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

This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.

开始日期2020-10-21

申办/合作机构

bluebird bio, Inc.

NCT04293185 / Recruiting临床3期

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

This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).

开始日期2020-02-14

申办/合作机构

bluebird bio, Inc.

NCT02140554 / Completed临床1/2期

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

This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD. The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using lovo-cel.

开始日期2015-02-02

申办/合作机构

bluebird bio, Inc.

100 项与 Lovotibeglogene autotemcel 相关的临床结果

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100 项与 Lovotibeglogene autotemcel 相关的专利（医药）

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项与 Lovotibeglogene autotemcel 相关的文献（医药）

2024-10-01·JOURNAL OF PHARMACY TECHNOLOGY

Gene Therapies for Sickle Cell Disease

Review

作者: Singh, Divita ; Weaver, Salome Bwayo ; Wilson, Kierra M.

Background: Sickle cell disease (SCD) is a prevalent autosomal recessive hemoglobinopathy affecting millions worldwide, particularly individuals of African ancestry. Sickle cell disease is a lifelong condition associated with a negative impact on quality of life and mortality, causing complications such as painful vaso-occlusive episodes, acute chest syndrome, stroke, long-term anemia, and end-organ damage. Currently, there are 4 U.S. Food and Drug Administration (FDA)-approved drugs, including hydroxyurea, l-glutamine, voxelotor, and crizanlizumab, which work to alleviate symptoms and prevent complications associated with SCD, albeit without addressing the underlying cause of SCD. Allogeneic hematopoietic stem cell transplant (HSCT) has shown promise as a curative approach to SCD but is limited by donor availability and associated complications. This paper aims to review the efficacy and safety of exagamglogene autotemcel and lovotibeglogene autotemcel for managing patients with SCD, including their place in therapy, cost, and accessibility in clinical care. Data Sources: The authors searched PubMed and Medline from 2017 to 2024, for primary literature on both exagamglogene autotemcel and lovotibeglogene autotemcel. Results: The authors identified relevant studies and summarized the data on the two gene therapies. Conclusion: Exagamglogene autotemcel and lovotibeglogene autotemcel are two management strategies that address the underlying cause of SCD and provide curative potential for patients with SCD.

2024-06-01·PHARMACOECONOMICS

Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States

Article

作者: Shah, Nirmish ; Gallagher, Meghan E ; Herring, William L ; Andemariam, Biree ; Paramore, Clark ; Mladsi, Deirdre ; Zhang, Lixin ; Morse, K C ; Dong, Olivia M ; Leiding, Jennifer W ; Chawla, Anjulika

BACKGROUND AND OBJECTIVE:

Gene therapies for sickle cell disease (SCD) may offer meaningful benefits for patients and society. This study evaluated the cost-effectiveness of lovotibeglogene autotemcel (lovo-cel), a one-time gene therapy administered via autologous hematopoietic stem cell transplantation, compared with common care for patients in the United States (US) with SCD aged ≥ 12 years with ≥ 4 vaso-occlusive events (VOEs) in the past 24 months.

METHODS:

We developed a patient-level simulation model accounting for lovo-cel and SCD-related events, complications, and mortality over a lifetime time horizon. The pivotal phase 1/2 HGB-206 clinical trial (NCT02140554) served as the basis for lovo-cel efficacy and safety. Cost, quality-of-life, and other clinical data were sourced from HGB-206 data and the literature. Analyses were conducted from US societal and third-party payer perspectives. Uncertainty was assessed through probabilistic sensitivity analysis and extensive scenario analyses.

