Drugs / betibeglogene autotemcel
betibeglogene autotemcel
also known as
LentiGlobin BB305 · beti-cel · Autologous cd34+ cell enriched population that contains hematopoietic stem cells transduced with lentiglobin bb305 lentiviral vector encoding the beta-a-t87q-globin gene · BB-305 · Betibeglogen autotemcel · LentiGlobin BB305 Drug Product · LentiGlobin BB305 vector · Zynteglo
targets HBB
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed beta-thalassemia and related diseases 1 milestone | |||||
| Filed | US (FDA) | ZYNTEGLO | beta-thalassemia and related diseases | — | “Bluebird submitted its Biologics License Application to the U.S. Food and Drug Administration (FDA) for betibeglogene autotemcel (beti-cel)” sanroccotherapeutics.com ↗ |
| Withdrawn Indication not stated 2 milestones | |||||
| Withdrawn | EU (EMA) | Zynteglo | — | 2022-03-24 | europa.eu ↗ |
| Approved · conditional | EU (EMA) | Zynteglo | — | 2019-05-29 | europa.eu ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/22 trials | ||||||
| Phase 1/2 | NCT01745120 | Aug 2013 → Feb 2018 | beta-thalassemia major | Genetix Biotherapeutics Inc. | Completed | No outcome recorded |
| Phase 1/2 | NCT02151526 | Jun 2013 → Feb 2019 | beta-thalassemia major, sickle cell disease | Genetix Biotherapeutics Inc. | Completed | No outcome recorded |
| Phase 32 trials | ||||||
| Phase 3 | NCT03207009 | Jun 2017 → Nov 2022 | beta thalassemia | Genetix Biotherapeutics Inc. | Completed | No outcome recorded |
| Phase 3 | NCT02906202 | Aug 2016 → Mar 2022 | beta-thalassemia major | Genetix Biotherapeutics Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2022-08-19 | San Rocco Therapeutics | Regulatory FDA Approves First Gene Therapy for Beta-Thalassemia Patients Who Require Regular Red Blood Cell Transfusions: $2.8m Cost sanroccotherapeutics.com ↗ |
| 2021-02-15 | San Rocco Therapeutics | Regulatory “UK’s NICE Rejects bluebird bio: Questions Value Price Tag & Nominal Clinical Evidence While Sponsor Shocked” sanroccotherapeutics.com ↗
A gene therapy for patients with transfusion-dependent beta thalassemia (TDT) didn’t fare well after a thorough review by the United Kingdom’s National Institute for Health and Care Excellence (NICE) turned down biotech firm bluebird bio’s Zynteglo (betibeglogene autotemcel; beti-cel). |
All press releases naming this drug 2 releases
| Date | Issuer | Release |
|---|---|---|
| 2022-08-19 | San Rocco Therapeutics | FDA Approves First Gene Therapy for Beta-Thalassemia Patients Who Require Regular Red Blood Cell Transfusions: $2.8m Cost sanroccotherapeutics.com ↗ |
| 2021-02-15 | San Rocco Therapeutics | “UK’s NICE Rejects bluebird bio: Questions Value Price Tag & Nominal Clinical Evidence While Sponsor Shocked” sanroccotherapeutics.com ↗ |
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 8 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | betibeglogene autotemcel | “Betibeglogene autotemcel (beti-cel) gene therapy uses autologous haematopoietic stem and progenitor cells (HSPCs) transduced with BB305 lentiviral vector to enable transfusion...” PMID 39527960 ↗ Nov 2024 |
| Known as | Autologous cd34+ cell enriched population that contains hematopoietic stem cells transduced with lentiglobin bb305 lentiviral vector encoding the beta-a-t87q-globin gene | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594311 ↗ |
| Known as | BB-305 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594311 ↗ |
| Known as | beti-cel | “Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia contains autologous CD34+ hematopoietic stem cells and progenitor cells transduced with...” PMID 34891223 ↗ Dec 20211“Betibeglogene autotemcel (beti-cel) gene therapy uses autologous haematopoietic stem and progenitor cells (HSPCs) transduced with BB305 lentiviral vector to enable transfusion...” PMID 39527960 ↗ Nov 2024 |
| Known as | Betibeglogen autotemcel | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594311 ↗ |
| Known as | LentiGlobin BB305 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01745120 ↗ |
| Known as | LentiGlobin BB305 Drug Product | “LentiGlobin BB305 Drug Product (autologous CD34+ cell-enriched population that contains cells transduced with LentiGlobin BB305 lentiviral vector encoding human βA-T87Q-globin)” NCT03207009 ↗ |
| Known as | LentiGlobin BB305 vector | “we obtained mobilized autologous CD34+ cells from 22 patients (12 to 35 years of age) with transfusion-dependent β-thalassemia and transduced the cells ex vivo with LentiGlobin...” PMID 29669226 ↗ Apr 2018 |
| Known as | Zynteglo | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594311 ↗ |
| Action | Restore | “lentiviral transfer of a marked β-globin (βA-T87Q) gene could substitute for long-term red-cell transfusions” PMID 29669226 ↗ Apr 2018 |
| Route | Intravenous | “LentiGlobin BB305 Drug Product was administered by intravenous (IV) infusion.” NCT02151526 ↗ |
| Target | HBB | “Hemoglobin beta chain exogenous gene” CHEMBL4594311 ↗ |