Lifileucel

1Q26 Total Revenue of ~$71M Delivers ~ 45% Year-over-Year Growth 2Q26 Revenue Guidance of $86M to $88M and FY26 of $350M to $370M 40% Confirmed Objective Response Rate in Metastatic Serous Endometrial Cancer SAN CARLOS, Calif., May 07, 2026 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today reported first quarter 2026 financial results, business achievements, and corporate updates. Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, “We are accelerating the adoption and commercial expansion for Amtagvi after record high demand. Iovance is well positioned through 2026 for long-term revenue growth, while advancing multiple ongoing and new clinical trials, including our registrational trial in advanced sarcomas now underway and encouraging initial data reported today for li in metastatic serous endometrial cancer. Internal manufacturing efficiencies, operational improvements, and cost reductions will benefit gross margin and propel future profitability, sustainable growth, and long-term value for patients and shareholders.” First Quarter 2026 Financial Highlights Continued Strength in Execution and Cost Discipline Total product revenue of ~$71 million increased by ~45% over 1Q25, reflecting significant performance improvements over the prior annual maintenance period. U.S. Amtagvi revenue was ~$60 million. Global Proleukin revenue was ~$11 million. Gross margin of 41% absorbed one-time costs for the annual maintenance period and the recent internal facility expansion. Consistent with 1Q25, revenue was affected by maintenance of the Iovance Cell Therapy Center ( i CTC). The facility has now been expanded to ensure continuous supply going forward during future maintenance periods. Research and Development (R&D) expenses decreased by 12% compared to 4Q25, driven by operational efficiencies and marking the third consecutive quarter of improvements. Successful centralization of manufacturing at i CTC, significant operational excellence initiatives focused on Amtagvi production, and R&D optimization should further reduce costs and improve gross margins in 2026 and 2027. Second Quarter 2026 and Full Year 2026 Guidance Strong Growth in Amtagvi Forecast for 2026 Total product revenue guidance for 2Q26 is $86 million to $88 million and for FY26 is $350 million to $370 million. U.S. Amtagvi revenue for 2Q26 is expected to be $79 million to $81 million, reflecting an expected ~23% increase over 4Q25 (the quarter prior to i CTC maintenance). Amtagvi Commercial Business Strong U.S. Commercial Business to Deliver Strong Growth in 2026 Increasing Amtagvi demand, catalyzed by real-world data, is driven by adoption and referrals toward earlier treatment. Recently published real world objective response rates were 52% in patients with two or fewer prior lines of therapy. Five-year follow-up clinical data demonstrated deep and durable responses in heavily pretreated patients, with a median duration of