RESULTS:

Patients treated with lovo-cel were predicted to survive 23.84 years longer on average (standard deviation [SD], 12.80) versus common care (life expectancy, 62.24 versus 38.40 years), with associated discounted patient quality-adjusted life-year (QALY) gains of 10.20 (SD, 4.10) and direct costs avoided of $1,329,201 (SD, $1,346,446) per patient. Predicted societal benefits included discounted caregiver QALY losses avoided of 1.19 (SD, 1.38) and indirect costs avoided of $540,416 (SD, $262,353) per patient. Including lovo-cel costs ($3,282,009 [SD, $29,690] per patient) resulted in incremental cost-effectiveness ratios of $191,519 and $124,051 per QALY gained from third-party payer and societal perspectives, respectively. In scenario analyses, the predicted cost-effectiveness of lovo-cel also was sensitive to baseline age and VOE frequency and to the proportion of patients achieving and maintaining complete resolution of VOEs.

CONCLUSIONS:

Our analysis of lovo-cel gene therapy compared with common care for patients in the US with SCD with recurrent VOEs estimated meaningful improvements in survival, quality of life, and other clinical outcomes accompanied by increased overall costs for the health care system and for broader society. The predicted economic value of lovo-cel gene therapy was influenced by uncertainty in long-term clinical effects and by positive spillover effects on patient productivity and caregiver burden.

Journal of Blood Medicine

Newer Modalities and Updates in the Management of Sickle Cell Disease: A Systematic Review

Review

作者: Khullar, Kaarvi ; Patel, Zeel ; Gadam, Srikanth ; Patel, Zeel Vishnubhai ; Prajjwal, Priyadarshi ; Patel, Divyakshi ; Bethineedi, Lakshmi Deepak ; Bethineedi, Lakshmi ; Patel, Hinal ; Marsool, Mohammed Dheyaa Marsool ; Aleti, Soumya ; Patel, Divyakshi J ; Khatri, Kanishka ; Amir, Omniat

Sickle cell disease (SCD), the most common autosomal recessive genetic disorder, affects the hemoglobin (Hb) chains in human red blood cells. It is caused by mutations in the β-globin genes, leading to the production of hemoglobin S, which results in the formation of sickle-shaped red blood cells (RBCs). These abnormal cells cause hemolysis, endothelial damage, and small vessel occlusion, leading to both acute and long-term complications. According to the World Health Organization's 2008 estimates, SCD affects approximately 2.28 per 1000 individuals globally. Despite this high prevalence, therapeutic advancements have been slow. For many years, the only FDA-approved medications for managing SCD complications were hydroxyurea and deferiprone. However, recent years have seen the approval of several new therapies, including L-glutamine (2017), voxelotor and crizanlizumab (2019), as well as exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) (2023). These treatments have proven effective in managing both the acute and chronic effects of SCD, including hemolytic anemia, chronic pain, stroke, vaso-occlusive crises, and multiple organ damage syndromes. This review explores the mechanisms of action, practical considerations, and side effects of these emerging therapies, drawing from a comprehensive search of databases such as PubMed, Medline, and Cochrane.

