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

/ Active, not 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 and progenitor stem cell (collectively referred to as hematopoietic stem and progenitor cells or HSPCs) transplantation 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 相关的转化医学

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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）

2025-05-01·PHARMACOECONOMICS

Innovative Payment Models for Sickle-Cell Disease Gene Therapies in Medicaid: Leveraging Real-World Data and Insights from CMMI’s Gene Therapy Access Model

Article

作者: Nair, Kavita ; Leonard, Jim ; DiStefano, Michael J ; Wright, Garth C ; Zemplenyi, Antal ; McQueen, R Brett ; Anderson, Kelly E

OBJECTIVE:

This study aims to evaluate the financial implications of implementing various payment models, including outcome-based agreements (OBAs), volume-based rebates, and guaranteed rebates, for the newly approved gene therapies, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), in the treatment of sickle-cell disease (SCD) from the perspective of Colorado Medicaid. The analysis specifically examines the cost of standard of care (SoC) for severe SCD, the impact of different eligibility criteria based on vaso-occlusive events (VOEs), and the potential financial impacts associated with rebate structures.

METHODS:

Data from the Colorado Department of Health Care Policy & Financing (HCPF) database was used to estimate the annual costs for Medicaid-enrolled patients with severe SCD from 2018 to 2023. Patients were selected based on various eligibility criteria, including the number of VOEs, acute chest syndrome events, and stroke diagnoses. Three-state Markov models (SCD, stable, and dead) were constructed to compare the costs of SoC and gene therapies. The durability of gene therapy effectiveness and the financial impact of OBAs, volume-based rebates, and guaranteed rebates were evaluated over a 6-year contract period, with scenarios reflecting different VOE criteria and treatment durability.

RESULTS:

The average annual SoC cost for severe SCD patients (N = 138) was US$45,941 (SD US$59,653), with higher costs associated with more frequent VOEs. Gene therapies exa-cel and lovo-cel, with one-off list prices of US$2.2 million and US$3.1 million, respectively, exhibited high upfront costs, resulting in a negative cumulative balance averaging - US$2.11 million for exa-cel and - US$3.00 million for lovo-cel per patient over 6 years compared with SoC. Outcome-based rebates could potentially save Medicaid approximately US$260K (uncertainty interval 88K-772K) per patient on average for exa-cel and US$367K (uncertainty interval 122K-1111K) for lovo-cel after they pay the full up-front cost. Volume-based and guaranteed rebates also offered potential savings but varied in impact based on contract duration and effectiveness of gene therapy.

CONCLUSIONS:

The study highlights critical considerations for Medicaid in negotiating OBAs for SCD gene therapies. Achieving budget neutrality over 6 years is unlikely due to low SoC costs. However, payment models can enhance value-based spending by linking high therapy costs and potential rebates to the health gains these treatments may offer. OBAs offer offsets contingent on therapy effectiveness durability and contract terms (such as length and price), while varying eligibility criteria impact budgets and outcomes. Medicaid real-world data is crucial for navigating complexities in defining eligible populations and structuring OBAs.

2025-05-01·ADVANCES IN THERAPY

Impact of Different Definitions of Vaso-Occlusion on Efficacy Assessments in Sickle Cell Disease Clinical Trials

Review

作者: Xuan, Fengjuan ; Imren, Suzan ; Eckrich, Michael J ; Li, Nanxin ; Locatelli, Franco ; Grupp, Stephan A ; Frangoul, Haydar

INTRODUCTION:

Patients with sickle cell disease (SCD) experience recurrent, severe pain events due to vaso-occlusion. Eliminating these acute pain events is a key outcome in SCD clinical trials; however, the definition of a vaso-occlusive crisis (VOC) or a vaso-occlusive event (VOE) has not been consistently applied, hampering comparisons of treatment efficacy between different therapeutic approaches. We have examined the degree to which differing definitions of vaso-occlusion in clinical trial endpoints impact efficacy outcomes.

METHODS:

Descriptions of clinical endpoints related to vaso-occlusion and pain events were reviewed from trials of exagamglogene autotemcel (exa-cel), lovotibeglogene autotemcel (lovo-cel), renizgamglogene autogedtemcel (reni-cel), hydroxyurea, L-glutamine, voxelotor, and crizanlizumab. Patient-level data from the published exa-cel Phase 3 pivotal trial (CLIMB SCD-121; data cut 14 Jun 2023) was used to evaluate efficacy outcomes based on differing endpoint definitions of vaso-occlusion.