response of 3 years. Demand and referral patterns are accelerating across a growing network of more than 90 U.S. and Canadian academic and community authorized treatment centers (ATCs). By year-end 2026, at least 110 ATCs will be activated. Amtagvi turnaround time is 32 days or less with the first scaled, centralized commercial manufacturing process for TIL therapy. This is significantly faster than any other TIL therapy in development. Amtagvi global expansion is advancing: Decisions on marketing authorization application (MAA) approvals are expected in Australia in the first half of 2026 and in Switzerland in the first half of 2027. In the United Kingdom, Iovance withdrew its initial MAA for li in May 2026 for procedural reasons. With the full agreement of the Medicines and Healthcare products Regulatory Agency (MHRA), Iovance will promptly resubmit the MAA with updated information for an expedited review by the MHRA, which is expected to be completed over the coming months. Iovance is working to resubmit an MAA to the European Medicines Agency (EMA) in 2026. Other regulatory submissions are planned in markets with a high prevalence of advanced melanoma, non-small cell lung cancer (NSCLC), and soft tissue sarcomas. Pipeline Updates New Data Across Several Pipeline Programs Anticipated Throughout 2026 Registrational Trials of Li Treatment in Solid Tumors IOV-END-201: Positive initial data in previously treated metastatic serous endometrial cancer: The confirmed objective response rate (cORR) by RECIST v1.1 was 40% and disease control rate was 100% in the first five evaluable patients with a median of 2 prior lines of therapy. All five patients were mismatch repair proficient and progressed on prior chemotherapy and checkpoint inhibitor therapy. These initial responses build on established differentiation of li from immune checkpoint inhibitors, including in melanoma, and demonstrate its advantages for solid tumor indications. Serous endometrial cancer is a difficult to treat subtype accounting for ~40% of the approximately 12,500 annual U.S. endometrial cancer deaths. 1 The second line setting represents an area of unmet medical need, with no therapy approved by FDA specifically for patients with serous endometrial carcinoma or for patients who have received prior PD-1 blocking antibodies. Engagement on an expedited approval pathway with the ongoing IOV-END-201 trial is planned with the U.S. Food and Drug Administration (FDA). IOV-LUN-202: initial results in previously treated, metastatic non-squamous NSCLC supported FDA Fast Track Designation, reflecting the high unmet medical need in this population. Upcoming milestones include: Updated data at a major medical meeting in 2026. Completion of enrollment in 2026 to support a supplemental Biologics License Application (sBLA). Potential for a U.S. accelerated approval and launch in the second half of 2027. IOV-SAR-201: a new registrational trial in