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更新于：2025-03-20

Betibeglogene autotemcel

更新于：2025-03-20

概要

概要

基本信息

药物类型

基因疗法、造血干细胞疗法

别名

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

+ [12]

靶点

β-globin

作用方式

刺激剂

作用机制

β-globin刺激剂(血红蛋白β链刺激剂)、基因转移

治疗领域

遗传病与畸形血液及淋巴系统疾病其他疾病

在研适应症

地中海贫血β地中海贫血镰状细胞血症

非在研适应症

输血依赖性β地中海贫血输血依赖性地中海贫血

原研机构

bluebird bio, Inc.

在研机构

bluebird bio, Inc.

非在研机构-

最高研发阶段批准上市

首次获批日期

欧盟 (2019-05-29),

输血依赖性β地中海贫血

最高研发阶段(中国)-

特殊审评优先药物（PRIME） (欧盟)、加速批准 (欧盟)、加速审评 (欧盟)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、快速通道 (美国)、突破性疗法 (美国)、优先审评 (美国)、再生医学先进疗法 (美国)

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关联

项与 Betibeglogene autotemcel 相关的临床试验

JPRN-jRCTc071190041

/ Not yet recruiting临床2期IIT

A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis - A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis

开始日期2019-01-04

申办/合作机构-

EUCTR2017-001275-23-GB

/ Unknown status临床2期IIT

Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase Deficiency

开始日期2017-09-21

申办/合作机构

Great Ormond Street Hospital Children's Charity

NCT03207009

/ Completed临床3期

A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

开始日期2017-06-08

申办/合作机构

bluebird bio, Inc.

100 项与 Betibeglogene autotemcel 相关的临床结果

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100 项与 Betibeglogene autotemcel 相关的转化医学

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100 项与 Betibeglogene autotemcel 相关的专利（医药）

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项与 Betibeglogene autotemcel 相关的文献（医药）

2025-01-14·Blood Advances

Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel

Article

作者: Jakoby, Donate ; Heine, Sabine ; Lobitz, Stephan ; Pavel, Petra ; Schmitt, Michael ; Ritsert, Mona-Lisa ; Mirza, Adil ; Dürken, Matthias ; Kulozik, Andreas ; Tao, Gloria ; Schmitt, Anita ; Kunz, Joachim ; Laier, Sascha ; Thakar, Himal ; Koscher, Leila ; Greil, Johann

AbstractGene addition and editing strategies for transfusion-dependent β-thalassemia have gained momentum as potentially curative treatment options, with studies showcasing their efficacy and safety. We report, to our knowledge, the first real-world application of betibeglogene autotemcel (beti-cel; Zynteglo) during its period of active license in Europe from January 2020 to March 2022 for patients aged ≥12 years without a β0/β0 genotype and without a HLA-matched sibling donor, before beti-cel marketing authorization was withdrawn by its holder because of nonsafety reasons. Among 15 screened patients, 4 opted out for fertility and safety concerns, 2 were excluded because of marked hepatic siderosis, and 1 had apheresis collection failure. Eight patients received beti-cel after busulfan myeloablative conditioning, all achieving transfusion independence within 8 to 59 days, with posttreatment hemoglobin levels ranging from 11.3 to 19.3 g/dL. No deaths occurred, but acute toxicity mirrored busulfan’s known effects. Posttreatment platelet management faced challenges because of HLA-antibodies in 3 patients. Monitoring up to month 24 revealed pituitary-gonadal endocrine dysfunction in all 3 female and in 2 of 5 male patients. Additionally, we observed unexpected posttreatment sequelae: 1 patient developed polycythemia that could not be explained by known genetic or acquired mechanisms, 1 patient developed posttreatment depression and anxiety prohibiting her from returning to work, and 1 patient developed fatigue severely compromising both quality of life and work capacity. This real-world experience corroborates beti-cel’s efficacy and safety and provides information on adverse events observed during real-world use of the therapy.

