCT03739931 Phase 1 (2018–2026) Novel mRNA-based therapeutic
agent encoding OX40L T cell co-stimulator, IL-23 and IL-36γ
pro-inflammatory cytokines
DLin-MC3-DMA, DSPC, Cholesterol, DSPE-PEG2000 Relapsed/refractory solid tumor malignancies or lymphoma Intratumoral (Deng et al., 2022; Manish et al., 2021) ModernaTX, Inc. Active, Not Recruiting
MEDI1191/Durvalumab NCT03946800 Phase 1 (2019–2023) LNP formulation therapy of IL-12 mRNA to induce a potent TH1-mediated anti-tumor response N/A Solid Tumors Intratumoral/IV (Hamid et al., 2021) MediImmune LLC Completed
mRNA-4157/Pembrolizumab NCT03313778

NCT03897881
Phase 1 (2017–2025)

Phase 2 (2019–2029)

LNP-encapsulated mRNA-4157 that encodes 34 different patient-specific neoantigens in combination with Pembrolizumab
N/A Solid tumors including melanoma, bladder carcinoma, HPV-neg HNSCC, NSCLC, SCLC, MSI-high, TMB-high cancers IM/IV (Burris et al., 2019; Julie et al., 2020; Weber et al., 2024) ModernaTX, Inc. Recruiting
SAR441000 NCT03871348 Phase 1 (2019–2024) A mixture of four mRNAs encoding IL-12sc, IFN-α-2b, GM-CSF and IL-15sushi as monotherapy and in combination with cemiplimab N/A Advanced solid tumors Intratumoral/IV (Oliver et al., 2020) Sanofi Active, Not recruiting
mRNA-2416 NCT03323398 Phase1/2 (2017-2021) LNP-encapsulated mRNA encoding human OX40L alone or in combination with Duvulamab N/A Relapsed/Refractory Solid Tumor Malignancies or Lymphoma and Ovarian Cancer Intratumoral (Jimeno et al., 2020) ModernaTX, Inc. Terminated
mRNA-5671/ V941 and Pembrolizumab NCT03948763 Phase 1 (2019–2022) LNP-encapsulated mRNA-5671 that encodes for most common KRAS substitutions (G12d, G12V, G13D, G12C) either alone or in combination with Pembrolizumab N/A Neoplasms IM/IV (Barbier et al., 2022) Merck Sharp & Dohme LLC Completed
NCI-4650 NCT03480152 Phase 1/2 (2018–2019) A mRNA based personalized cancer vaccine that targets up to 15 tumor-associated antigens N/A Melanoma, Colon Cancer, Gastrointestinal Cancer, Genitourinary cancer, hepatocellular cancer IM (Cafri et al., 2020) National Cancer Institute Terminated



Cardiovascular disease
mRNA-0184 NCT05659264 Phase 1 (2022–2025) LNP-encapsulated mRNA-0184 that encodes for relaxin hormone N/A Chronic Heart Failure IV (Soroudi et al., 2024) ModernaTX, Inc. Recruiting



Viral diseases/Influenza
mRNA-1345/ Afluria®Quadrivalent/mRNA-1273.214 NCT05330975 Phase 3 (2022–2024) mRNA-1345 co-administered with seasonal influenza vaccine (Afluria) to evaluate the impact of co-administration on immune respose of RSV-A and severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) N/A Respiratory Syncytial Virus in adults over 50 and SARS-COV2 IM (Li et al., 2023; Wilson et al., 2023) ModernaTX, Inc. Active, not recruiting
mRNA-1345 NCT05127434 Phase 2/3
2021–2025
LNPs with encapsulated mRNA encoding for a stabilized prefusion F glycoprotein N/A Respiratory Syncytial Virus (RSV) IM Moderna TX, Inc Active, not recruiting
mRNA-1273
mRNA-1010
mRNA-1345
mRNA −1647
FLUAD®
NCT05397223 Phase 1 (2022–2026) LNPs encapsulating mRNA-1273, mRNA-1010, mRNA-1345, and mRNA-1647 to observe systemic reactogenicity, adverse effects and adverse reactions N/A Respiratory Syncytial Virus in healthy adults, SARS-CoV-2, Cytomegalovirus IM (Lee et al., 2023a) ModernaTX, Inc. Active, not recruiting
mRNA-1273
mRNA-1273.351
NCT04405076 Phase 2 (2020−2021) LNP-encapsulated mRNA-1273 vaccine encoding a pre-fusion stabilized form of the SARS-CoV-2 spike protein (S—2P). N/A SARS-COV-2
Booster dose
IM (Choi et al., 2021) ModernaTX, Inc. Completed
Spikevax mRNA-1273 NCT04283461

NCT04470427
Phase 1 (2020−2022)