RESULTS:

In the seven clinical trials reviewed, definitions of vaso-occlusion and/or pain events varied by care setting, duration of care, treatments used, and associated complications, with the frequency and duration of medical facility visits for acute pain events being most dissimilar between trials. Definitions of severe VOCs (exa-cel), VOC (voxelotor), and sickle cell-related pain crises (SCPCs; crizanlizumab and L-glutamine) included pain events requiring a medical facility visit of any duration, whereas the definition of painful crises (hydroxyurea) required a medical facility visit of > 4 h and the definition of severe vaso-occlusive events (VOEs; lovo-cel and reni-cel) required a hospital or emergency room (ER) observation unit visit lasting ≥ 24 h or ≥ 2 visits to a day unit or ER over a 72-h period. Based on the definition of severe VOCs, 29/30 patients [96.7%; 95% confidence interval (CI): 82.8, 99.9] in the CLIMB SCD-121 trial were considered free from severe VOCs for ≥ 12 consecutive months, whereas when the severe VOEs definition was applied to the same data, all patients (30/30; 100.0%; 95% CI: 88.4, 100.0) were considered free from severe VOEs for ≥ 12 consecutive months.

CONCLUSION:

Differences exist in definitions of vaso-occlusion and pain events used in SCD clinical trials. Severe VOCs (exa-cel), VOC (voxelotor), and SCPCs (crizanlizumab and L-glutamine) were more broadly inclusive than severe VOEs (lovo-cel and reni-cel) or painful crisis (hydroxyurea). Clinically, these differences resulted in differing numbers of patients being considered free from vaso-occlusion pain events, underscoring the challenge in comparing frequencies of pain events across SCD clinical trials.

2025-01-01·CYTOTHERAPY

Sickle cell disease gene therapy drug expenses and reimbursement: a litmus test for commercial pricing strategy and patient access for curative therapies

Article

作者: Galipeau, Jacques ; Hume, Ashley ; Grilley, Bambi J

Durable gene therapies newly approved by the Food and Drug Administration for sickle cell disease offer the promise of cure with subsequent improvement in quality of life through avoidance of repeated hospitalizations and worsening of disabilities in these patients. In the United States, following Food and Drug Administration approval, lovotibeglogene autotemcel (Lyfgenia, bluebird bio, Inc, Sommerville, MA, USA) and exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals Inc, Boston, MA, USA) were launched in 2024 for treatment of patients 12 years and older with frequent vaso-occlusive crises. Here we will discuss the first treatment with lovotibeglogene as a test case serving to highlight the multiple drug reimbursement pathways in the United States for cell and gene products for orphan disorders and their impact on patient adoption and access.

259

项与 Lovotibeglogene autotemcel 相关的新闻（医药）

2025-04-17

·抗体圈

蓝鸟生物收购遇波折：Ayrmid 出价 “流产”

