寡核甘酸市场 - 竞争情形(2025年)

"寡核苷酸市场 - 竞争格局 (2025)" 报告全面分析了寡核苷酸领域 280 多家公司和 320 多种药物的竞争格局。报告涵盖了按产品类型、阶段、给药途径和分子类型进行的治疗评估。此外，报告还重点介绍了该领域未上市的研发线产品。

对象地区

• 作为对象的世界

寡核甘酸理解

寡核甘酸概要

寡核苷酸是由重复核苷酸单体组成的寡聚体，这些单体由脱氧核糖或核糖、含氮碱基和磷酸骨架组成。寡核苷酸具有独特的能力，可以特异性地与其互补序列（例如 DNA 和 RNA）结合，形成双股体，以及（较少见的）高阶杂合体。这项特性使得寡核苷酸可用作探针来辨识特定的DNA或RNA序列。寡核苷酸（ON）是短链核酸聚合物，通常由13至25个核苷酸组成，可与目标DNA或RNA杂交。它们分为反义寡核苷酸（ASO）、小干扰RNA（siRNA）、微小RNA（miRNA）和适体等类别，目前正在探索其在神经退化性疾病、癌症甚至罕见疾病中的应用。

它们也正在临床试验中用于治疗皮肤、胃肠道和荷尔蒙失调。寡核苷酸可透过多种途径调控基因表达，包括RNAi、RNase H介导的切割降解标靶基因、剪接调控、非编码RNA抑制、基因活化和程序性基因编辑。寡核苷酸疗法，包括反义寡核苷酸 (ASO)、小干扰RNA (siRNA) 和剪接转换寡核苷酸，是另一类具有突破性潜力的药物，有望治疗多种遗传和后天疾病。儘管这些分子前景广阔，但它们的临床成功与能够克服其固有生物学局限性的高效递送系统的开发密切相关。由于寡核苷酸分子量大、亲水性强且带负电荷，其在到达细胞内标靶方面面临巨大障碍。此外，它们易被体液中的核酸酶降解、组织穿透能力有限、肾臟快速清除以及需要组织特异性靶向，进一步增加了全身递送的复杂性。

寡核苷酸因其高特异性、可编程性和易于合成，彻底改变了诊断和治疗。在诊断领域，它们可用作PCR/qPCR的引子、FISH等杂交检测的探针，以及微阵列和基于CRISPR的检测等先进平台的组件。适体可结合特定生物标记以实现标靶成像，并经过化学修饰以提高其体内稳定性。在治疗领域，寡核苷酸包括适体（例如，哌加他尼）、反义寡核苷酸（例如，nusinersen）和siRNA（例如，patisiran），它们透过调节或沉默基因表现来靶向治疗疾病。它也是mRNA/DNA疫苗、miRNA抑制剂和基于CRISPR的基因编辑的基础。儘管存在脱靶效应和快速清除等挑战，但GalNAc偶联和AI引导设计等创新技术已改善了药物的递送、特异性和临床疗效，从而推动了精准医疗的进步。

报告的亮点

• 2025年3月，专注于开发基于RNA编辑的新型基因药物（用于治疗罕见疾病和高发病）的临床阶段生物製药公司Korro Bio, Inc.宣布，美国食品药品监督管理局 (FDA) 已授予其在研药物KRRO-110孤儿药资格认定，用于治疗α-1抗胰蛋白酶缺乏症(AATD)。
• 2025年1月，Arrowhead Pharmaceuticals, Inc.宣布，美国食品药物管理局 (FDA) 已接受其在研药物百忧解(prozacilan)的新药申请，该药物用于治疗家族性乳糜微粒血症综合症(FCS)，这是一种严重且罕见的遗传性疾病。
• 2025年2月，临床阶段生物技术公司AusperBio Therapeutics, Inc.和Ausper Biopharma Co., Ltd.宣布了其主要候选化合物AHB-137正在进行的临床开发的最新进展。 AHB-137是一种反义寡核苷酸(ASO)疗法，旨在实现慢性乙型肝炎(CHB)的功能性治癒。
• 2024年12月，Vir Biotechnology, Inc.宣布，tobevibart和elebsiran分别获得美国食品药物管理局(FDA)颁发的突破性疗法认定和欧洲药品管理局(EMA)颁发的治疗慢性丁型肝炎(CHD)的优先疗法认定。
• 2024年12月，FDA授予Stoke Therapeutics公司的在研药物STK-001突破性疗法认定，用于治疗Dravet综合征，这是一种罕见的、经基因确诊的癫痫病。
• 2024年11月，Ionis Pharmaceuticals宣布，美国食品药物管理局 (FDA) 已受理donidalorsen的新药申请 (NDA)。 donidalorsen是一种RNA标靶药物，用于预防12岁及以上成人和儿童遗传性血管性水肿(HAE)患者的癫痫发作。 FDA依据 "处方药使用者付费法案" (PDUFA)，将核准日期设定为2025年8月21日。
• 2024年10月，Ribocure Pharmaceuticals AB和苏州瑞博生命科技有限公司获得瑞典药品管理局 (MPA) 的批准，将在瑞典启动RBD5044的II期临床试验。 RBD5044是一种针对APOC3的降脂siRNA药物。该研究将评估其在混合性血脂异常患者的疗效和安全性。
• 2024年9月，Wave Life Sciences公司开发的用于治疗杜氏肌肉营养不良症（DMD）的外显子跳跃寡核苷酸WVE-N531获得了美国食品药品监督管理局（FDA）的孤儿药资格认定。 DMD适用于治疗53号外显子跳跃的疾病。
• 2024年9月，日本新药株式会社的子公司NS Pharma, Inc.获得了美国食品药物管理局（FDA）的孤儿药资格认定。 2024年9月，日本新药株式会社的子公司NS Pharma Co., Ltd.宣布，正在开发的用于治疗杜氏肌肉营养不良症的NS050/NCNP-03已获得美国食品药物管理局（FDA）的罕见儿科疾病资格认定。
• 2024年5月，Sunhawk Vision Biotech宣布已获得美国FDA批准，开始进行儿童近视控制的II期临床试验。
• 2024年5月，Imvax, Inc.宣布完成IGV-001针对新诊断胶质母细胞瘤(ndGBM)患者的随机、多中心、双盲、安慰剂对照IIb期临床试验的招募工作。