Phase 3 (2020–2022)
LNP-mRNA-1273 is a lipid-encapsulated mRNA-based vaccine that encodes for a full-length, prefusion stabilized spike (S) protein of SARS-CoV-2 SM-102, and 3 commercially available lipids, cholesterol, DSPC, and PEG2000 DMG. SARS-CoV-2 IM (Baden et al., 2021) National Institute of Allergy and Infectious Diseases (NIAID) Completed
mRNA-1283 NCT05137236 Phase 2 (2021−2023) Next generation SARS-CoV2 vaccine composed of LNP encapsulated mRNA-1283 N/A SARS-CoV-2 IM (Yassini et al., 2023) ModernaTX, Inc. Completed
Comirnaty (BNT162b2) NCT04368728 Phase 1/2/3 (2020−2023) LNP-encapsulated nucleoside-modified mRNA that encodes membrane-anchored, full-length SARS-CoV-19 S-protein ALC-0315, ALC-0519, DSPC Cholesterol SARS-CoV-2 IM (Polack et al., 2020b) (Lamb, 2021) BioNTech SE Completed
CV-NCOV NCT04449276 Phase 1 (2020–2021) LNP-formulated SARS-CoV-2 vaccine containing mRNA encoding perfusion conformation-stabilized full-length SARS-CoV-2 spike protein DSPC, cationic lipid, PEG-lipid conjugate, cholesterol SARS-CoV-2 IM (Kremsner et al., 2021) CureVac Completed
ARCoV NCT04847102 Phase 3 (2021–2023) mRNA-LNP that encodes the receptor binding domain of the S-protein of SARS-CoV2 Ionizable lipid (Lipid 9001), (1,2-DSPC, cholesterol and PEG-lipid SARS-CoV-2 IM (Zhang et al., 2020a) (Chen et al., 2022) Walvax Biotechnology Co., Ltd. Unknown Status
mRNA-1010 NCT04956575 Phase 1/2 (2021−2022) LNP encapsulating quadrivalent seasonal influenza vaccine encoding membrane-bound HA surface glycoproteins of four influenza strains (A/H1N1, A/H3N2, B/Victoria, and B/Yamagata) recommended by the WHO for cell- or recombinant vaccines N/A Seasonal influenza A: H1N1 and H3N2
Influenza B: strains including Victoria-lineage and Yamagata lineage
IM (Lee et al., 2023a) ModernaTX, Inc. Completed
mRNA −1647
mRNA-1443
NCT03382405
Phase 1 (2017–2020)

LNP encapsulated mRNA-1647 comprises six mRNAs encoding for CMV antigens given along with LNP-encapsulated mRNA-1443 encoding pp65 from T cells of the CMV antibody
N/A Cytomegalovirus (CMV) IM (Jung et al., 2022) ModernaTX, Inc. Completed
mRNA-1647 NCT04232280 Phase 2 (2020–2023) LNP encapsulating mRNA-1647 cytomegalovirus vaccine in CMV-seronegative and CMV-seropositive healthy adults N/A IM (Panther et al.) Completed
NCT05085366 Phase 3 (2021–2026) Active, not recruiting
mRNA-1653 NCT04144348 Phase 1 (2019–2022) LNP encapsulated bivalent vaccine comprised of nucleoside-modified mRNA encoding full-length membrane-bound fusion proteins of hMPV and PIV3 N/A Human Metapneumovirus and Human parainfluenza virus IM (August et al., 2022; Nelson et al., 2020; Schnyder Ghamloush et al., 2024) ModernaTX, Inc. Completed
mRNA-1440 (VAL-506440) NCT03076385 Phase 1 (2015–2018) LNP-formulated modified mRNA-based vaccine encoding hemagglutinin (HA)proteins of H10N8 or H7N9 influenza strain N/A Influenza A (H10N8 and H7N9) IM (Bahl et al., 2017; Feldman et al., 2019) ModernaTX, Inc. Completed
mRNA-1851 (VAL-339851) NCT03345043 Phase 1 (2016–2018) LNP encapsulated mRNA-1851 that encodes for the HA protein of H7N9 N/A Influenza A virus H7N9 subtype IM (Shi et al., 2022) ModernaTX, Inc. Completed
CV7202 NCT03713086 Phase 1 (2018–2021) RABV-G mRNA using initial formulation CV7201 antigen with cationic protein protamine encapsulated in LNP Ionizable amino lipid, DSPC, PEG-2000-DMG, Cholesterol Rabies virus glycoprotein (RABV-G) IM (Aldrich et al., 2021; Shi et al., 2022) CureVac Completed
mRNA-1325 NCT03014089 Phase 1 (2016–2019) LNP-encapsulated modified mRNA vaccine encoding premembrane and envelope E structural proteins (prME) from a Micronesia 2007 Zika virus isolate Proprietary ionizable lipids, DSPC, cholesterol, and PEG-lipid Zika Virus IM (Richner et al., 2017) (Bollman et al., 2023) ModernaTX, Inc. Completed
mRNA-1944 NCT03829384 Phase 1 (2019–2021) LNP-encapsulated mRNA-1944 that encodes for the heavy and light chains of CHKV-24 antibody Proprietary high purity PEG-2000 stearate monoester, IAL (proprietary ionizable amino lipid), cholesterol, DSPC Chikungunya virus IV (August et al., 2021) ModernaTX, Inc. Completed
mRNA-1893 NCT04917861 Phase 2 (2021–2024) LNP-encapsulated mRNA-1893 vaccine encoding the envelope E structural proteins (prME) from the RIO-U1 Zika virus isolate Proprietary ionizable lipid, DSPC, cholesterol, and PEG lipid Zika virus IV (Bollman et al., 2023; Essink et al., 2023; Li et al., 2024) ModernaTX, Inc. Active, not recruiting
mRNA-1215 NCT05398796 Phase 1 (2022–2024) LNP encapsulated mRNA-1215 that encodes for Nipah perfusion F protein and G protein N/A Nipah Virus (NiV) Infection IM (Rodrigue et al., 2024; Wang et al., 2023c) ModernaTX, Inc. Active, not recruiting
H1ssF-3928 NCT03814720