undifferentiated pleomorphic sarcoma (UPS) and dedifferentiated liposarcoma (DDLPS) is now underway, driven by positive early data with a cORR of 50% in the first six evaluable patients. Site activation and enrollment are on track to begin in the third quarter of 2026. Iovance is actively engaging with FDA on a path to expedited approval for li in UPS and DDLPS. TILVANCE-301: A Phase 3 randomized trial of li and pembrolizumab in frontline advanced melanoma. Sites are actively enrolling patients across a broad global footprint. An early interim analysis based on cORR is intended for a potential sBLA in frontline advanced melanoma. TILVANCE-301 is also the confirmatory trial to support full approval in second line advanced melanoma. Next Generation Pipeline An Investigational New Drug (IND) application was submitted to FDA for a Phase 1/2 basket trial of IOV-5001, a second-generation IL-12 tethered TIL therapy, to begin enrolling in 2H 2026. Cohorts include advanced colorectal cancer, triple negative and estrogen receptor low breast cancers, and other highly prevalent solid tumors representing more than 100,000 U.S. deaths annually. 2 IOV-5001 is designed to remodel the suppressive tumor microenvironment (TME) and activate immunologically cold tumors to support TIL responses. A first-generation IL-12 secreted TIL therapy showed a cORR of 63% in 16 melanoma patients at cell doses much lower than used with typical TIL therapies as well as those safely achievable with IOV-5001. 3 A Phase 1/2 trial, IOV-GM1-201, is enrolling using IOV-4001, a PD-1 inactivated TIL therapy, in previously treated advanced melanoma and NSCLC. IOV-4001 is engineered to resist inhibitory signals and enhance the ability of TIL therapies to fight and kill cancer in the TME. A Phase 1 safety cohort using IOV-3001 is advancing through multiple dose levels in the Phase 1/2 trial of our second-generation, modified IL-2 analog for the TIL treatment regimen. IOV-3001 selectively expands effector T cells while avoiding activation of regulatory T cells, with the potential for a lower dose IL-2 regimen with reduced adverse events. IOV-3001 exhibits favorable pharmacokinetics and is expected to be superior to Proleukin as a component of future TIL regimens. Multiple investigator-sponsored clinical trials of li are enrolling in cutaneous squamous and Merkel cell carcinomas as well as other new solid tumor indications. Corporate Updates Iovance currently owns or licenses nearly 400 granted or allowed U.S. and international patents and patent rights for Amtagvi and other TIL-related technologies, as well as more than 1,000 patent applications worldwide, which are expected to provide exclusivity into 2042 for Amtagvi and beyond for pipeline therapies. Dr. Friedrich Graf Finckenstein, Chief Medical Officer, will retire from Iovance in June 2026. The company thanks Dr. Finckenstein for his service and contributions to the development of Amtagvi and other pipeline products. A new Chief Medical Officer is expected to be announced in the near term. Iovance’s cash position was ~$319 million on March 31, 2026. 