寡核甘酸市場 - 競爭情形(2025年)

"寡核苷酸市場 - 競爭格局 (2025)" 報告全面分析了寡核苷酸領域 280 多家公司和 320 多種藥物的競爭格局。報告涵蓋了按產品類型、階段、給藥途徑和分子類型進行的治療評估。此外，報告還重點介紹了該領域未上市的研發線產品。

對象地區

• 作為對象的世界

寡核甘酸理解

寡核甘酸概要

寡核苷酸是由重複核苷酸單體組成的寡聚體，這些單體由脫氧核糖或核糖、含氮鹼基和磷酸骨架組成。寡核苷酸具有獨特的能力，可以特異性地與其互補序列（例如 DNA 和 RNA）結合，形成雙股體，以及（較少見的）高階雜合體。這項特性使得寡核苷酸可用作探針來辨識特定的DNA或RNA序列。寡核苷酸（ON）是短鏈核酸聚合物，通常由13至25個核苷酸組成，可與目標DNA或RNA雜交。它們分為反義寡核苷酸（ASO）、小幹擾RNA（siRNA）、微小RNA（miRNA）和適體等類別，目前正在探索其在神經退化性疾病、癌症甚至罕見疾病中的應用。

它們也正在臨床試驗中用於治療皮膚、胃腸道和荷爾蒙失調。寡核苷酸可透過多種途徑調控基因表達，包括RNAi、RNase H介導的切割降解標靶基因、剪接調控、非編碼RNA抑制、基因活化和程序性基因編輯。寡核苷酸療法，包括反義寡核苷酸 (ASO)、小幹擾RNA (siRNA) 和剪接轉換寡核苷酸，是另一類具有突破性潛力的藥物，有望治療多種遺傳和後天疾病。儘管這些分子前景廣闊，但它們的臨床成功與能夠克服其固有生物學局限性的高效遞送系統的開發密切相關。由於寡核苷酸分子量大、親水性強且帶負電荷，其在到達細胞內標靶方面面臨巨大障礙。此外，它們易被體液中的核酸酶降解、組織穿透能力有限、腎臟快速清除以及需要組織特異性靶向，進一步增加了全身遞送的複雜性。

寡核苷酸因其高特異性、可編程性和易於合成，徹底改變了診斷和治療。在診斷領域，它們可用作PCR/qPCR的引子、FISH等雜交檢測的探針，以及微陣列和基於CRISPR的檢測等先進平台的組件。適體可結合特定生物標記以實現標靶成像，並經過化學修飾以提高其體內穩定性。在治療領域，寡核苷酸包括適體（例如，哌加他尼）、反義寡核苷酸（例如，nusinersen）和siRNA（例如，patisiran），它們透過調節或沉默基因表現來靶向治療疾病。它也是mRNA/DNA疫苗、miRNA抑制劑和基於CRISPR的基因編輯的基礎。儘管存在脫靶效應和快速清除等挑戰，但GalNAc偶聯和AI引導設計等創新技術已改善了藥物的遞送、特異性和臨床療效，從而推動了精準醫療的進步。