近日，基因疗法公司蓝鸟生物（bluebird bio）的收购进程陷入了戏剧性的转折。原本看似有望获得更高报价的蓝鸟生物，因爱尔兰投资公司 Ayrmid 未能按预期提交有约束力的收购要约，不得不重新回归与凯雷集团（Carlyle）和 SK 资本伙伴（SK Capital Partners）的原始交易，这一事件在生物医药投资领域引发了广泛关注。Ayrmid “爽约”，蓝鸟生物收购计划生变蓝鸟生物在基因疗法领域一直备受瞩目，然而其市场表现却不尽人意。今年 2 月，蓝鸟生物与凯雷集团和 SK 资本伙伴达成收购协议，这两家投资公司将以每股 3 美元，总计 3000 万美元的价格收购蓝鸟生物，使其私有化，交易原计划于 2025 年上半年完成。3 月下旬，Ayrmid 突然杀出，主动提出以每股 4.5 美元的价格收购蓝鸟生物，较原始报价高出 50%，并且承诺根据销售里程碑再支付每股 6.84 美元。这一出价看似诱人，给蓝鸟生物的股东们带来了更多希望。双方随后展开了为期两周的尽职调查，期间蓝鸟生物还应 Ayrmid 的要求将调查期延长了 4 天。但令人意外的是，调查结束后，Ayrmid 不仅未能提交有约束力的收购要约，还承认自身缺乏完成收购所需的资金。蓝鸟生物困境凸显，原始交易成 “救命稻草”蓝鸟生物董事会主席马克・瓦雄（Mark Vachon）表示，公司已与 Ayrmid 进行了两次接触，但均未得到有约束力或资金充足的报价。他还指出，若不能获得某种形式的资金注入，蓝鸟生物将面临贷款违约的风险。在这种情况下，蓝鸟生物董事会重申对凯雷集团和 SK 资本伙伴原始交易的支持。蓝鸟生物曾经估值颇高，在 2018 年时峰值接近 125 亿美元。尽管该公司获得了美国食品药品监督管理局（FDA）对多种基因疗法的批准，如治疗镰状细胞病的 Lyfgenia、治疗 β - 地中海贫血的 Zynteglo 以及治疗脑肾上腺白质营养不良的 Skysona，但这些疗法在市场上的接受度较低，销售缓慢，导致公司财务状况逐渐恶化。到 2025 年第一季度，蓝鸟生物的资金即将耗尽，分析师们担忧其在今年难以实现收支平衡。因此，与凯雷集团和 SK 资本伙伴的交易，成为了蓝鸟生物的 “救命稻草”。行业影响与展望：投资需谨慎，企业求突破Ayrmid 的 “爽约” 给蓝鸟生物的发展带来了不确定性，也为生物医药投资领域敲响了警钟。对于企业来说，在寻求外部投资和并购机会时，需要更加谨慎地评估潜在合作伙伴的实力和诚意；而对于投资者而言，在做出投资决策前，更要全面考量目标公司的财务状况、市场前景以及潜在风险。蓝鸟生物未来的发展仍然充满挑战，即使顺利完成与凯雷集团和 SK 资本伙伴的交易，如何提升现有基因疗法的市场表现，开发更具竞争力的产品，依旧是摆在其面前的难题。此次事件也让行业更加关注基因疗法领域的投资环境和企业的可持续发展，未来，基因疗法企业在吸引投资和实现商业成功的道路上，需要不断探索新的模式和策略。参考来源：https://www.biospace.com/deals/bluebirds-second-suitor-ayrmid-fails-to-produce-an-offer识别微信二维码，添加抗体圈小编，符合条件者即可加入抗体圈微信群！请注明：姓名+研究方向！本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

基因疗法并购

2025-04-17

·Pharmaceutical Technology

Bluebird bio reaffirms Carlyle deal as Ayrmid misses deadline

bluebird has been under financial pressure for several years. Credit: SOPA Images via Getty Images. Bluebird bio (bluebird) has reaffirmed its support for a proposed acquisition by Carlyle and SK Capital after rival bidder Ayrmid failed to deliver a binding offer or secure financing by the extended deadline. The announcement marks the second time Ayrmid has initiated discussions with bluebird without producing a fully financed proposal. Bluebird said it had engaged with Ayrmid for three weeks following the private equity firm’s unsolicited bid in March, including extending the original deadline to allow more time for due diligence and deal structuring. However, Ayrmid did not submit a definitive proposal by the agreed-upon 15 April cut-off date and has yet to secure the necessary capital to proceed. As a result, bluebird’s board of directors is reiterating its unanimous support for the acquisition agreement with Carlyle and SK Capital, which was first announced in February 2025. The board is urging shareholders to tender their shares by the updated deadline of 2 May, extending the previous expiration date of 18 April. bluebird’s board chairman Mark Vachon said: “Ayrmid’s proposal remains highly conditional. bluebird has engaged with Ayrmid on two separate occasions—neither of which has resulted in a binding or fully-financed offer. The board unanimously reaffirms its support of the previously announced agreement with Carlyle and SK Capital in the strongest possible terms.” Bluebird has been under significant financial pressure for several years. The company currently faces the risk of defaulting on a $175m loan from Hercules Capital, secured in 2024 to support ongoing operations. Without a new capital injection, the board said, the company’s liquidity position remains precarious. Carlyle and SK Capital’s proposal offers $3 per share in cash, along with a potential additional payment of $6.84 per share if certain clinical and commercial milestones are met. Ayrmid‘s offer, disclosed last month, proposed $4.50 per share upfront and the same milestone-based payout structure. However, bluebird confirmed that Ayrmid has not demonstrated access to committed funding. bluebird noted that Ayrmid is continuing to seek financing and may provide further updates in the coming days. bluebird’s financial position has deteriorated over time, despite achieving several key regulatory milestones. The company currently markets three US Food and Drug Administration (FDA)- approved gene therapies – Zynteglo (betibeglogene autotemcel) for transfusion-dependent beta-thalassemia, Skysona (elivaldogene autotemcel) for cerebral adrenoleukodystrophy, and Lyfgenia (lovotibeglogene autotemcel) for sickle cell disease – but has struggled with slow uptake, high costs, and reimbursement hurdles. In 2023, the company suffered a major setback when the FDA denied a priority review voucher (PRV) for Lyfgenia. Bluebird had already arranged to sell the voucher to Novartis for $103m, but the agency ruled that the therapy’s active ingredient had previously been used in Zynteglo, for which the company had already received a PRV in 2022. The loss of the expected funds deepened bluebird’s financial strain. To fill the gap, bluebird pursued emergency financing through a $150m public stock offering underwritten by Goldman Sachs and JP Morgan, as well as up to $100m in receivables financing from Alterna Capital Solutions. The company has also undergone repeated cost-cutting efforts, including workforce reductions of 30% in 2022 and an additional 25% last year. Despite these efforts, bluebird’s therapies continue to face stiff competition. Lyfgenia is priced at $3.1m per patient, while a competing treatment, Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and CRISPR Therapeutics, is listed at $2.2m. Both therapies were approved on the same day in December 2023 to treat sickle cell disease. Going public in 2013, bluebird has seen its stock decline sharply in recent years. Following the 16 April update, shares closed down by 7%. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic You have a right to withdraw your consent at any time, by clicking here . We may still continue to send you service-related and other non-promotional communications. For more information relating to our privacy practices, we invite you to review our privacy policy .

