Table 1.

Selected self-replicating RNA clinical trials.

Therapeutic area	Name	Antigen/protein	Phase	VRP or srRNA	species	Formulation	ROA	Dose (IU or ug)	Entity	Trial number	Results
Infectious Disease	AVX601	CMV gB, pp65/IE1	I	VRP	VEEV	None	IM	107, 108	AlphaVax	NCT00439803	Well-tolerated; Antibody and polyfunctional antigen-specific T cell responses
	AVX101	HIV Gag	I	VRP	VEEV	None	IM	104-108	AlphaVax	NCT00063778, NCT00097838	Well-tolerated, low immunogenicity
	AVX502	Influenza HA	I/II	VRP	VEEV	None	IM, SC	Not listed	AlphaVax	NCT00440362	Well-tolerated; 86% seroconversion and T cell responses.
	ARCT-021	SARS-CoV-2	III	srRNA	VEEV	LNP	IM	5	Arcturus	Initiation H2-2021	Interim data: >90% seroconversion after a single dose for IgG antibodies
	ARCT-154	SARS-CoV-2	I/II/III	srRNA	VEEV	LNP	IM	5	Arcturus	NCT05012943	No data reported
	LNP-nCoVsaRNA	SARS-CoV-2	I	srRNA	VEEV	LNP	IM	0.1-10	ICL	ISRCTN17072692	Well-tolerated; Neutralizing antibody seroconversion varied from 15-48% depending on dose level.
	CORAL	SARS-CoV-2	I	srRNA	VEEV	LNP	IM	10, 30	Gritstone	NCT04776317	Homologous srRNA regimen and heterologous with ChAd
	BNT162c2	SARS-CoV-2	I/II	srRNA	VEEV	LNP	IM	Not listed	BioNTech	NCT04380701	Single dose
	CoV2 SAM	SARS-CoV-2	I	srRNA	VEEV-SINV	LNP	IM	Not listed	GSK	NCT04758962	No data reported
	GSK4108771A	HSV-2	I	srRNA	VEEV-SINV	LNP	IM	Not listed	GSK	NCT04762511	Terminated
	RG SAM	Rabies	I	srRNA	VEEV-SINV	CNE	IM	Not listed	GSK	NCT04062669	No data reported
Oncology	AVX901	HER2	I	VRP	VEEV	None	IM	4×108	AlphaVax	NCT01526473	Well-tolerated; HER2-specific memory T cell population correlated with improved progression free survival
	AVX701	CEA	I	VRP	VEEV	None	IM	0.4 × 108-4 × 108	AlphaVax	NCT00529984	Well-tolerated; Prolonged survival, T cell responses detected
	PSMA-VRP	PSMA	I	VRP	VEEV	None	IM	0.9 × 107, 0.36 × 108	AlphaVax	PMID: 23246260	Well-tolerated; No clinical benefit due to suboptimal dosing
	LSFV-IL-12	IL-12	I/II	VRP	SFV	liposome	IV or IP	1 × 108, 1 × 109	Regulon	PMID: 12952295	No grade III or IV toxicities
	CYN102	NYESO-1	I	VRP	SINV	None	IV or IP	Not listed	Cynvec	Initiation planned 2021	Not yet initiated
	Vvax001	HPV16 E6 E7	I	VRP	SFV	None	IM	5 × 105-2.5 × 108	ViciniVax B.V	NCT03141463	Well-tolerated; immune responses observed in all participants
	GRT-C901/2	Neoantigens	II	srRNA	VEEV	LNP	IM	Not listed	Gritstone	NCT03639714	ChAd prime + /− srRNA: Interim data: 44% molecular response rate by ctDNA analysis in CRC.
	GRT-C903/4	Shared neoantigens	II	srRNA	VEEV	LNP	IM	Not listed	Gritstone	NCT03953235	ChAd prime + /− srRNA + /− checkpoint inhibitors. Unconfirmed partial response in NSCLC. Doses well-tolerated