2024-11-01·The Lancet

Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial

Article

作者: Walters, Mark C ; Kwiatkowski, Janet L ; Tao, Ge ; Locatelli, Franco ; Elliot, Heidi ; Hongeng, Suradej ; Lee, Ji ; Thakar, Himal L ; Thuret, Isabelle ; Kulozik, Andreas E ; Sauer, Martin G ; Yannaki, Evangelia ; Thompson, Alexis A ; Ali, Shamshad ; Lal, Ashutosh ; Thrasher, Adrian J ; Lodaya, Ankit ; Colvin, Richard A ; Kunz, Joachim B

BACKGROUNDTransfusion-dependent β-thalassaemia (TDT) is a severe disease, resulting in lifelong blood transfusions, iron overload, and associated complications. Betibeglogene autotemcel (beti-cel) gene therapy uses autologous haematopoietic stem and progenitor cells (HSPCs) transduced with BB305 lentiviral vector to enable transfusion independence.METHODSHGB-212 was a non-randomised, multicentre, single-arm, open-label, phase 3 study of beti-cel in patients with TDT conducted at eight centres in France, Germany, Greece, Italy, the UK, and the USA. Patients with β⁰/β⁰, β⁰/β^+IVS-I-110, or β^+IVS-I-110/β^+IVS-I-110 genotypes, clinically stable TDT, and a transfusion history of at least 100 mL/kg per year of packed red blood cells (pRBCs) or at least eight transfusions of pRBCs per year in the 2 years before enrolment were eligible for participation. After undergoing HSPC mobilisation and busulfan-based, pharmacokinetic-adjusted myeloablative conditioning, patients were infused with beti-cel and followed up for 24 months. The primary efficacy outcome was transfusion independence, defined as weighted average haemoglobin level of 9 g/dL or above without pRBC transfusions for 12 or more months. The primary outcome was measured in all patients who received an infusion of beti-cel (transplant population); safety was evaluated in all patients who initiated study treatment (intention-to-treat population). Patients were eligible to enrol in the ongoing 13-year long-term follow-up study (for a total of 15 years), LTF-303 (registered at ClinicalTrials.gov, NCT02633943). This trial, HGB-212, was registered at ClinicalTrials.gov (NCT03207009), and is complete.FINDINGSFrom June 8, 2017, to March 12, 2020, 20 patients were screened for eligibility. One patient was ineligible and one withdrew consent before HSPC mobilisation and myeloablative conditioning. Of the 18 patients who received beti-cel, ten (56%) were male and eight (44%) were female; 13 (72%) were younger than 18 years at the time of informed consent, and five (28%) were older than 18 years. 12 (67%) patients had β⁰/β⁰ genotypes, three (17%) had β⁰/ β^+IVS-I-110, and three (17%) had β^+IVS-I-110/β^+IVS-I-110. As of Jan 30, 2023, all patients enrolled in the long-term follow-up study and the median follow-up was 47·9 months (range 23·8-59·0). All 18 patients were evaluable for transfusion independence, with 16 (89%) of 18 reaching and maintaining transfusion independence to last follow=up (estimated effect size 89·9% [95% CI 65·3-98·6]). All patients had at least one adverse event after beti-cel infusion. There were no serious adverse events considered to be related to beti-cel, and no deaths.INTERPRETATIONThese data demonstrate that beti-cel can allow patients with genotypes that cause severe β-thalassaemia (β⁰/β⁰, β⁰/β^+IVS-I-110, or β^+IVS-I-110/β^+IVS-I-110) to reach transfusion independence. Beti-cel offers the potential to attain near-normal haemoglobin levels for those with severe forms of TDT, and a potentially curative option without the risks and limitations of allogeneic HSPC transplantation. Patients are being followed up for a total of 15 years to assess the durability of transfusion independence and long-term safety profile of beti-cel.FUNDINGBluebird Bio, Somerville, MA, USA.

