[{"Additional Information":"","Comment":"not for rare disease","Company":"Celgene","Condition/indication":"Relapsed or Refractory Multiple Myeloma","DrugBank ID":"DB16665","Gene":"","Generic Name":"Idecabtagene vicleucel","HGNCID":"","Market":"US, Canada, EU, UK, Japan, Israel, Switzerland","Marketed drug":"Abecma\u2122","Mechanism":"Idecabtagene vicleucel / autologous CAR T -cellsyu76h, CAR T cells recognize the B-cell maturation antigen (BCMA) and kill BCMA-expressing tumor cells. A lentiviral vector, developed by bluebird bio, is used to engineer the CAR T cells.","Modality":"Cell therapy","Type":"Ex vivo GTMP"},{"Additional Information":"","Comment":"not for rare disease","Company":"Ferring Pharmaceuticals","Condition/indication":"high-risk Bacillus Calmette-Gu\u00e9rin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS)","DrugBank ID":"DB17381","Gene":"","Generic Name":"Nadofaragene firadenovec","HGNCID":"","Market":"US","Marketed drug":"Adstiladrin","Mechanism":"A recombinant adenovirus (rAd-IFNa/Syn3) delivers human interferon alfa-2b cDNA into the bladder epithelium. Treats patients with certain types of bladder cancer.","Modality":"Gene therapy","Type":"Adenovirus"},{"Additional Information":"","Comment":"not for rare disease","Company":"SSM Cardinal Glennon Children's Medical Center","Condition/indication":"","DrugBank ID":"","Gene":"","Generic Name":"cord blood injectable suspension for intravenous use","HGNCID":"","Market":"","Marketed drug":"ALLOCORD","Mechanism":"Blood cell transplant derived from umbilical cord and placenta.","Modality":"Cell therapy","Type":"Cell (allogeneic)"},{"Additional Information":"withdrawn by EMA","Comment":"not for rare disease","Company":"Takeda Pharma","Condition/indication":"Complex perianal fistulas in patients with Crohn\u2019s disease","DrugBank ID":"DB16581","Gene":"","Generic Name":"Darvadstrocel","HGNCID":"","Market":"","Marketed drug":"Alofisel\u2122","Mechanism":"Darvadstrocel / Mesenchymal stem cells from fat tissue of adult donors","Modality":"Cell therapy","Type":"SCTMP"},{"Additional Information":"","Comment":"","Company":"Sarepta Therapeutics","Condition/indication":"Duchenne muscular dystrophy","DrugBank ID":"DB14984","Gene":"DMD","Generic Name":"Casimersen","HGNCID":2928.0,"Market":"US","Marketed drug":"Amondys 45","Mechanism":"exon skipping","Modality":"RNA therapy","Type":"blockmer, PMO"},{"Additional Information":"","Comment":"","Company":"AIM ImmunoTech","Condition/indication":"Chronic fatigue syndrome","DrugBank ID":"","Gene":"","Generic Name":"rintatolimod","HGNCID":"","Market":"Argentina","Marketed drug":"Ampligen","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Iovance Biotherapeutics, Inc.","Condition/indication":"adult patients with unresectable or metastatic melanoma","DrugBank ID":"DB17107","Gene":"","Generic Name":"Lifileucel","HGNCID":"","Market":"US","Marketed drug":"AMTAGVI","Mechanism":"Engineered T cells for the treatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor.","Modality":"Cell therapy","Type":"Cell (modified)"},{"Additional Information":"","Comment":"","Company":"ALNYLAM PHARMS INC","Condition/indication":"Hereditary Transthyretin-mediated amyloidosis","DrugBank ID":"DB16699","Gene":"TTR","Generic Name":"Vutrisiran","HGNCID":12405.0,"Market":"US, EU, UK, Brazil, Japan","Marketed drug":"Amvuttra","Mechanism":"knockdown","Modality":"RNA therapy","Type":"siRNA"},{"Additional Information":"","Comment":"not for rare disease","Company":"Suzhou Abogen Biosciences","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"COVID-19 vaccine, Suzhou Abogen\nBiosciences","HGNCID":"","Market":"Indonesia","Marketed drug":"ARCoV","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Arcturus Therapeutics","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"COVID-19 mRNA vaccine, Arcturus","HGNCID":"","Market":"Japan, EU","Marketed drug":"ARCT-154","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Autolus Limited","Condition/indication":"acute lymphoblastic leukemia","DrugBank ID":"DB17362","Gene":"","Generic Name":"Obecabtagene autoleucel","HGNCID":"","Market":"US, UK, EU","Marketed drug":"AUCATZYL","Mechanism":"","Modality":"Cell therapy","Type":""},{"Additional Information":"","Comment":"","Company":"Belief BioMed","Condition/indication":"Hemophilia B","DrugBank ID":"","Gene":"","Generic Name":"dalnacogene ponparvovec","HGNCID":"","Market":"China","Marketed drug":"BBM-H901","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"","Company":"Pfizer, Inc.","Condition/indication":"adults with moderate to severe hemophilia B","DrugBank ID":"DB16783","Gene":"F9","Generic Name":"Fidanacogene elaparvovec","HGNCID":3551.0,"Market":"US/Canada/EMA","Marketed drug":"BEQVEZ","Mechanism":"AAV-mediated gene therapy for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid.","Modality":"Gene therapy","Type":"AAVRh74var"},{"Additional Information":"","Comment":"not for rare disease","Company":"Juno Therapeutics","Condition/indication":"Relapsed or Refractory Large B-cell lymphoma Non-Hodgkin lymphoma Follicular lymphoma","DrugBank ID":"DB16582","Gene":"","Generic Name":"Lisocabtagene