244

项与 Lovotibeglogene autotemcel 相关的新闻（医药）

2024-11-18

·药渡

基因疗法先驱大规模裁员，CGT公司为何财源不畅、裁员不断？

来源：药渡撰文：哥哈骎编辑：维他命在2024年，细胞与基因治疗（CGT）领域面临着前所未有的挑战，许多生物科技和制药公司因资金压力和市场不确定性纷纷宣布大规模裁员。这一现象不仅反映了行业内的激烈竞争和财务困境，也突显了公司在寻求创新与可持续发展的过程中所面临的艰难抉择。尽管一些细胞基因疗法的治疗费用高达数百万美元，令外界认为这些产品为公司带来了丰厚的利润，但实际情况却并非如此。许多CGT公司在推出新疗法后，仍面临着高昂的研发成本、市场推广压力以及患者接受度等多重挑战，生存环境远未与其标价相匹配。基因疗法开发的先驱之一——Bluebird也一度被迫走上了大规模裁员的道路，为CGT领域商业化开发的困境又做了一个注解。 PART. 01 CGT疗法先驱Bluebird 宣布大规模裁员毫无疑问，Bluebird bio已成为基因治疗领域的先锋，特别是在为严重遗传疾病开发和商业化疗法方面。他们开发并商业化了多种突破性的细胞和基因疗法，包括用于输血依赖性β-地中海贫血（TDT）的betibeglogene autotemcel（Zynteglo）以及分别于2019年和2022年获得EMA和FDA批准、用于治疗脑型肾上腺脑白质营养不良症（CALD）的Skysona™ (elivaldogene autotemcel)。他们成功地将基因治疗的前景从临床研究带入现实世界，证明并扩大了基因治疗的商业模式。然而在CGT商业化的道路上，Bluebird同很多其它先进疗法开发商一样遭遇了转化率方面的巨大挑战。Bluebird bio旗下的CALD基因疗法Skysona，截至今年8月初只有4名患者开始使用。还有19名患者开始使用其输血依赖性地中海贫血的基因疗法Zynteglo，这同Bluebird最初的预计相去甚远。 9月24日，Bluebird Bio宣布将裁减25%的员工以进行重组，目的是节省资金并寻求镰状细胞病及其他疾病的治疗开发。Bluebird Bio已获得FDA对包括镰状细胞基因治疗Lyfgenia在内的治疗方法的批准，但尚未看到可观的财务回报，并仍在持续亏损。尽管Bluebird没有明确裁员人数，但该公司在6月时报告拥有375名全职员工，这意味着裁员人数将接近100人。 Bluebird没有透露受影响员工的具体职位或裁员是否全部发生在位于马萨诸塞州索美维尔的总部。Bluebird Bio首席执行官Andrew Obenshain在新闻稿中表示：“为了支持更集中优先事项而减少员工数量的决定是在对我们组织的需求和能力进行详细审查后作出的，我们对每一位为实现我们创始愿景、让基因治疗成为受到严重遗传疾病影响的患者和家庭现实而努力的员工深表感谢。” Bluebird预计重组将使运营费用降低20%，并计划在2025年第三季度开始实现每季度收支平衡，届时重组预计完成。该公司股价今年已损失超过一半。 Bluebird此前在2022年裁减了三分之一的员工，并警告投资者未来财务状况不确定。2023年Lyfgenia基因治疗获得FDA批准，但未能达到公司预期的效果，因为Vertex Pharmaceuticals /CRISPR Therapeutics针对镰状细胞病的基因疗法exagamglogene autotemcel（exa-cel）也在同一天也获得了批准，但后者同样遭遇到了商业化的问题。 Bluebird Bio的高管表示，目前已有41名患者开始使用他们的治疗方法。Bluebird在Q3已有20名患者开始治疗，这一数字较上个季度的12名和今年第一季度的9名显著增长。Bluebird预计在第四季度将有40名患者开始治疗。裁员后的Bluebird将更加专注于Lyfgenia、Zynteglo和Skysona的商业转化。 PART. 02 2024年大规模裁员的 CGT开发公司在经历了多款CGT产品获批上市之后，2024年专注于这些先进疗法开发的生物制药公司，以及生物技术公司反而陷入了大规模裁员的泥淖之中。尽管这些基因疗法动辄上百万美元一剂，然而商业化进展却举步维艰。除了Bluebird最近的大规模裁员之外，还有多家CGT开发商今年也不得不被动地选择“精兵简政”的策略来应对商业化的拖后腿。 1 BioMarin 基因疗法开发的另一位先驱BioMarin已于今年8月再次宣布大规模裁员，将225名岗位裁撤，占其全球员工总数的7%。