A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)

The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in participants with CALD. A participant's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the participant following myeloablative conditioning. Enrollment and treatment in Study ALD-104 have been completed and further enrollment in this study is not expected, although participants follow-up remains ongoing in the long-term follow-up Study LTF-304 (NCT02698579).

开始日期2019-01-24

申办/合作机构

bluebird bio, Inc.

NCT01896102 / Completed临床2/3期

A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells were collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells were transplanted back into the participant following myeloablative conditioning. Participants in this study will be continuously followed in study LTF-304.

开始日期2013-08-21

申办/合作机构

bluebird bio, Inc.

100 项与 Elivaldogene Autotemcel (Bluebird Bio) 相关的临床结果

登录后查看更多信息

100 项与 Elivaldogene Autotemcel (Bluebird Bio) 相关的转化医学

登录后查看更多信息

100 项与 Elivaldogene Autotemcel (Bluebird Bio) 相关的专利（医药）

登录后查看更多信息

项与 Elivaldogene Autotemcel (Bluebird Bio) 相关的文献（医药）

2024-10-10·New England Journal of Medicine

Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy

Article

作者: Dietz, Andrew C ; Thakar, Himal L ; Thrasher, Adrian J ; Williams, David A ; Smith, Nicholas ; Duncan, Christine N ; Gupta, Ashish O ; Downey, Gerald F ; Platzbecker, Uwe ; Musolino, Patricia L ; Demopoulos, Laura ; Eichler, Florian ; Sankar, Raman ; Kühl, Jörn-Sven ; Amartino, Hernan ; De Oliveira, Satiro ; Loes, Daniel J ; Orchard, Paul J ; Dalle, Jean-Hugues ; Gissen, Paul ; Sevin, Caroline ; Lund, Troy C ; Folloni Fernandes, Juliana ; Hussain, Shaun A ; Aubourg, Patrick ; McNeil, Elizabeth

BACKGROUNDCerebral adrenoleukodystrophy is a severe form of X-linked adrenoleukodystrophy characterized by white-matter disease, loss of neurologic function, and early death. Elivaldogene autotemcel (eli-cel) gene therapy, which consists of autologous CD34+ cells transduced with Lenti-D lentiviral vector containing ABCD1 complementary DNA, is being tested in persons with cerebral adrenoleukodystrophy.METHODSIn a phase 2-3 study, we evaluated the efficacy and safety of eli-cel therapy in boys with early-stage cerebral adrenoleukodystrophy and evidence of active inflammation on magnetic resonance imaging (MRI). The primary efficacy end point was survival without any of six major functional disabilities at month 24. The secondary end points included overall survival at month 24 and the change from baseline to month 24 in the total neurologic function score.RESULTSA total of 32 patients received eli-cel; 29 patients (91%) completed the 24-month study and are being monitored in the long-term follow-up study. At month 24, none of these 29 patients had major functional disabilities; overall survival was 94%. At the most recent assessment (median follow-up, 6 years), the neurologic function score was stable as compared with the baseline score in 30 of 32 patients (94%); 26 patients (81%) had no major functional disabilities. Four patients had adverse events that were directly related to eli-cel. Myelodysplastic syndrome (MDS) with excess blasts developed in 1 patient at month 92; the patient underwent allogeneic hematopoietic stem-cell transplantation and did not have MDS at the most recent follow-up.CONCLUSIONSAt a median follow-up of 6 years after lentiviral gene therapy, most patients with early cerebral adrenoleukodystrophy and MRI abnormalities had no major functional disabilities. However, insertional oncogenesis is an ongoing risk associated with the integration of viral vectors. (Funded by Bluebird Bio; ALD-102 and LTF-304 ClinicalTrials.gov numbers NCT01896102 and NCT02698579, respectively.).

2024-10-10·New England Journal of Medicine

Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy

Article

作者: Harris, Marian H ; Duncan, Christine N ; Orchard, Paul J ; Thakar, Himal L ; Beckman, Amy ; Grzywacz, Bartosz ; Lodaya, Ankit ; Kinney, Melissa A ; Pierciey, Francis J ; Colvin, Richard A ; Williams, David A ; Slauson, Sarah ; Bonner, Melissa ; Dietz, Andrew C ; Foos, Marianna ; Gupta, Ashish O ; Kühl, Jörn-Sven ; Eichler, Florian S ; Floro, Nicole ; Downey, Gerald F ; Bledsoe, Jacob R ; Parsons, Geoffrey ; Prasad, Vinod K

