预约演示
更新于：2026-09-07
Lovotibeglogene autotemcel
更新于：2026-09-07
概要
基本信息
非在研机构- |
最高研发阶段批准上市 |
首次获批日期 美国 (2023-12-08), |
最高研发阶段(中国)- |
特殊审评- |
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结构/序列
Sequence Code 559582938
关联
4
项与 Lovotibeglogene autotemcel 相关的临床试验NCT04628585
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector
NCT04293185
A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the BB305 Lentiviral Vector in Subjects With Sickle Cell Disease
NCT02140554
A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease
100 项与 Lovotibeglogene autotemcel 相关的临床结果
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100 项与 Lovotibeglogene autotemcel 相关的转化医学
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100 项与 Lovotibeglogene autotemcel 相关的专利（医药）
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18
项与 Lovotibeglogene autotemcel 相关的文献（医药）2026-03-01Molecular Therapy-Nucleic Acids
CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges
Review
作者: Bhide, Pradeep G ; Duke, Leanne ; Kantor, Boris
The gene therapy landscape has evolved substantially in recent years, beginning with the approval of the first adeno-associated virus-based gene therapy, Luxterna, in 2017. Since then, the US FDA has approved nearly 30 new viral gene therapy programs, with notable examples including Zolgensma, Spinraza, Hemgenix, Zynteglo, Lyfgenia, Kymriah, Skysona, and Tecelra. Remarkably, all these products rely on delivery via adeno-associated vectors (AAVs) and lentiviral vectors (LVs). Improvements in viral-mediated gene transfer efficiency and clinical-scale manufacturing, together with immense commercial interest, have greatly propelled the clinical adoption of gene therapy products. In recent years, clustered regularly interspaced short palindromic repeats (CRISPR) and its related Cas proteins (CRISPR-Cas) have made significant advances in gene therapy, offering next-generation approaches for curative gene editing to treat genetic diseases and disorders. In this review, we examine the range of these therapeutics and their viral carriers, focusing primarily on LVs and AAVs. We provide a snapshot of the current status of the field and highlight some of the current challenges in the clinical application of gene therapy, with particular emphasis on viral CRISPR-Cas-based technologies and their future potential.
2026-01-01Cardiovascular & hematological disorders drug targets
Updated Review of Current Therapeutic Approaches for the Management of Sickle Cell Disease
Review
作者: Bhardwaj, Harish ; Jangde, Rajendra Kumar ; Puri, Devwati ; Verma, Bhupendra
Sickle cell disease is a severe genetic blood disorder marked by the production of abnormal
hemoglobin (HbS), leading to sickle-shaped red blood cells that obstruct blood flow and
cause various problems, such as the increased risk of infection, persistent anemia, acute pain episodes,
and organ damage. Roughly 100,000 Americans suffer from SCD, with approximately
40,000 of them being children. Black people have the highest frequency of the disease. There are
six Food and Drug Administration (FDA)-approved drugs, hydroxyurea, L-glutamine, crizanlizumab-
TMCA, voxelotor, Casgevy, and Lyfgenia, that are used for the prophylaxis and treatment
of serious complications of sickle cell disease. Current treatment approaches focus on symptom
management, including pain control, hydroxyurea to reduce pain crises, and transfusions for severe
anemia. Based on the clinical trial results, L-glutamine and crizanlizumab-TMCA prevent
cell damage and hemoglobin sickling by reducing the sickle cell crisis episodes. At the same time,
voxelotor improves hemoglobin oxygen-binding capacity in patients with SCD. Novel therapies,
such as gene therapy and clustered regularly interspaced short palindromic repeats/CRISPR-associated
protein 9 (CRISPR-Cas9) technology, aim to correct the genetic defect. At the same time,
stem cell and bone marrow transplants offer potential cures but are limited by the availability of
donors and side effects. Ongoing research seeks to enhance treatment options and develop potential
cures for SCD. This review attempts to present a comprehensive overview of the current therapeutic
approaches and newly developed innovative medicines to combat and potentially eradicate
SCD with an emphasis on their mechanisms, efficacy, and clinical implications.
