UNION Therapeutics A/S

TORL BioTherapeutics , the antibody-drug conjugate startup led by former Celgene chief Mark Alles , has brought aboard a chief medical officer with big pharma chops. Aran Maree will be overseeing clinical development at the Los Angeles biotech, born out of Herceptin founder Dennis Slamon ’s lab at UCLA. Maree departs a nearly 20-year career at Johnson & Johnson , where he was chief medical officer of the medtech unit before stepping into the same post at the pharmaceuticals division, which was recently renamed J&J Innovative Medicine . The J&J veteran’s move over to TORL comes as the biotech is just weeks into its first potentially registrational trial in CLDN6 positive platinum-resistant ovarian cancer. The well-funded startup is testing an ADC called TORL-1-23. The pipeline also contains early-stage ADCs against CLDN18.2, CDH17 and DLK1. Other immunotherapies are also in the works. Alles and crew raised a $158 million round in April 2023 . Almost a year to the day, they raised another round of the exact same size. They’ve kept a low profile the entire time, wishing not to speak with the news media. — Kyle LaHucik → Samsung Bioepis has promoted Kyung-Ah Kim to president and CEO, becoming the first female exec in the Samsung universe to hold this title that isn’t part of the founding family. Kim has worked at the biosimilar maker since 2015 and had been development division leader for the past three years. Her predecessor, Christopher Hansung Ko , was CEO since its founding in February 2012 and will be the new leader of Samsung’s Future Business Division. The FDA approved Samsung Bioepis’ Soliris biosimilar Epysqli in July, and the company will soon launch its Stelara biosimilar in the US. → Iskra Reic will lead AstraZeneca ’s China business as EVP of international. She’ll take over for detained exec Leon Wang while he is still under investigation in China, a market that CEO Pascal Soriot called “an important part of innovation in the industry” last month. Reic is a 23-year AstraZeneca vet who ran the vaccines and immune therapies unit after a stint as EVP for Europe and Canada. → On the same day President-elect Donald Trump nominated Marty Makary to lead the FDA, a commish from his first administration picked up a board seat. Stephen Hahn — the current CEO of Flagship company Harbinger Health — has joined the board at Faeth Therapeutics , a cancer startup that lists Sid Mukherjee and Lewis Cantley as founders. Faeth has also appointed Rivus Pharmaceuticals CEO and ex- Aimmune boss Jayson Dallas to the board. “Faeth’s approach of leveraging metabolic research in combination with AI has opened up metabolism as a new pillar in the treatment of cancer,” Hahn said in a statement . → Franklin Biolabs , a contract research organization that Jim Wilson founded after leaving his leadership post at the University of Pennsylvania’s Gene Therapy Program, has installed a CEO. Vatsala Naageshwaran got promoted to head of business development for US CGT & lab services for Pharmaron and had a long career with Absorption Systems before it was sold to Pharmaron in late 2020. Brazilian health research institute Fiocruz made a commitment of up to $100 million for Wilson’s other venture, GEMMABio , in an effort to make gene therapies more accessible globally. → Scott Wolchko will retire as president and CEO of Fate Therapeutics on Jan. 1, and R&D chief Bob Valamehr will take over, according to a Black Friday release . Wolchko was the San Diego biotech’s first CFO and vaulted to the top spot in 2015, succeeding former Escient Pharmaceuticals chief Christian Weyer . Valamehr has led R&D since 2018 and his promotion will coincide with his 15th anniversary at the company. You may recall that Fate, which has several CAR-T and CAR NK candidates in its pipeline, ended its development deal with J&J in January 2023 and subsequently laid off nearly half of its employees. → Paragon Therapeutics continues to churn out public biotechs — and now has a CEO at the mother ship. Susanna High is the former COO of bluebird bio and, more recently, Dyne Therapeutics . She also had several roles that spanned more than nine years at Alnylam , including SVP, strategy and business integration. Two Paragon companies — Jade Biosciences and Crescent Biopharma — reverse merged their way onto the Nasdaq in October, and