Drug Type Gene therapy, Hematopoietic stem cell therapy |
Synonyms 2009 EGT-produced vector, Autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiglobin BB305 lentiviral vector encoding the beta-A-T87Q-globin gene, Autologous CD34+ cells encoding βA-T87Q-globin gene + [11] |
Target |
Action stimulants |
Mechanism β-globin stimulants(Hemoglobin beta chain stimulants), Gene transference(Gene transference) |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization- |
Drug Highest PhaseApproved |
First Approval Date European Union (29 May 2019), |
RegulationBreakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), PRIME (European Union), Regenerative Medicine Advanced Therapy (United States), Accelerated assessment (European Union), Priority Review (United States) |
KEGG | Wiki | ATC | Drug Bank |
---|---|---|---|
- | Betibeglogene autotemcel | - |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Thalassemia | United States | 17 Aug 2022 | |
Transfusion-dependent Beta Thalassemia | European Union | 29 May 2019 | |
Transfusion-dependent Beta Thalassemia | Iceland | 29 May 2019 | |
Transfusion-dependent Beta Thalassemia | Liechtenstein | 29 May 2019 | |
Transfusion-dependent Beta Thalassemia | Norway | 29 May 2019 | |
Beta-Thalassemia | United Kingdom | - |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Anemia, Sickle Cell | NDA/BLA | United States | 25 Apr 2023 | |
Transfusion-dependent Thalassemia | Phase 3 | United States | 08 Aug 2016 | |
Transfusion-dependent Thalassemia | Phase 3 | France | 08 Aug 2016 | |
Transfusion-dependent Thalassemia | Phase 3 | Germany | 08 Aug 2016 | |
Transfusion-dependent Thalassemia | Phase 3 | Italy | 08 Aug 2016 | |
Transfusion-dependent Thalassemia | Phase 3 | Thailand | 08 Aug 2016 | |
Transfusion-dependent Thalassemia | Phase 3 | United Kingdom | 08 Aug 2016 |
Phase 3 | 18 | oytutimrfu(hcidntkyvf) = aamlozedxu ezysnjsdcn (xfhhtqwsrx ) View more | Positive | 08 Nov 2024 | |||
Not Applicable | - | vygaszyvbd(gdlpedpbqu) = Most patients (51/66 [77.3%]) had ≥1 adverse event (AE) attributed to mobilization and apheresis. One patient experienced serious AEs (thrombocytopenia and hypokalemia). zvhkfixcrk (pjyurvenvi ) View more | - | 01 Feb 2024 | |||
Beti-cel gene therapy | |||||||
HGB-210 (BusinessWire) Manual | Not Applicable | 47 | jjlhxejpae(tdjthqgcbk) = cgimgwrnrw niklpbdnvr (rysdlyijve ) View more | Positive | 09 Dec 2023 | ||
FDA Manual | Phase 1/2 | 45 | zdmitnfzvv(ipunylitkd) = ubsrotfbje ojsxgxnwnu (kmuvwhbbnp, 71 - 97) View more | Positive | 08 Dec 2023 | ||
Phase 1/2 | BB305 lentiviral vector (LVV) | - | (Group A) | oacldziefg(vaiwyqephm) = The most common SAEs in Groups A and C are shown in Table 2. Two AML events were reported in Group A, and none in Group C. ykcvcmziyz (rqqnonnxfk ) | Positive | 01 Mar 2022 | |
(Group C) | |||||||
Phase 1/2 | 35 | LentiGlobin for SCD (bb1111) gene therapy | ikjpkcydcn(kxysnxfsir) = wgetosgwxl uhchobjrnz (tdxksbjvjb ) | Positive | 01 Mar 2022 | ||
Not Applicable | 2 | Beti-cel gene therapy | alijonmrix(fwmlbmwtse) = Post-infusion AEs for pt 1 included febrile neutropenia, elevated C-reactive protein, pruritus, gingivitis, mild mucositis, and vertigo. odmnkknctb (zrqqytfcew ) View more | Positive | 01 Mar 2022 | ||
Not Applicable | - | Beti-cel gene therapy | mdfcfewpwh(mpgougodhe) = Seven pts experienced DP-related adverse events (AEs) occurring between infusion and engraftment (abdominal pain [n=5], dyspnea [n=1], hot flush [n=1], non-cardiac chest pain [n=1], and tachycardia [n=1]), and 4 pts experienced DP-related AEs occurring between engraftment and 2 y post-infusion (thrombocytopenia [n=3], erythroid dysplasia [n=1], extremity pain[n=1], leukopenia [n=1], and neutropenia [n=1]). No DP-related AEs occurred >2 y post-infusion. qzskkpitvm (hhnlnrozjz ) View more | - | 01 Mar 2022 | ||
Phase 3 | 37 | Beti-cel gene therapy | dffzjklimb(fltyfwxles) = lbfajkscuu olttsbthet (nyernpbbyq ) | Positive | 01 Mar 2022 | ||
Phase 3 | 23 | sgypqhsked(qlkjuddiln) = sfwxmnxfec lamcmodlis (gffpkwhesg ) View more | Positive | 11 Dec 2021 |