2024-05-01·Blood Reviews

Revolutionising healing: Gene Editing's breakthrough against sickle cell disease

Review

作者: Strouboulis, John ; Dimitrievska, Marija ; Nicolaides, Kypros H ; Jacków-Malinowska, Joanna ; Shangaris, Panicos ; Miccio, Annarita ; El Hoss, Sara ; Bansal, Dravie ; Vitale, Marta

Recent advancements in gene editing illuminate new potential therapeutic approaches for Sickle Cell Disease (SCD), a debilitating monogenic disorder caused by a point mutation in the β-globin gene. Despite the availability of several FDA-approved medications for symptomatic relief, allogeneic hematopoietic stem cell transplantation (HSCT) remains the sole curative option, underscoring a persistent need for novel treatments. This review delves into the growing field of gene editing, particularly the extensive research focused on curing haemoglobinopathies like SCD. We examine the use of techniques such as CRISPR-Cas9 and homology-directed repair, base editing, and prime editing to either correct the pathogenic variant into a non-pathogenic or wild-type one or augment fetal haemoglobin (HbF) production. The article elucidates ways to optimize these tools for efficacious gene editing with minimal off-target effects and offers insights into their effective delivery into cells. Furthermore, we explore clinical trials involving alternative SCD treatment strategies, such as LentiGlobin therapy and autologous HSCT, distilling the current findings. This review consolidates vital information for the clinical translation of gene editing for SCD, providing strategic insights for investigators eager to further the development of gene editing for SCD.

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项与 Betibeglogene autotemcel 相关的新闻（医药）

2025-03-17

·FierceBiotech

AstraZeneca goes in vivo, penning $1B deal for Belgian off-the-shelf cell therapy biotech

AstraZeneca has “been looking at the potential for in vivo technology for a little while,” according to the company\'s executive vice president of oncology hematology R&D.\n AstraZeneca has entered the “off-the-shelf” cell therapy space, penning a $1 billion deal to acquire EsoBiotec and its lentiviral vector platform.The deal will see AstraZeneca scoop up all outstanding equity of the Belgian biotech for a total consideration of $1 billion—split between an upfront payment of $425 million and up to $575 million in developmental and regulatory milestones.Bringing EsoBiotech onboard will give AstraZeneca access to the biotech’s Engineered NanoBody Lentiviral (ENaBL) platform, which uses lentiviruses to deliver genetic instructions to T cells to recognize and destroy either tumor cells for cancer treatment or potentially autoreactive cells for immune-mediated diseases.The resulting cell therapies can be administered via an intravenous injection as opposed to the standard process for autologous cell therapies, where the cells are removed from their body, genetically modified and then readministered.“What this means is that it can offer scalability to a much larger number of patients who can have access to this transformative cell therapy treatment, and the delivery of the treatment can happen in just minutes rather than the many-weeks process, which is the current reality,” Susan Galbraith, Ph.D., executive vice president for oncology hematology R&D at AstraZeneca, told journalists on a call Monday morning.AstraZeneca currently has seven cell therapy programs in the clinic, led by a BCMA and CD19 dual-targeting CAR-T for multiple myeloma acquired as part of 2023’s Gracell acquisition. However, executives at the Big Pharma told Fierce as far back as 2022 that they were also interested in exploring the off-the-shelf, also known as allogeneic, cell therapy space. Galbraith confirmed on the call that AstraZeneca had “been looking at the potential for in vivo technology for a little while” but said the company had been deterred by the “many technical barriers,” including the fact that the viral capsids used to deliver mRNA often don’t last long in the peripheral circulation.That’s where EsoBiotec comes in. The company’s lead program, which targets BCMA, is already in the clinic, and, while “only a few patients have been treated” so far, according to Galbraith, “the data we\'ve seen gives us confidence.”Specifically, AstraZeneca was impressed by data presented by EsoBiotech at the J.P. Morgan Healthcare Conference from the first patient to be dosed in the program, which demonstrated “really quite impressive cell kinetics,” Galbraith said.“What this enables us to do as a platform is take the learnings from the autologous programs that we have, apply them and then scale them at pace into the off-the-shelf platform,” she added.Lentiviral vectors have attracted interest for their ability to infect dividing and nondividing cells, in addition to their low cytotoxicity rate. Orchard Therapeutics’ approved metachromatic leukodystrophy treatment Libmeldy uses the vectors, as does bluebird bio’s sickle cell therapy Zynteglo.Novartis got in on the action last year, paying an undisclosed amount to leverage Vyriad\'s active targeting lentiviral vector platform to discover and develop new in vivo CAR-T cell therapy candidates.Mont-Saint-Guibert, Belgium-based Esobiotech is a relatively new entrant in the space, with CEO Jean-Pierre Latere, Ph.D., explaining on the call that the company was set up four years ago with the “the mission of making in vivo cell therapies much more accessible, more effective and really affordable for patients.”