Oruka Therapeutics did the same in April. → Volker Herrmann is out as CEO of Flagship’s Sonata Therapeutics , and about 20 other staffers have also been let go, Fierce Biotech reported. Flagship brought two of its companies together, Inzen Therapeutics and Cygnal Therapeutics , to form Sonata two years ago. Herrmann had led Inzen, while Cygnal chief Pearl Huang eventually took the CEO job at Dunad Therapeutics . Herrmann spent more than 15 years at Pfizer and joined Flagship from SQZ Biotechnologies , where he was COO for nearly two years. → Arcutis Biotherapeutics medical chief Patrick Burnett and chief commercial officer Todd Edwards have both been promoted to EVP, and Kent Taylor has been elevated to SVP of sales. Burnett is a Novartis vet who left Verrica Pharmaceuticals to take the CMO job at Arcutis in August 2020; Peer Review informed you about Edwards’ appointment in September 2023, when he took over for Ken Lock after three years with Incyte ; and Taylor — an Incyte vet in his own right — followed Edwards two months later as VP of sales. → OSE Immunotherapeutics co-founder Dominique Costantini is retiring at the end of the year. The French I/O and I&I player has selected Fiona Olivier as chief corporate affairs and investor relations officer, and research head Aurore Morello earns a spot on the executive committee. Costantini, the former CEO who also chaired the board at OSE, had been director of development and strategy. “Many of us have benefited from Dominique’s mentorship and guidance over the years and we have grown stronger as a result,” CEO Nicolas Poirier said in a statement . Olivier spent more than three years with Sanofi as global head of corporate affairs for the General Medicines division. She had several job titles over an eight-year period at AbbVie , including head of global patient relations and international public affairs. → CAR-T cell therapy developer AffyImmune has recruited Deyaa Adib as CMO. Adib just had a very short stay at FibroGen and has also held this position at Triumvira Immunologics . AffyImmune, a company in ORI Capital ’s portfolio, is under new management for the second time this year — in July, Daniel Janse took over for Matt Britz as CEO. Britz had been elevated to the top spot in March after two years as operating chief. → At another CAR-T shop, ex- Bellicum and Artiva Biotherapeutics CEO Tom Farrell has been named president of Shanghai-based Oricell . “Tom is a transformative leader who has consistently delivered results. We believe he will undoubtedly accelerate our clinical development momentum in the US,” CEO Helen Yang said in a statement . Oricell raised a $45 million Series B1 in February 2023 on top of a $120 million haul. → Merck ’s macrocyclic peptide partner Unnatural Products has tapped Simon Bailey as COO and president of R&D. Bailey left protein degradation biotech Plexium in March after more than four years as head of drug discovery and was SVP of research for Intercept from 2016-19. His 20-year career with Pfizer culminated in his role as senior director for worldwide medicinal chemistry in oncology. → Iovance Biotherapeutics has promoted Raj Puri to chief regulatory officer. Puri spent two decades as a director at CBER in the Office of Tissues and Advanced Therapies before coming to Iovance in March 2022 as EVP, regulatory strategy and translational medicine. Iovance fought through multiple delays to receive the first-ever FDA approval for a tumor-infiltrating lymphocyte with its cell therapy Amtagvi . → Synthetic cell specialist Slingshot Biosciences has welcomed Nimisha Srivastava as COO. Srivastava held a number of roles over a decade at Twist Bioscience , where she was VP of engineering and eventually head of R&D. Slingshot also said last month that it had named Tiffany Salas-Morris as chief commercial officer. → Sapience Therapeutics , which is testing its beta-catenin antagonist ST316 in colorectal cancer patients, has recruited Augustine Bourne as VP, business development. He previously worked for Mirati as its business development and corporate strategy leader when Bristol Myers Squibb purchased the Krazati maker for $4.8 billion. In other Sapience news, ex- Replimune CBO Pamela Esposito now has a seat on its board of directors. → Swiss cancer vaccine developer Nouscom has recruited Grünenthal and MorphoSys alum Eckhard