2025-09-01STEM CELLS AND DEVELOPMENT
Advances in Sickle Cell Disease Treatment: A Comparative Review of Hematopoietic Stem Cell Transplantation and Gene Therapy (Casgevy and Lyfgenia)
Review
作者: Kawari, Mohammed ; Abdelazim, Omer Tarig Fathi ; Sharafeldin, Abu-Baker Khalid ; Hasan, Zahra Abdulla Isa Yusuf ; Toorani, Zainab Abdulmajeed
Sickle cell disease (SCD), affecting approximately 2.1% of Bahrain’s population, is a prevalent inherited disorder that necessitates effective treatments and long-term management. This review highlights two innovative gene therapies (Casgevy and Lyfgenia) and compares their efficacy and safety with hematopoietic stem cell transplantation (HSCT)—the only curative option currently available for SCD. While HSCT offers a 90% success rate with suitable donors, its limitations include donor scarcity and toxicity. Gene therapies like Casgevy and Lyfgenia show promising efficacy in reducing SCD complications while bypassing such limitations. In the Kingdom of Bahrain, the Bahrain Oncology Center approved Casgevy in December 2023 and completed its first patient treatment in mid-February 2025, making Bahrain an early adopter. This milestone marks a crucial moment in the history of both SCD and gene therapies and thus warrants exploring the considerations revolving around their implementation. Although these therapies seem to offer hope for patients ineligible for HSCT, their long-term outcomes remain unassessed—further studies with extended follow-up are needed to confirm their safety and durability.
100 项与 Lovotibeglogene autotemcel 相关的药物交易
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外链
| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| D12765 | - | - | - |
研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 镰状细胞血症 | 美国 | 2023-12-08 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 急性胸部综合征 | 临床2期 | 法国 | 2013-06-07 | |
| β地中海贫血 | 临床2期 | 法国 | 2013-06-07 | |
| 输血依赖性β地中海贫血 | 临床2期 | 法国 | 2013-06-07 |
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临床结果
临床结果
适应症
分期
评价
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N/A | 55 | Lovotibeglogene Autotemcel (Lovo-cel) (Lovo-cel) | 膚積網願顧鬱壓蓋醖鹹(積醖窪願醖鏇製廠願鏇) = 繭遞鹹簾遞築夢憲積廠 鹹範製鏇廠積廠壓醖製 (醖醖夢夢蓋鹽淵積遞憲 ) | 积极 | 2024-12-08 | ||
临床1/2期 | 47 | Lovotibeglogene Autotemcel (Lovo-cel) (Lovo-cel) | 製壓醖鹽蓋網繭獵淵鬱(鏇齋鹹顧簾觸憲網顧鹹) = 觸鏇鏇製鏇顧醖鑰廠鏇 鹹鬱夢衊壓蓋淵醖鹹艱 (築壓膚糧獵顧憲網襯鹹 ) 更多 | 积极 | 2024-02-01 | ||
临床1/2期 | 35 | Lovo-cel infusion | 觸簾夢鏇觸鑰廠網鏇窪(製憲積製選膚廠獵網淵) = Two patients with findings that initially raised suspicion of myelodysplastic syndrome (MDS) underwent further assessments in March 2022. Presentation included neutropenia in one patient, and persistent anemia despite adequate HbA T87Q production, bone marrow morphology showing erythroid-specific dysplasia consistent with dyserythropoiesis seen in other hemoglobinopathies, and low levels of trisomy 8 (≤7.7%) only observed by fluorescence in situ hybridization analysis in both patients. There were no clinical symptoms suggestive of hematologic malignancies (no driver mutations or clonal process [vector related or otherwise] as shown by integration site analysis and next-generation sequencing, and no chromosomal abnormalities on karyotyping). One patient maintained total Hb of 9.8 g/dL at Month 18 without transfusion; the other required chronic transfusions to maintain 10.3 g/dL at Month 18 with otherwise normal complete blood counts. These were the only patients with two α-globin deletions (− α 3.7/− α 3.7). We propose that α-globin trait may contribute to anemia and dyserythropoiesis after lovo-cel infusion. 鏇顧鑰簾獵窪憲選淵簾 (遞蓋顧鬱壓襯鹹壓餘餘 ) 更多 | 积极 | 2023-02-01 | ||
临床1/2期 | 35 | LentiGlobin | 鹽範觸餘糧簾壓顧構餘(醖夢網遞積繭窪糧齋網) = 襯鹽範齋鬱壓築繭蓋鑰 鏇夢鑰網獵蓋醖膚選構 (淵鏇網鹽壓衊齋鹹窪積 ) 更多 | 积极 | 2021-12-12 | ||
临床1/2期 | 7 | (LentiGlobin BB305 Drug Product for SCD) | 顧淵觸窪鏇獵壓夢壓繭 = 膚鏇網廠製網衊願淵淵 壓糧淵繭齋選鏇築艱夢 (壓膚廠廠獵壓齋鏇襯憲, 鏇遞遞壓築製鹹遞簾餘 ~ 餘廠遞鏇艱廠餘衊襯鑰) 更多 | - | 2020-03-10 | ||
(LentiGlobin BB305 Drug Product for TDT) | 顧淵觸窪鏇獵壓夢壓繭 = 積膚壓夢憲齋鬱鑰衊繭 壓糧淵繭齋選鏇築艱夢 (壓膚廠廠獵壓齋鏇襯憲, 鹽願鏇繭醖鹹壓鑰製築 ~ 廠廠構顧鏇糧網餘範簾) 更多 |
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