maraleucel","HGNCID":"","Market":"US, Japan, EU, Switzerland, UK, Canada","Marketed drug":"Breyanzi","Mechanism":"CAR T cells engineered with lentivirus to attack CD19-expressing tumor cells. Treats patients with certain kinds of large B-cell lymphoma.","Modality":"Cell therapy","Type":"Ex vivo GTMP, Cell (autologous, modified)"},{"Additional Information":"","Comment":"not for rare disease","Company":"Janssen Biotech, Inc.","Condition/indication":"Relapsed or Refractory Multiple Myeloma","DrugBank ID":"DB16738","Gene":"","Generic Name":"Ciltacabtagene autoleucel","HGNCID":"","Market":"US, EU, UK, Japan, Brazil, Australia, Canada, China","Marketed drug":"CARVYKTI","Mechanism":"CAR T cells engineered with lentivirus to attack BCMA-expressing tumor cells. Treats patients with certain kinds of relapsed or refractory multiple myeloma. Ciltacabtagene autoleucel / autologous CAR T cells directed to B cell maturation antigen (BCMA)","Modality":"Cell therapy","Type":"Ex vivo GTMP, Cell (autologous, modified)"},{"Additional Information":"","Comment":"","Company":"Vertex Pharmaceuticals","Condition/indication":"sickle cell disease, beta thalassaemia","DrugBank ID":"DB15572","Gene":"HBB","Generic Name":"Exagamglogene autotemcel","HGNCID":4827.0,"Market":"US, UK, Bahrain, Saudi Arabia, EU, Canada,\nSwitzerland; Qatar","Marketed drug":"Casgevy","Mechanism":"CD34+ hematopoietic stem cells engineered with electroporation of CRISPR/Cas9 RNP complexes to decrease expression of BCL11A to increase fetal hemoglobin production. Treats patients with certain kinds of sickle cell disease.","Modality":"Cell/Gene therapy","Type":"Ex vivo, Cell (autologous, modified)"},{"Additional Information":"","Comment":"not for rare disease","Company":"Cleveland Cord Blood Center","Condition/indication":"","DrugBank ID":"","Gene":"","Generic Name":"HPC, Cord Blood","HGNCID":"","Market":"","Marketed drug":"CLEVECORD","Mechanism":"Transplant of hematopoietic progenitor cells (HPCs) from human cord blood.","Modality":"Cell therapy","Type":"Cell (allogeneic)"},{"Additional Information":"","Comment":"not for rare disease","Company":"BioNTech","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"tozinameran","HGNCID":"","Market":"UK, Bahrain, Israel, Canada, US, Rwanda, Serbia, United Arab Emirates, Macao, Taiwan,\nMexico, Kuwait, Singapore, Saudi Arabia, Chile, Switzerland, EU, Ghana, Colombia,\nPhilippines, Indonesia, Australia, Hong Kong, Peru, South Korea, New Zealand, Japan, Brazil,\nSri Lanka, Vietnam, South Africa, Thailand, Oman, Egypt, Malaysia","Marketed drug":"Comirnaty","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"CSPC Pharmaceutical","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"COVID-19 vaccine, CSPC\nPharmaceutical","HGNCID":"","Market":"China","Marketed drug":"CSPC Pharmaceutical\nCOVID-19 vaccine","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"","Company":"Ionis Pharmaceuticals, Inc.","Condition/indication":"Hereditary angioedema","DrugBank ID":"\nDB18751","Gene":"","Generic Name":"Donidalorsen","HGNCID":"","Market":"US","Marketed drug":"Dawnzera","Mechanism":"prophylaxis to prevent attacks of hereditary angioedema (HAE), knockdown of  KLKB1 gene","Modality":"RNA therapy","Type":"gapmer"},{"Additional Information":"","Comment":"","Company":"Daiichi Sankyo","Condition/indication":"Malignant glioma","DrugBank ID":"","Gene":"","Generic Name":"teserpaturev","HGNCID":"","Market":"Japan","Marketed drug":"Delytact","Mechanism":"","Modality":"Gene therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Duke University School of Medicine","Condition/indication":"","DrugBank ID":"","Gene":"","Generic Name":"HPC, Cord Blood","HGNCID":"","Market":"","Marketed drug":"DUCORD","Mechanism":"Transplant of HPCs from human cord blood.","Modality":"Cell therapy","Type":"Cell (allogeneic)"},{"Additional Information":"","Comment":"not for rare disease","Company":"Atara Biotherapeutics","Condition/indication":"Second-line treatment for transplant recipients who develop, as a result of immunosuppression treatment, Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disease (PTLD)","DrugBank ID":"DB17072","Gene":"","Generic Name":"Tabelecleucel","HGNCID":"","Market":"US/EMA","Marketed drug":"Ebvallo\u2122","Mechanism":"Tabelecleucel / allogeneic, EBV-specific T-cells from matched donor","Modality":"","Type":"SCTMP"},{"Additional Information":"","Comment":"","Company":"SAREPTA","Condition/indication":"Duchenne muscular dystrophy","DrugBank ID":"DB16802","Gene":"DMD","Generic Name":"Delandistrogene moxeparvovec","HGNCID":2928.0,"Market":"US, United Arab Emirates, Qatar, Kuwait, Bahrain,\nOman, Israel, Japan","Marketed drug":"Elevidys","Mechanism":"Recombinant AAVrh74 delivers a micro-dystrophin gene to treat patients with certain types of Duchenne muscular dystrophy.","Modality":"Gene therapy","Type":"In vivo, AAVrh74"},{"Additional Information":"","Comment":"not for rare disease","Company":"Neurotech Pharmaceuticals","Condition/indication":"macular telangiectasia type 2","DrugBank ID":"DB05344","Gene":"","Generic Name":"Revakinagene taroretcel","HGNCID":"","Market":"US","Marketed drug":"Encelto","Mechanism":"allogeneic encapsulated cell-based gene