寡核苷酸寡核苷酸：公司与产品简介（目录）

1. 公司概况：诺华

诺华国际股份公司是一家总部位于瑞士的全球医疗保健公司，也是全球最大的製药公司之一。诺华公司成立于1996年，由汽巴-嘉基公司和山德士公司合併而成，致力于研究、开发、生产和销售广泛的医疗保健产品，专注于药品、仿製药和生物类似药。诺华致力于提供永续的医疗保健解决方案，包括减少对环境的影响并改善全球药品的可近性。诺华也致力于为中低收入国家提供负担得起的药品。

产品概述：LEQVIO

LEQVIO (Inclisiran) 是由诺华公司开发的首创siRNA（小干扰RNA）疗法，作用于PCSK9（前蛋白转化酶枯草桿菌蛋白酶9型）的mRNA。与其他疗法不同，LEQVIO能够抑制肝臟中目标蛋白的产生，从而增加肝臟对LDL-C的摄取并将其从血液中清除。 2023年，LEQVIO（通用名：inclisiran）获得厚生劳动省核准，用于治疗家族性和非家族性高胆固醇血症，且心血管事件风险较高的患者。给药方案为首次注射，三个月后再注射，每六个月维持注射一次。临床试验表明，LEQVIO与他汀类药物联合使用可降低约50%的低密度脂蛋白胆固醇（LDL-C）水平。

2. 公司简介：安斯泰来製药

安斯泰来製药是一家总部位于日本东京的日本跨国製药公司，以其创新的医疗保健解决方案而闻名。安斯泰来製药成立于2005年，由山之内製药和藤泽製药合併而成，专注于肿瘤学、泌尿学、免疫学和神经科学等领域。安斯泰来製药专注于研发，致力于透过推进治疗手段来满足尚未满足的医疗需求，进而改善病患的治疗效果。安斯泰来製药也积极寻求全球合作伙伴关係和联盟，以加速药物研发，并扩大其在北美、欧洲和亚洲等关键地区的市场占有率。

产品概述：IZERVAY

Avacincaptad pegol，以 IZERVAY 为商品名上市，已获准用于治疗地图样萎缩。 Avacincaptad pegol 是一种与支链聚乙二醇 (PEG) 分子共价连接的 RNA 适体。其主要功能是抑制补体因子 C5，C5 是参与 AMD 相关发炎过程的补体级联的关键组成部分。透过抑制 C5 裂解为活性片段（C5a 和 C5b），Avacincaptad pegol 旨在减轻发炎并减缓地图样萎缩 (GA) 的进展。 IZERVAY 于 2023 年 8 月 4 日获得美国食品药物管理局 (FDA) 批准，用于治疗继发于老年黄斑部病变 (AMD) 的 GA，目前正在接受欧洲药品管理局 (EMA) 的审查。该药物也正在接受 Stargardt 病治疗评估。

3. 公司概况：Alnylam Pharmaceuticals

Alnylam Pharmaceuticals 是一家总部位于麻萨诸塞州剑桥市的生物製药公司，专注于 RNA 干扰 (RNAi) 疗法的发现、开发和商业化。该公司成立于 2002 年，致力于开发 RNAi 技术，以灭活与疾病相关的特定基因。Alnylam 以在罕见遗传、心臟代谢和肝臟感染性疾病治疗领域开创性地而闻名。 Alnylam 的产品线涵盖多种罕见遗传、心臟代谢和肝臟感染性疾病，拥有多个已核准的产品，包括 ONPATTRO(R)、GIVLAARI(R)、OXLUMO(R) 和 AMVUTTRA(R)。公司专注于科学创新、策略合作伙伴关係和全球扩张，致力于为治疗选择有限的患者带来全新的治疗方案。

产品概述：AMVUTTRA

AMVUTTRA(R)（布特里斯兰）是一种 RNAi 疗法，可快速敲低甲状腺素蛋白 (TTR)，从而解决转甲状腺素蛋白 (ATTR) 淀粉样变性的根本病因。 AMVUTTRA 由医疗保健专业人员每季皮下注射一次，已在超过 15 个国家/地区获批上市，用于治疗患有遗传性转甲状腺素蛋白介导的淀粉样变性多发性神经病变 (hATTR-PN) 的成年患者，并在美国获批上市，用于治疗患有野生型或遗传性转甲状腺素蛋白介导的淀粉样心肌病变的成年患者，旨在降低心血管死亡率、心血管住院率和急诊心臟衰竭就诊率。

4. 公司概况：Ionis Pharmaceuticals

Ionis Pharmaceuticals 成立于 1989 年，总部位于加州卡尔斯巴德。它是一家专注于 RNA 标靶治疗的生物技术公司。该公司率先开发反义技术，用于治疗多种疾病，包括神经系统疾病、心血管疾病和罕见疾病。 Ionis 专注于利用其专有平台（包括先进的反义和 RNA 干扰 (RNAi) 技术）来发现和开发基于 RNA 的疗法。公司专注于精准医疗，并与大型製药公司合作，以推进其产品线。凭藉着完全自主开发和合作的多元化专案组合，Ionis 不断拓展其在生物製药领域的影响力。 Ionis 已在纳斯达克上市，专注于开发 RNA 标靶疗法，为患者提供创新疗法。