4 The current cash position, bolstered by expense reductions, is expected to fund operations well into 2028. Webcast and Conference Call Management will host a conference call and live audio webcast to discuss these results and provide a corporate update today at 8:30 a.m. ET. To listen to the live or archived audio webcast, please register at . The live and archived webcast can be accessed in the Investors section of the Company’s website, IR.Iovance.com, for one year. 1.Hamilton, C., Cheung, M., Osann, K. et al. Uterine papillary serous and clear cell carcinomas predict for poorer survival compared to grade 3 endometrioid corpus cancers. Br J Cancer 94, 642–646 2. Surveillance, Epidemiology, and End Results Program Cancer Stat Facts (accessed May 2026). 3. Zhang L, Rosenberg SA, et al, Clin Cancer Res 2015;21(10):2278–2288. 4. Cash, cash equivalents, short-term investments, and restricted cash as of March 31, 2026. About Iovance Biotherapeutics, Inc. Iovance Biotherapeutics, Inc. aims to be the global leader in innovating, developing, and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. Iovance’s Amtagvi ® is the first FDA-approved T cell therapy for a solid tumor indication. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer. For more information, please visit . Amtagvi ® and its accompanying design marks, Proleukin ® , Iovance ® , and IovanceCares™ are trademarks and registered trademarks of Iovance Biotherapeutics, Inc. or its subsidiaries. All other trademarks and registered trademarks are the property of their respective owners. Information on Iovance’s broad, industry-leading patent portfolio is available on the Intellectual Property page on . Forward-Looking Statements Certain matters discussed in this press release are “forward-looking statements” of Iovance Biotherapeutics, Inc. (hereinafter referred to as the “Company,” “we,” “us,” or “our”) within the meaning of the Private Securities Litigation Reform Act of 1995 (the “PSLRA”). Without limiting the foregoing, we may, in some cases, use terms such as “predicts,” “believes,” “potential,” “achievable,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “forecast,” “guidance,” “outlook,” “may,” “can,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes and are intended to identify forward-looking statements. Forward-looking statements are based on assumptions and assessments made in light of management’s experience and perception of historical trends, current conditions, expected future developments, and other factors believed to be appropriate. Forward-looking statements in this press release are made as of the date of this press release, and we undertake no duty to update or revise any such statements, whether as a result of new information, future events or