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From Wikipedia, the free encyclopedia
This article contains a list of commercially available
gene therapies
.
Gene therapies
See also:
Gene therapy § List of gene therapies for treatment of disease
Alipogene tiparvovec
(Glybera): AAV-based treatment for
lipoprotein lipase deficiency
(no longer commercially available)
Axicabtagene ciloleucel
(Yescarta): treatment for large
B-cell lymphoma
[1]
Beremagene geperpavec
(Vyjuvek): treatment of wounds.
[2]
Betibeglogene autotemcel
(Zynteglo): treatment for
beta thalassemia
[3]
Brexucabtagene autoleucel
(Tecartus): treatment for
mantle cell lymphoma
and
acute lymphoblastic leukemia
[4]
[5]
Cambiogenplasmid
(Neovasculgen): treatment for vascular endothelial growth factor
peripheral artery disease
Ciltacabtagene autoleucel
(Carvykti): treatment for
multiple myeloma
[6]
Delandistrogene moxeparvovec
(Elevidys): treatment for
Duchenne muscular dystrophy
[7]
[8]
Elivaldogene autotemcel
(Skysona): treatment for cerebral
adrenoleukodystrophy
[9]
Etranacogene dezaparvovec
(Hemgenix): AAV-based treatment for
hemophilia B
[10]
Exagamglogene autotemcel
(Casgevy): treatment for
sickle cell disease
.
[11]
Gendicine
: treatment for head and neck squamous cell
carcinoma
Idecabtagene vicleucel
(Abecma): treatment for multiple myeloma
[12]
Lovotibeglogene autotemcel
(Lyfgenia): treatment for
sickle cell disease
.
[11]
Nadofaragene firadenovec
(Adstiladrin): treatment for bladder cancer
[13]
Onasemnogene abeparvovec
(Zolgensma): AAV-based treatment for
spinal muscular atrophy
[14]
Strimvelis
: treatment for
adenosine deaminase deficiency
(ADA-SCID)
Talimogene laherparepvec
(Imlygic): treatment for
melanoma
in patients who have recurring skin lesions
[15]
Tisagenlecleucel
(Kymriah): treatment for B cell
lymphoblastic leukemia
[16]
Valoctocogene roxaparvovec
(Roctavian): treatment for
hemophilia A
[17]
[18]
[19]
Voretigene neparvovec
(Luxturna): AAV-based treatment for
Leber congenital amaurosis
[20]
See also
FDA-approved CAR T cell therapies
References
^
"FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma"
(Press release). U.S.
Food and Drug Administration
(FDA). 24 March 2020
. Retrieved
16 December
2022
.
^
"FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa"
.
U.S.
Food and Drug Administration
(FDA)
(Press release). 19 May 2023
. Retrieved
28 May
2023
.
This article incorporates text from this source, which is in the
public domain
.
^
"FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions"
(Press release). U.S.
Food and Drug Administration
(FDA). 17 August 2022
. Retrieved
16 December
2022
.
^
"FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL"
(Press release). U.S.
Food and Drug Administration
(FDA). 24 July 2020
. Retrieved
24 July
2020
.
^
"FDA approves brexucabtagene autoleucel for relapsed or refractory B-cell precursor acute lymphoblastic leukemia"
. U.S.
Food and Drug Administration
(FDA). 1 October 2021
. Retrieved
2 October
2021
.
^
"FDA approves ciltacabtagene autoleucel for relapsed or refractory multiple myeloma"
. U.S.
Food and Drug Administration
(FDA). 7 March 2022
. Retrieved
16 March
2022
.
^
"FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy"
(Press release). U.S.
Food and Drug Administration
(FDA). 22 June 2023
. Retrieved
22 June
2023
.
This article incorporates text from this source, which is in the
public domain
.
^
"Sarepta Therapeutics Announces FDA Approval of Elevidys, the First Gene Therapy to Treat Duchenne Muscular Dystrophy"
(Press release). Sarepta Therapeutics. 22 June 2023
. Retrieved
22 June
2023
– via Business Wire.
^
"Skysona"
. U.S.
Food and Drug Administration
(FDA). 24 October 2022
. Retrieved
16 December
2022
.
^
"FDA Approves First Gene Therapy to Treat Adults with Hemophilia B"
(Press release). U.S.