therapy used for the treatment of macular telangiectasia type 2","Modality":"","Type":""},{"Additional Information":"","Comment":"","Company":"Sarepta Therapeutics","Condition/indication":"Duchenne muscular dystrophy","DrugBank ID":"DB06014","Gene":"DMD","Generic Name":"Eteplirsen","HGNCID":2928.0,"Market":"US","Marketed drug":"Exondys","Mechanism":"exon skipping","Modality":"RNA therapy","Type":"blockmer, PMO"},{"Additional Information":"","Comment":"not for rare disease","Company":"Nanjing IASO Biotechnology","Condition/indication":"Multiple myeloma","DrugBank ID":"","Gene":"","Generic Name":"equecabtagene autoleucel","HGNCID":"","Market":"China, Hong Kong","Marketed drug":"Fucaso","Mechanism":"","Modality":"Gene therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Shenzhen SiBiono GeneTech","Condition/indication":"Head and neck cancer","DrugBank ID":"","Gene":"","Generic Name":"recombinant p53 gene","HGNCID":"","Market":"China","Marketed drug":"Gendicine","Mechanism":"","Modality":"Gene therapy","Type":""},{"Additional Information":"","Comment":"","Company":"Gennova Biopharmaceuticals","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"COVID-19 vaccine, Gennova\nBiopharmaceuticals","HGNCID":"","Market":"India","Marketed drug":"Gennova COVID-19\nvaccine","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Organogenesis Inc.","Condition/indication":"Topical (non-submerged) application to a surgically created vascular wound bed in the treatment of mucogingival conditions in adults (venous leg ulcers)","DrugBank ID":"","Gene":"","Generic Name":"Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen","HGNCID":"","Market":"","Marketed drug":"GINTUIT","Mechanism":"Cellularized scaffold for topical application to a surgically created wound bed to treat certain mucogingival conditions. Allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen","Modality":"Cell therapy","Type":"TEP"},{"Additional Information":"","Comment":"multiple enzyme deficiences","Company":"ALNYLAM PHARMS INC","Condition/indication":"Acute Hepatic Porphyria","DrugBank ID":"DB15066","Gene":"x","Generic Name":"Givosiran","HGNCID":"","Market":"US/Canada/EMA","Marketed drug":"Givlaari","Mechanism":"knockdown","Modality":"RNA therapy","Type":"siRNA"},{"Additional Information":"withdrawn","Comment":"","Company":"Chiesi Farmaceutici","Condition/indication":"lipoprotein lipase deficiency","DrugBank ID":"DB13521","Gene":"LPL","Generic Name":"Alipogene tiparvovec","HGNCID":6677.0,"Market":"","Marketed drug":"Glybera (withdrawn)","Mechanism":"","Modality":"Gene therapy","Type":"In vivo AAV1"},{"Additional Information":"","Comment":"not for rare disease","Company":"New York Blood Center","Condition/indication":"","DrugBank ID":"DB11054","Gene":"","Generic Name":"Human cord blood hematopoietic progenitor cell","HGNCID":"","Market":"US","Marketed drug":"HEMACORD","Mechanism":"Transplant of HPCs from human cord blood.","Modality":"Cell therapy","Type":"Cell (allogeneic)"},{"Additional Information":"","Comment":"","Company":"CSL Behring LLC, UniQure","Condition/indication":"Adult patients with hemophilia B","DrugBank ID":"DB16791","Gene":"F9","Generic Name":"Etranacogene dezaparvovec","HGNCID":3551.0,"Market":"US, EU, UK, Canada, Switzerland, Australia, Hong\nKong, Saudi Arabia, South Korea, Taiwan","Marketed drug":"HEMGENIX","Mechanism":"Recombinant AAV5 delivers F9 to treat patients with certain kinds of Hemophilia B. Etranacogene dezaparvovec / AAV5 vector containing the cDNA of human coagulation factor IX","Modality":"Gene therapy","Type":"In vivo GTMP AAV5"},{"Additional Information":"","Comment":"","Company":"Hrain Biotechnology","Condition/indication":"Relapsed or refractory large B-cell lymphoma","DrugBank ID":"","Gene":"","Generic Name":"renikeolunsai","HGNCID":"","Market":"China","Marketed drug":"Hicara","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Holostem Advanced Therapies","Condition/indication":"Patients with corneal damage due to (chemical) burns","DrugBank ID":"DB18660","Gene":"","Generic Name":"Ex vivo expanded autologous human corneal epithelial cells containing stem cells","HGNCID":"","Market":"EMA","Marketed drug":"Holoclar\u2122","Mechanism":"Ex vivo expanded autologous human corneal epithelial cells containing stem cells","Modality":"Cell therapy","Type":"SCTMP"},{"Additional Information":"","Comment":"not for rare disease","Company":"Clinimmune Labs, University of Colorado Cord Blood Bank","Condition/indication":"","DrugBank ID":"","Gene":"","Generic Name":"","HGNCID":"","Market":"","Marketed drug":"HPC, Cord Blood","Mechanism":"Transplant of HPCs from human cord blood.","Modality":"Cell therapy","Type":"Cell (allogeneic)"},{"Additional Information":"","Comment":"not for rare disease","Company":"Bloodworks","Condition/indication":"","DrugBank ID":"","Gene":"","Generic Name":"","HGNCID":"","Market":"","Marketed drug":"HPC, Cord Blood - Bloodworks","Mechanism":"Transplant of HPCs from human cord blood.","Modality":"Cell therapy","Type":"Cell (allogeneic)"},{"Additional Information":"","Comment":"not for rare disease","Company":"LifeSouth Community Blood Centers, Inc.","Condition/indication":"","DrugBank