Niemeier as CBO. Niemeier just ended a three-year run as business chief at Danish biotech Union Therapeutics , and he was head of business development & portfolio management for Pieris Pharmaceuticals . “Cancer vaccines are, let’s say, back in fashion,” Nouscom CEO Marina Udier told Endpoints News in November 2023, when the biotech announced its Series C funding. → Incendia Therapeutics has appointed Bradford Smith as its first finance chief. Smith has previously been CFO for a number of companies, including Homology Medicines , Ocular Therapeutix , OmniGuide , NeuroMetrix and Synarc . Smith also sits on the boards of Lyra Therapeutics and eGenesis . → Massachusetts-based ITF Therapeutics has recruited Erica Monaco as COO. Monaco joins with previous COO experience from Medicus Pharma and Biofrontera , where she also served as CEO and CFO). Earlier in her career, Monaco was with Sun Pharmaceuticals and Deloitte . → Portuguese CRO FairJourney Biologics has brought aboard Marc van Dijk as CSO, responsible for the company’s antibody discovery and engineering divisions. Dijk also served in the role at MiNK Therapeutics , and earlier in his career, Dijk was with Genmab as VP of antibody technology and chief technology officer at 4-Antibody and Agenus . → CRISPR Therapeutics veteran Chris Gemmiti has landed at Cellino as SVP of therapeutics development. Gemmiti was in charge of CMC for Casgevy , CRISPR and Vertex ’s sickle cell gene therapy, and became VP of technical operations in April 2022. Cellino recently caught our attention by appointing Recursion CEO Chris Gibson to its board of directors. → Looking for the next breakthrough in psychedelic therapy, MindMed has hired yet another exec, bringing in Javier Muniz as VP of research and development strategy. He just finished up a 10-year career with the FDA, where he was associate director for therapeutic review in the agency’s Division of Psychiatry within the Office of Neuroscience. This is MindMed’s third notable leadership move in the past few months, following the additions of chief regulatory and quality assurance officer Gregg Pratt and corporate affairs chief Stephanie Fagan . → Former Bristol Myers exec Kristen Hege leads a new crop of clinical advisory board members at Gilead ’s cancer partner Cartography Biosciences . Kevin Parker ’s crew has also welcomed Affini-T Therapeutics CMO Dirk Nagorsen , UCSF’s Bridget Keenan and Josep Tabernero from the Vall d´Hebron University Hospital in Barcelona. → Beam Therapeutics has added Chirfi Guindo to the board of directors. Guindo had a 27-year career with Merck before joining Biogen in 2017, then returned to Merck in July 2022 as chief marketing officer for the Human Health division. Click here for Andrew Dunn’s Slack interview from February with CEO John Evans , who also spoke to Endpoints for an early look at Beam’s gene-edited sickle cell data. → Ex- Decibel Therapeutics CEO Laurence Reid has sewn up a spot on the board of directors at hereditary angioedema biotech KalVista Pharmaceuticals . Reid sold Decibel to Regeneron for $109 million in August 2023 and chairs the board at Broken String Biosciences . The FDA has assigned a June 17 decision date for KalVista’s HAE drug sebetralstat . → Backed by The Column Group , 8VC and others, California ADC developer Hexagon Bio has elected Ideaya Biosciences CEO Yujiro Hata to its board of directors. This was Hexagon’s first press release of any kind since its $77 million Series B in February 2023. → Longtime EY vet Fran Schulz has joined the board of directors at Tim Lu ’s Senti Bio , as Seer chief Omid Farokhzad exits. Shares $SNTI went through the roof on Monday, rising more than 600% and settling on a 363% jump after Senti Bio disclosed Phase 1 data for its CAR-NK cell therapy SENTI-202 . → Day One Biopharmaceuticals co-founder and R&D leader Sam Blackman is headed to the board of directors at Bonum Therapeutics , the Seattle-based I/O biotech that sprouted from Good Therapeutics and nabbed a $93 million Series A two years ago. Blackman will retire from Day One at the end of the year. → Rare disease AI company Healx has named Abcam founder Jonathan Milner as non-executive chairman, taking over from co-founder David Brown , who will remain on the board. Despite Milner’s opposition , Abcam completed its acquisition of Danaher exactly one year ago.