并购优先审批上市批准细胞疗法基因疗法

2025-03-24

·BiG生物创新社

天价基因疗法，狂热追捧中踩下急刹

作者｜与安 2025年3月，罗氏制药（Roche）宣布对旗下基因疗法子公司Spark Therapeutics启动“根本性重组”，全额减记24亿美元商誉。财务数据显示，罗氏已累计为 Spark 投入超10亿美元的重组成本，其中包括2024 年1.84亿美元的前期重组费用和2025年预计达3.4亿美元的追加支出。然而，这场持续六年的资本实验最终却惨淡收场，成为基因疗法领域最惨痛的收购案例之一。而这并非个例。过去三个月，基因治疗领域在希望与阵痛中交织前行，此前昭示熊熊野心的各家药企中，不乏惨淡收场的身影。跨国药企的管线重组、战略收缩与突破转型，再次将这一赛道的复杂性与不确定性推至台前，同时也代表着曾受资本狂热吹捧的基因疗法领域正逐步向理性发展回归转折。 01 43亿美元的深刻教训技术瓶颈下散场的资本盛宴 2019年，罗氏以43亿美元天价收购Spark Therapeutics，看中的是其全球首个获批的遗传性视网膜病变疗法Luxturna，以及血友病A基因疗法SPK-8011（dirloctocogene samoparvovec）的3期临床潜力。彼时Spark市值仅22亿美元，而罗氏以溢价125%的代价收购，创下当时基因疗法领域最大并购案，折射出行业对基因疗法商业化的狂热预期。然而截至 2024 年，Luxturna 年销售额仅 2050 万美元，同比暴跌 59%.此外，SPK-8011 项目则因安全性问题被紧急叫停，不达预期的商业表现和无法突破的技术难题，直接让这项大手笔并购案从"战略收购" 降级为 "产品交易"。迅速击穿资本泡沫的主要是三重瓶颈。首先是载体免疫性难题。Luxturna依赖的AAV2血清型载体，暴露了基因疗法最顽固的技术瓶颈之一，即预存免疫与后天免疫的双重绞杀。全球约30%-60%人群因自然感染携带AAV中和抗体，导致Luxturna治疗前必须进行抗体筛查。但Spark采用的"血清型切换"策略并未突破根本限制，数据显示超60%患者治疗后仍产生新中和抗体，直接导致疗效持久性不足。而后天免疫的影响在于，即使患者通过预存免疫筛查，治疗后仍可能因免疫系统激活产生新的抗AAV2免疫反应。其次是产能不足。Spark费城工厂病毒载体年产能仅5000剂，在适应症人群本就狭窄（全球约6000名患者）的情况下，覆盖率仍旧不足85%。患者等待期过长，市场渗透未达预期。最后则是临床表现的滑铁卢，完成收购时被列为重点的SPK-8011因安全性问题被叫停，于去年年底宣布终止研究。当病毒载体的免疫原性、生产工艺、长期安全性三大技术关卡未获突破时，资本堆砌反而加速了泡沫破裂。 02 火热势头下的行业寒冬难以走出的商业化困境与此同时，罗氏的受挫并非个例。从去年开始，基因治疗领域接连出现“利空”消息，整体成绩远低于预期，市场表现陷入疲软，多家行业明星宣布因进展不佳关停管线或业务重组。 2月21日，辉瑞宣布将停止进一步开发和商业化血友病B基因疗法Beqvez（SPK-9001）。这款药物于2024年4月刚获FDA批准，定价高达350万美元（约合人民币2537万元），在2024年全美最贵药物前十排行榜中，仅次于定价 425 万美元的 Lenmeldy，并列第二。 2024年全美最贵十款药物而Beqvez从获批上市到决定撤回，还不满一年时间。辉瑞表示，如此决定是因为“患者及医生对血友病基因疗法表现出的兴趣有限”，上市至今，尚未有患者接受过该药治疗。与此同时，辉瑞彻底清空了基因疗法管线。随着Beqvez退出，该司已没有任何基因疗法处于临床或商业化阶段。实际上，彼时将基因治疗领域列为“优先发展领域”的辉瑞，近三年来正在逐步撤离基因治疗领域。2023年7月，该司将早期研发阶段的基因疗法打包以高达10亿美元的价格卖给了阿斯利康旗下的罕见病部门Alexion；2024年6月，其针对杜氏肌营养不良症（DMD）的基因疗法fordadistrogene movaparvovec因CIFFREO Ⅲ期试验结果未达主要终点，陷入停滞；而到9月，更是撤回了市场上所有批次的镰状细胞贫血疗法Oxbryta，同时终止所有相关试验。因其临床数据表明，该药在镰状细胞患者群体中的总体效益不再足以覆盖其风险。至年底，辉瑞则终止了与Sangamo Therapeutics的全球合作及许可协议，放弃共同开发治疗A型血友病的基因疗法候选药物giroctocogene fitelparvovec（Giro-vec），尽管该药在去年完成的AFFINE III期临床试验中已取得积极结果，并已准备向FDA和EMA提交监管申请。临时放弃的主要原因大致有二，一是BioMarin同适应症的基因疗法Roctavian商业化进展不佳，市场表现惨淡，可谓现成的前车之鉴；二是辉瑞血友病新型抗体疗法Marstacimab于不久前才获批上市，作为给药便利性更加的抗体，对于基因疗法的市场空间也造成了一定冲击。