ID":"","Gene":"","Generic Name":"","HGNCID":"","Market":"","Marketed drug":"HPC, Cord Blood - LifeSouth","Mechanism":"Transplant of HPCs from human cord blood.","Modality":"Cell therapy","Type":"Cell (allogeneic)"},{"Additional Information":"","Comment":"not for rare disease","Company":"MD Anderson Cord Blood Bank","Condition/indication":"","DrugBank ID":"","Gene":"","Generic Name":"","HGNCID":"","Market":"","Marketed drug":"HPC, Cord Blood - MD Anderson Cord Blood Bank","Mechanism":"Transplant of HPCs from human cord blood.","Modality":"Cell therapy","Type":"Cell (allogeneic)"},{"Additional Information":"","Comment":"not for rare disease","Company":"Amgen","Condition/indication":"melanoma","DrugBank ID":"DB13896","Gene":"","Generic Name":"Talimogene laherparepvec","HGNCID":"","Market":"US, EU, UK, Australia","Marketed drug":"Imlygic","Mechanism":"Recombinant HSV-1 delivers GM-CSF to treat patients with certain unresectable melanoma lesions. Talimogene laherparepvec / HSV-1 oncolytic virus containing the cDNA of human GM-CSF","Modality":"Gene therapy","Type":"In vivo, HSV-1"},{"Additional Information":"","Comment":"","Company":"Juventas Cell Therapy","Condition/indication":"Acute lymphocytic leukemia","DrugBank ID":"","Gene":"","Generic Name":"inaticabtagene autoleucel","HGNCID":"","Market":"China","Marketed drug":"inaticabtagene\nautoleucel","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Astellas Pharma Inc.","Condition/indication":"age-related macular degeneration","DrugBank ID":"DB15165","Gene":"","Generic Name":"Avacincaptad pegol","HGNCID":"","Market":"US, Japan","Marketed drug":"Izervay","Mechanism":"inhibitor","Modality":"RNA therapy","Type":"aptamer"},{"Additional Information":"","Comment":"","Company":"PTC Therapeutics","Condition/indication":"aromatic L\u2011amino acid decarboxylase (AADC) deficiency","DrugBank ID":"DB16780","Gene":"DDC","Generic Name":"Eladocagene exuparvovec","HGNCID":2719.0,"Market":"US/EMA","Marketed drug":"KEBILIDI (eladocagene exuparvovec-tneq)","Mechanism":"","Modality":"Gene therapy","Type":"In vivo"},{"Additional Information":"","Comment":"not for rare disease","Company":"Novartis","Condition/indication":"B cell lymphoblastic leukemia, Acute Lymphoblastic Leukemia (ALL)","DrugBank ID":"DB13881","Gene":"","Generic Name":"Tisagenlecleucel","HGNCID":"","Market":"US, EU, UK, Japan, Australia, Canada, South Korea,\nSwitzerland","Marketed drug":"Kymriah","Mechanism":"CAR T cells engineered with lentivirus to attack CD19-expressing tumor cells. Treats patients with certain kinds of relapsed or refractory follicular lymphoma.","Modality":"Cell therapy","Type":"Cell (autologous, modified)"},{"Additional Information":"withdrawn","Comment":"","Company":"Genzyme","Condition/indication":"Homozygous Familial Hypercholesterolemia","DrugBank ID":"DB05528","Gene":"APOB, LDLR, PCSK9","Generic Name":"Mipomersen","HGNCID":"","Market":"US, Mexico, Argentina, South Korea","Marketed drug":"Kynamro (withdrawn)","Mechanism":"knockdown","Modality":"RNA therapy","Type":"MOE gapmer"},{"Additional Information":"","Comment":"not for rare disease","Company":"CellTrans Inc.","Condition/indication":"","DrugBank ID":"DB17961","Gene":"","Generic Name":"Donislecel","HGNCID":"","Market":"US","Marketed drug":"LANTIDRA","Mechanism":"Pancreatic islet cells from a deceased donor are transplanted to treat patients with certain types of type I diabetes.","Modality":"Cell therapy","Type":"Cell (allogeneic)"},{"Additional Information":"","Comment":"not for rare disease","Company":"Fibrocell Technologies","Condition/indication":"","DrugBank ID":"DB11051","Gene":"","Generic Name":"Azficel-T","HGNCID":"","Market":"Withdrawn","Marketed drug":"LAVIV (withdrawn)","Mechanism":"Fibroblasts to improve the appearance of moderate to severe nasolabial fold wrinkles.","Modality":"Cell therapy","Type":"Cell (autologous, expanded)"},{"Additional Information":"","Comment":"","Company":"Orchard Therapeutics (Europe) Limited","Condition/indication":"metachromatic leukodystrophy","DrugBank ID":"DB17538","Gene":"ARSA","Generic Name":"Atidarsagene autotemcel","HGNCID":713.0,"Market":"EU, UK, Switzerland, US","Marketed drug":"LENMELDY","Mechanism":"Ex vivo gene therapy for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).","Modality":"Cell/Gene therapy","Type":"Gene (ex vivo, autologous)"},{"Additional Information":"","Comment":"multiple genetic disorders","Company":"Novartis","Condition/indication":"Heterozygous Familial Hyperchloesterolemia","DrugBank ID":"DB14901","Gene":"APOB, LDLR, PCSK9","Generic Name":"Inclisiran","HGNCID":"","Market":"EU, UK, Australia, Canada, Israel, US, Saudi Arabia, Japan, China","Marketed drug":"Leqvio","Mechanism":"knockdown","Modality":"RNA therapy","Type":"siRNA"},{"Additional Information":"","Comment":"","Company":"Spark Therapeutics","Condition/indication":"biallelic RPE65 mutation associated Leber congenital amaurosis","DrugBank ID":"DB13932","Gene":"RPE65","Generic Name":"Voretigene neparvovec","HGNCID":10294.0,"Market":"US, EU, UK, Australia, Canada, South Korea, Japan","Marketed drug":"Luxturna","Mechanism":"Recombinant AAV2 delivers RPE65 to treat patients with