NCT05755620
Phase 1 (2019–2021)

Phase 1 (2023–2025)
VRC H1ssF 3928 mRNA-LNP vaccine encoding influenza H1 hemagglutinin stem N/A Influenza IM (Andrews et al., 2023; Widge et al., 2023) National Institute of Allergy and Infectious Diseases (NIAID) Completed

Recruiting
DCVC H1 HA NCT05945485 Phase 1 (2023–2024) mRNA-LNP vaccine encoding full length H1 HA of influenza A/California/07/2009 (H1N1) N/A Influenza A/H1N1 IM National Institute of Allergy and Infectious Diseases (NIAID) Recruiting
AVX502 NCT00440362 Phase 1/2 (2007) Alphavirus Replicon Vaccine Expressing Influenza HA protein N/A Influenza IM/SC AlphaVax, Inc. Completed
mRNA-1189 NCT05164094 Phase 1 (2021–2025) LNP-encapsulated mRNAs that encode Epstein-Barr Virus (EBV) envelope glycoproteins gp42, gp220, gH and gL N/A Epstein-Barr Virus (EBV) IM (Zhong et al., 2022) ModernaTX, Inc Active, Not Recruiting



Genetic disorders
mRNA-3704 NCT03810690 Phase 1/2 (2019–2020) LNP-encapsulated mRNA encoding human methylmalonyl-CoA mutase (hMUT) N/A Methylmalonic acidemia IV (Hou et al., 2021) ModernaTX, Inc. Withdrawn
mRNA-3705 NCT05295433 Phase 1/2 (2022–2034) LNP-encapsulated mRNA encoding hMUT SM-86, DSPC, cholesterol, and OL-56 [polyethylene glycol-lipid conjugate] Methylmalonic Acidemia IV (Baek et al., 2024; Suzuki et al., 2023) ModernaTX, Inc Recruiting
mRNA-3927 NCT04159103
NCT05130437
Phase1/2 (2021−2031) LNP-encapsulated dual mRNA therapy that encodes for (propionic-CoA mutase) PCC-A and PCC-B subunit proteins restoring PCC enzyme in liver SM-86, DSPC, cholesterol, and OL-56 [polyethylene glycol-lipid conjugate] Propionic Acidemia IV (Attarwala et al., 2023; Baek et al., 2024) ModernaTX, Inc. Recruiting
MRT5201 NCT03767270 Phase 1/2 (2019–2022) LNP-encapsulated codon-optimized human OTC mRNA N/A Ornithine Transcarbamylase Deficiency IV Translate Bio, Inc. Withdrawn
MRT5005 NCT03375047 Phase 1/2 (2018–2021) Aerosolized LNP-encapsulated a codon-optimized CFTR mRNA N/A Cystic Fibrosis Nebulization (Barbier et al., 2018; Hou et al., 2021; Rowe et al., 2023) Translate Bio, Inc. Unknown Status
ARCT-810 NCT04442347 Phase 1 (2020–2023) Human ornithine transcarbamylase (hOTC) mRNA-LNP N/A Ornothine Transcarbamylase Deficiency IV (Yamazaki et al., 2023) Arcturus Therapeutics, Inc. Active, not recruiting
NCT05526066 Phase 2 (2022–2024) Recruiting



