Last update 02 Oct 2025

Betibeglogene autotemcel

Overview

Basic Info

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)
Originator Organization
Active Organization
Inactive Organization-
License Organization-
Drug Highest PhaseApproved
First Approval Date
European Union (29 May 2019),
RegulationPriority Review (United States), Breakthrough 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)
Login to view timeline

External Link

KEGGWikiATCDrug Bank
-Betibeglogene autotemcel-

R&D Status

Approved
10 top approved records.
Login
to view more data
IndicationCountry/LocationOrganizationDate
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
-
Developing
10 top R&D records.
Login
to view more data
IndicationHighest PhaseCountry/LocationOrganizationDate
Anemia, Sickle CellNDA/BLA
United States
25 Apr 2023
Transfusion-dependent ThalassemiaPhase 3
United States
08 Aug 2016
Transfusion-dependent ThalassemiaPhase 3
France
08 Aug 2016
Transfusion-dependent ThalassemiaPhase 3
Germany
08 Aug 2016
Transfusion-dependent ThalassemiaPhase 3
Italy
08 Aug 2016
Transfusion-dependent ThalassemiaPhase 3
Thailand
08 Aug 2016
Transfusion-dependent ThalassemiaPhase 3
United Kingdom
08 Aug 2016
Login to view more data

Clinical Result

Indication
Phase
Evaluation
View All Results
Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
63
ufddkegjmk(xlvlpvvonp) = pmhryqsyti kabqrdjcpq (cmwylelywl, 0.1 - 4.9)
Positive
07 Dec 2024
ufddkegjmk(xlvlpvvonp) = doxtgkpdwg kabqrdjcpq (cmwylelywl, 0.1 - 4.8)
Phase 3
18
bvchmklqgo(giyjtpjeix) = esroorvhlq jtsmfsdnqs (bdfopxswci )
Positive
08 Nov 2024
Phase 3
63
Beti-cel gene therapy
uzuyppluru(hmfzisfcso) = ronvpssfpa jfvcampbcb (crctcgxrsj )
Positive
01 Feb 2024
Not Applicable
-
hssvxndnkp(bepoqsldya) = Most patients (51/66 [77.3%]) had ≥1 adverse event (AE) attributed to mobilization and apheresis. One patient experienced serious AEs (thrombocytopenia and hypokalemia). gbdfmzivzq (crcbdupgtg )
-
01 Feb 2024
Beti-cel gene therapy
Not Applicable
51
swnizmoonn(ycbqxmefkk) = kshmvdqthh adpxlteyxf (sotgglimbf, -22.3 to 10.5)
Positive
10 Dec 2023
Not Applicable
47
utgzayudaz(prejcxtskh) = rlokzdwwji nbqrfbflsc (zasswqzdev )
Positive
09 Dec 2023
FDA
ManualManual
Phase 1/2
45
ektedhcqho(rkcuftrqsu) = vcecaakkux cebzuxlakx (jdiiaqvckg, 71 - 97)
Positive
08 Dec 2023
Not Applicable
63
betibeglogene autotemcel (beti-cel) Gene Therapy
plapfvdywu(kdwjtrpwam) = rhdjeiudui hnjleyaruk (kgztvepekp, 19 - 191)
Positive
01 Feb 2023
Phase 1/2
35
LentiGlobin for SCD (bb1111) gene therapy
oigurzucfn(rdwwbefqyw) = yvetjilxcy yzojedjegs (bgkjfeoquy )
Positive
01 Mar 2022
Not Applicable
2
Beti-cel gene therapy
tnstgsgqqg(plqvuaxwfr) = Post-infusion AEs for pt 1 included febrile neutropenia, elevated C-reactive protein, pruritus, gingivitis, mild mucositis, and vertigo. nlofhhkxvw (uxqmctjmpj )
Positive
01 Mar 2022
Login to view more data

Translational Medicine

Boost your research with our translational medicine data.
Boost your research with our translational medicine data.

Deal

Boost your decision using our deal data.
Boost your decision using our deal data.

Core Patent

Boost your research with our Core Patent data.
Boost your research with our Core Patent data.

Clinical Trial

Identify the latest clinical trials across global registries.
Identify the latest clinical trials across global registries.

Approval

Accelerate your research with the latest regulatory approval information.
Accelerate your research with the latest regulatory approval information.

Regulation

Understand key drug designations in just a few clicks with Synapse.
Understand key drug designations in just a few clicks with Synapse.
AI Agents Built for Biopharma Breakthroughs
Accelerate discovery. Empower decisions. Transform outcomes.
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.
Bio
Bio Sequences Search & Analysis
Sign up for free
Chemical
Chemical Structures Search & Analysis
Sign up for free