产品概述：TEGSEDI

Tegsedi (Inoterzen) 是一种反义寡核苷酸疗法，用于治疗患有遗传性转甲状腺素蛋白介导的淀粉样变性 (hATTR) 并发展为 1 期或 2 期多发性神经病变的成年患者。该药物透过抑制转甲状腺素蛋白 (TTR) 的产生来减缓神经病变的进展。 Tegsedi 每週一次，每次 284 毫克，皮下注射，但由于可能出现严重副作用，包括血小板数量减少和肾臟炎症，因此需要定期监测。常见的副作用包括注射部位反应、噁心、头痛、疲劳和发烧。在美国，Tegsedi 仅透过名为 Tegsedi 风险评估和缓解策略 (REMS) 的受限项目提供，以确保安全使用。 Tegsedi 是一种每週一次的自行给药皮下製剂，已在美国、欧洲、加拿大和巴西获准用于治疗 ATTRv-PN 患者。

寡核苷酸公司及产品简介（管线治疗）

1. 公司概况：诺华製药

诺华製药是一家总部位于瑞士的全球领先医疗保健公司，致力于重塑医学，改善和延长人们的生活。诺华凭藉其强大的创新处方药产品组合，专注于肿瘤学、免疫学、神经科学和心血管疾病等关键治疗领域。诺华始终致力于研发，利用先进的科学和数位技术提供高价值疗法。诺华业务遍及150多个国家，透过策略合作伙伴关係、丰富的创新疗法管线以及对卓越营运和以患者为中心的创新的明确承诺，持续推动永续成长。

产品概述：Pelacarsen

Pelacarsen (TQJ230)，也称为IONIS-APO(a)-LRx、AKCEA-APO(a)-LRx和TQJ230，是一种在研反义药物，旨在提供一种直接降低肝臟载脂蛋白(a)和脂蛋白(a) (Lp(a))（一种高度致动脉粥样硬化、易结的脂蛋白凝结）。脂蛋白a升高被认为是冠状动脉疾病、心臟病发作、中风和周边动脉疾病的独立遗传原因。 Pelacarsen 是一种旨在降低脂蛋白a的在研反义药物，由 Ionis 发现，并于 2019 年授权给诺华公司。该药物目前处于治疗高脂蛋白血症的 III 期临床试验阶段。

2. 公司概况：Oncotelic

Oncotelic Therapeutics 是一家临床阶段的生物製药公司，专注于开发针对癌症和罕见疾病的创新疗法。该公司总部位于加州阿古拉山，利用 RNA 疗法和免疫肿瘤学方法治疗难治性肿瘤和纤维化疾病。该公司的主要项目包括治疗胶质母细胞瘤、黑色素瘤、特发性肺纤维化等。 Oncotelic 结合 TGF-B 抑制和寡核苷酸疗法的专业知识，精准调控疾病路径。 Oncotelic 致力于透过内部研发和合作研究，提供有效的疗法，满足尚未满足的医疗需求。

产品概述：Travedersen

OT-101（也称为 travedersen）是由 Oncotelic 开发的一种新型反义寡脱氧核苷酸 (ODN)，用于治疗胰腺癌、恶性黑色素瘤、结直肠癌、高级别胶质瘤 (HGG) 以及其他过度表达转化生长因子 β2 (TGF-B2) 的恶性肿瘤，包括前列腺癌和肾细胞癌。 Travedersen 是一种合成的 18 聚体硫代磷酸酯寡脱氧核苷酸 (S-ODN)，与人类 TGF-B2 基因的信使核糖核酸 (mRNA) 互补。癌症过度表现TGF-B，抑制宿主对癌症的先天免疫反应。 OT-101治疗可解锁TGF-B的掩蔽效应，使先天性或治疗性免疫能够攻击并消除癌症。 OT-101已完成胰臟癌和黑色素瘤的II期临床试验，胶质母细胞瘤的II期临床试验也证实其有效性和安全性。

3. 公司概况：Wave Life Sciences

Wave Life Sciences是一家生物技术公司，致力于释放RNA药物改变人类健康的广泛潜力。 Wave的RNA药物平台PRISM®融合了多种模式、化学创新和对人类遗传学的深刻洞察，在治疗罕见疾病和常见疾病方面取得了科学突破。其RNA标靶工具包包括编辑、剪接、RNA干扰和反义沉默，为Wave提供了无与伦比的能力，能够设计并持续交付最能解决疾病生物学问题的候选药物。 Wave 多元化的研发管线包括杜氏肌肉营养不良症、α-1 抗胰蛋白酶缺乏症和亨丁顿舞蹈症的临床项目，以及肥胖症的临床前项目。秉承 "重塑无限可能" 的理念，Wave 正朝着一个让人类潜能不再受疾病困扰的世界迈进。

产品概述：WVE-N531

WVE-N531 是一种外显子跳跃寡核苷酸，旨在为适合进行 53 号外显子跳跃疗法的杜氏肌肉营养不良症儿童开发一种疾病修饰疗法。 WVE-N531 采用 Wave 一流的寡核苷酸化学修饰技术，包括 PN 骨架化学修饰技术。 WVE-N531 已获得美国食品药物管理局 (FDA) 授予的孤儿药资格和儿科罕见疾病资格。目前，该药物正处于治疗强直性肌肉营养不良症的 II 期临床试验阶段。

