Table 1 Detailed information on genes with randomized clinical trial evidence

From: Transcriptome-wide Mendelian randomization during CD4⁺ T cell activation reveals immune-related drug targets for cardiometabolic diseases

Gene^a	Disease	Drug names	Indications	Trial status	Phase	Reactome immune pathway counts
TGFB1	CAD	BINTRAFUSP ALFA	Breast cancer; cervical cancer; gastrointestinal/stomach cancer; biliary cancer; non-small cell lung cancer; urethral cancer	Active	Phase III Clinical Trial	29
LIPA	CAD	Sebelipase alfa	Lysosomal acid lipase deficiency	Success	Launched	39
LIPA	CAD	Lysosomal acid lipase, Large S	Atherosclerosis	Ceased	Preclinical	39
DNAJC15	CAD	MCJ agonist, Mitotherapeutix	Breast cancer; unspecified solid cancer	Ceased	Preclinical	32
RAC1	Both	JK50561	Alzheimer’s disease; amnesia; dementia, vascular; intellectual disability; posterior cortical atrophy	Active	Phase I Clinical Trial	38
KHK	Both	PF-06835919	Diabetes; non-alcoholic steatohepatitis	Ceased	Phase II Clinical Trial	23
PARP1	CAD	AL-309	Diabetic neuropathy	Ceased	Preclinical	28
PARP1	CAD	Davunetide	Alzheimer’s disease; corticobasal degeneration; Parkinson’s disease; progressive supranuclear palsy; schizophrenia	Ceased	Phase III Clinical Trial	28
ERAP2	CAD	Grey Wolf Therapeutics	Cancer	Active	Preclinical	23
CD151	CAD	F-50065	Cancer	Ceased	Preclinical	34
HLA-C	CAD	KIR/HLA-C inhibitor, Interprotein	Cancer	Ceased	Preclinical	27
HLA-B	CAD	Anti-B27 antibody, ImmunOs Therapeutics	Autoimmune disease, unspecified	Active	Preclinical	5
LAP3	T2D	TOSEDOSTAT	Myelodysplastic syndrome; acute myeloid leukemia; neoplasm; non-small cell lung carcinoma; pancreatic carcinoma	Completed	Phase II Clinical Trial	32
UBA6	T2D	TAK-243	Diffuse large B-cell lymphoma; cancer, solid, unspecified	Ceased	Phase I Clinical Trial	26
GCK	T2D	Dorzagliatin	Type 2 diabetes; Type 1 diabetes; diabetic nephropathy; non-alcoholic steatohepatitis	Active	Launched	17
HRH2	T2D	Icotidine	Cardiovascular disease	Ceased	Phase I Clinical Trial	19
FADS1	T2D	Dihomo-gamma-linolenic acid, DS Biopharma	Cancer, solid, unspecified; acne; eczema, atopic; pruritus; radio/chemotherapy-induced injury, unspecified; rosacea; wound healing; infection, coronavirus, novel coronavirus	Active	Phase II Clinical Trial	18
HLA-DRB1	T2D	ZYBK-2	Rheumatoid arthritis	Active	Phase I Clinical Trial	32
CFD	T2D	Danicopan	Paroxysmal nocturnal haemoglobinuria; macular degeneration, age-related, dry; C3 glomerulopathy; chronic obstructive pulmonary disease; glomerulonephritis; hemolytic uremic syndrome; myasthenia gravis	Active	Phase III Clinical Trial	22
STK17B	T2D	MU-D201	Diffuse large B-cell lymphoma; leukemia	Active	Preclinical	22
GPX7	T2D	CV-03	Gastrointestinal/stomach/esophageal cancer	Ceased	Preclinical	22
CD52	T2D	Alemtuzumab	Multiple sclerosis; cancer,leukemia, chronic lymphocytic; cancer, lymphoma, non-Hodgkin’s; cancer, lymphoma, T-cell; arthritis, rheumatoid	Active	Launched	28
ERN1	T2D	Inositol requiring enzyme-1alpha inhibitors, Mannkind	Autoimmune disease, unspecified; cancer, myeloma	Ceased	Preclinical	30
CALR	T2D	Calreticulin antibody, MyeloPro	Unspecified	Ceased	Preclinical	37
MDM4	T2D	ALRN-6924	Cancer	Active	Phase II Clinical Trial	23
MICB	T2D	ADI-925	Cancer, hematological, unspecified, Cancer, solid, unspecified	Active	Preclinical	34
DPP7	CAD	CRWCYVOHVXAEM; F-LBPRGKRZSA-N	Carcinoma, liver cirrhosis, idiopathic pulmonary fibrosis	Active	Patented	35

^aThe IMPC database search, literature search, and pathway analysis showed mechanism evidence based on the immune system, related to Supplementary Data 25.

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Table 1 Detailed information on genes with randomized clinical trial evidence

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