细胞疗法免疫疗法并购临床研究

2025-03-11

·药时代

基因疗法先驱蓝鸟生物陨落记：从300亿美金独角兽到2900万"贱卖"背后的行业警示

正文共：1489字 1图预计阅读时间：4分钟 2025年2月，医药资本圈被一则重磅消息震动——曾被誉为"基因疗法三冠王"的蓝鸟生物（bluebird bio）以首付款2900万美元、潜在总价1亿美元的价格被凯雷与SK Capital收入囊中。这个数字仅为其2018年巅峰市值的千分之三，更讽刺的是，收购方提出的首付款在三个月内从1亿美元腰斩至不足三成，为这场资本寒冬中的"跳楼价"并购再添戏剧性注脚。一、天价疗法的陨落之路作为全球首个攻克β地中海贫血的基因疗法开创者，蓝鸟生物曾手握令同行艳羡的资本故事：2013年登陆纳斯达克后市值直逼300亿美元，三款FDA获批疗法累计定价超800万美元，被视作改写罕见病治疗史的里程碑。然而现实给了理想主义一记重击——其明星产品Zynteglo在欧洲因疑似引发白血病遭紧急叫停，177万美元的"天价"更让医保体系望而却步，最终被迫撤离欧洲市场。后续推出的300万美元级疗法Skysona和Lyfgenia同样陷入"叫好不叫座"困境，2024年前三季度仅57名患者接受治疗，对应4528万美元营收难掩2.12亿美元亏损的残酷现实。二、资本博弈下的"割肉"求生收购拉锯战暴露了生物科技企业的脆弱性：2024年9月，现金流濒临断裂的蓝鸟启动第二轮裁员，试图通过削减25%人力成本续命，却未能阻止资本市场的抛弃。凯雷最初1亿美元收购邀约看似救命稻草，却在财务尽调后骤降至2900万美元，附加条款要求销售额达6亿美元才能解锁剩余对价——这个看似苛刻的条件，实则暗藏对基因疗法商业化困境的精准预判。值得玩味的是，收购协议中特别设置了"FDA优先审评券"奖励机制，这张价值1.5亿美元的市场通行证，恰恰是蓝鸟三次申请未果的遗憾。三、寒冬中的行业镜像蓝鸟的困境折射出CGT（细胞与基因治疗）赛道集体遇冷的现状：据弗若斯特沙利文数据，全球基因治疗市场虽保持71%的年复合增长率，但2025年305亿美元规模预期下，企业正经历"从烧钱研发到变现乏力"的阵痛。国内Biotech同样面临生存考验，2024年亘喜生物、普方生物等相继被跨国药企收购，最高交易额达130亿元，而License-out（对外授权）总金额突破525亿美元的背后，实则是本土创新药企"断臂求生"的无奈。四、幸存者的启示录在这场行业大洗牌中，少数突围者提供了另一种范本：某药企凭借双抗药物半年狂揽14.7亿元收入，百利天恒更通过与BMS的84亿美元授权合作登顶盈利榜首。这些案例揭示生物科技企业的生存法则——既要保持技术前瞻性，更需建立清晰的商业化路径。正如业内人士所言："当资本潮水退去，能自我造血的Biotech才有资格讲述下一个十年故事。" 这场估值缩水99%的收购案，终将成为教科书级的行业警示：在基因治疗这个充满理想主义色彩的领域，科学突破与商业现实的距离，或许比双螺旋结构更为复杂。当资本耐心耗尽，留下的不仅是企业估值的残局，更是对整个创新药生态系统的深度拷问。参考资料： How bluebird's buyout offer went from $100M upfront to $29M曾经估值高达100亿美元，bluebird bio公司将被私募股权公司以2900万美元收购蓝鸟生物：“优等生”的落寞与自救 FDA两场专家咨询委员会后，蓝鸟生物和CRISPR的「股价」头对头变化... 高票通过！FDA专家咨询委员会首日，蓝鸟生物「闯关成功」，这次他们能缓过来吗？以邻为师 | 从LogicBio、蓝鸟生物，看遭遇到FDA临床暂停禁令时，该如何趋吉避凶？ CFO递交辞呈，资金即将耗尽，蓝鸟生物处境堪忧... 半年报公布后，基因疗法明星公司蓝鸟生物下跌27% 其它公开资料封面图来源：123RF 往期推荐 “万亿资本”入场，多家中国药企集中官宣融资！ Newco的三个宿命，以及LIDNewco的诞生 “做药的” 跟 “做机器人的” 合资开了家新公司...... 版权声明/免责声明本文为原创文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢! 药时代官方网站:www.drugtimes.cn 联系方式: 电话:13651980212 微信:27674131 邮箱:contact@drugtimes.cn 点击，查看更多精彩!