从 Biotech 蜕变为 BioPharma 并非易事，真正能成功转型的寥寥无几。 然而，信达生物不仅以丰富的产品线完成了蜕变，还布下一盘大棋局，储备了梯队合理的管线，冲刺「2027 年国内产品收入达到 200 亿元」的目标。 冲刺 200 亿目标 信达生物距离 200 亿目标还有多远？ 凭借「自主研发+战略引进」双轮驱动的策略，如今信达的商业化产品已扩充至 11 款，覆盖了多个疾病领域，其中抗肿瘤产品 9 个、自免产品 1 个、心血管产品 1 个。 2024 年前三季度，信达实现产品总收入超过 61 亿元，较上年同期增长约 51%，主要得益于信迪利单抗和其他产品的出色表现，以及新产品的加速增长及贡献增加。 信迪利单抗是信达的业绩支柱产品，目前已斩获 7 个适应症，今年上半年实现销售额达 2.4 亿美元（约合人民币 16.95 亿元），同比增长 46%，第三季度仍保持强劲增长势头。而且，信迪利单抗的增长尚未触及天花板，信达正在通过开展联合疗法、持续拓展新适应症，延长产品高销售额的生命周期。 今年 4 月，信迪利单抗联合呋喹替尼治疗二线子宫内膜癌的上市申请获国家药监局受理；而且，还有多项 Ⅲ 期研究在进行中，包括针对非小细胞肺癌围手术期、联合 IBI310（CTLA-4）用于结直肠癌新辅助治疗。 信达生物 11 款获批上市的产品 数据来源：Insight 数据库 信达生物能否在业绩层面更胜一筹，是市场最为关注的焦点。 就现有产品而言，抗肿瘤领域的奥雷巴替尼、氟泽雷塞，以及心血管领域的托莱西单抗均颇有市场前景。 奥雷巴替尼是亚盛医药研发的中国首个三代 BCR-ABL 抑制剂，目前已获批 2 项适应症：治疗任何 TKI 耐药、并伴有 T315I 突变的慢性髓细胞白血病（CML）慢性期（-CP）和加速期（-AP）的成年患者，以及治疗对一代和二代 TKI 耐药和/或不耐受的 CML-CP 成年患者。 奥雷巴替尼在中国的商业化推广由亚盛和信达共同负责，自 2021 年 11 月上市以来已累计实现含税销售额近 5 亿元。尤其是今年 6 月，武田制药以总额 13 亿美元拿下了奥雷巴替尼在大中华区以外的全球权益，更加凸显其市场潜力。 氟泽雷塞是劲方医药研发的中国首个 KRAS G12C 抑制剂，已于今年 8 月获批用于治疗至少接受过一种系统性治疗的 KRAS G12C 突变型晚期 NSCLC 成人患者，填补了 40 年中国 KRAS G12C 突变靶向治疗的空白。信达在 2021 年以交易总额 3.12 亿美元获得了氟泽雷塞在中国地区的权益，并拥有全球开发和商业化权益的选择权。 托莱西单抗是信达布局心血管疾病领域的首款产品，也是首个国产 PCSK9 抑制剂，已于去年 8 月获批用于治疗原发性高胆固醇血症（包括杂合子型家族性和非家族性高胆固醇血症）和混合型血脂异常。 除已上市产品外，后续管线的商业化落地也将是信达生物冲刺 200 亿目标的新增长引擎。 抗肿瘤管线全面开花 近两年还有 2 款新产品获批 抗肿瘤产品是信达生物实现收入大幅增长的「功臣」，后续管线组合自然也十分丰富，覆盖了小分子、单抗、多抗、ADC 等多种药物形式，涉及 ROS1、EGFR、ClDN18.2、HER3 和 B7-H3 等众多靶点。其中，进展最快的他雷替尼（IBI344）、利厄替尼，均已提交上市申请。 他雷替尼是信达从葆元医药（已被 Nuvation Bio 收购）引进的新一代潜在最佳 ROS1 抑制剂，拥有大中华区的权益，目前已向 NMPA 提交 2 项适应症的上市申请： 包括用于经 ROS1-TKI 治疗失败的 ROS1 阳性局部晚期或转移性 NSCLC 成人患者，以及一线治疗上述适应症。两个适应症均有望在今年 Q4 获批。 