这是BioMarin继今年5月裁减170名员工之后的又一次大规模裁员。 BioMarin在基因疗法开发领域占据着重要地位，是该行业的标杆型企业之一。BioMarin推出的Roctavian是全球首个获批用于治疗血友病A的基因疗法，标志着基因治疗在罕见病领域的重要进展。 BioMarin将最新一轮裁员归因于“组织重组”以及公司更专注于A型血友病基因疗法Roctavian的战略，还有管线项目BMN 293的提前终止，BMN 293是一种针对遗传性肥厚性心肌病的基因疗法。Roctavian于2023年6月获得批准，但首位接受该商业产品的患者直到2023年12月才在加州遗传出血性疾病中心（CIBD）接受治疗。Roctavian 2023年销售额远低于BioMarin最初预测的1亿至2亿美元，只为他们带来了350万美元的收入。 2 2seventy bio 由Bluebird拆分产生的2seventy bio也在今年进行了多次重组，他们的多发性骨髓瘤CAR-T细胞疗法Abecma（idecabtagene vicleucel）2021年获得FDA批准。今年年初，2seventy bio再次拆分，将临床前和临床细胞疗法项目转移给Regeneron，导致160名员工转投Regeneron，剩余的员工将专注于Abecma的商业开发，并裁员55人，将员工人数缩减至65名。通过此次重组和裁员，2seventy预计2024年每年可节省约1.5亿美元，2025年可节省2亿美元，从而将现金流延长至2027年以后。这笔交易标志着2seventy的新战略路径，他们在2021年从Bluebird Bio剥离出来，专注于肿瘤产品后一直举步维艰。在剥离了管线资产后，2seventy bio将专注于Abecma的适应症和标签扩展。今年6与月份，2seventy bio又一次“瘦身”，将其A型血友病项目以及自身免疫性疾病的体内基因编辑技术megaTAL以4000万美元的价格卖给诺和诺德。作为剥离的一部分，目前参与这些项目的2seventy员工也将前往诺和诺德从事科学研究。 3 Tome Biosciences 2023年底刚刚成立的基因编辑初创公司Tome Biosciences今年8月也宣布将于11 月裁员131人。Tome Biosciences是带着2.13亿美元融资入场的，但却一次性地几乎将全部员工裁撤。有消息称，不利的市场力量迫使Tome逐步关闭其业务。这家初创公司正在以降低的产能运营，保持核心专业知识。 Tome于2023年12月成立，获得了2.13亿美元的A轮和B轮融资，推进其可编程基因组整合（PGI）平台，该平台使用专有酶插入或编写基因。然而，投资者在基因编辑领域却有些意兴阑珊，导致Tome除了裁员之外几乎别无选择。 4 Intergalactic Therapeutics 另一家专注于基因疗法的初创公司Intergalactic Therapeutics也在8月宣告裁员，不同的是，他们裁撤了所有员工并申请破产，破产的原因是Intergalactic Therapeutics的资金链断裂。 Intergalactic的开发方向是不通过病毒作为递送系统的基因疗法。之所以难以为继，还是因为大环境下生物技术投资者几乎没有表现出押注早期前景资产的意愿，这使得基因治疗尤其容易受到影响。 5 Homology Medicines Intergalactic宣布破产的前一周，基因编辑生物技术公司Homology Medicines表示将裁员87%，停止研究并寻找买家。 PART. 03 CGT开发公司缘何捧着金饭碗裁员？拥有上市天价疗法的CGT公司在2024年屡屡陷入大规模裁员的泥淖，可能存在以下几个主要原因： 1 高昂的研发成本与失败率研发投入：CGT的研发过程复杂，通常涉及长时间的临床试验和大量资金投入。公司需要为实验室研究、临床试验、患者招募等各个环节付出高额费用。高失败率：由于CGT的复杂性，临床试验的失败率较高。一旦治疗方案未能达到预期效果，投入的资金就会化为泡影，增加公司财务压力。 2 监管审批的复杂性严格的监管要求：CGT产品在上市前需经过严格的监管审查，这一过程可能延迟产品上市，影响公司的现金流和盈利能力。审批延迟的影响：审批过程中可能出现的延迟或要求追加试验，都会导致公司不得不重新评估其业务策略和财务状况，从而引发裁员。 3 市场需求与定价压力需求波动：尽管CGT疗法在某些情况下具有革命性，但市场需求并不总是稳定。