BACKGROUNDGene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti-D) has shown efficacy in clinical studies for the treatment of cerebral adrenoleukodystrophy. However, the risk of oncogenesis with eli-cel is unclear.METHODSWe performed integration-site analysis, genetic studies, flow cytometry, and morphologic studies in peripheral-blood and bone marrow samples from patients who received eli-cel therapy in two completed phase 2-3 studies (ALD-102 and ALD-104) and an ongoing follow-up study (LTF-304) involving the patients in both ALD-102 and ALD-104.RESULTSHematologic cancer developed in 7 of 67 patients after the receipt of eli-cel (1 of 32 patients in the ALD-102 study and 6 of 35 patients in the ALD-104 study): myelodysplastic syndrome (MDS) with unilineage dysplasia in 2 patients at 14 and 26 months; MDS with excess blasts in 3 patients at 28, 42, and 92 months; MDS in 1 patient at 36 months; and acute myeloid leukemia (AML) in 1 patient at 57 months. In the 6 patients with available data, predominant clones contained lentiviral vector insertions at multiple loci, including at either MECOM-EVI1 (MDS and EVI1 complex protein EVI1 [ecotropic virus integration site 1], in 5 patients) or PRDM16 (positive regulatory domain zinc finger protein 16, in 1 patient). Several patients had cytopenias, and most had vector insertions in multiple genes within the same clone; 6 of the 7 patients also had somatic mutations (KRAS, NRAS, WT1, CDKN2A or CDKN2B, or RUNX1), and 1 of the 7 patients had monosomy 7. Of the 5 patients with MDS with excess blasts or MDS with unilineage dysplasia who underwent allogeneic hematopoietic stem-cell transplantation (HSCT), 4 patients remain free of MDS without recurrence of symptoms of cerebral adrenoleukodystrophy, and 1 patient died from presumed graft-versus-host disease 20 months after HSCT (49 months after receiving eli-cel). The patient with AML is alive and had full donor chimerism after HSCT; the patient with the most recent case of MDS is alive and awaiting HSCT.CONCLUSIONSHematologic cancer developed in a subgroup of patients who were treated with eli-cel; the cases are associated with clonal vector insertions within oncogenes and clonal evolution with acquisition of somatic genetic defects. (Funded by Bluebird Bio; ALD-102, ALD-104, and LTF-304 ClinicalTrials.gov numbers, NCT01896102, NCT03852498, and NCT02698579, respectively.).

2023-12-01·Biochemistry (Moscow)

Viral Vectors in Gene Replacement Therapy

Review

作者: Karabelsky, Alexander ; Ivanov, Roman ; Egorov, Alexander D. ; Minskaia, Ekaterina ; Galieva, Alima

Throughout the years, several hundred million people with rare genetic disorders have been receiving only symptom management therapy. However, research and development efforts worldwide have led to the development of long-lasting, highly efficient, and safe gene therapy for a wide range of hereditary diseases. Improved viral vectors are now able to evade the preexisting immunity and more efficiently target and transduce therapeutically relevant cells, ensuring genome maintenance and expression of transgenes at the relevant levels. Hematological, ophthalmological, neurodegenerative, and metabolic therapeutic areas have witnessed successful treatment of hemophilia and muscular dystrophy, restoration of immune system in children with immunodeficiencies, and restoration of vision. This review focuses on three leading vector platforms of the past two decades: adeno-associated viruses (AAVs), adenoviruses (AdVs), and lentiviruses (LVs). Special attention is given to successful preclinical and clinical studies that have led to the approval of gene therapies: six AAV-based (Glybera® for lipoprotein lipase deficiency, Luxturna® for retinal dystrophy, Zolgensma® for spinal muscular atrophy, Upstaza® for AADC, Roctavian® for hemophilia A, and Hemgenix® for hemophilia B) and three LV-based (Libmeldy® for infantile metachromatic leukodystrophy, Zynteglo® for β-thalassemia, and Skysona® for ALD). The review also discusses the problems that arise in the development of gene therapy treatments, which, nevertheless, do not overshadow the successes of already developed gene therapies and the hope these treatments give to long-suffering patients and their families.

248

项与 Elivaldogene Autotemcel (Bluebird Bio) 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