RPE65-mutation-associated retinal dystrophy.","Modality":"Gene therapy","Type":"In vivo AAV2"},{"Additional Information":"","Comment":"","Company":"Bluebird Bio","Condition/indication":"sickle cell disease","DrugBank ID":"DB18680","Gene":"HBB","Generic Name":"Lovotibeglogene autotemcel","HGNCID":4827.0,"Market":"US","Marketed drug":"Lyfgenia","Mechanism":"Hematopoietic stem cells engineered with a lentivirus, BB305, expressing modified \u00dfA-globin gene. Treats patients with certain kinds of sickle cell disease.","Modality":"Cell/Gene therapy","Type":"Ex vivo Cell (autologous, modified)"},{"Additional Information":"","Comment":"not for rare disease","Company":"Vericel Corp.","Condition/indication":"The repair of single or multiple symptomatic, full-thickness cartilage defects of the knee with or without bone involvement in adults","DrugBank ID":"","Gene":"","Generic Name":"autologous cultured chondrocytes on porcine collagen membrane","HGNCID":"","Market":"","Marketed drug":"MACI","Mechanism":"Cellularized scaffold to repair cartilage defects of the knee. Matrix-applied characterised autologous cultured chondrocytes","Modality":"Cell therapy","Type":"Cell (autologous, expanded)"},{"Additional Information":"","Comment":"not for rare disease","Company":"OSI/Eyetech Pharmaceuticals, New York, NY and Pfizer, New York, NY","Condition/indication":"neovascular (wet) age-related macular degeneration","DrugBank ID":"DB04895","Gene":"","Generic Name":"Pegaptanib","HGNCID":"","Market":"Withdrawn","Marketed drug":"Macugen","Mechanism":"inhibitor","Modality":"RNA therapy","Type":"aptamer"},{"Additional Information":"","Comment":"not for rare disease","Company":"Moderna Therapeutics","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"COVID-19 next generation vaccine, Moderna\nTherapeutics","HGNCID":"","Market":"US","Marketed drug":"mNexspike","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Moderna Therapeutics","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"COVID-19 bivalent original/Omicron\nvaccine, Moderna","HGNCID":"","Market":"UK, Canada, Taiwan, Switzerland, Japan, EU, Australia, South\nKorea, Singapore, US","Marketed drug":"Moderna Spikevax Bivalent\nOriginal/Omicron vaccine","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Moderna Therapeutics","Condition/indication":"Respiratory syncytial virus prophylaxis","DrugBank ID":"","Gene":"","Generic Name":"respiratory syncytial virus vaccine, Moderna\nTherapeutics","HGNCID":"","Market":"US, EU, Canada, Qatar, Taiwan, UAE, UK, Australia,\nSwitzerland, Japan","Marketed drug":"mRESVIA","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"ArtGene Therapeutics","Condition/indication":"vascular endothelial growth factor peripheral artery disease","DrugBank ID":"","Gene":"","Generic Name":"Cambiogenplasmid","HGNCID":"","Market":"Russian Federation, Ukraine","Marketed drug":"Neovasculgen","Mechanism":"","Modality":"","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Gamida Cell Ltd.","Condition/indication":"","DrugBank ID":"DB17132","Gene":"","Generic Name":"Omidubicel","HGNCID":"","Market":"US","Marketed drug":"OMISIRGE","Mechanism":"Transplant of nicotinamide-modified HPCs from human cord blood.","Modality":"Cell therapy","Type":"Cell (allogeneic, modified)"},{"Additional Information":"","Comment":"not for rare disease","Company":"Shanghai Sunway Biotech","Condition/indication":"Head and neck cancer; nasopharyngeal cancer","DrugBank ID":"","Gene":"","Generic Name":"E1B/E3 deficient adenovirus","HGNCID":"","Market":"China","Marketed drug":"Oncorine","Mechanism":"","Modality":"Gene therapy","Type":""},{"Additional Information":"","Comment":"","Company":"ALNYLAM PHARMS INC","Condition/indication":"Hereditary Transthyretin-mediated amyloidosis","DrugBank ID":"DB14582","Gene":"TTR","Generic Name":"Patisiran","HGNCID":12405.0,"Market":"US, EU, UK, Japan, Canada, Switzerland, Brazil, Taiwan, Israel, Turkey, Australia","Marketed drug":"Onpattro","Mechanism":"knockdown","Modality":"RNA therapy","Type":"siRNA"},{"Additional Information":"","Comment":"","Company":"ALNYLAM PHARMS INC","Condition/indication":"Primary Hyperoxaluria Type 1","DrugBank ID":"DB15935","Gene":"AGXT","Generic Name":"Lumasiran","HGNCID":341.0,"Market":"US/Canada/EMA/Brazil/UK","Marketed drug":"Oxlumo","Mechanism":"knockdown","Modality":"RNA therapy","Type":"siRNA"},{"Additional Information":"","Comment":"","Company":"Precigen","Condition/indication":"Recurrent respiratory papillomatosis (RRP)","DrugBank ID":"","Gene":"","Generic Name":"zopapogene imadenovec","HGNCID":"","Market":"USA","Marketed drug":"Papzimeos","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Dendreon Corp.","Condition/indication":"Metastatic castrate-resistant hormone-refractory prostate cancer","DrugBank ID":"DB06688","Gene":"","Generic Name":"Sipuleucel-T","HGNCID":"","Market":"US","Marketed drug":"PROVENGE","Mechanism":"Antigen presenting cells are activated and used to treat certain types of prostate cancer. Sipuleucel-T / autologous dendritic cells","Modality":"Cell therapy","Type":"Cell (autologous, activated/expanded) SCTMP"},{"Additional Information":"","Comment":"","Company":"Biogen","Condition/indication":"SOD1-associated