siRNA
ALN-TTR01 NCT01148953 Phase 1 (2010−2012) First gen formulation of LNPs to deliver siRNAs DLin-MC3- DMA, DSPC, PEG2000-C-DMG, Cholesterol Transthyretin (TTR) Mediated amyloidosis (ATTR) IV (Coelho et al., 2013; Schoenmaker et al., 2021a; Zatsepin et al., 2016) Alnylam Pharmaceuticals Completed
ALN-TTR02 NCT01559077 Phase 1 (2012) Second gen formulation of LNPs to deliver siRNAs DLin-MC3- DMA, DSPC, PEG2000-C-DMG, Cholesterol Transthyretin (TTR) Mediated amyloidosis (ATTR) IV (Adams et al., 2018; Coelho et al., 2020; Coelho et al., 2013; Schmidt et al., 2022; Suhr et al., 2015; Zatsepin et al., 2016) Alnylam Pharmaceuticals Completed
NCT01617967 Phase 2 (2012–2014) Completed
NCT01961921 Phase 2 (2013–2016) Completed
NCT01960348 Phase 3 (2013–2017) Completed
NCT03862807 Phase 3 (2019–2020) Completed
NCT02510261 Phase 3 (2015–2022) Completed
DCR-MYC NCT02110563 Phase 1 (2014–2016) DCR-MYC is a LNP-formulated Dicer substrate siRNA (DsiRNA) that silences MYC mRNA N/A Solid tumors, multiple myeloma, non-Hodgkins lymphoma IV (Chipumuro et al., 2016; Tolcher et al., 2015) Dicerna Pharmaceuticals Terminated
NCT02314052 Phase 1/2 (2015–2016) Hepatocellular carcinoma Terminated
ND-L02-s0201 NCT01858935 Phase 1 (2013–2014) LNP-encapsulated anti- hsp47 siRNA formulation designed to reversibly inhibit the expression of HSP47 Six key lipid components including cationic, helper and targeting lipids (names of lipids not specified) Safety, Tolerability and Pharmacokinetics in Healthy Normal Subjects IV (Liu et al., 2021) Bristol-Myers Squibb Completed
NCT02227459 Phase 1/2 (2014–2016) Moderate to Extensive Hepatic Fibrosis Bristol-Myers Squibb Completed
NCT03538301 Phase 2 (2018–2022) Idiopathic Pulmonary Fibrosis Nitto Denko Corporation Completed
TKM-080301 NCT01437007 Phase 1 (2011−2012) LNP containing siRNA against PLK1 gene products Four lipid components Hepatocellular carcinoma Intra-arterial /IV (Ramanathan et al., 2013) National Cancer Institute (NCI) Completed
NCT01262235 Phase 1/2 (2010–2015) Adrenocortical carcinoma (Demeure et al., 2016) Arbutus Biopharma Corporation Completed
NCT02191878 Phase 1/2 (2014–2016) Hepatocellular carcinoma (El Dika et al., 2019) Arbutus Biopharma Corporation Completed
NBF-006 NCT03819387 Phase 1 (2019–2024) LNP encapsulating siRNA targeting glutathione-S-transferase Pi N/A NSCLC, Pancreatic, Colorectal cancer IV (Hattab et al., 2021) Nitto BioPharma., Completed
BMS-986263 NCT03142165 Phase 1 (2017) BMS-986263, a retinoid-conjugated lipid nanoparticle delivering small interfering RNA designed to target heat shock protein (HSP)-47 mRNA, for the treatment of advanced fibrosis. N/A Hepatic Cirrhosis -Hepatic Impairment IV (Qosa et al., 2023a; Qosa et al., 2023b) Bristol-Myers Squibb Completed
NCT03420768 Phase 2 (2018–2019) Completed
NCT04225936 Phase 1 (2020–2021) Completed
NCT04267393 Phase 2 (2021–2024) Terminated



CRISPR/Cas9 (sgRNA)
NTLA-2001 NCT04601051

NCT06128629
Phase 1 (2020–2026)

Phase 3 (2023–2028)
LNP-encapsulated single guide RNA (sgRNA) targeting human TTR and a human-codon optimized mRNA sequence of S. pyogens Cas9 protein N/A Transthyretin (ATTR) amyloidosis with cardiomyopathy IV (Gillmore et al., 2021) Intellia Therapeutics Active, not recruiting

Recruiting
VERVE-101 NCT05398029 Phase 1 (2022–2024) LNPs for a CRISPR-based gene editing that inactivates the PCSK9 gene N/A Heterozygous Familial Hypercholesteremia and Cardiovascular Disease IV (Lee et al., 2023b) Verve Therapeutics Recruiting
VERVE-102 NCT06164730 Phase 1 (2024–2026) LNP-encapsulated CRISPR-based treatment using a guide RNA targeting PCSK-9 gene N/A Heterozygous Familial Hypercholesteremia or Premature Coronary Artery Disease IV Verve Therapeutics Not yet recruiting