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Table 2 Clinical products produced using ionizable nanocarriers

From: Ionizable drug delivery systems for efficient and selective gene therapy

Ionizable nanomaterials

Nucleic acid

Application

Product

Clinical Phase

SM-102

mRNA

COVID-19 vaccine

Spikevax®

Approved in 2021

Acuitas ALC-0315

mRNA

COVID-19 vaccine

Comirnaty®

Approved in 2021

MC3

siRNA

ATTR

Onpattro®

Approved in 2018

ALC-0315

mRNA

COVID-19 vaccine

CVnCoV

III

A9

mRNA

COVID-19 vaccine

LNP-nCo VsaRNA

I

CL1

mRNA

COVID-19 vaccine

ChulaCov19

I

ATX

mRNA

COVID-19 vaccine

LUNAR-COV19

(ARCT-021)

III

-

mRNA

COVID-19 vaccine

ARCoV

III

COVID-19 vaccine

PTX-COVID19B

II

ATX

mRNA

Ornithine

transcarbamylase

deficiency

LUNAR-OTC

(ARCT-810)

II

Cystic fibrosis

LUNAR-CF

(ARCT-032)

Preclinical

Influenza

LUNAR-FLU

Preclinical

Hemophilia B

LUNAR®

Preclinical

LPO1

sgRNA

ATTR

NTLA-2001

I

  1. SM-102 Heptadecan-9-yl 8-[(2-hydroxyethyl)(6-oxo-6-(undecyloxy) hexyl) amino] octanoate, MC3 (6Z,9Z,28Z,31Z)heptatriaconta-6,9,28,31-tetraen-19-yl-4-(dimethylamino)butanoate, ALC-0315 6-[(2-hexyldecanoyl)oxy]-N-{6-[(2-hexyldecanoyl)oxy]hexyl}-N-(4-hydroxybutyl)hexan-1-aminium, COVID-19 coronavirus disease 2019, ATTR transthyretinamyloidosis, mRNA messenger RNA, sgRNA small guide RNA