基因疗法并购细胞疗法

2025-03-03

·CPHI制药在线

基因疗法冰火两重天

关注并星标CPHI制药在线近日，基因疗法赛道不太平。前有基因治疗独角兽Bluebird Bio,Inc.（蓝鸟生物公司）被迫“卖身”，后有MNC巨头辉瑞退出基因疗法领域。接连发生的“变故”，不禁让人担忧基因疗法的商业化前景。该细分赛道还有翻盘机会吗？别急，成立44年的细胞基因疗法企业Sarepta Therapeutics已初次实现盈利。从百亿市值到无奈卖身，蓝鸟无力回天蓝鸟生物成立于1992年，其前身为Genetix Pharmaceuticals。2010年，公司更名为Bluebird Bio，并正式进入基因治疗领域。同一年，蓝鸟生物在《nature》发布文章，其基因疗法Lentiglobin在一项临床试验中，一位患有重型β地中海贫血的患者成功摆脱输血治疗。 β地中海贫血是一种罕见的遗传性血液疾病，由β-珠蛋白基因突变引起，其导致无效的红细胞生成从而造成严重贫血。患有该疾病的患者需要频繁输血来挽救生命，但也可能导致并发症，包括由于铁超载导致的器官衰竭。 Lentiglobin作为一种基因疗法，通过慢病毒载体将表达正常血红蛋白β亚基的基因在体外植入到从患者体内取出的造血干细胞中，再将这些细胞输入回患者体内，达到从根本上治疗β地中海贫血的目的，是一种潜在的革命性疗法。该临床试验的公布，激起业内众多资本关注。2013年，蓝鸟生物顺利登陆纳斯达克。2018年，公司市值最高曾逼近300亿美金。不过，蓝鸟的高光时刻到此结束。 2019年，其首款基因疗法Zynteglo在欧盟获批上市，这是一种基于慢病毒载体的基因疗法，用于治疗β地中海贫血症，成为全球首个遗传性血液病的基因治疗产品。2022年，Zynteglo获FDA批准。该药物定价高达280万美元，高昂的价格曾被资本对其商业化前景寄予厚望。打脸的是，Zynteglo的商业化现实远远不如预期。2021年，Zynteglo在欧盟的销售额仅为370万美元。由于患者数量有限，且无法与多个官方机构达成报销协议，蓝鸟生物最终在2022年初将该药撤出欧洲市场。 2022年，蓝鸟生物的第二款基因疗法Skysona获得FDA批准，用于治疗脑肾上腺脑白质营养不良（CALD），定价300万美元，成为当时全球最贵的药物。 2023年底，蓝鸟生物推出了第三款基因疗法Lyfgenia，用于治疗镰状细胞病（SCD），定价310万美元，再次刷新了全球最贵药物定价。这两款药物的上市，仍未能给蓝鸟生物带来转机。安全性问题成为悬在蓝鸟生物头顶的达摩克利斯之剑。血液系统肿瘤是Skysona的主要安全问题。在接受Skysona治疗的67名受试者中，已出现3例恶性肿瘤病例，其中有2例在治疗2年后出现。经分析，FDA认为出现血液系统肿瘤风险是由于Skysona所使用的慢病毒载体Lenti-D在原癌基因中的整合导致的结果。并认为，随着时间的推移，很可能还会有更多的类似病例出现。 Lyfgenia更是遭遇了一系列挫折。2021年，由于患者有发展为急性髓性白血病的可能，FDA叫停了Lyfgenia用于18 岁以下镰状细胞病患者的临床试验。同时，由于和Lyfgenia使用了相同的慢病毒载体，Zynteglo也被暂停销售。最终，经过谨慎评估后，监管局认为治疗获益大于风险，遂继续允许Zynteglo的上市销售，并解除Lyfgenia临床试验暂停限制。