4. 公司概况：Autotelic Bio

Autotelic Bio 是一家成立于 2015 年的韩国临床阶段生物技术公司，专注于开发下一代核酸疗法，尤其是反义寡核苷酸 (ASO)。该公司利用其专有的 ASODE 和 CAT 平台，旨在提供具有更高疗效和安全性的标靶优化 ASO 疗法。该公司多元化的研发管线包括用于治疗癌症、特发性肺纤维化 (IPF) 和代谢性疾病的联合用药候选药物。截至 2022 年 3 月，Autotelic Bio 已获得 1,820 万美元融资，包括由 UTC Investment 和 Stonebridge Ventures 等投资者领投的 1,260 万美元 B 轮融资。该公司旨在透过攻克先前无法治癒的癌症靶点，成为癌症治疗的全球领导者。

产品概述：ATB 301

ATB-301 是由 Autotelic Bio 公司开发的一种针对转化生长因子 β2 (TGF-β2) 的在研反义寡核苷酸 (ASO) 疗法。目前正在评估该疗法与重组白细胞介素 2（阿地白介素）联合用于治疗晚期或转移性实体瘤，包括胰腺癌和肾细胞癌。该疗法旨在抑制 TGF-β2，从而调节肿瘤微环境并增强免疫反应。在胰臟癌模型中，该药物已显示出透过抑制 TGF-β2 来抑制肿瘤生长并增加癌组织中 CD8 T 细胞的功能。目前，该药物正处于胰臟癌治疗的 I 期临床试验阶段。

5.公司概况：Tallac Therapeutics

Tallac Therapeutics 是一家总部位于加州的临床阶段生物製药公司，专注于开发新型癌症免疫疗法。该公司专有的 Toll 样受体激动剂抗体偶联物 (TAAC) 平台旨在直接在肿瘤微环境中刺激先天性和适应性免疫反应。 Tallac 的主导计画旨在靶向激活 Toll 样受体 9 (TLR9​​)，以增强抗肿瘤免疫力，同时最大限度地降低全身毒性。 Tallac 利用其平台开发下一代免疫肿瘤疗法，正在推进一系列候选药物研发管线，旨在治疗各种实体瘤并改善患者预后。

产品概述：TAC001

TAC-001 是一种新型抗体-寡核苷酸偶联物，可发挥全身性 TLR9 激动作用，并靶向激活 B 细胞的免疫功能，而 B 细胞在癌症免疫中发挥关键作用。在临床前研究中，全身给药的TAC-001作为单一药物在多种假基因肿瘤模型（包括免疫抑製或抗药性模型）中显示出活性，可完全清除肿瘤并产生免疫记忆。

6. 公司概况：ARTHEx Biotech

ARTHEx Biotech是一家临床阶段的生物技术公司，专注于透过调节基因表现开发创新药物。我们的先导化合物ATX-01正在I-IIa期ArthemiR™研究中进行评估，用于治疗一种罕见的神经肌肉疾病－1型强直性肌肉营养不良症(DM1)。 ArtemiR研究由EIC加速器计画共同资助，资助协议编号为N0 190181217。 Artemis正在推进其内部药物发现引擎，以识别和开发基于核酸的疗法，用于治疗其他存在巨大未满足医疗需求的疾病，包括遗传性疾病。公司总部位于西班牙瓦伦西亚。

产品概述：ATX-01

ATX-01 是一种抗 miR 寡核苷酸，旨在靶向参与 DM1 病理的 microRNA 23b (miR-23b)。已证明 ATX-01 在人类 DM1 成肌细胞系和两种小鼠模型中具有独特的双重作用机制，可降低毒性 DMPK mRNA 并增加 MBNL 蛋白的产生。 ATX-01 由 ARTHEx 的内部发现引擎发现，该引擎旨在识别、设计和优化新型基因表现调节剂，并确保优先递送至受疾病影响的目标组织。该药物目前正处于 I/II 期临床开发阶段，用于治疗第 1 型强直性肌肉营养不良症。

DelveInsight 寡核苷酸分析展望

• 各公司寡核苷酸合作详细商业评估分析

本报告对该药物进行了详细的商业评估，包括合作、协议、许可和收购趋势。报告还包含细分领域，以表格形式对企业合作（许可/伙伴关係）、企业学术合作和收购进行了分析。

• 寡核甘酸竞争情形

本报告对各公司（按疗法、开发阶段和技术）进行了比较评估。

目录

• 简介
• 函数
• 作用机制
• 结构
• 用途
• 寡核苷酸合作研究公司分析
• 公司比较评估（依疗法、开发阶段和技术）
• 依产品类型评估
• 依阶段和产品类型评估
• 依给药途径评估
• 以分期和给药途径评估
• 依分子类型评估
• 依阶段和分子类型评估
• 公司概况
• 产品描述
• 研发活动
• 产品开发活动
• 比较分析

DelveInsight's, "Oligonucleotides - Competitive landscape, 2025," report provides comprehensive insights about 280+ companies and 320+ drugs in Oligonucleotides Competitive landscape. It covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered:

• Global coverage

Oligonucleotides: Understanding

Oligonucleotides: Overview

Oligonucleotides are oligomers composed of repeating nucleotide monomers, comprising deoxyribose or ribose sugar, nitrogenous bases, and a phosphate backbone. Oligonucleotides possess a unique capability to bind specifically to their complements, such as DNA or RNA, leading to the formation of duplexes or, less frequently, higher-order hybrids. This characteristic enables the use of Oligonucleotides as probes for identifying specific DNA or RNA sequences. Oligonucleotide (ON) is a short strand of nucleic acid polymers mostly comprising of thirteen to twenty-five nucleotides, which can hybridize to targeted DNA or RNA. They are categorized into classes including antisense Oligonucleotides (ASOs), small interfering RNA (siRNA), microRNA (miRNAs), and aptamer, which are currently being explored for their use in neurodegenerative disorders, cancer, and even orphan diseases.