otherwise. Forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties, and other factors, many of which are outside of our control, that may cause actual results, levels of activity, performance, achievements, and developments to be materially different from those expressed in or implied by these forward-looking statements. Important factors that could cause actual results, developments, and business decisions to differ materially from forward-looking statements are described in the sections titled "Risk Factors" in our filings with the U.S. Securities and Exchange Commission, including our most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, and include, but are not limited to, the following substantial known and unknown risks and uncertainties inherent in our business: the risks related to our ability to successfully commercialize our products; the acceptance by the market of our products and product candidates, if approved, and their potential pricing and/or reimbursement by payors, and whether such acceptance is sufficient to support continued commercialization or development of our products or product candidates; the risk regarding our ability to manufacture our therapies at our i CTC facility, including the risk that our ability to increase manufacturing capacity at our facility may adversely affect our commercial launch; the risk that the successful development or commercialization of our products may not generate sufficient revenue from product sales, and we may not become profitable in the near term, or at all; the risks related to the timing of and our ability to successfully develop, submit, obtain, or maintain regulatory authority approval of our product candidates; whether clinical trial results from our pivotal studies and cohorts, and meetings with regulatory authorities may support registrational studies and subsequent approvals by regulatory authorities, including the risk that the planned registrational trial in advanced sarcomas may not support approval; preliminary and interim clinical results, which may include efficacy and safety results, from ongoing clinical trials or cohorts may not be reflected in the final analyses of our ongoing clinical trials or subgroups within these trials or in other prior trials or cohorts; the risk that we may be required to conduct additional clinical trials or modify ongoing or future clinical trials based on feedback from regulatory authorities; the risk that our interpretation of the results of our clinical trials or communications with regulatory authorities may differ from the interpretation of such results or communications by such regulatory authorities; the risk that clinical data from ongoing clinical trials of Amtagvi will not continue or be repeated in ongoing or planned clinical trials or may not support regulatory approval or renewal of authorization; the risk that unanticipated expenses may decrease our estimated cash balances and forecasts and