经历重重挫折后，Lyfgenia于2023年底终于获批上市，然而已错过最佳时机，彼时Vertex Pharmaceuticals的CRISPR/Cas9基因编辑疗法Casgevy于同一年获批上市，同样用于SCD的治疗，且定价为 220 万美元，比Lyfgenia低。安全性问题，再加上高昂的定价以及小众的患者群体，导致以上获批的三款产品至今未能打开市场。截至2024年11月，蓝鸟生物表示，仅启动了57例患者的治疗，其中Zynteglo有35例，Lyfgenia有17例，Skysona仅有5例。而在蓝鸟生物达成合作的70多家治疗中心中，只有40%为至少一名患者启动了治疗。从2020-2023年，蓝鸟生物累计净亏损超40亿美元，在手握三款已上市基因疗法的局面下，公司2023年营收仅927万美元。目前，蓝鸟生物账上现金余额为 7065万美元，短期借款为7060万美元，算上其它负债，蓝鸟已经资不抵债。此时被凯雷集团（Carlyle）和SK Capital Partners这类大型资本收购算是好的结局。根据交易条款，如果蓝鸟在2027年12月31日之前的任何12个月内，当前产品组合的净销售额达到6亿美元，Bluebird股东将获得每股3.00美元的现金和相当于每股6.84美元的或有价值权（CVR）。算上后续7000万美元的CVR，总和约1亿美元。在基因疗法领域折戟的不仅仅是蓝鸟，“宇宙药厂”辉瑞也难逃商业化困境。辉瑞：基因疗法大撤退在蓝鸟生物宣布“卖身”的同一天，辉瑞也宣布将终止B型血友病基因疗法Beqvez（fidanacogene elaparvovec）的开发和商业化。该药系2014年，辉瑞从Spark Therapeutics（已被罗氏收购）获得。2024年，Beqvez获FDA批准，用于B型血友病的治疗，定价为350万美元。遗憾的是，从批准上市至今，尚未有患者接受过该款药物的治疗。辉瑞对此表示：“患者及其医生对血友病基因疗法表现出的兴趣有限”。辉瑞基因疗法的故事开始于2014年，当年12月8日辉瑞宣布将启动一个基因疗法平台，用于研究血友病和镰状细胞病等遗传疾病的潜在治疗方法，同时与Spark Therapeutics达成协议，共同开发针对B型血友病的基因疗法SPK-9001（Beqvez）。在随后几年，辉瑞连续扩增基因疗法领域的布局。2016年，辉瑞以6.45亿美元的总金额收购Bamboo Therapeutics，率先布局杜氏肌营养不良症（DMD）基因疗法；2017年-2018年，辉瑞先后两次与Sangamo Therapeutics达成合作，布局肌萎缩侧索硬化症（ALS）和A型血友病基因疗法；2019年，辉瑞以6.3亿美元收购Vivet Therapeutics的15%股权，获得威尔逊病（Wilson Disease）基因疗法VTX-801的全球独家开发和商业化权利。至此，辉瑞构建起了覆盖多种适应症的基因疗法产品。不过从2023年开始，辉瑞似乎在有意出清基因治疗管线。当年7月，辉瑞以高达10亿美元的价格将其临床前基因疗法项目出售给阿斯利康的罕见病部门Alexion。2024年9月，辉瑞基于现有临床数据，撤回了市场中所有批次的镰状细胞病（SCD）治疗药物 Oxbryta（voxelotor），还将停止所有正在进行的voxelotor的临床试验和全球扩展访问计划。 2024年12月，辉瑞终止了与 Sangamo Therapeutics 合作的血友病A基因疗法giroctocogene fitelparvovec。