Table 5.

Summary of on-going clinical studies of mRNA for other applications.

Name of product	Payload (for example, antigen or protein)	Disease	Route of administration	NCT Number	Study phase	Trial status	Sponsor/collaborators	Vehicle
MRT5005	Cystic fibrosis transmembrane conductance regulator	Cystic fibrosis	Inhalation	NCT03375047	I/II	Unknown status	Translate Bio	LNP
LUNAR-OTC (ARCT-810)	Ornithine transcarbamylase	Ornithine transcarbamylase deficiency	i.v.	NCT04442347	Ib	Active, not recruiting	Arcturus	LUNAR®
mRNA-3927	Propionyl-CoA carboxylase	Propionic acidemia	i.v.	NCT04159103	I/II	Recruiting	Moderna	LNP
NTLA-2001	Cas9	Transthyretin amyloidosis	i.v.	NCT04601051	I	Recruiting	Intellia Therapeutics	LNP
NTLA-2001	Cas9	Transthyretin amyloidosis	i.v.	NCT05697861	Observational	Recruiting	Intellia Therapeutics	LNP
NTLA-2002	Cas9	Hereditary Angioedema	i.v.	NCT05120830	I/II	Recruiting	Intellia Therapeutics	LNP

Note: i.v., intravenous.