increase our estimated capital requirements; the risk that we may not be able to recognize revenue for our products; the risk that Proleukin revenues, and other factors such as the number of ATCs, may not serve as a leading indicator for Amtagvi revenues; the risks regarding our anticipated operating and financial performance, including our financial guidance and projections; the effects of global and domestic geopolitical factors or public health events; and other factors, including general economic conditions and regulatory developments, not within our control. Any financial guidance provided in this press release assumes the following: no material change in our ability to manufacture our products; no material change in payor coverage; no material change in revenue recognition policies; no new business development transactions not completed as of the period covered by this press release; and no material fluctuation in exchange rates. IOVANCE BIOTHERAPEUTICS, INC. Selected Condensed Consolidated Balance Sheets (in thousands) March 31, 2026 (unaudited) December 31, 2025 Cash, cash equivalents, and investments $ 313,443 $ 296,980 Restricted cash $ 5,992 $ 5,980 Total assets $ 925,665 $ 913,170 Stockholders' equity $ 721,754 $ 698,583 Condensed Consolidated Statements of Operations (in thousands, except per share information) For the Three Months Ended March 31, 2026 (unaudited) 2025 (unaudited) Revenue Product revenue, net $ 71,430 $ 49,324 Total revenue 71,430 49,324 Costs and expenses* Cost of sales ** $ 42,498 $ 42,715 Research and development ** 62,487 75,965 Selling, general and administrative ** 38,949 43,800 Depreciation and amortization 8,539 8,065 Total costs and expenses 152,473 170,545 Loss from operations (81,043 ) (121,221 ) Other income Interest and other income, net 1,333 3,220 Net Loss before income taxes (79,710 ) (118,001 ) Income tax (expense) benefit 665 1,838 Net Loss $ (79,045 ) $ (116,163 ) Net Loss Per Share of Common Stock, Basic and Diluted $ (0.19 ) $ (0.36 ) Weighted-Average Shares of Common Stock Outstanding, Basic and Diluted 418,511 322,868 *Non-cash stock-based compensation included in cost of sales and operating expenses: Cost of sales $ 1,019 $ 2,420 Research and development 5,117 9,917 Selling, general and administrative 5,133 10,578 Total stock-based compensation included in costs and expenses $ 11,269 $ 22,915 ** Excludes depreciation and amortization CONTACTS Investors IR@iovance.com 650-260-7120 ext. 150 Media PR@iovance.com 650-260-7120 ext. 150