患者对新疗法的接受度受多种因素影响，包括疗法的有效性、治疗的复杂性和风险等。保险报销限制：许多CGT疗法价格昂贵，尽管临床效果显著，但保险公司可能对这些治疗的报销存在限制，导致患者无法负担，进一步影响销售预期。 4 技术与疗法的替代性竞争加剧：随着新疗法和技术的不断涌现，CGT公司面临越来越激烈的竞争。这迫使公司投入更多资金进行创新和研发，以保持市场竞争力。市场动态变化：新兴技术（如基因编辑、免疫疗法等）的发展可能会改变治疗市场格局，迫使现有CGT公司重新评估其产品线和市场定位。 5 投资者压力高期望的投资回报：在CGT领域，投资者通常期待高回报，因此公司必须在财务表现和市场表现上快速见效。若未能满足这些期望，可能导致股价下跌和投资者信心下降。资金链紧张：由于资金压力，许多CGT公司在财务上可能处于困境，裁员成为降低开支、改善现金流的措施之一。 6 战略重组业务方向调整：面对市场挑战和内部资源配置问题，公司可能决定重新审视其产品组合和业务战略，聚焦于更有前景的项目。资源优化：裁员可以帮助公司减少冗余，优化人力资源配置，以提高整体运营效率。尽管CGT公司拥有天价疗法，但在复杂的市场环境和经济压力下，它们仍然面临巨大挑战。这些挑战不仅影响到公司的财务状况，还可能导致裁员，以应对持续的市场压力和确保公司的长期可持续发展。 PART. 04 未来展望在2024年，CGT领域的生物科技和制药公司面临显著的裁员潮，这一现象不仅反映出行业的深刻变革与挑战，也暴露了在高研发成本和市场不确定性下，企业在创新与财务健康之间的艰难抉择。然而，尽管当前环境严峻，CGT在罕见病和遗传性疾病治疗中的不可替代性，预示着这一领域仍蕴藏着光明的前景。未来，随着保险覆盖的不断扩大和商业化进程的顺利推进，CGT的市场潜力将进一步释放。政策的支持和市场的接受度将为企业的创新提供更为坚实的基础，使得CGT疗法能够更广泛地惠及患者。这不仅意味着对罕见病患者的治疗选择将大幅增加，还将为公司创造可持续的商业模式，推动更多创新治疗方案的开发。展望未来，CGT开发的前景乐观。随着技术的不断进步和对疗效的深入验证，行业将迎来一波新的投资热潮。企业在这一过程中需要紧密关注市场需求，灵活应对政策变化，通过与保险公司和监管机构的合作，推动疗法的准入与覆盖，确保患者能够获得所需的治疗。总之，尽管当前的裁员潮带来了挑战，但在保险覆盖和商业化推进顺利的背景下，CGT领域的发展仍将展现出无限的可能性。通过持续的创新与合作，CGT将不仅为罕见病和遗传性疾病患者带来希望，也将为整个医疗行业开辟出更加光明的未来。参考文献： 1. Armstrong, A. UPDATE: 2seventy splits once more, sending cell therapy pipeline to Regeneron in 'dramatic' change. Fierce Biotech. 30. 01. 2024. 2. Masson, G. 2seventy bio divests again, this time sending hemophilia and gene editing science to Novo Nordisk. Fierce Biotech. 26. 06. 2024. 3. Liu, A. BioMarin cuts deeper with 225 layoffs under CEO's new corporate strategy. Fierce Pharma. 29. 08. 2024. 4. Manalac, T. Tome to Lay Off Almost All of Staff After Scaling Back Operations. Biospace. 26. 08. 2024. 专栏作者哥哈骎南开大学本科、硕士，德国比勒菲尔德大学博士。多肽化学、多肽API GMP生产专家、欧洲制药公司首席科学家；拥有Lean Six Sigma黑带认证；著有专著《Side Reactions in Peptide Synthesis》。 *声明：本文仅是介绍医药疾病领域研究进展或简述研究概况或分享医药相关讯息，并非也不会进行治疗或诊断方案推荐，也不对相关投资构成任何建议。内容如有疏漏，欢迎沟通指出！大卖203亿美元的「司美格鲁肽」与捷报频传的「诺和诺德」百济神州三季报：营收71.39亿，泽布替尼大卖49亿元！ Cell：新型靶向蛋白降解技术来袭！解读TRIM21介导的多聚蛋白降解技术