总得来说，就是彼时看来，这款药物似乎难以实现大卖，而三个月后折戟的Beqvez也确实证实了这一点。而就在辉瑞宣布从基因疗法退场的前一天，曾经估值百亿的蓝鸟生物宣布卖身私募资金，一同踩下急刹。 2018年初，凭借β地中海贫血疗法Zynteglo（定价280万美元）、脑肾上腺脑白质营养不良疗法Skysona（定价300万美元）和镰状细胞病疗法Lyfgenia（定价310万美元），蓝鸟生物一时声名鹊起，股价飙升至每股2300美元，市值突破300亿美元。其“一次性治愈”叙事吸引了黑石、富达等顶级资本加持，成为首个基因疗法领域“十角兽”企业。然而，高昂定价和狭窄的患者群体让其商业化之路变得十分困难。受限于保险公司等支付方在高昂定价下的报销审批挑战，三款核心产品2023年总销售额为2910万美元，至今未实现盈利，其中，Zynteglo 2023年销售额为1670万美元，截至2024年11月累计治疗35例患者；Lyfgenia则仅完成4例患者治疗，虽与Vertex的CRISPR疗法同日获批，却因价格高出90万美元且因存在安全问题遭FDA发出黑框警告，市场份额遭到挤压。去年10月，蓝鸟宣布裁员25%并启动重组，但现金流缺口仍无法填补。最终，该司以2900万美元（不足巅峰市值0.1%）被私募基金凯雷/SK资本收购，对赌条款上限仅1亿美元，蓝鸟最终“断翅”。 03 市场洗牌中的希望与曙光大额盈利并非不可能一度受挫的基因疗法领域也并非毫无商业进展。与前文几款药物相比，Sarepta Therapeutics的Elevidys表现似乎截然不同。这是全球首款DMD基因疗法，可对携带任何类型DMD致病基因变异的患者生效，很好地填补了临床上的空白。值得注意的是，这还是和罗氏联合开发的疗法，同样高成本、高定价，售价320万美元，位列全美最贵药物第四（见图1）。2023年6月获FDA批准，8月正式上市销售，2024年便首度扭亏为盈。 2024年6月，Serepta扩大了该药的适应症，从原来的仅限于 4～5 岁具有行走能力的儿童，扩展为 4 岁及以上的儿童（无论是否有行走能力），进一步拓宽了市场覆盖面。至第三季度，Sarepta实现了净利润同比增长113%的好成绩，一举达到7619万美元。而诺华在AAV基因疗法上的投入则更是成效亮眼，针对脊髓性肌萎缩症（SMA）的Zolgensma可谓销冠，据其财报显示，2024年的年销售额仍然取得了12.14亿美元的好成绩。 Zolgensma2024年净销售额或许还有一大关键原因在于适应症的选择。反观让多位巨头或挣扎前行、或认栽止损的血友病基因疗法，市场表现不佳主要还是因为有凝血因子替代疗法和双抗药物可供患者选择。自身售价不菲，疗效也并无绝对优势，在可替代方案充足的情况下，难以打开市场的情况或许也并非难以预料。然而罗氏并未完全放弃。一方面，Spark的剩余业务将整合至集团，重组不影响Luxturna的持续供应，费城创新中心的一部分研发职能也仍然予以保留；另一方面，通过与Dyno Therapeutics达成的10亿美元合作，利用后者人工智能平台开发神经疾病新型AAV载体，罗氏将转向开发针对神经疾病的下一代基因疗法。基因疗法的故事远未终章。尽管挑战重重，部分企业仍以战略调整与技术升级寻找生机。2025年的春天，基因治疗领域仍在冰火之间跋涉。但随着临床技术的进步、监管政策的完善和支付体系的创新，当基因治疗的商业化摆脱纯资本驱动，构建起“技术-临床-支付”的完整闭环，这一领域仍然有望在迎来更为明朗开阔的前景，重唱高歌，再次高速前行。参考文献 https://www.fiercepharma.com/pharma/roche-overhauls-spark-gene-therapy-unit-recording-24b-full-impairment https://www.fiercepharma.com/pharma/pfizer-empties-gene-therapy-portfolio-discontinues-hemophilia-treatment-beqvez https://www.fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy https://www.pharmaceutical-technology.com/features/the-most-expensive-drugs-in-the-us/ 公众号文章 BioSeedin柏思荟辉瑞“泪洒”基因疗法 https://news.bioon.com/article/98498658028f.html 各家药企官网新闻动态和财报数据共建Biomedical创新生态圈！如何加入BiG会员？