ALS","DrugBank ID":"DB14782","Gene":"SOD1","Generic Name":"Tofersen","HGNCID":11179.0,"Market":"US, EU, Japan, China, Canada; South Korea","Marketed drug":"Qalsody","Mechanism":"knockdown","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"","Company":"Immuneel Therapeutics","Condition/indication":"B-cell Non-Hodgkin\u201a\u00c4\u00f4s Lymphoma (B-NHL)","DrugBank ID":"","Gene":"","Generic Name":"varnimcabtagene autoleucel","HGNCID":"","Market":"India","Marketed drug":"Qartemi","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"","Company":"ALNYLAM PHARMS INC","Condition/indication":"Hemophilia A or B","DrugBank ID":"DB15002","Gene":"F8, F9","Generic Name":"Fitusiran","HGNCID":"","Market":"US","Marketed drug":"Qfitlia","Mechanism":"knockdown","Modality":"RNA therapy","Type":"GalNac siRNA"},{"Additional Information":"","Comment":"","Company":"Arrowhead Pharmaceuticals","Condition/indication":"Familial chylomicronemia syndrome","DrugBank ID":"\nDB18997","Gene":"LPL","Generic Name":"Plozasiran","HGNCID":6677.0,"Market":"US","Marketed drug":"Redemplo","Mechanism":"Plozasiran is an apolipoprotein C-III (apoC-III)-directed small interfering ribonucleic acid (siRNA).","Modality":"RNA therapy","Type":"siRNA"},{"Additional Information":"","Comment":"not for rare disease","Company":"StemCyte, Inc.","Condition/indication":"","DrugBank ID":"","Gene":"","Generic Name":"HPC, Cord Blood","HGNCID":"","Market":"","Marketed drug":"REGENECYTE","Mechanism":"","Modality":"Cell therapy","Type":""},{"Additional Information":"","Comment":"","Company":"JW Therapeutics","Condition/indication":"Diffuse large B-cell lymphoma; follicular lymphoma; mantle\ncell lymphoma","DrugBank ID":"","Gene":"","Generic Name":"relmacabtagene autoleucel","HGNCID":"","Market":"China, Macao","Marketed drug":"Relma-cel","Mechanism":"","Modality":"Gene therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Enzyvant Therapeutics GmbH","Condition/indication":"Pediatric patients with Congenital Athymia","DrugBank ID":"DB16736","Gene":"","Generic Name":"Allogeneic processed thymus tissue","HGNCID":"","Market":"US","Marketed drug":"RETHYMIC","Mechanism":"Thymus tissue implant to improve immune function in patients with certain types of congenital athymia. Human allogeneic processed thymus tissue-agdc","Modality":"Cell therapy","Type":"Cell (allogeneic) TEP"},{"Additional Information":"","Comment":"","Company":"Epeius Biotechnologies","Condition/indication":"Solid tumors","DrugBank ID":"","Gene":"","Generic Name":"mutant cyclin-G1 gene","HGNCID":"","Market":"Philippines","Marketed drug":"Rexin-G","Mechanism":"","Modality":"Gene therapy","Type":""},{"Additional Information":"","Comment":"","Company":"Dicerna Pharmaceuticals, Inc","Condition/indication":"Primary Hyperoxaluria Type 1","DrugBank ID":"DB17635","Gene":"AGXT","Generic Name":"Nedosiran","HGNCID":341.0,"Market":"US","Marketed drug":"Rivfloza","Mechanism":"knockdown","Modality":"RNA therapy","Type":"siRNA"},{"Additional Information":"","Comment":"","Company":"BioMarin International Limited","Condition/indication":"hemophilia A","DrugBank ID":"DB15561","Gene":"F8","Generic Name":"Valoctocogene roxaparvovec","HGNCID":3546.0,"Market":"US/EMA","Marketed drug":"Roctavian","Mechanism":"Recombinant AAV5 delivers a B-domain deleted SQ form of F8 to treat patients with certain types of hemophilia A.","Modality":"Gene therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Walvax Biotechnology","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"COVID-19 mRNA vaccine, Walvax\nBiotechnology","HGNCID":"","Market":"China","Marketed drug":"RQ-3033","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Mesoblast, Inc.","Condition/indication":"steroid-refractory acute graft-versus-host disease (SR-aGvHD)","DrugBank ID":"DB18032","Gene":"","Generic Name":"Allogenic bone marrow-derived mesenchymal stem cells","HGNCID":"","Market":"US","Marketed drug":"RYONCIL (remestemcel-L-rknd)","Mechanism":"","Modality":"Cell therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Geron Corporation","Condition/indication":"myelodysplastic syndrome","DrugBank ID":"DB14909","Gene":"","Generic Name":"Imetelstat","HGNCID":"","Market":"US","Marketed drug":"Rytelo","Mechanism":"inhibitor","Modality":"RNA therapy","Type":"ASO"},{"Additional Information":"","Comment":"","Company":"bluebird bio, Inc.","Condition/indication":"cerebral adrenoleukodystrophy","DrugBank ID":"DB16746","Gene":"ABCD1","Generic Name":"Elivaldogene autotemcel","HGNCID":61.0,"Market":"US","Marketed drug":"Skysona","Mechanism":"Hematopoietic stem cells engineered with Lenti-D vectors that express the ABCD1 gene. Treats patients with a kind of cerebral adrenoleukodystrophy. Elivaldogene autotemcel / autologous CD34+ cells tranduced with retroviral vector containing The cDNA of the human ABCD1","Modality":"Gene therapy","Type":"Ex vivo GTMP"},{"Additional Information":"","Comment":"not for rare disease","Company":"CO.DON Ag","Condition/indication":"Symptomatic articular cartilage defects of the femoral condyle and the patella of the knee","DrugBank ID":"DB10997","Gene":"","Generic Name":"Autologous cultured