They are also undergoing clinical trials for the treatment of dermatological, gastrointestinal, and hormonal disorders. Oligonucleotides can be used to modulate gene expression via a range of processes including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation, non-coding RNA inhibition, gene activation and programmed gene editing. Oligonucleotide therapeutics-including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and splice-switching oligonucleotides-represent a transformative class of drugs with the potential to treat a wide spectrum of genetic and acquired diseases. Despite their promise, the clinical success of these molecules is tightly linked to the development of efficient delivery systems that can overcome their inherent biological limitations. Due to their large molecular weight, hydrophilicity, and negative charge, oligonucleotides face significant barriers in reaching intracellular targets. Additionally, their susceptibility to degradation by nucleases in biological fluids, limited ability to penetrate tissues, rapid renal clearance, and the need for tissue-specific targeting further complicate systemic delivery.

Oligonucleotides have revolutionized diagnostics and therapeutics due to their high specificity, programmability, and ease of synthesis. In diagnostics, they serve as primers in PCR/qPCR, probes in hybridization assays like FISH, and components in advanced platforms like microarrays and CRISPR-based tests. Aptamers enable targeted imaging by binding specific biomarkers, while chemical modifications enhance their stability in vivo. Therapeutically, oligonucleotides include aptamers (e.g., pegaptanib), antisense oligonucleotides (e.g., nusinersen), and siRNAs (e.g., patisiran), targeting diseases by modulating or silencing gene expression. They also underpin mRNA/DNA vaccines, miRNA inhibitors, and CRISPR-based gene editing. Despite challenges like off-target effects and rapid clearance, innovations such as GalNAc conjugation and AI-guided design are improving delivery, specificity, and clinical outcomes, driving progress in precision medicine.

Report Highlights:

• In March 2025, Korro Bio, Inc. a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to the investigational medicine KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD).
• In January 2025, Arrowhead Pharmaceuticals, Inc. announced that the US Food and Drug Administration (FDA) had accepted the New Drug Application (NDA) for investigational plozasiran for the treatment of familial chylomicronemia syndrome (FCS), a severe and rare genetic disease.
• In February 2025, AusperBio Therapeutics, Inc. and Ausper Biopharma Co., Ltd. (together AusperBio), a clinical-stage biotechnology company, announced recent progress in the ongoing clinical development of its lead candidate AHB-137, an antisense oligonucleotide (ASO) therapeutic for functional cure of chronic Hepatitis B (CHB)
• In December 2024, Vir Biotechnology, Inc. announced that tobevibart and elebsiran have received U.S. Food and Drug Administration (FDA) Breakthrough Therapy designation and European Medicines Agency (EMA) Priority Medicines (PRIME) designation for the treatment of chronic hepatitis delta (CHD).
• In December 2024, The FDA had granted breakthrough therapy designation to Stoke Therapeutics investigational antisense agent STK-001 for the treatment of genetically confirmed Dravet syndrome (DS), a rare epilepsy disorder.
• In November 2024, Ionis Pharmaceuticals that the US Food and Drug Administration (FDA) had accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. The FDA has set an action date of August 21, 2025 under the Prescription Drug User Fee Act (PDUFA).
• In October 2024, Ribocure Pharmaceuticals AB and Suzhou Ribo Life Science Ltd (Ribo) received authorization from the Swedish Medicinal Product Agency (MPA), to initiate a Phase II clinical trial in Sweden with the lipid-lowering siRNA drug RBD5044 that targets APOC3. The trial will evaluate efficacy and safety in patients with mixed dyslipidemia
• In September 2024, WVE-N531, an exon-skipping oligonucleotide developed by Wave Life Sciences for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping, received Orphan Drug Designation from the US Food and Drug Administration (FDA).
• In September 2024, NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that the Food and Drug Administration (FDA) has granted rare pediatric disease designation to NS050/NCNP-03 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne).
• In May 2024, Sunhawk Vision Biotech announced that it has received authorization from the US FDA to commence a Phase II clinical trial for myopia control in children.
• In May 2024, Imvax, Inc., announced the completion of enrollment in its randomized, multicenter, double-blind, placebo-controlled Phase IIb clinical trial of IGV-001 in patients with newly diagnosed glioblastoma (ndGBM).

Oligonucleotides: Company and Product Profiles (Marketed Therapies)

1. Company Overview: Novartis

Novartis International AG is a global healthcare company based in Switzerland, and one of the largest pharmaceutical companies in the world. Founded in 1996 through the merger of Ciba-Geigy and Sandoz, Novartis is involved in the research, development, manufacturing, and marketing of a broad range of healthcare products, with a focus on pharmaceuticals, generics, and biosimilar. Novartis has committed to sustainable healthcare solutions, including efforts to reduce environmental impact and improve global access to medicines. It also works on initiatives to provide affordable drugs in low- and middle-income countries.

Product Description: LEQVIO

LEQVIO (inclisiran), a first-in-class small interfering RNA (siRNA)-based therapeutic developed by Novartis, is directed to PCSK9 (proprotein convertase subtilisin kexin type 9) mRNA. LEQVIO works differently from other therapies by preventing the production of the target protein in the liver, increasing hepatic uptake of LDL-C, and clearing it from the bloodstream. In 2023, LEQVIO (inclisiran) was approved by MHLW for familial and non-familial hypercholesterolemia and for patients who are at a high risk of developing cardiovascular events. The dosing regimen includes an initial injection, a second dose at three months, followed by maintenance doses every six months. Clinical studies have demonstrated that LEQVIO, in combination with statins, can reduce LDL-C levels by approximately 50%.