截图来源：Insight 数据库 利厄替尼是奥赛康研发的第三代 EGFR 抑制剂，已提交 2 项适应症的上市申请： 包括既往经 EGFR-TKI 治疗时或治疗后出现疾病进展，并且经检测确认存在 EGFR T790M 突变阳性，或原发性 EGFR T790M 突变阳性的局部晚期或转移性 NSCLC 成人患者的治疗，预计 2026 Q3 获批。 一线治疗具有 EGFR 外显子 19 缺失（19DEL）或外显子 21 置换突变（L858R）的局部晚期或转移性 NSCLC 成人患者，预计 2025 Q1 获批。 今年 10 月，信达与奥赛康达成战略合作，将在中国地区独家推广销售利厄替尼，并向奥赛康支付首付款、注册里程碑和销售里程碑款项。 截图来源：Insight 数据库 信达在肿瘤领域的亮点还有多抗： 拥有全球首创 PD-1/IL-2α 双抗融合蛋白 IBI363 目前正在中国、美国、澳大利亚同时开展Ⅰ/Ⅱ期研究，今年 9 月还获得 FDA 快速通道资格认定，用于治疗黑色素瘤。该药在解决免疫治疗（IO）抵抗和冷肿瘤患者的未满足临床需求方面具有巨大潜力。 全球首个且目前唯一进入临床阶段的 DR5×CEA 双抗 IBI3004。该药正在澳洲和中国内地开展治疗结直肠癌等实体瘤的Ⅰ/Ⅱ期研究； 全球首款靶向 B7-H3/EGFR 的双抗 IBI334，正在开展针对晚期实体瘤的 I/II 期临床研究； 还有一款靶向 GPRC5D/BCMA/CD3 的三抗 IBI3003，正在国内开发用于治疗复发或难治性多发性骨髓瘤的 I 期研究，进度处于全球第一梯队。 为了深度布局「IO+ADC」，目前信达已有 7 款 ADC 药物进入临床阶段，包括 IBI343（CLDN18.2 ADC）、IBI354（HER2 ADC）、IBI130（TROP2 ADC）、IBI129（B7 H3 ADC）、IBI133（HER3 ADC）、IBI3009（DLL-3 ADC）、IBI3001（全球首创 EGFR/B7 H3 双抗 ADC）。 IBI343 进度最快，正在开展国际多中心 Ⅲ 期研究，并已在 I 期试验中展现出积极疗效：6 mg/kg 剂量组针对 CLDN18.2 高表达胃癌患者的 ORR、DCR 分别为 36.7%、93.3%，中位无进展生存期长达 6.8 个月。 IBI354 在 I/II 期试验展示出对乳腺癌、卵巢癌等多个癌种积极的早期疗效信号和优异的安全性； IBI130 正在澳洲开展 I/II 期试验； IBI133 正在国内和澳洲开展 I/II 期试验； IBI3009 正在澳洲开展针对小细胞肺癌和其他神经内分泌癌的Ⅰ期试验。 自免、代谢、眼科， 研发版图再扩张 除肿瘤管线外，信达生物还在自免、眼科、心血管及代谢领域撒下火种。 自免领域，管线的靶点差异化优势显著，包括 IBI112（IL-23p19 抑制剂）、IBI353（Orismilast）、IBI355（CD40L 抗体）、IBI356（OX40L 单抗）、IBI3002（全球首创 IL-4Rα/TSLP 双抗）。 其中，IBI112（匹康奇拜单抗）进度最快，已在中国申报上市用于治疗中重度斑块状银屑病，预计在 2026 年 Q1 获批；针对溃疡性结肠炎的 II 期临床也已达到主要终点。 Orismilast 是治疗炎症性皮肤病的潜在同类最佳 PDE4 抑制剂，已在中度至重度银屑病研究中显示出积极结果，信达在 2021 年以总额 2.67 亿美元从 UNION Therapeutics 引进了大中华区权益。 代谢领域中，信达布局的 GLP-1R/GCGR 双重激动剂玛仕度肽，有望成为下一个爆发点。 目前已提交用于减重和 2 型糖尿病两大适应症的上市申请，预计 2025 年 Q3 可获批，并有多个 III 期研究在进行中。 