Table 1.

Gene therapies approved by the SFDA, FDA or EMA

Product	Indications	Approval	Delivery route	Delivery vector	Mechanism	References
Gendicine	head and neck cancer	2003	intratumoral injection	recombinant Adenovirus vector	deliver functional copies of the p53 gene	Peng et al.47
Glybera	familial lipoprotein lipase deficiency	2012	intravenous infusion	AAV vector	deliver functional copies of the LPL gene	Gaudet et al.48
Strimvelis	ADA-SCIDa	2016	ex vivo/HSCe	gamma-retroviral vector	deliver functional copies of the ADA gene	Cicalese et al.49
Luxturna	LCAb or retinitis pigmentosa	2017	subretinal injection	AAV2	deliver functional copies of the RPE65 gene	Russell et al.51
Zolgensma	spinal muscular atrophy (SMA)	2019	intravenous infusion	AAV9	deliver functional copies of the SMN1 gene	Mendell et al.54
Zynteglo	transfusion-dependent β-thalassemia	2019	ex vivo/HSCe	gamma-retroviral vector	deliver functional copies of the β-globin gene	Locatelli et al.55
ROCTAVIAN	hemophilia A	2019	intravenous infusion	AAV5	deliver functional copies of FVIII gene	Ozelo et al.56
Libmeldy	metachromatic leukodystrophy (MLD)	2020	intrathecal injection	lentiviral vector	deliver functional copies of the arylsulfatase A gene	Sessa et al.57
ADSTILADRIN	high-grade, BCG-unresponsive NMIBCc	2022	intravesical	adenovirus vector	deliver functional copies of the IFNα2b gene	Boorjian et al.58
HEMGENIX	hemophilia B	2022	intravenous infusion	AAV5	deliver functional copies of the factor IX gene	Pipe et al.59
SKYSONA	boys with early, active CALDd	2022	ex vivo/HSCe	lentiviral vector	deliver functional copies of the ABCD1 gene	Eichler et al.60
ELEVIDYS	duchenne muscular dystrophy (DMD)	2023	intravenous infusion	AAV vector	deliver functional copies of an engineered dystrophin gene	Mendell et al.61
VYJUVEK	dystrophic epidermolysis bullosa	2023	tropical	non-replicating Herpes simplex virus	deliver functional copies of the COL7A1 gene	Guide et al.52
CASGEVY	sickle cell diseases	2024	ex vivo/HSCe	electroporation	deliver CRISPR/Cas9-sgRNA RNP to disrupt the BCL11A enhancer & restore γ-globin expression	Frangoul et al.62
LYFGENIA	sickle cell diseases	2024	ex vivo/HSCe	lentiviral vector	deliver functional copies of the β-globin gene	Kanter et al.63

^aADA-SCID: severe combined immunodeficiency due to adenosine deaminase deficiency.

^bLCA: Leber Congenital Amaurosis.

^cBCG-unresponsive NMIBC: Bacillus Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC).

^dCALD: cerebral adrenoleukodystrophy.

^eHSC: hematopoietic stem cell.