2. Company Overview: Company Overview: Astellas Pharma

Astellas Pharma Inc. is a Japanese multinational pharmaceutical company headquartered in Tokyo, known for its innovative approach to healthcare solutions. Established in 2005 through the merger of Yamanouchi Pharmaceutical Co. and Fujisawa Pharmaceutical Co., Astellas focuses on areas such as oncology, urology, immunology, and neuroscience. The company is committed to improving patient outcomes by advancing therapies that address unmet medical needs, with a significant emphasis on research and development. Astellas also actively pursues global partnerships and collaborations to accelerate drug discovery and expand its market presence across key regions, including North America, Europe, and Asia.

Product Description: IZERVAY

Avacincaptad pegol, marketed under the brand name IZERVAY, is an approved medication specifically designed for the treatment of geographic atrophy. Avacincaptad pegol is an RNA aptamer that is covalently linked to a branched polyethylene glycol (PEG) molecule. Its primary function is to inhibit complement factor C5, a key component in the complement cascade involved in inflammatory processes associated with AMD. By blocking the cleavage of C5 into its active fragments (C5a and C5b), avacincaptad pegol aims to reduce inflammation and slow the progression of GA. IZERVAY was approved by the US Food and Drug Administration on August 4, 2023, for the treatment of GA secondary to AMD and is currently under review by the European Medicines Agency. The drug is also being evaluated for the treatment of Stargardt disease.

3. Company Overview: Company Overview: Alnylam Pharmaceuticals

Alnylam Pharmaceuticals is a biopharmaceutical company headquartered in Cambridge, Massachusetts, focused on the discovery, development, and commercialization of RNA interference (RNAi) therapeutics. Founded in 2002, the company is a recognized pioneer in RNAi technology, which enables the silencing of specific genes associated with disease. Alnylam's pipeline targets a range of rare genetic, cardio-metabolic, and hepatic infectious diseases, with several approved products, including ONPATTRO(R), GIVLAARI(R), OXLUMO(R), and AMVUTTRA(R). The company maintains a strong emphasis on scientific innovation, strategic partnerships, and global expansion, aiming to transform the treatment landscape for patients with limited therapeutic options.

Product Description: AMVUTTRA

AMVUTTRA(R) (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection by a healthcare professional, AMVUTTRA is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and is approved in the U.S. for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.

4. Company Overview: Company Overview: Ionis Pharmaceuticals

Ionis Pharmaceuticals, Inc., founded in 1989 and headquartered in Carlsbad, California, is a biotechnology company specializing in RNA-targeted therapeutics. The company has been a pioneer in the development of antisense technology, aiming to address a broad range of diseases, including neurological disorders, cardiovascular conditions, and rare diseases. Ionis focuses on discovering and developing RNA-based therapies using its proprietary platform, which includes advanced antisense and RNA interference (RNAi) technologies. The company emphasizes precision medicine and partners with major pharmaceutical firms to advance its pipeline. With a diversified portfolio of wholly owned and partnered programs, Ionis continues to expand its presence in the biopharmaceutical sector. It is publicly traded on the Nasdaq and remains focused on advancing RNA-targeted approaches to deliver transformative therapies to patients.

Product Description: TEGSEDI

Tegsedi (inotersen) is an antisense oligonucleotide therapy designed to treat adults with hereditary transthyretin-mediated amyloidosis (hATTR) experiencing stage 1 or 2 polyneuropathy. It functions by reducing the production of transthyretin (TTR) protein, thereby slowing the progression of nerve damage. Administered as a 284 mg subcutaneous injection once weekly, Tegsedi requires regular monitoring due to potential serious side effects, including low platelet counts and kidney inflammation. Common side effects encompass injection site reactions, nausea, headache, fatigue, and fever. In the United States, Tegsedi is available only through a restricted program called the Tegsedi Risk Evaluation and Mitigation Strategy (REMS) Program to ensure safe use. TEGSEDI is a once weekly, self-administered subcutaneous medicine approved in the U.S., Europe, Canada and Brazil for the treatment of patients with ATTRv-PN.

Oligonucleotides: Company and Product Profiles (Pipeline Therapies)

1. Company Overview: Novartis Pharmaceuticals

Novartis Pharmaceuticals is a leading global healthcare company headquartered in Switzerland, dedicated to reimagining medicine to improve and extend people's lives. Operating through a robust portfolio that includes innovative prescription medicines, Novartis focuses on key therapeutic areas such as oncology, immunology, neuroscience, and cardiovascular disease. The company leverages advanced science and digital technologies to deliver high-value treatments while maintaining a strong commitment to research and development. With a presence in over 150 countries, Novartis continues to drive sustainable growth through strategic partnerships, a deep pipeline of novel therapies, and a clear focus on operational excellence and patient-centric innovation.

Product Description: Pelacarsen

Pelacarsen (TQJ230), also known as IONIS-APO(a)-LRx, AKCEA-APO(a)-LRx, and TQJ230, is an investigational antisense medicine designed to reduce apolipoprotein(a) in the liver to offer a direct approach for reducing lipoprotein(a), or Lp (a) - a very atherogenic and thrombogenic form of LDL. Elevated Lp (a) is recognized as an independent genetic cause of coronary artery disease, heart attack, stroke, and peripheral arterial disease. Pelacarsen, an investigational antisense medicine designed to lower Lp(a), was discovered by Ionis and licensed to Novartis in 2019. Currently the drug is in Phase III for the treatment of Hyperlipoproteinaemia.