chondrocytes","HGNCID":"","Market":"US/EMA","Marketed drug":"Spherox\u2122","Mechanism":"Spheroids of human autologous matrix-associated chondrocytes","Modality":"Cell therapy","Type":"SCTMP"},{"Additional Information":"","Comment":"not for rare disease","Company":"Moderna Therapeutics","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"COVID-19 vaccine,\nModerna","HGNCID":"","Market":"US, Canada, Israel, EU, Switzerland, Singapore, Qatar, Vietnam, UK, Philippines, Thailand,\nJapan, South Korea, Brunei, Paraguay, Taiwan, Botswana, India, Indonesia, Saudi Arabia,\nMexico, Australia, Nigeria, Colombia","Marketed drug":"Spikevax","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"","Company":"Ionis Pharmaceuticals, Inc.","Condition/indication":"Spinal Muscular Atrophy","DrugBank ID":"DB13161","Gene":"SMN1","Generic Name":"Nusinersen","HGNCID":11117.0,"Market":"US, EU, UK, Canada, Japan, Brazil, Switzerland, Australia, South Korea, China, Argentina,\nColombia, Taiwan, Turkey, Hong Kong, Israel","Marketed drug":"Spinraza","Mechanism":"exon inclusion","Modality":"RNA therapy","Type":"blockmer, full MOE"},{"Additional Information":"","Comment":"not for rare disease","Company":"Mallinckrodt plc","Condition/indication":"Adults with thermal burns containing intact dermal elements for which surgical intervention is clinically indicated","DrugBank ID":"DB16709","Gene":"","Generic Name":"Allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen","HGNCID":"","Market":"US","Marketed drug":"StrataGraft\u2122","Mechanism":"Allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen Cellularized scaffold of keratinocytes and dermal fibroblasts in murine collagen to treat patients with some types of thermal burns.","Modality":"Cell therapy","Type":"TEP"},{"Additional Information":"","Comment":"","Company":"Orchard Tx","Condition/indication":"Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)","DrugBank ID":"DB17594","Gene":"ADA","Generic Name":"Strimvelis","HGNCID":186.0,"Market":"EMA, UK","Marketed drug":"Strimvelis\u2122","Mechanism":"Autologous CD34+ cells transduced with a retroviral vector containing the ADA cDNA encoding human adenosine deaminase (ADA)","Modality":"Gene therapy","Type":"Ex vivo GTMP"},{"Additional Information":"","Comment":"not for rare disease","Company":"Humacyte Global, Inc.","Condition/indication":"","DrugBank ID":"","Gene":"","Generic Name":"acellular tissue engineered vessel-tyod","HGNCID":"","Market":"","Marketed drug":"SYMVESS","Mechanism":"acellular tissue engineered vessel indicated for use in adults as a vascular conduit for extremity arterial injury","Modality":"","Type":""},{"Additional Information":"","Comment":"","Company":"CSPC Pharmaceutical","Condition/indication":"Infection, coronavirus, novel coronavirus\nprophylaxis","DrugBank ID":"","Gene":"","Generic Name":"Bivalent COVID-19 mRNA vaccine, CSPC\nPharmaceutical","HGNCID":"","Market":"China","Marketed drug":"SYS-6006.32","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"not for rare disease","Company":"Kite Pharma","Condition/indication":"Mantle Cell Lymphoma Acute Lymphoblastic Leukemia (ALL)","DrugBank ID":"DB15699","Gene":"","Generic Name":"Brexucabtagene autoleucel","HGNCID":"","Market":"US, EU, UK, Australia, Canada","Marketed drug":"Tecartus\u2122","Mechanism":"Brexucabtagene autoleucel / autologous CAR T cells directed to CD19","Modality":"Cell therapy","Type":"Ex vivo GTMP Cell (autologous, modified)"},{"Additional Information":"","Comment":"not for rare disease","Company":"Adaptimmune","Condition/indication":"synovial sarcoma","DrugBank ID":"DB18592","Gene":"","Generic Name":"Afamitresgene autoleucel","HGNCID":"","Market":"US","Marketed drug":"Tecelra","Mechanism":"","Modality":"","Type":"Ex vitro"},{"Additional Information":"Canada - cancelled post-market","Comment":"","Company":"Ionis Pharmaceuticals, Inc.","Condition/indication":"Hereditary Transthyretin-mediated amyloidosis","DrugBank ID":"DB14713","Gene":"TTR","Generic Name":"Inotersen","HGNCID":12405.0,"Market":"US/Canada (cancelled post-market)/EMA/Brazil","Marketed drug":"Tegsedi","Mechanism":"knockdown","Modality":"RNA therapy","Type":"MOE gapmer"},{"Additional Information":"","Comment":"","Company":"Ionis Pharmaceuticals, Inc.","Condition/indication":"familial chylomicronemia syndrome (FCS)","DrugBank ID":"DB18728","Gene":"LPL","Generic Name":"Olezarsen","HGNCID":6677.0,"Market":"US, EU","Marketed drug":"Tryngolza","Mechanism":"knockdown","Modality":"RNA therapy","Type":"GalNAc ASO"},{"Additional Information":"","Comment":"","Company":"PTC Tx","Condition/indication":"Patients > 18 months with aromatic L-amino acid decarboxylase deficiencies","DrugBank ID":"DB16780","Gene":"DCC","Generic Name":"Eladocagene exuparvovec","HGNCID":2701.0,"Market":"EU, UK, Israel, US","Marketed drug":"Upstaza\u2122","Mechanism":"Eladocagene exuparvovec / AAV2 vector containing the cDNA of human dopa decarboxylase (DCC)","Modality":"","Type":"In vivo GTMP"},{"Additional Information":"","Comment":"","Company":"NIPPON SHINYAKU","Condition/indication":"Duchenne muscular dystrophy","DrugBank ID":"DB15005","Gene":"DMD","Generic Name":"Viltolarsen","HGNCID":2928.0,"Market":"US, Japan","Marketed