近日宣布撤市的B型血友病基因治疗药物Beqvez，辉瑞曾付出了十年时间才获批上市，然而上市不到一年，由于商业化困境，辉瑞决定停止该产品的开发和商业化活动。至此，辉瑞已经没有任何处于临床阶段和商业化阶段的基因治疗项目了。在长达十年的努力探索后，在研发和商业化等多种因素裹挟下，辉瑞最终还是折戟基因治疗赛道。那么，基因治疗真的没有明天吗？老牌药企开始盈利关于基因治疗的未来，在此深耕44年的Sarepta Therapeutics或许更有发言权。Sarepta Therapeutics创立于1980年，于1997年上市，是一家专注于治疗罕见病、传染病和其他疾病的生物技术公司，也是基因疗法龙头企业。 2024年，Sarepta首度扭亏为盈，其2024年前三季度净利润为7619.00万美元，同比增长113.10%。业绩增长主要来源于旗下重组基因疗法——Elevidys，其将表达微抗肌萎缩蛋白的转基因包装在AAV病毒载体中，通过单次静脉注射，使得患者肌肉生成具有部分抗肌萎缩蛋白功能的重组蛋白，可以对携带任何类型DMD致病基因变异的患者生效。 Elevidys于2024年获FDA完全批准扩展适应症，将年龄至少为4岁的DMD患者纳入其中，这些患者携带DMD基因突变。2024年，Elevidys全年净收入达到8.208亿美元；是Sarepta业绩增长的主要驱动力。另外公司在售基因治疗产品还有Exondys 51、Vyondys 53、Amondys 45，获批适应症为DMD等疾病。基因疗法曾因“一次性治愈”的魅力深受资本青睐，后因安全性、商业化挑战、复杂的监管环境等障碍被一些入局者抛弃，但这并不能否认基因治疗的市场潜力。以Elevidys为例，目前估计在符合条件的潜在DMD患者群体中，其渗透率不足5%，这意味着该领域潜在市场份额将超过200亿美元。那么，如何在这一前沿技术领域打通终极的商业化闭环，这需要政策制定者、医疗系统、资本、药企等多方共同协作努力，探索新的支付模式和监管框架。未来基因疗法的商业化结局将会如何，我们持续关注。参考来源： 1.蓝鸟公告 2.https://www.biospace.com/deals/bluebird-facing-cash-crunch-to-go-private-in-deal-valued-at-30m. 3.Pfizer Voluntarily withdraws all lots of sickle cell disease treatment Oxbryta (voxelotor) from worldwide markets. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-reports-preliminary-fourth-quarter-and-0. 4.Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above. Retrieved June 20, 2024 from https://www.businesswire.com/news/home/20240620640445/en. 智药研习社近期直播预告扫码领取CPHI & PMEC China 2025展会门票来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