2024-11-14

·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

2024-11-14

·Firstwordpharma

Bluebird bio needs cash injection to fund operations beyond Q1 next year

Bluebird bio revealed in its third-quarter report Thursday that it remains in a tight financial position and needs to secure more funds in order to avoid running out of cash by early 2025 — casting a shadow over otherwise encouraging signs of commercial progress for its gene therapy portfolio.The announcement puts pressure on the company to garner additional financing within the next few months to bridge the gap to its projected cash flow break-even in the second half of next year."Based on our current forecasts, which assumes continued cost saving initiatives, successfully renegotiating key contracts and continued collaborative engagement from Hercules, we expect our existing cash and cash equivalents will enable us to fund our operations into the first quarter of 2025," stated Chief Financial Officer James Sterling during the company's earnings call.This past March, bluebird inked a $175-million, five-year term loan facility with Hercules Capital, securing funding that the company said was expected to extend its cash runway for 24 months. Bluebird later announced it would let go of about a quarter of its staff in an effort to scale back operating expenses by 20% as its trio of gene therapies struggled to get off the ground.The company ended September with $118.7 million in cash and cash equivalents, including $48 million in restricted cash — a stark contrast to its 2018 position when it held $1.6 billion (see – Vital Signs: The last flight of the bluebird).Seventeen starts for LyfgeniaThe update comes amid a backdrop of third-quarter momentum. Bluebird reported 57 patient starts completed to date in 2024 across its three approved gene therapies: 35 for Zynteglo in beta-thalassemia, 17 for sickle cell disease treatment Lyfgenia (compared to only four in Q2), and 5 for Skysona in cerebral adrenoleukodystrophy. An additional 17 starts are scheduled through year-end.Tom Klima, chief commercial and operating officer, highlighted strong early indicators for 2025, noting "30 patient starts already scheduled in 2025," with the "vast majority" of those in the first quarter. He also pointed to a near-perfect conversion rate from scheduled to treated patients. "So far, it's very high… Essentially, 100% are converting to a start. The only variable is just the time."He added, "We have plans to double capacity for Lyfgenia in 2026 based on anticipated demand."Meanwhile, the company's network of qualified treatment centres (QTCs) has more than 70 locations in the US, with approximately 30 having initiated for one of its therapies. "I think the most exciting part of that is we have about 40 QTCs that are looking to start their first patients. So, I think it's a nice indicator of growth ahead," Klima said.

基因疗法

100 项与 Elivaldogene Autotemcel (Bluebird Bio) 相关的药物交易

登录后查看更多信息

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
肾上腺脑白质营养不良	美国	bluebird bio, Inc.	2022-09-16

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
肾上腺脑白质营养不良	临床1期	欧盟	bluebird bio, Inc.	2021-07-16
肾上腺脑白质营养不良	临床1期	挪威	bluebird bio, Inc.	2021-07-16
肾上腺脑白质营养不良	临床1期	列支敦士登	bluebird bio, Inc.	2021-07-16
肾上腺脑白质营养不良	药物发现	冰岛	bluebird bio, Inc.	2021-07-16

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02698579 \| NCT01896102 (ALD-102, Pubmed \| N Engl J Med) 人工标引	临床2/3期	肾上腺脑白质营养不良	32	Elivaldogene autotemcel (eli-cel) gene therapy	(構獵築蓋鹽製顧獵窪窪) = 鏇觸鬱膚顧繭憲窪夢蓋範範範鏇繭觸獵顧積獵 (積獵鬱醖襯餘網繭觸夢 ) 更多	积极	2024-10-10
NCT03852498 \| NCT01896102 (ALD-104, Tandem Meetings ASTCT and CIBMTR2024) 人工标引	N/A	肾上腺脑白质营养不良 CD34+	67	Elivaldogene Autotemcel	(廠製網構鑰網鏇顧構獵) = Few SAEs were reported during mobilization/apheresis; incidence was expectedly higher in conditioning and neutropenic periods, consistent with myeloablative transplant. No patients developed sinusoidal obstruction syndrome or new seizures; all survived to hospital discharge. As of Sep 6, 2023, 5 patients who received eli-cel were subsequently diagnosed with myelodysplastic syndrome. 艱鬱鑰夢製鏇窪糧壓襯 (構鬱獵鹹醖糧觸顧獵獵 )	积极	2024-02-01
NCT01896102 (ALD-102, BusinessWire) 人工标引	临床2/3期	肾上腺脑白质营养不良	32	Elivaldogene Autotemcel	顧襯構壓獵鹽蓋築築築(獵餘積醖範廠鏇願廠襯) = 艱觸淵艱廠築選膚廠積鏇獵憲淵鹹築艱築簾網 (顧網艱遞構憲窪蓋鏇獵 ) 更多	积极	2021-03-15
EUCTR2011-001953-10-DE \| NCT01896102 (EBMT2018)	临床2/3期	肾上腺脑白质营养不良 ABCD1	21	Lenti-D gene therapy	(廠顧鑰顧製鹽繭膚憲選) = 遞願築鑰襯鏇網壓範廠鬱製廠鬱蓋餘鹽選願鏇 (鹹觸憲鬱鹹鹽廠範顧網 ) 更多	积极	2018-09-24
NCT01896102 (STARBEAM, Pubmed \| Br Dent J) 人工标引	临床2/3期	肾上腺脑白质营养不良	17	elivaldogene tavalentivec	膚夢憲餘餘願鏇膚築淵(淵齋願鑰獵選夢糧膚淵) = 遞襯淵夢積簾鹽壓顧襯醖願鬱膚淵衊觸願憲齋 (構鑰壓簾窪憲廠蓋鑰顧 )	积极	2017-10-26