2. Company Overview: Company Overview: Oncotelic

Oncotelic Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for cancer and rare diseases. Headquartered in Agoura Hills, California, the company leverages RNA therapeutics and immuno-oncology approaches to target difficult-to-treat tumors and fibrotic conditions. Its lead programs include treatments for glioblastoma, melanoma, and idiopathic pulmonary fibrosis, among others. Oncotelic integrates expertise in TGF-B inhibition and oligonucleotide therapeutics to modulate disease pathways with precision. Through a combination of in-house R&D and collaborative partnerships, the company aims to deliver impactful therapies that address significant unmet medical needs.

Product Description: Trabedersen

OT-101, also referred to Trabedersen, is a novel antisense oligodeoxynucleotide (ODN) developed by Oncotelic for the treatment of patients with pancreatic carcinoma, malignant melanoma, colorectal carcinoma, high-grade glioma (HGG), and other transforming growth factor beta 2 (TGF-B2) overexpressing malignancies (e.g., prostate carcinoma, renal cell carcinoma, etc.). Trabedersen is a synthetic 18-mer phosphorothioate oligodeoxynucleotide (S-ODN) complementary to the messenger ribonucleic acid (mRNA) of the human TGF-B2 gene. Cancers overexpress TGF-B, which suppresses host innate immune response to the cancers. Treatment with OT-101 lifts the TGF-B cloaking effect and allows innate or therapeutic immunity to attack and eliminate the cancers. The company had completed Phase II for pancreatic cancer and melanoma, and Phase II in glioblastoma with robust efficacy and safety.

3. Company Overview: Company Overview: Wave Life Sciences

Wave Life Sciences is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave's RNA medicines platform, PRISM(R), combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. Its toolkit of RNA-targeting modalities includes editing, splicing, RNA interference and antisense silencing, providing Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave's diversified pipeline includes clinical programs in Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington's disease, as well as a preclinical program in obesity. Driven by the calling to "Reimagine Possible", Wave is leading the charge toward a world in which human potential is no longer hindered by the burden of disease.

Product Description: WVE-N531

WVE-N531 is an exon skipping oligonucleotide being developed as a disease modifying treatment for boys with Duchenne muscular dystrophy amenable to exon 53 skipping. WVE-N531 was designed using Wave's best-in-class oligonucleotide chemistry modifications, including PN backbone chemistry. WVE-N531 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food & Drug Administration. Currently, the drug is in Phase II stage of Clinical trial evaluation for the treatment of Myotonic dystrophy.

4. Company Overview: Company Overview: Autotelic Bio

Autotelic Bio is a clinical-stage South Korean biotechnology company founded in 2015, specializing in the development of next-generation nucleic acid-based therapeutics, particularly antisense oligonucleotides (ASOs). Utilizing proprietary platforms like ASODE and CATs, the company aims to deliver targeted and optimized ASO therapies with enhanced efficacy and safety profiles. Its diversified pipeline includes candidates for oncology, idiopathic pulmonary fibrosis (IPF), and fixed-dose combinations for metabolic disorders. As of March 2022, Autotelic Bio has secured USD 18.2 million in funding, with a USD 12.6 million Series B round led by investors such as UTC Investment and Stonebridge Ventures. The company aspires to become a global leader in cancer treatment by addressing previously undruggable targets.

Product Description: ATB 301

ATB-301 is an investigational antisense oligonucleotide (ASO) therapy developed by Autotelic Bio, targeting Transforming Growth Factor Beta 2 (TGF-B2). It is being evaluated in combination with recombinant interleukin-2 (Aldesleukin) for the treatment of advanced or metastatic solid tumors, including pancreatic cancer and renal cell carcinoma. The therapy aims to inhibit TGF-B2 to modulate the tumor microenvironment and enhance immune responses. The drug demonstrated suppression of tumor growth and increase of CD8 T cells in cancer tissue through inhibition of TGF-B2 in pancreatic cancer model. Currently, the drug is in Phase I stage of Clinical trial evaluation for the treatment of Pancreatic Cancer.

5. Company Overview: Company Overview: Tallac Therapeutics

Tallac Therapeutics is a clinical-stage biopharmaceutical company based in California, focused on developing novel immunotherapies for cancer. The company's proprietary Toll-like receptor agonist antibody conjugate (TAAC) platform is designed to stimulate innate and adaptive immune responses directly within the tumor microenvironment. Tallac's lead programs aim to activate Toll-like receptor 9 (TLR9) in a targeted manner, with the goal of enhancing anti-tumor immunity while minimizing systemic toxicity. By leveraging its platform to create next-generation immuno-oncology treatments, Tallac is advancing a pipeline of therapeutic candidates intended to address various solid tumors and improve patient outcomes.

Product Description: TAC001

TAC-001 is a novel antibody-oligonucleotide conjugate designed to deliver systemic TLR9 agonism with targeted immune activation of B cells, which plays a key role in cancer immunity. In preclinical studies, systemically administered TAC-001 is active as a single agent across a number of syngeneic tumor models including ones with immune suppression and resistance, leading to complete tumor eradication and immune memory.

6. Company Overview: Company Overview: ARTHEx Biotech

ARTHEx Biotech is a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression. The Company's lead investigational compound, ATX-01, is being evaluated for the treatment of myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder, in the Phase I-IIa ArthemiR(TM)trial. ArthemiR trial is co-funded by EIC Accelerator program under the Grant Agreement N0 190181217. ARTHEx is advancing its in-house discovery engine to identify and develop nucleic acid-based therapies for other disorders with high unmet medical need, including genetically-driven diseases. The Company headquarters are in Valencia, Spain.