基因疗法细胞疗法临床研究财报

2024-11-15

·BioPharmaDive

Halozyme bids for Evotec; BeiGene gets a new name

Today, a brief rundown of news involving Evotec, Bluebird bio and the company formerly known as BeiGene, as well as updates from Leerink Partners and Abeona Therapeutics that you may have missed.Halozyme Therapeutics has made an unsolicited bid to buydrug discovery specialist Evotec for 11 euros per share in cash, an offer worth some 2 billion euros in total. In a press release, Halozyme said the combined company could offer increased scale and become a highly attractive strategic partner to the drug industry. Evotec, in its own statement, said it would carefully analyze this expression of interest. Ned PagliaruloBluebird bio has recorded its first revenue from sales of Lyfgenia, the companys gene therapy for sickle cell disease that won U.S. approval late last year. The process to prepare and receive Lyfgenia is lengthy, so only recently did the first patient actually get an infusion, at which point Bluebird books revenue for the $3.1 million therapy.Another 16 patients have started the process to receive treatment. The company is counting on climbing demand for Lyfgenia and its two other gene therapies, Zynteglo and Skysona, as it works to break even. Currently, it has enough cash to fund operations into the first quarter. Ned PagliaruloCancer drugmaker BeiGene will now operate under the name BeOne Medicines, a change that it says reflects its commitment to oncology research. The name also distances the company from its roots in China, a connection that may be less helpful as lawmakers in the U.S. increasingly view such ties as a national security risk. BeOne, which trades on the Hong Kong and Shanghai stock exchanges as well as the Nasdaq, is headquartered in California and in Switzerland. Ned PagliaruloAdaptimmune will lay off a third of its workforce in a bid to save about $300 million and break even financially in 2027, the company said Wednesday. Adaptimmune in August won U.S. clearance for its first product, a cell therapy called Tecelra for a type of soft tissue cancer. Going forward, the company will focus resources on that launch and another program in development for similar cancers, while partnering off two unrelated projects in preclinical testing. Its also shutting down a Phase 2 study in ovarian tumors.Ben FidlerLeerink Partnershas named two senior managing directors, Grant Curry and Jason Truman, to join its biopharma M&A team. Truman previously worked in a similar role at Guggenheim Securities, while Curry was a managing director at Gordon Dyal & Co. Leerink, which reemerged via a management buy-out after the collapse of SVB Financial Group,hopes to grow its M&A business. Outside of the big banks, Centerview Partners, Lazard and PJT Partners currently win the most deal business from drugmakers, according to BioPharma Dive data. Ned PagliaruloBy the end of next April, Abeona Therapeutics should know whether the Food and Drug Administration will approve its gene therapy pz-cel for a rare wound healing condition known as recessive dystrophic epidermolysis bullosa. The agency rejected pz-cel this past April, asking Abeona for more information on how its produced.The company then resubmitted its application with new information and, on Thursday, secured a target decision date of April 29, 2025 from the agency. Ned Pagliarulo '