drug":"Viltepso","Mechanism":"exon skipping","Modality":"RNA therapy","Type":"blockmer, PMO"},{"Additional Information":"","Comment":"","Company":"Krystal Biotech","Condition/indication":"dystrophic epidermolysis bullosa (DEB)","DrugBank ID":"DB17831","Gene":"COL7A1","Generic Name":"Beremagene geperpavec","HGNCID":2214.0,"Market":"US, EU, Japan","Marketed drug":"Vyjuvek","Mechanism":"Recombinant HSV-1 delivers the collagen type VII gene (COL7) to treat certain wounds of patients with dystrophic epidermolysis bullosa with mutations in the COL7A1 gene.","Modality":"Gene therapy","Type":"In vivo HSV-1"},{"Additional Information":"","Comment":"","Company":"Sarepta Therapeutics","Condition/indication":"Duchenne muscular dystrophy","DrugBank ID":"DB15593","Gene":"DMD","Generic Name":"Golodirsen","HGNCID":2928.0,"Market":"US","Marketed drug":"Vyondys 53","Mechanism":"exon skipping","Modality":"RNA therapy","Type":"blockmer, PMO"},{"Additional Information":"","Comment":"","Company":"AstraZeneca and Ionis","Condition/indication":"Hereditary Transthyretin-mediated amyloidosis","DrugBank ID":"DB16199","Gene":"TTR","Generic Name":"Eplontersen","HGNCID":12405.0,"Market":"US, Canada, EU, UK","Marketed drug":"Wainua","Mechanism":"knockdown","Modality":"RNA therapy","Type":"GaNac MOE gapmer"},{"Additional Information":"First approved gene therapy developed by patient organisation/charity","Comment":"","Company":"Fondazione Telethon ETS","Condition/indication":"Wiskott\u2013Aldrich syndrome","DrugBank ID":"","Gene":"WAS","Generic Name":"Etuvetidigene autotemcel","HGNCID":12731.0,"Market":"US","Marketed drug":"Waskyra","Mechanism":"autologous CD34+ haematopoietic stem cells transduced ex vivo with a lentiviral vector encoding human Wiskott-Aldrich syndrome protein","Modality":"Gene therapy","Type":"ex vivo"},{"Additional Information":"","Comment":"","Company":"Akcea and Ionis","Condition/indication":"Familial Chylomicronemia","DrugBank ID":"DB15067","Gene":"LPL","Generic Name":"Volanesorsen","HGNCID":6677.0,"Market":"EU, UK, Brazil, Canada","Marketed drug":"Waylivra","Mechanism":"knockdown","Modality":"RNA therapy","Type":"MOE gapmer"},{"Additional Information":"","Comment":"not for rare disease","Company":"Kite pharma","Condition/indication":"large B-cell lymphoma","DrugBank ID":"DB13915","Gene":"","Generic Name":"Axicabtagene ciloleucel","HGNCID":"","Market":"US, EU, UK, Japan, Canada, China, Australia, South\nKorea","Marketed drug":"Yescarta","Mechanism":"CAR T cells engineered with gammaretrovirus to attack CD19-expressing tumor cells. Treats patients with certain kinds of relapsed or refractory large B-cell lymphoma.","Modality":"Cell therapy","Type":"Ex vitro Cell (autologous, modified)"},{"Additional Information":"","Comment":"not for rare disease","Company":"MolMed S.p.A.","Condition/indication":"Add on treatment for patients who have received a haploidentical hematopoietic stem cell transplant","DrugBank ID":"","Gene":"","Generic Name":"nalotimagene carmaleucel","HGNCID":"","Market":"Withdrawn","Marketed drug":"Zalmoxis\u2122","Mechanism":"Allogeneic T cells genetically modified with a retroviral vector encoding for a truncated form of the human low affinity nerve growth factor receptor (\u0394LNGFR) and the herpes simplex I virus thymidine kinase (HSV-TK Mut2)","Modality":"","Type":"Ex vivo GTMP"},{"Additional Information":"","Comment":"","Company":"Abeona Therapeutics","Condition/indication":"Recessive dystrophic epidermolysis bullosa (RDEB)","DrugBank ID":"","Gene":"","Generic Name":"prademagene zamikeracel","HGNCID":"","Market":"US","Marketed drug":"Zevaskyn","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"","Company":"CARsgen Therapeutics","Condition/indication":"Relapsed or refractory multiple myeloma","DrugBank ID":"","Gene":"","Generic Name":"zevorcabtagene autoleucel","HGNCID":"","Market":"China","Marketed drug":"zevorcabtagene\nautoleucel","Mechanism":"","Modality":"RNA therapy","Type":""},{"Additional Information":"","Comment":"Itvisma ","Company":"Novartis Gene Therapies","Condition/indication":"spinal muscular atrophy type I","DrugBank ID":"DB15528","Gene":"SMN1","Generic Name":"Onasemnogene abeparvovec","HGNCID":11117.0,"Market":"US, EU, UK, Japan, Australia, Canada, Brazil, Israel,\nTaiwan, South Korea, China","Marketed drug":"Zolgensma","Mechanism":"Recombinant AAV9 delivers SMN1 to treat patients with certain types of spinal muscular atrophy. Onasemnogene abeparvovec / AAV9 vector containing the SMN1 transgene","Modality":"Gene therapy","Type":"In vivo AAV9"},{"Additional Information":"","Comment":"","Company":"Bluebird Bio","Condition/indication":"Transfusion-dependent beta thalassemia","DrugBank ID":"DB16900","Gene":"HBB","Generic Name":"Betibeglogene autotemcel","HGNCID":4827.0,"Market":"US","Marketed drug":"Zynteglo\u2122","Mechanism":"Betibeglogene autotemcel / Lentiviral vector containing the cDNA of the human HBB gene. Hematopoietic stem cells engineered with a lentivirus, LentiGlobin BB305, to express HBB. Treats patients with certain types of \u00df-thalassemia.","Modality":"Cell/Gene therapy","Type":"Ex vivo GTMP Cell (autologous, modified)"}]