報告的亮點

• 2025年3月，專注於開發基於RNA編輯的新型基因藥物（用於治療罕見疾病和高發病）的臨床階段生物製藥公司Korro Bio, Inc.宣布，美國食品藥品監督管理局 (FDA) 已授予其在研藥物KRRO-110孤兒藥資格認定，用於治療α-1抗胰蛋白酶缺乏症(AATD)。
• 2025年1月，Arrowhead Pharmaceuticals, Inc.宣布，美國食品藥物管理局 (FDA) 已接受其在研藥物百憂解(prozacilan)的新藥申請，該藥物用於治療家族性乳糜微粒血症綜合症(FCS)，這是一種嚴重且罕見的遺傳性疾病。
• 2025年2月，臨床階段生物技術公司AusperBio Therapeutics, Inc.和Ausper Biopharma Co., Ltd.宣布了其主要候選化合物AHB-137正在進行的臨床開發的最新進展。 AHB-137是一種反義寡核苷酸(ASO)療法，旨在實現慢性乙型肝炎(CHB)的功能性治癒。
• 2024年12月，Vir Biotechnology, Inc.宣布，tobevibart和elebsiran分別獲得美國食品藥物管理局(FDA)頒發的突破性療法認定和歐洲藥品管理局(EMA)頒發的治療慢性丁型肝炎(CHD)的優先療法認定。
• 2024年12月，FDA授予Stoke Therapeutics公司的在研藥物STK-001突破性療法認定，用於治療Dravet綜合徵，這是一種罕見的、經基因確診的癲癇病。
• 2024年11月，Ionis Pharmaceuticals宣布，美國食品藥物管理局 (FDA) 已受理donidalorsen的新藥申請 (NDA)。 donidalorsen是一種RNA標靶藥物，用於預防12歲及以上成人和兒童遺傳性血管性水腫(HAE)患者的癲癇發作。 FDA依據 "處方藥使用者付費法案" (PDUFA)，將核准日期設定為2025年8月21日。
• 2024年10月，Ribocure Pharmaceuticals AB和蘇州瑞博生命科技有限公司獲得瑞典藥品管理局 (MPA) 的批准，將在瑞典啟動RBD5044的II期臨床試驗。 RBD5044是一種針對APOC3的降脂siRNA藥物。該研究將評估其在混合性血脂異常患者的療效和安全性。
• 2024年9月，Wave Life Sciences公司開發的用於治療杜氏肌肉營養不良症（DMD）的外顯子跳躍寡核苷酸WVE-N531獲得了美國食品藥品監督管理局（FDA）的孤兒藥資格認定。 DMD適用於治療53號外顯子跳躍的疾病。
• 2024年9月，日本新藥株式會社的子公司NS Pharma, Inc.獲得了美國食品藥物管理局（FDA）的孤兒藥資格認定。 2024年9月，日本新藥株式會社的子公司NS Pharma Co., Ltd.宣布，正在開發的用於治療杜氏肌肉營養不良症的NS050/NCNP-03已獲得美國食品藥物管理局（FDA）的罕見兒科疾病資格認定。
• 2024年5月，Sunhawk Vision Biotech宣布已獲得美國FDA批准，開始進行兒童近視控制的II期臨床試驗。
• 2024年5月，Imvax, Inc.宣布完成IGV-001針對新診斷膠質母細胞瘤(ndGBM)患者的隨機、多中心、雙盲、安慰劑對照IIb期臨床試驗的招募工作。

寡核苷酸寡核苷酸：公司與產品簡介（目錄）

1. 公司概況：諾華

諾華國際股份公司是一家總部位於瑞士的全球醫療保健公司，也是全球最大的製藥公司之一。諾華公司成立於1996年，由汽巴-嘉基公司和山德士公司合併而成，致力於研究、開發、生產和銷售廣泛的醫療保健產品，專注於藥品、仿製藥和生物類似藥。諾華致力於提供永續的醫療保健解決方案，包括減少對環境的影響並改善全球藥品的可近性。諾華也致力於為中低收入國家提供負擔得起的藥品。

產品概述：LEQVIO

LEQVIO (Inclisiran) 是由諾華公司開發的首創siRNA（小幹擾RNA）療法，作用於PCSK9（前蛋白轉化酶枯草桿菌蛋白酶9型）的mRNA。與其他療法不同，LEQVIO能夠抑制肝臟中目標蛋白的產生，從而增加肝臟對LDL-C的攝取並將其從血液中清除。 2023年，LEQVIO（通用名：inclisiran）獲得厚生勞動省核准，用於治療家族性和非家族性高膽固醇血症，且心血管事件風險較高的患者。給藥方案為首次注射，三個月後再注射，每六個月維持注射一次。臨床試驗表明，LEQVIO與他汀類藥物聯合使用可降低約50%的低密度脂蛋白膽固醇（LDL-C）水平。

2. 公司簡介：安斯泰來製藥

安斯泰來製藥是一家總部位於日本東京的日本跨國製藥公司，以其創新的醫療保健解決方案而聞名。安斯泰來製藥成立於2005年，由山之內製藥和藤澤製藥合併而成，專注於腫瘤學、泌尿學、免疫學和神經科學等領域。安斯泰來製藥專注於研發，致力於透過推進治療手段來滿足尚未滿足的醫療需求，進而改善病患的治療效果。安斯泰來製藥也積極尋求全球合作夥伴關係和聯盟，以加速藥物研發，並擴大其在北美、歐洲和亞洲等關鍵地區的市場佔有率。