Product Description: ATX-01

ATX-01 is an antimiR oligonucleotide designed to target microRNA 23b (miR-23b), which is involved in the pathogenesis of DM1. It has been demonstrated, in human DM1 myoblast cell lines and in two murine models, that ATX-01 has a unique, dual mechanism of action which reduces toxic DMPK mRNA and increases MBNL protein production. ATX-01 was discovered through ARTHEx's in-house discovery engine, which was built to identify, design and optimize novel gene expression modulators and ensure their preferential delivery to target tissues affected by the disease. Currently the drug is in Phase I/II stage of its clinical development for the treatment of Myotonic Dystrophy Type 1.

Oligonucleotides Analytical Perspective by DelveInsight

• In-depth Commercial Assessment: Oligonucleotides Collaboration Analysis by Companies

The Report provides in-depth commercial assessment of drugs that have been included, which comprises collaboration, agreement, licensing and acquisition - deals values trends. The sub-segmentation is described in the report which provide company-company collaboration (licensing/partnering), company academic collaboration and acquisition analysis in tabulated form.

• Oligonucleotides Competitive Landscape

The report comprises of comparative assessment of Companies (by therapy, development stage, and technology).

Oligonucleotides Report Assessment

• Company Analysis
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions:

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Oligonucleotides drugs?
• How many Oligonucleotides drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Oligonucleotides?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Oligonucleotides therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Oligonucleotides and their status?
• What are the key designations that have been granted to the emerging and approved drugs?

Key Players

• Novartis
• Astellas
• Alnylam Pharmaceuticals
• Ionis Pharmaceuticals
• 4D Molecular Therapeutics
• Avidity Biosciences
• Suzhou Ribo Life Science
• Amgen
• GSK
• ProQR Therapeutics
• Stoke Therapeutics
• MiNA Therapeutics
• Sylentis
• GSK
• Silexion Therapeutics
• Novo Nordisk A/S
• Bio-Path Holdings
• Sunhawk Vision Biotech
• Isarna Therapeutics
• Sirnaomics
• Laboratoire Thea
• Dyne Therapeutics
• Vertex Pharmaceuticals
• Korro Bio
• Praxis-Precision-Medicines
• Vico Therapeutics
• BioMarin Pharmaceutical
• TransCode Therapeutics
• TME therapeutics
• ARTHEx Biotech
• aptaTargets
• CSPC Zhongnuo Pharmaceutical
• ExoRNA Bioscience
• Visirna Therapeutics
• AiCuris
• Comanche Biopharma
• Tallac Therapeutics

Key Products

• LEQVIO
• IZERVAY
• AMVUTTRA
• TEGSEDI
• 4D-150
• Delpacibart Etedesiran
• RBD1007
• Olpasiran
• GSK3228836
• Sepofarsen
• STK-001
• MTL-CEBPA
• SYL-1801
• Loder
• CDR132L
• BP1001
• SHJ002
• ISTH0036
• STP705
• Ultevursen
• DYNE-101
• VX-670
• KRRO 110
• PRAX-222
• VO659
• BMN 351
• TTX-MC138
• TME151
• ATX-01
• ApTOLL
• SYH2062
• ER2001
• VSA012
• AIC468
• CBP-4888
• ALTA-002

Table of Contents

Introduction

Executive Summary

Oligonucleotides: Overview

• Introduction
• Function
• Mechanism of Action
• Structure
• Applications

Oligonucleotides -Analytical Perspective: In-depth Commercial Assessment

• Oligonucleotides Collaboration Analysis by Companies

Competitive Landscape

• Comparative Assessment of Companies (by therapy, development stage, and technology)

Therapeutic Assessment

• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type

Oligonucleotides: Company and Product Profiles (Marketed Therapies)

Novartis Pharmaceuticals

• Company Overview

LEQVIO

• Product Description
• Research and Development Activities
• Product Developmental Activities

Oligonucleotides: Company and Product Profiles (Pipeline Therapies)

Late Stage Products (Phase III)

• Comparative Analysis

Novartis Pharmaceuticals

• Company Overview

Pelacarsen

• Product Description
• Research and Development Activities
• Product Developmental Activities

Mid Stage Products (Phase II)

• Comparative Analysis

Oncotelic

• Company Overview

Trabedersen

• Product Description
• Research and Development Activities
• Product Developmental Activities

Early Stage Products (Phase I)

• Comparative Analysis

Autotelic Bio

• Company Overview

ATB 301

• Product Description
• Research and Development Activities
• Product Developmental Activities

Preclinical and Discovery Stage Products

• Comparative Analysis

Company Name

• Company Overview

Product Name

• Product Description
• Research and Development Activities
• Product Developmental Activities

Inactive Products

• Comparative Analysis

Oligonucleotides- Unmet needs

Oligonucleotides - Market drivers and barriers

List of Tables

• Table 1 Total Products for Oligonucleotides
• Table 2 Late Stage Products
• Table 3 Mid Stage Products
• Table 4 Early Stage Products
• Table 5 Pre-clinical & Discovery Stage Products
• Table 6 Assessment by Product Type
• Table 7 Assessment by Stage and Product Type
• Table 8 Assessment by Route of Administration
• Table 9 Assessment by Stage and Route of Administration
• Table 10 Assessment by Molecule Type
• Table 11 Assessment by Stage and Molecule Type
• Table 12 Inactive Products

List of Figures

• Figure 1 Total Products for Oligonucleotides
• Figure 2 Late Stage Products
• Figure 3 Mid Stage Products
• Figure 4 Early Stage Products
• Figure 5 Preclinical and Discovery Stage Products
• Figure 6 Assessment by Product Type
• Figure 7 Assessment by Stage and Product Type
• Figure 8 Assessment by Route of Administration
• Figure 9 Assessment by Stage and Route of Administration
• Figure 10 Assessment by Molecule Type
• Figure 11 Assessment by Stage and Molecule Type
• Figure 12 Inactive Products