Table 1.

Examples of gene therapy products and their status concerning approval for use in the clinic

Name	Developer	Indication	In/ex vivo	Approval date	Status	Technique	URL for more info
Luxturna (voretigene neparvovec)	Spark Therapeutics	Leber congenital amaurosis; retinitis pigmentosa	In vivo	N/A	Positive CHMP opinion 20 September 2018	Inserting functional gene with an AAV	https://www.ema.europa.eu/medicines/human/summaries-opinion/luxturna
Yescarta (axicabtagene ciloleucel)	Kite Pharma	Diffuse large B-cell lymphoma (DLBCL); primary mediastinal B-cell lymphoma	Ex vivo	N/A	Positive CHMP opinion 28 June 2018	Inserting a new CAR-T gene with a retrovirus	https://tinyurl.com/ydcefrm4
Kymriah (tisagenlecleucel)	Novartis	Acute lymphoblastic leukaemia; DLBCL	Ex vivo	N/A	Positive CHMP opinion 28 June 2018	Inserting a new CAR-T gene with a lentivirus	https://tinyurl.com/y92q7e3e
Zalmoxis	MolMed	Stem cell transplantation in serious blood cancers	Ex vivo	June 2016	Approved	Inserting an overactive NGFR gene with a retrovirus	https://tinyurl.com/ycw9fx34
Strimvelis	GSK	ADA-SCID	Ex vivo	April 2016	Approved	Inserting functional gene with a retrovirus	https://tinyurl.com/ycsa5yf4
Imlygic (talimogene laherparepvec)	Amgen	Melanoma	Ex vivo	October 2015	Approved	Oncolytic gene therapy with HSV	https://tinyurl.com/y7lrjyyv
Glybera (alipogene tiparvovec)	uniQure	Lipoprotein lipase deficiency	In vivo	November 2012	Withdrawn in 2017	Inserting functional gene with parvovirus	https://tinyurl.com/y977x5tz