Figure and Table | Raising Gene Therapy for Unmet Medical Needs in Japan

Table 1. Gene Therapy Products Approved in the EU and the USA (as of January 24, 2019).

From: Raising Gene Therapy for Unmet Medical Needs in Japan

Descriptive name (Trade name)	Jurisdiction approved in	Developmental incentives	Intervention type	Indications	Remarks
Alipogene tiparvovec (Glybera)	EU 2012	Orphan Medicine, Conditional Marketing Approval.	In vivo gene therapy	Severe pancreatitis due to lipoprotein lipase deficiency (LPLD)	A non-integrative AAV1 vector is administered intramuscularly to deliver a normal copy of LPL gene to muscle cells. However, its marketing was withdrawn in 2017.
Talimogene laherparepvec (IMLYGIC)	USA 2015, EU in 2015	Special Protocol Assessment, Priority Review, Orphan Drug (USA)	In vivo gene therapy	Melanoma	An oncolytic HSV1 is directly injected into patients’ melanoma tumors, delivering GM-CSF gene to the cells.
Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence (Strimvelis)	EU 2016	Orphan Medicine, Paediatric Investigation Plan	Ex vivo gene therapy	Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), where bone-marrow transplants are unavailable.	A retrovirus vector delivers a normal copy of ADA gene to CD34+ cells. The modified cells are infused intravenously.
Allogeneic T cells genetically modified with a retroviral vector encoding for a truncated form of the human low affinity nerve growth factor receptor (ΔLNGFR) and the herpes simplex I virus thymidine kinase (HSV-TK Mut2) (Zalmoxis)	EU 2016	Orphan Medicine	Ex vivo gene therapy	Prevention of complications in hematopoietic stem cell Transplantation.	Allogeneic T cells are genetically modified with a retroviral vector coding HSV-TK and ΔLNGFR genes so that these genes are integrated in the genome of the cells. If graft vs host disease (GvHD) occurs, the cell product can be inactivated by administrating ganciclovir.
Tisagenlecleucel (KYMRIAH)	USA 2017, EU 2018	Priority Review, Breakthrough Therapy, Orphan Drug (USA)	Ex vivo gene therapy	Patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.	Autologous T cells are genetically modified using a lentiviral vector to encode an anti-CD19 CAR, including a murine anti-CD19 single chain antibody fragment (scFv) and two human signaling domains (CD3-ζ and 4-1BB). The modified cells are administered intravenously.
Tisagenlecleucel (KYMRIAH)	USA 2017, EU 2018	Priority Review, Breakthrough Therapy, Orphan Drug (USA)	Ex vivo gene therapy	Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.
Axicabtagene ciloleucel (YESKARTA)	USA 2017, EU 2018	Priority Review, Breakthrough Therapy, Orphan Drug (USA)	Ex vivo gene therapy	Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.	Autologous T cells are genetically modified, using a lentiviral vector, to encode an anti-CD19 CAR, including a murine scFv and two human signaling domains (CD3-ζ and CD28). The CAR T cells are administered intravenously.
Voretigene neparvovec-rzyl (LUXTURNA)	USA 2017, EU 2018	Priority Review, Breakthrough Therapy, Orphan Drug, Rare Pediatric Disease Designation (USA)	In vivo gene therapy	Patients with biallelic RPE65 mutation-associated retinal dystrophy.	A non-integrative AAV2 vector is administered subretinally to deliver a normal copy of RPE65 gene to retinal cells.

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