说到治病吃药，大家最先想到的都是药片、针剂，可如今医学界早已迎来全新突破——细胞治疗，作为继小分子化学药、大分子抗体药之后的第三代生物医药核心技术，正在彻底改变难治性疾病的治疗格局。 不同于传统药物只能对症缓解、长期服药，细胞治疗是把活细胞当成“药物”，将自体或异体健康细胞，经过体外改造、扩增、筛选后，再回输到患者体内，通过细胞替代、精准杀伤、免疫调节三种方式，从根源上干预肿瘤、遗传病、退行性疾病等疑难病症，不少重症患者甚至能靠它实现长期缓解、甚至临床治愈。 截至2026年5月，全球已有超20款细胞治疗药物获主流药监机构批准上市，国内也已有8款CAR-T细胞治疗产品正式获批，数百款新药进入临床冲刺阶段。今天就用大白话讲清楚，细胞治疗到底分哪几类，哪些药已经能在医院用上，哪些新药未来几年有望落地，普通人也能一眼看懂。 目前细胞治疗主要分为三大类，每一类都对应着不同的疾病治疗方向，也有着各自成熟的上市药物和前沿在研管线。 一、免疫细胞治疗：肿瘤患者的“生命希望”，商业化最成熟 免疫细胞治疗是当下临床应用最多、获批产品最丰富的赛道，核心就是帮人体重启免疫系统，精准干掉癌细胞，尤其在肿瘤治疗上效果惊人，也是目前细胞治疗里上市药物最多的品类。 1. CAR-T细胞治疗 ：血液肿瘤“克星”，上市产品最丰富 CAR-T可以理解为给免疫T细胞装上了“GPS导航定位器”，通过基因工程改造，让T细胞能绕过癌细胞的伪装，直接精准找到肿瘤细胞，不留死角地将其消灭。它对血液肿瘤疗效格外显著，复发难治性白血病、淋巴瘤、多发性骨髓瘤患者，总缓解率能达到80%-90%，也是目前唯一实现大规模商业化的基因修饰型细胞治疗技术。 【已正式获批上市的药物】 目前全球已有超15款CAR-T产品获批上市，截至2026年5月，中国NMPA已批准8款CAR-T产品，覆盖淋巴瘤、白血病、多发性骨髓瘤三大核心适应症，患者在国内三甲医院就能规范使用： · 国内首款获批CAR-T：阿基仑赛注射液，靶向CD19，用于复发难治性大B细胞淋巴瘤； · 国产全流程自主研发首款：伊基奥仑赛注射液（商品名福可苏®），靶向BCMA，于2023年6月在国内获批，用于复发难治性多发性骨髓瘤； · 首款出海的国产CAR-T：西达基奥仑赛注射液（商品名Carvykti），靶向BCMA，2022年2月获FDA批准上市，2024年8月获NMPA批准，多发性骨髓瘤客观缓解率高达98%； · 其他获批产品：瑞基奥仑赛注射液、纳基奥仑赛注射液（曾用名赫基仑赛，是国内首个获批治疗白血病的CAR-T产品，成人适应症2023年11月获批） 、泽沃基奥仑赛注射液、雷尼基奥仑赛注射液、普基奥仑赛注射液， 均已在国内正式上市； · 全球首款获批CAR-T：诺华的Kymriah，2017年获FDA批准，开启了细胞治疗的商业化时代，适应症覆盖儿童急性淋巴细胞白血病、大B细胞淋巴瘤。 【重磅临床在研项目（2026年关键突破）】 目前CAR-T的研发核心，集中在攻克实体瘤、通用型现货产品两大方向，多款新药已进入上市冲刺阶段： · 全球首款实体瘤CAR-T：科济药业舒瑞基奥仑赛注射液（CT041），靶向CLDN18.2，2025年5月被NMPA纳入优先审评，2025年6月上市申请获CDE受理，预计2026年第二季度正式获批，适应症为晚期胃癌/食管胃结合部腺癌，III期临床数据显示，较标准治疗显著延长无进展生存期（mPFS 3.25个月 vs 1.77个月，HR 0.366） ，将打破CAR-T仅对血液肿瘤有效的行业瓶颈； · 胃癌治疗新选择：易慕峰生物IMC002，同样靶向CLDN18.2，针对晚期胃癌的临床数据显示，据企业披露， 客观缓解率达到66.7%，其中有患者肿瘤病灶完全消失，完全缓解状态持续超60周； · 通用型CAR-T突破：茂行生物MT026，针对复发性脑胶质瘤的临床数据据披露登顶国际顶刊，MT027获FDA批准开展II期临床，无需患者自体细胞定制，可实现现货量产，攻克了传统CAR-T“一人一药”的痛点； · 国内首款体内CAR-T：SYS6055注射液，2026年1月底获批临床，能在患者体内直接生成CAR-T细胞，无需体外制备，大幅降低治疗成本和周期。 2.TCR-T细胞治疗 ：实体瘤新希望，上市冲刺进行中 和CAR-T不同，TCR-T能识别癌细胞内部的抗原，完美填补了CAR-T无法靶向胞内靶点的空白，理论上可靶向肿瘤细胞中超过90%的蛋白质，是攻克实体瘤的核心突破路线。 【上市情况】 截至2026年5月，全球暂无正式获批上市的TCR-T产品，国产新药处于全球第一研发梯队，最快有望在2026-2027年实现全球首款产品落地。 【重磅临床在研项目】 · 国产首款冲刺上市：香雪生命科学TAEST16001，针对软组织肉瘤，已进入确证性临床关键阶段，是国内首个获得IND批件并纳入CDE突破性治疗品种名单的TCR-T产品，预计2026年提交NDA，同时布局了食管癌、肝癌、胃癌等多类实体瘤适应症； · 全球前沿技术：据研发团队披露，中科院团队研发的超级TCR技术，将细胞制备周期缩短至72小时，精准度提升50倍，成本降低80%，在胰腺癌治疗中肿瘤缩小率达到78%； · 星汉德生物SCG101、可瑞生物CRTE7A2-01等多款产品，已进入II/III期临床，针对乙肝相关肝癌、宫颈癌等实体瘤，均展现出优异的疗效和安全性。 3. TIL细胞治疗 ：天然抗癌“特种兵”，首款产品已落地 这是一种不用基因改造的免疫疗法，从患者自身肿瘤组织里，分离出天生能识别癌细胞的浸润T细胞，体外大规模扩增后再回输体内，能有效应对肿瘤异质性难题，是实体瘤治疗的又一核心赛道。 【已正式获批上市的药物】 全球首款、目前唯一获批上市的TIL产品，是美国Iovance公司的Lifileucel，2023年获FDA批准，适应症为晚期黑色素瘤，为传统治疗耐药的患者带来了长期生存希望。 【重磅临床在研项目】 国产TIL疗法已跻身全球前列，多款产品进入关键临床阶段： · 君赛生物GC101，是全球首款“免IL-2、免高强度清淋”的天然TIL疗法，核心适应症为黑色素瘤，已进入关键II期临床，预计2026年提交BLA，有望成为中国首个获批上市的TIL疗法；同时布局非小细胞肺癌，2026年3月公布Ib期临床数据并启动II期临床，相关数据入选2026年ASCO年会最高规格突破摘要，也是本届大会唯一获此殊荣的细胞治疗企业，打破了欧美长期垄断； · 据披露，沙砾生物GT101，是国内首个获批注册临床的TIL药物，针对宫颈癌已进入II期临床，预计2027年提交上市申请； · 此外，国内多款产品针对肝癌、结直肠癌、宫颈癌等实体瘤的III期临床正在推进，有望在未来2-3年实现国产TIL产品上市。 4.其他前沿免疫疗法 除了三大核心路线，还有多款免疫细胞疗法处于临床研发阶段，是未来行业的重要突破方向： · CAR-NK细胞治疗：给自然杀伤细胞（NK）装上导航，安全性比CAR-T更高，细胞因子风暴风险显著降低，无需严格配型，可实现标准化现货量产，目前全球暂无获批上市产品；2026年3月，恩瑞恺诺KN5501注射液获批临床，一次性获批原发性干燥综合征、系统性硬化症、难治性狼疮肾炎3项自身免疫病适应症，是全球首个获批临床的通用型CD19靶向CAR-NK疗法；此外，国健呈诺、药明巨诺、传奇生物的多款CAR-NK产品，已进入II/III期临床，覆盖血液肿瘤、实体瘤、自身免疫病三大领域； · CAR-M、Treg细胞、DC疫苗：均处于临床研发阶段，其中CAR-M在实体瘤组织穿透、Treg在红斑狼疮、类风湿关节炎等自身免疫病治疗领域，已展现出巨大潜力，多款产品进入I/II期临床。 二、干细胞治疗：人体“修复师”，专攻组织损伤与慢病 如果说免疫细胞是抗癌战士，那干细胞就是人体的“万能修复师”，依靠自我更新和多向分化能力，变成身体需要的各类功能细胞，修复受损器官、调节免疫炎症，和免疫细胞治疗形成完美互补，主要用于组织修复、慢病管理和退行性疾病治疗。 