截图来源：Insight 数据库 同类竞品中，今年前九个月司美格鲁肽、替尔泊肽分别实现销售额高达 203.24 亿美元、110.28 亿美元。根据国联证券研报，相比于司美格鲁肽及替尔泊肽，玛仕度肽使用了更简便的给药方案，可快速到达最大维持剂量，提升用药早期的减重效果，也减少了患者调整药物剂量的次数，提高患者依从性。 此外，信达还在心血管及代谢领域布局了 IBI128（替古索司他片）、IBI-3016（AGT siRNA）。 其中，替古索司他片是一款治疗痛风的全新的黄嘌呤氧化酶抑制剂（XOI），信达在 2022 年以超 9500 万美元从韩国 LG 化学引进了中国的权益，目前 LG 化学正在开展两项 Ⅲ 期研究。 IBI3016 是一款靶向血管紧张素原（AGT）的变革性 siRNA 创新疗法，具备药效持久且安全耐受性良好的优势，用于治疗患者数量庞大的高血压，目前正在开展 I 期研究。 在眼科领域，信达即将迎来首个商业化产品——替妥尤单抗（IBI311）。 替妥尤单抗已提交用于治疗甲状腺眼病（TED）的上市申请，是中国首个申报上市的 IGF-1R 抗体，预计 2025 年 Q2 获批。 目前在研的同类竞品不多、竞争格局良好，而且全球首款且唯一获批的 IGF-1R 单抗 Tepezza，2022 年销售额高达近 20 亿美元，足见该类药物的市场价值。 截图来源：Insight 数据库 除替妥尤单抗外，信达还针对眼科领域的明星靶点 VEGF 进行了深度布局，拥有全球首创抗 VEGF-抗补体双特异性融合蛋白 IBI302（Ⅲ期临床）、IBI324（VEGF/Ang2 双抗）、IBI333（VEGF-A/VEGF-C 双抗）等多个管线。 IBI302 在针对新生血管性年龄相关性黄斑变性（nAMD）的临床 II 期研究中展现出了积极的治疗效果，以及长间隔给药的潜力，有望解决以往 VEGF 药物需频繁注射的问题。 要知道，罗氏治疗 nAMD 的法瑞西单抗（Vabysmo），之所以能在短时间内实现惊人的高速增幅，在今年前三季度实现超 30 亿美元的销售额，就是因为提供了便捷的给药方式。 结语 多个疾病领域全面开花的棋局，可谓相当的有看头。 可如果把目光放得更长远，信达若想继续走出持续高增长轨迹，必然还得做好国际化市场，毕竟这是关乎未来发展的关键战役。 但从现在的情形看，信达的海外管线推进速度还是不够快，大多仍处于早期临床阶段。反观，许多 Biotech 的收入结构都逐渐进阶成了「商业化+源源不断的合作收入」，而信达在 BD 出海、在国际化的布局方面，显然还需要加快速度。 参考资料： 1. 信达生物财报、公告、官微 2.Insight 数据库 3. 国联证券研报 封面来源：企业 Logo 免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。 编辑：vvy PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn 多样化功能、可溯源数据…… Insight 数据库网页版等你体验 点击阅读原文，立刻解锁！