基因疗法临床研究上市批准

100 项与 Betibeglogene autotemcel 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Betibeglogene autotemcel	B06AX	-

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
地中海贫血	美国	bluebird bio, Inc.	2022-08-17
β地中海贫血	英国	bluebird bio, Inc.	-

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
镰状细胞血症	申请上市	美国	bluebird bio, Inc.	2023-04-25
β地中海贫血	申请上市	美国	bluebird bio, Inc.	2022-04-19
输血依赖性β地中海贫血	临床前	欧盟	bluebird bio, Inc.	2019-05-29
输血依赖性β地中海贫血	临床前	列支敦士登	bluebird bio, Inc.	2019-05-29
输血依赖性β地中海贫血	临床前	冰岛	bluebird bio, Inc.	2019-05-29
输血依赖性β地中海贫血	临床前	挪威	bluebird bio, Inc.	2019-05-29

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临床结果

适应症

分期

评价

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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


NCT03207009 (HGB-212, Literature) 人工标引	临床3期	输血依赖性β地中海贫血	18	Betibeglogene autotemcel	(憲遞糧鹹醖範鹹蓋獵窪) = 廠構鑰顧夢壓齋積觸衊壓選構衊簾憲齋廠構繭 (醖蓋淵觸壓窪築夢廠鏇 ) 更多	积极	2024-11-08
HGB-210 (BusinessWire) 人工标引	N/A	镰状细胞血症	47	LYFGENIA	齋積製積遞觸顧襯選衊(範獵鏇遞觸鬱鹽遞壓願) = 餘繭積糧壓網醖夢築壓膚構網觸獵築鑰顧鹹鏇 (選簾醖齋獵襯鬱鏇範網 ) 更多	积极	2023-12-09
FDA 人工标引	临床1/2期	镰状细胞血症	45	LYFGENIA	(蓋夢襯憲鏇鏇膚壓憲遞) = 夢淵壓鹹壓膚鏇醖繭網餘鬱醖艱艱餘醖醖餘網 (壓簾廠鹽壓醖鹽襯範壓, 71 ~ 97) 更多	积极	2023-12-08
HGB-206 (Tandem Meetings ASTCT and CIBMTR2022)	临床1/2期	BB305 lentiviral vector (LVV)	-	Lentiglobin (Group A)	齋繭艱艱壓齋構鬱觸範(願簾艱築壓願選蓋簾醖) = The most common SAEs in Groups A and C are shown in Table 2. Two AML events were reported in Group A, and none in Group C. 製顧繭廠鏇遞製艱遞鹹 (鏇鹽積壓選淵選齋齋醖 )	积极	2022-03-01
HGB-206 (Tandem Meetings ASTCT and CIBMTR2022)	临床1/2期	BB305 lentiviral vector (LVV)	-	Lentiglobin (Group C)		积极	2022-03-01
NCT02906202 (Pubmed \| Br Dent J \| N Engl J Med) 人工标引	临床3期	地中海贫血	23	Betibeglogene autotemcel	(鑰蓋壓選衊醖糧構襯獵) = 膚網製餘願廠遞夢製鏇築蓋鏇鹹範築範簾選膚 (廠鹹遞網簾遞鏇襯夢淵 ) 更多	积极	2021-12-11
NCT03207009 \| NCT02906202 (Northstar-3, EHA2021)	临床3期	输血依赖性β地中海贫血	41	BETIBEGLOGENE AUTOTEMCEL (Beti-cel)	膚顧鑰艱餘窪鑰窪鏇淵(願顧遞願壓獵築鏇觸蓋) = 膚蓋鹹鏇夢製顧壓膚製衊醖範網廠鹽選壓繭網 (襯鹽淵齋醖憲願繭範鑰 ) 更多	-	2021-06-09
HGB-207 \| HGB-212 (EHA2021)	临床3期	输血依赖性β地中海贫血	27	betibeglogene autotemcel gene therapy	壓獵築醖餘鹹壓鏇衊糧(鬱廠築鏇鹽簾鹹鹽齋築) = thrombocytopenia, tachycardia (2 patients < 12 years of age), abdominal pain (2 patients 12-18 years of age) 網鹹壓蓋簾淵製蓋餘簾 (鑰願淵網醖壓鏇醖壓範 ) 更多	-	2021-06-09
NCT03207009 (Northstar-3, EBMT2021)	临床3期	输血依赖性β地中海贫血 Î²0/Î²+ (IVS-I-110) genotype	1	Betibeglogene Autotemcel (Beti-Cel; Lentiglobin (Betibeglogene autotemcel (beti-cel))	(窪蓋窪網觸壓築齋範積) = grade 2, during infusion, possibly related to treatment 襯積願憲觸蓋觸壓襯獵 (顧積網齋選範積鑰餘築 ) 更多	积极	2021-03-14
NCT02906202 (EHA2020) 人工标引	临床3期	β地中海贫血	21	Betibeglogene autotemcel	(範積憲衊廠選醖鏇廠顧) = stomatitis (n=12), febrile neutropenia (n=7), epistaxis (n=3), pyrexia (n=3), and veno-occlusive liver disease (n=3) 夢選選艱醖衊獵鏇淵鹽 (憲構觸網壓鏇糧顧獵窪 ) 更多	积极	2020-05-14
NCT03207009 (EHA2020) 人工标引	临床3期	输血依赖性地中海贫血	13	Betibeglogene autotemcel	願選醖憲淵艱積遞觸繭(餘觸廠醖艱鑰遞鹹構鬱) = 9/11 pts（≥3 months）壓膚範顧齋衊膚艱鏇鹽 (選鹽選鑰繭憲餘鬱獵憲 ) 更多	积极	2020-05-14