Food and Drug Administration
(FDA). 22 November 2022
. Retrieved
16 December
2022
.
^
a
b
"FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease"
.
U.S.
Food and Drug Administration
(FDA)
(Press release). 8 December 2023.
Archived
from the original on 8 December 2023
. Retrieved
8 December
2023
.
^
"Abecma"
. U.S.
Food and Drug Administration
(FDA). 21 April 2021
. Retrieved
16 December
2022
.
^
"FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer"
(Press release). U.S.
Food and Drug Administration
(FDA). 16 December 2022
. Retrieved
16 December
2022
.
^
"FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality"
(Press release). U.S.
Food and Drug Administration
(FDA). 24 March 2020
. Retrieved
16 December
2022
.
^
"Imlygic (talimogene laherparepvec)"
. U.S.
Food and Drug Administration
(FDA). 6 July 2022
. Retrieved
16 December
2022
.
^
"FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome"
. U.S.
Food and Drug Administration
(FDA). 30 August 2017
. Retrieved
16 December
2022
.
^
"First gene therapy to treat severe haemophilia A"
(Press release).
European Medicines Agency
(EMA). 24 June 2022.
Archived
from the original on 26 June 2022
. Retrieved
26 June
2022
.
^
"Roctavian: Pending EC decision"
.
European Medicines Agency
(EMA). 23 June 2022.
Archived
from the original on 26 June 2022
. Retrieved
26 June
2022
.
^
"Roctavian"
. Union Register of medicinal products. 25 August 2022
. Retrieved
6 September
2022
.
^
"FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss"
.
U.S. Food and Drug Administration
(Press release). 24 March 2020
. Retrieved
16 December
2022
.
External links
Daley, Jim (1 January 2020).
"Gene Therapy Arrives"
.
Scientific American
. Retrieved
9 June
2020
.
Portal
:
Medicine
Categories
:
Gene therapy
Applied genetics
Bioethics
Biotechnology
Medical genetics
Molecular-biology-related lists
Gene delivery
Genetic engineering
Hidden categories:
Source attribution
Articles with short description
Short description is different from Wikidata
From Wikipedia, the free encyclopedia
This article contains a list of commercially available
gene therapies
.
Gene therapies
See also:
Gene therapy § List of gene therapies for treatment of disease
Alipogene tiparvovec
(Glybera): AAV-based treatment for
lipoprotein lipase deficiency
(no longer commercially available)
Axicabtagene ciloleucel
(Yescarta): treatment for large
B-cell lymphoma
[1]
Beremagene geperpavec
(Vyjuvek): treatment of wounds.
[2]
Betibeglogene autotemcel
(Zynteglo): treatment for
beta thalassemia
[3]
Brexucabtagene autoleucel
(Tecartus): treatment for
mantle cell lymphoma
and
acute lymphoblastic leukemia
[4]
[5]
Cambiogenplasmid
(Neovasculgen): treatment for vascular endothelial growth factor
peripheral artery disease
Ciltacabtagene autoleucel
(Carvykti): treatment for
multiple myeloma
[6]
Delandistrogene moxeparvovec
(Elevidys): treatment for
Duchenne muscular dystrophy
[7]
[8]
Elivaldogene autotemcel
(Skysona): treatment for cerebral
adrenoleukodystrophy
[9]
Etranacogene dezaparvovec
(Hemgenix): AAV-based treatment for
hemophilia B
[10]
Exagamglogene autotemcel
(Casgevy): treatment for
sickle cell disease
.
[11]
Gendicine
: treatment for head and neck squamous cell
carcinoma
Idecabtagene vicleucel
(Abecma): treatment for multiple myeloma
[12]
Lovotibeglogene autotemcel
(Lyfgenia): treatment for
sickle cell disease
.
[11]
Nadofaragene firadenovec
(Adstiladrin): treatment for bladder cancer
[13]
Onasemnogene abeparvovec
(Zolgensma): AAV-based treatment for
spinal muscular atrophy
[14]
Strimvelis
: treatment for
adenosine deaminase deficiency
(ADA-SCID)
Talimogene laherparepvec
(Imlygic): treatment for
melanoma
in patients who have recurring skin lesions
[15]
Tisagenlecleucel
(Kymriah): treatment for B cell
lymphoblastic leukemia
[16]
Valoctocogene roxaparvovec
(Roctavian): treatment for
hemophilia A
[17]
[18]
[19]
Voretigene neparvovec
(Luxturna): AAV-based treatment for
Leber congenital amaurosis
[20]
See also
FDA-approved CAR T cell therapies
References
^
"FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma"
(Press release). U.S.