▎药明康德内容团队编辑 潜在“first-in-class”特应性皮炎疗法2期临床结果积极 UNION Therapeutics今天宣布，其潜在“first-in-class”口服疗法orismilast在治疗中度至重度特应性皮炎成人患者的2b期临床试验ADESOS中获得积极结果。基于这一进展，该公司计划推进这款疗法进入3期临床试验。  研究中随机分配的233名患者的数据表明，与安慰剂组（n=55）相比，orismilast的20 mg（n=58）、30 mg（n=61）和40 mg（n=59）剂量组在第16周时让更多患者达到了研究者总体评估（IGA）评分为0或1的缓解标准（比例分别为26.3%、24.3%、30.9%和9.5%；所有p<0.05）。此外，所有治疗组在第2周时的瘙痒数值评分（NRS）均表现出显著的≥4点的减轻，与安慰剂相比获得显著改善（p<0.05）。 Orismilast是一种新一代高效磷酸二酯酶4（PDE4）抑制剂，靶向与炎症相关的PDE4B/D亚型，表现出对Th1、Th2和Th17通路的强效抑制作用。它在炎症级联反应的早期阶段起作用，诱导对多种皮肤和免疫疾病相关的多种细胞因子的广泛抗炎作用。Orismilast的开发旨在比上市的PDE4抑制剂具有更强的抗炎特性，这得益于其对PDE4B和D亚型的更高选择性。 新锐完成4600万欧元B轮融资，开发基于慢病毒载体的基因疗法 Genespire今日宣布完成4600万欧元（约5200万美元）的B轮融资，获得资金将支持该公司的核心候选产品GENE202的开发，直至进入治疗甲基丙二酸血症（MMA）的1/2期临床试验。MMA是一种破坏某些氨基酸和脂肪代谢的严重遗传疾病，通常在婴儿早期发病，伴随高死亡率，临床表现包括肌无力、癫痫、发育迟缓和器官损伤。目前，尚无针对MMA的改变疾病进程的治疗方法。 GENE202是一种现货型基因疗法，利用了该公司的免疫屏蔽慢病毒载体（ISLV）平台。GENE202设计为可静脉注射，使患者的肝脏终生产生该疗法。ISLV的作用机制使Genespire的治疗方法特别适用于当前面临最紧迫未满足医疗需求的遗传病儿童患者，同时也能惠及成人患者。 本轮B轮融资还将加强公司产品管线，促进针对多种其他遗传疾病候选疗法的发现和临床前研究。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] UNION therapeutics announce results from the ADESOS Phase 2b study in atopic dermatitis confirming the potential of orismilast as a first-in-class safe and efficacious oral treatment. Retrieved September 25, 2024, from https://www.prnewswire.com/news-releases/union-therapeutics-announce-results-from-the-adesos-phase-2b-study-in-atopic-dermatitis-confirming-the-potential-of-orismilast-as-a-first-in-class-safe-and-efficacious-oral-treatment-302258828.html [2] Genespire raises €46.6 million (~$52 million) in a Series B round to advance its first pediatric in-vivo gene therapy into the clinic. Retrieved September 25, 2024, from https://www.genespire.com/wp-content/uploads/2024/09/240925-Genespire-Series-B-press-release-FINAL.pdf 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新