基因疗法并购临床3期

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

未上市

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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	美国	bluebird bio, Inc.	2015-02-02
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)	遞壓鹽糧觸夢膚積鬱膚(糧鹹餘窪廠選蓋壓遞衊) = 網餘鏇齋夢齋築築構築憲衊鏇遞積夢餘襯膚鑰 (簾憲觸選衊夢壓窪鹹製 ) 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	窪築淵糧顧獵顧築鏇簾(齋繭衊遞餘襯築簾鑰夢) = 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. 壓願願鹽選壓構鹽構製 (襯築構膚襯構顧夢膚醖 ) 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)	遞廠遞餘夢構壓憲鏇廠 = 廠簾觸壓願鬱艱襯築襯憲願構壓鑰獵醖鑰願蓋 (餘廠窪獵遞築膚簾淵顧, 範襯構選蓋繭選憲鑰衊 ~ 積獵簾艱簾衊膚廠範選) 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)	遞廠遞餘夢構壓憲鏇廠 = 醖簾醖蓋壓艱願壓網鹹憲願構壓鑰獵醖鑰願蓋 (餘廠窪獵遞築膚簾淵顧, 衊顧願膚壓選選鹽鹽糧 ~ 顧鬱選鬱蓋觸築鹹醖衊) 更多	-	2020-03-10