Food and Drug Administration
(FDA). 24 March 2020
. Retrieved
16 December
2022
.
^
"FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa"
.
U.S.
Food and Drug Administration
(FDA)
(Press release). 19 May 2023
. Retrieved
28 May
2023
.
This article incorporates text from this source, which is in the
public domain
.
^
"FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions"
(Press release). U.S.
Food and Drug Administration
(FDA). 17 August 2022
. Retrieved
16 December
2022
.
^
"FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL"
(Press release). U.S.
Food and Drug Administration
(FDA). 24 July 2020
. Retrieved
24 July
2020
.
^
"FDA approves brexucabtagene autoleucel for relapsed or refractory B-cell precursor acute lymphoblastic leukemia"
. U.S.
Food and Drug Administration
(FDA). 1 October 2021
. Retrieved
2 October
2021
.
^
"FDA approves ciltacabtagene autoleucel for relapsed or refractory multiple myeloma"
. U.S.
Food and Drug Administration
(FDA). 7 March 2022
. Retrieved
16 March
2022
.
^
"FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy"
(Press release). U.S.
Food and Drug Administration
(FDA). 22 June 2023
. Retrieved
22 June
2023
.
This article incorporates text from this source, which is in the
public domain
.
^
"Sarepta Therapeutics Announces FDA Approval of Elevidys, the First Gene Therapy to Treat Duchenne Muscular Dystrophy"
(Press release). Sarepta Therapeutics. 22 June 2023
. Retrieved
22 June
2023
– via Business Wire.
^
"Skysona"
. U.S.
Food and Drug Administration
(FDA). 24 October 2022
. Retrieved
16 December
2022
.
^
"FDA Approves First Gene Therapy to Treat Adults with Hemophilia B"
(Press release). U.S.
Food and Drug Administration
(FDA). 22 November 2022
. Retrieved
16 December
2022
.
^
a
b
"FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease"
.
U.S.
Food and Drug Administration
(FDA)
(Press release). 8 December 2023.
Archived
from the original on 8 December 2023
. Retrieved
8 December
2023
.
^
"Abecma"
. U.S.
Food and Drug Administration
(FDA). 21 April 2021
. Retrieved
16 December
2022
.
^
"FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer"
(Press release). U.S.
Food and Drug Administration
(FDA). 16 December 2022
. Retrieved
16 December
2022
.
^
"FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality"
(Press release). U.S.
Food and Drug Administration
(FDA). 24 March 2020
. Retrieved
16 December
2022
.
^
"Imlygic (talimogene laherparepvec)"
. U.S.
Food and Drug Administration
(FDA). 6 July 2022
. Retrieved
16 December
2022
.
^
"FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome"
. U.S.
Food and Drug Administration
(FDA). 30 August 2017
. Retrieved
16 December
2022
.
^
"First gene therapy to treat severe haemophilia A"
(Press release).
European Medicines Agency
(EMA). 24 June 2022.
Archived
from the original on 26 June 2022
. Retrieved
26 June
2022
.
^
"Roctavian: Pending EC decision"
.
European Medicines Agency
(EMA). 23 June 2022.
Archived
from the original on 26 June 2022
. Retrieved
26 June
2022
.
^
"Roctavian"
. Union Register of medicinal products. 25 August 2022
. Retrieved
6 September
2022
.
^
"FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss"
.
U.S. Food and Drug Administration
(Press release). 24 March 2020
. Retrieved
16 December
2022
.
External links
Daley, Jim (1 January 2020).
"Gene Therapy Arrives"
.
Scientific American
. Retrieved
9 June
2020
.
Portal
:
Medicine
Categories
:
Gene therapy
Applied genetics
Bioethics
Biotechnology
Medical genetics
Molecular-biology-related lists
Gene delivery
Genetic engineering
Hidden categories:
Source attribution
Articles with short description
Short description is different from Wikidata
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