截至2026年3月，全球已有12款干细胞治疗产品经主流药监机构正式批准上市，国内已有185款干细胞药物获得NMPA临床试验默示许可，覆盖神经、代谢、骨科、生殖等多个系统的慢病与重症。 1. 间充质干细胞（MSC） ：应用最广泛的“万能细胞” 间充质干细胞来源非常广泛，脐带、骨髓、脂肪中都能提取，免疫原性低，不用严格配型，安全性极高，是全球获批数量最多的干细胞类型，核心作用为免疫调节、抗炎与组织修复。 【已正式获批上市的药物】 · 国内首款获批干细胞药物：天津昂赛细胞的人脐带间充质干细胞注射液，2024年获NMPA批准，适应症为激素难治性急性移植物抗宿主病（aGVHD）； · 全球获批产品：韩国Cartistem，用于膝骨关节炎，是全球首款获批的软骨修复干细胞药物；欧盟Alofisel，用于克罗恩病肛瘘；此外，美国、澳大利亚、日本还有多款MSC产品获批，覆盖急性心梗、呼吸窘迫综合征、骨关节炎等适应症，全球已有数款MSC产品获批。 【重磅临床在研项目】 · 膝骨关节炎治疗：据披露，贝来生物人脐带间充质干细胞注射液，完成了国内最大规模的III期临床，512例患者随访数据显示，患者疼痛评分降低68%，62%的患者通过MRI证实软骨再生，已完成III期临床试验，正推进新药上市申请（NDA） ，预计2026年第四季度获批，有望成为国内首款膝骨关节炎干细胞药物；西比曼生物异体脂肪间充质祖细胞注射液，也已进入III期临床，同步实现症状改善和软骨结构修复； · 慢病治疗突破：国内已有多款MSC产品获批临床，针对2型糖尿病、绝经后骨质疏松症、帕金森病、特发性肺纤维化、失代偿期肝硬化等高发慢病，部分产品已进入II期临床，展现出优异的组织修复和功能改善效果。 2.造血干细胞（HSC）治疗： 血液疾病“根治方案” 这是临床应用最久的经典干细胞技术，通过移植健康造血干细胞，重建患者的造血和免疫系统，是白血病、再生障碍性贫血、地中海贫血等血液系统重症的根治性手段之一，早已挽救无数患者生命。 【已正式获批上市的药物】 美国FDA批准的Hemacord，是全球首款获批的脐血造血干细胞产品，用于造血系统恶性肿瘤的移植治疗；国内也已有多款造血干细胞相关产品获批上市，用于血液系统疾病的临床治疗。 3. 诱导多能干细胞（iPSC）治疗 ：2026年迎来全球商业化里程碑 这项技术能把普通的成体细胞（比如皮肤细胞）逆转成干细胞状态，再定向分化成任意功能细胞，实现标准化、现货型量产，彻底解决细胞供体短缺的难题，2026年也被业内称为“iPSC商业化元年”。 【已正式获批上市的药物】 2026年，日本连续批准两款全球首创iPSC治疗产品，实现了行业里程碑式突破： · Amchepri（Amchepry®） ：全球首款获批的iPSC治疗产品，2026年3月6日获日本厚生劳动省有条件、限时批准，适应症为帕金森病，通过iPSC分化的多巴胺能神经元前体细胞植入脑部，替代受损神经元，临床试验显示患者细胞存活率超90% ，运动功能与纹状体多巴胺合成水平显著提升； · Reheart（RiHeart®） ：全球首款iPSC来源心肌细胞治疗产品，适应症为重症心力衰竭，通过微创植入心肌细胞薄片，修复受损心脏，患者1年内心功能显著改善。 【重磅临床在研项目】 国产iPSC研发处于全球第一梯队，士泽生物、中盛溯源、霍德生物等企业的多款产品，已进入临床阶段，覆盖帕金森病、重症心衰、脊髓损伤、角膜损伤等适应症，预计未来3-5年将迎来国产iPSC产品的上市爆发期。 4. 胚胎干细胞（ESC）治疗 截至2026年5月，全球暂无正式获批上市的胚胎干细胞产品，核心研发集中在神经退行性疾病领域：美国FDA批准的bemdaneprocel，针对帕金森病已在2026年1月启动III期关键性临床研究；据披露，国内赛隽生物的CG-BM1，针对阿尔茨海默病，2026年2月获批临床，是国内首款获批的阿尔茨海默病干细胞疗法。 三、其他体细胞治疗：精准攻克单一器官疾病 除了免疫细胞和干细胞，还有针对特定器官的细胞替代治疗，精准解决单病种疑难病症，多款产品已获批上市，成为特定疾病的根治性方案。 【已正式获批上市的药物】 · 胰岛细胞治疗：美国FDA 2023年获批的Lantidra，是全球首款异体胰岛细胞疗法，能实现1型糖尿病的功能性治愈，患者术后可彻底摆脱长期注射胰岛素控糖； · 软骨细胞治疗：美国MACI、欧盟ChondroCelect获批上市，用于膝关节软骨损伤修复，通过自体软骨细胞体外扩增后植入，修复软骨缺损，帮助患者恢复关节功能； · 角膜上皮细胞治疗：欧盟获批的Holoclar，用于中重度角膜缘干细胞缺乏症导致的失明，是全球首款获批的组织工程干细胞产品，该产品已于2015年前后在欧洲获批，帮助无数角膜损伤患者重见光明。 【重磅临床在研项目】 · 巨核细胞治疗：据披露，2026年3月，血霁生物的同种异体巨核细胞注射液，10天内连续获NMPA、FDA IND批准，适应症为血小板减少症，是全球首个同种异体巨核细胞治疗产品，无需基因编辑，可快速、长效提升患者血小板数量； · 国内多款胰岛细胞治疗产品已进入临床阶段，针对1型糖尿病，部分产品已实现患者术后脱离胰岛素，预计未来3年有望实现国产产品上市； · 国内多款软骨细胞、视网膜色素上皮细胞产品获批临床，针对膝关节损伤、黄斑变性等疾病，已进入II期临床阶段。 四、细胞治疗核心优势、现存挑战与未来趋势 核心优势 1. 潜在治愈性：很多传统治疗无效的难治性疾病，单次细胞输注就能实现长期缓解甚至根治，不用终身服药，这是传统药物无法比拟的优势； 2. 精准度高：能靶向传统药物无法触及的病灶和靶点，对晚期肿瘤、罕见遗传病、退行性疾病等“无药可医”的疾病，有着独一无二的治疗效果； 3. 长效持久：活细胞进入体内后能持续增殖、分化，长期发挥治疗作用，还能自适应身体的病理环境变化，实现长期的病情控制。 现存挑战 目前细胞治疗也面临两大核心难题：一是部分产品价格偏高，以某国产CAR-T产品为例，单次治疗价格约120万元，让不少家庭望而却步，不过随着国产药物密集上市、 以及医保谈判和商业保险创新支付体系的推进（2026年首版商保创新药目录已纳入5款CAR-T产品） ，价格正在大幅下探，未来可及性会持续提升；二是实体瘤治疗仍需全面突破，肿瘤微环境抑制、靶点稀缺、肿瘤异质性等问题，还需要技术进一步迭代升级。 未来发展方向 未来3-5年，细胞治疗会迎来全面爆发期：一是通用型现货细胞会成为核心竞争方向，不用再等待自体细胞制备，治疗成本会大幅降低，真正走进普通患者的治疗方案；二是CRISPR基因编辑技术会和细胞治疗深度融合，实现更精准的基因修饰，进一步提升治疗的疗效与安全性；三是适应症会全面拓展，从血液肿瘤、罕见病，向糖尿病、骨关节炎、帕金森病、阿尔茨海默病等高发慢病快速延伸，成为更多疾病的一线治疗方案。 细胞治疗这一医学黑科技，早已不是实验室里的概念，而是实实在在拯救生命的成熟疗法。随着技术不断迭代，未来将会有更多细胞药物上市，彻底改变人类对抗疾病的方式，让更多疑难病不再是不治之症！ #细胞治疗 #医学科普 #抗癌新技术 #健康科普 #新药前沿