基因疗法并购高管变更上市批准临床2期

2024-11-14

·Endpoints

As bluebird projects more commercial demand, the biotech continues to seek cash

Bluebird reported that 17 people with sickle cell disease have started the process of receiving its gene therapy Lyfgenia. Bluebird said in its third-quarter earnings Thursday that it recognized revenue from Lyfgenia, though it did not specify how much. In total, the gene therapy biotech reported $10.6 million in revenue, but it said it expects at least double that — $25 million — next quarter. The company dosed its first sickle cell patient with Lyfgenia, the New York Times reported in September. Vertex Pharmaceuticals, which markets the gene-edited sickle cell and thalassemia therapy Casgevy, reported that it dosed its first patient in the third quarter and recorded $2 million in revenue. That patient was a 13-year-old thalassemia patient from Saudi Arabia , Vertex confirmed to Endpoints News via email. On Thursday, bluebird also said that 35 people have started the process of receiving Zynteglo, a similar therapy for thalassemia. It reported five patient starts for Skysona, a gene therapy for cerebral adrenoleukodystrophy. The biotech added it has 17 additional starts across its gene therapy portfolio scheduled for the rest of the year, along with 30 scheduled in 2025. “Patient starts more than doubled from our second to third quarter update, providing clear evidence that our commercial launches continued to accelerate,” bluebird CEO Andrew Obenshain said in a press statement. “We remain focused on securing additional cash resources to extend our runway, which we believe would enable us to achieve this vision and reach cash flow break-even in the second half of 2025.” Bluebird announced in September that it was laying off 25% of its workforce and cutting expenses by 20%. Earlier in the year, the biotech had to rework the terms of a loan with Hercules capital, and it currently has cash to fund operations into the first quarter of 2025. When asked in an analyst call about how the company was going to get more cash, CFO James Sterling said the “strategy to extend the runway includes renegotiating key contracts and other cost initiatives, and our partnership with Hercules is key in helping move those forward.” Bluebird is also at risk of being delisted from Nasdaq and is trying to implement a reverse stock split to avoid that. It’s convening a stockholder meeting on Dec. 4 to vote on the reverse stock split.

基因疗法

100 项与 Lovotibeglogene autotemcel 相关的药物交易

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适应症	国家/地区	公司	日期
镰状细胞血症	美国	bluebird bio, Inc.	2023-12-08

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适应症	最高研发状态	国家/地区	公司	日期
β地中海贫血	临床2期	法国	bluebird bio, Inc.	2013-06-07
输血依赖性β地中海贫血	临床2期	法国	bluebird bio, Inc.	2013-06-07

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02140554 (HGB-210, Tandem Meetings ASTCT and CIBMTR2024) 人工标引	临床1/2期	镰状细胞血症	47	Lovotibeglogene Autotemcel (Lovo-cel) (Lovo-cel)	(積餘夢製鹹繭襯築繭鬱) = 願糧蓋糧窪窪醖鑰積淵襯繭遞築壓繭襯糧築醖 (鏇醖築鹽繭襯餘窪膚淵 ) 更多	积极	2024-02-01
NCT02140554 (HGB-206, Tandem Meetings ASTCT and CIBMTR2023)	临床1/2期	镰状细胞血症 HbA T87Q \| neutropenia \| trisomy 8 ... 更多	35	Lovo-cel infusion	構廠範餘衊選夢廠積築(網廠願願艱鹽襯餘鏇餘) = 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. 顧窪遞壓鹽網獵壓齋糧 (觸範鹽築壓淵積鑰顧廠 ) 更多	积极	2023-02-01
NCT02151526 (CTgov)	临床1/2期	β地中海贫血 \| 输血依赖性β地中海贫血 \| 镰状细胞血症	7	LentiGlobin BB305 (LentiGlobin BB305 Drug Product for SCD)	鑰壓網憲製醖蓋衊觸觸(築夢網鹹壓艱獵觸積齋) = 艱醖淵廠廠築鬱構鑰壓鹹窪獵襯襯淵觸窪壓鑰 (製鹹艱壓選範製繭積遞, 糧憲簾製齋鹽壓襯糧衊 ~ 壓積範艱構選製鑰築築) 更多	-	2020-03-10
NCT02151526 (CTgov)	临床1/2期	β地中海贫血 \| 输血依赖性β地中海贫血 \| 镰状细胞血症	7	LentiGlobin BB305 (LentiGlobin BB305 Drug Product for TDT)	鑰壓網憲製醖蓋衊觸觸(築夢網鹹壓艱獵觸積齋) = 廠糧繭觸積遞鏇蓋鏇網鹹窪獵襯襯淵觸窪壓鑰 (製鹹艱壓選範製繭積遞, 簾淵鹹網醖齋鑰糧蓋鹽 ~ 艱鑰膚鏇蓋齋願窪簾夢) 更多	-	2020-03-10