產品概述：IZERVAY

Avacincaptad pegol，以 IZERVAY 為商品名上市，已獲準用於治療地圖樣萎縮。 Avacincaptad pegol 是一種與支鏈聚乙二醇 (PEG) 分子共價連接的 RNA 適體。其主要功能是抑制補體因子 C5，C5 是參與 AMD 相關發炎過程的補體級聯的關鍵組成部分。透過抑制 C5 裂解為活性片段（C5a 和 C5b），Avacincaptad pegol 旨在減輕發炎並減緩地圖樣萎縮 (GA) 的進展。 IZERVAY 於 2023 年 8 月 4 日獲得美國食品藥物管理局 (FDA) 批准，用於治療繼發於老年黃斑部病變 (AMD) 的 GA，目前正在接受歐洲藥品管理局 (EMA) 的審查。該藥物也正在接受 Stargardt 病治療評估。

3. 公司概況：Alnylam Pharmaceuticals

Alnylam Pharmaceuticals 是一家總部位於麻薩諸塞州劍橋市的生物製藥公司，專注於 RNA 幹擾 (RNAi) 療法的發現、開發和商業化。該公司成立於 2002 年，致力於開發 RNAi 技術，以滅活與疾病相關的特定基因。Alnylam 以在罕見遺傳、心臟代謝和肝臟感染性疾病治療領域開創性地而聞名。 Alnylam 的產品線涵蓋多種罕見遺傳、心臟代謝和肝臟感染性疾病，擁有多個已核准的產品，包括 ONPATTRO(R)、GIVLAARI(R)、OXLUMO(R) 和 AMVUTTRA(R)。公司專注於科學創新、策略合作夥伴關係和全球擴張，致力於為治療選擇有限的患者帶來全新的治療方案。

產品概述：AMVUTTRA

AMVUTTRA(R)（布特里斯蘭）是一種 RNAi 療法，可快速敲低甲狀腺素蛋白 (TTR)，從而解決轉甲狀腺素蛋白 (ATTR) 澱粉樣變性的根本病因。 AMVUTTRA 由醫療保健專業人員每季皮下注射一次，已在超過 15 個國家/地區獲批上市，用於治療患有遺傳性轉甲狀腺素蛋白介導的澱粉樣變性多發性神經病變 (hATTR-PN) 的成年患者，並在美國獲批上市，用於治療患有野生型或遺傳性轉甲狀腺素蛋白介導的澱粉樣心肌病變的成年患者，旨在降低心血管死亡率、心血管住院率和急診心臟衰竭就診率。

4. 公司概況：Ionis Pharmaceuticals

Ionis Pharmaceuticals 成立於 1989 年，總部位於加州卡爾斯巴德。它是一家專注於 RNA 標靶治療的生物技術公司。該公司率先開發反義技術，用於治療多種疾病，包括神經系統疾病、心血管疾病和罕見疾病。 Ionis 專注於利用其專有平台（包括先進的反義和 RNA 幹擾 (RNAi) 技術）來發現和開發基於 RNA 的療法。公司專注於精準醫療，並與大型製藥公司合作，以推進其產品線。憑藉著完全自主開發和合作的多元化專案組合，Ionis 不斷拓展其在生物製藥領域的影響力。 Ionis 已在納斯達克上市，專注於開發 RNA 標靶療法，為患者提供創新療法。

產品概述：TEGSEDI

Tegsedi (Inoterzen) 是一種反義寡核苷酸療法，用於治療患有遺傳性轉甲狀腺素蛋白介導的澱粉樣變性 (hATTR) 並發展為 1 期或 2 期多發性神經病變的成年患者。該藥物透過抑制轉甲狀腺素蛋白 (TTR) 的產生來減緩神經病變的進展。 Tegsedi 每週一次，每次 284 毫克，皮下注射，但由於可能出現嚴重副作用，包括血小板數量減少和腎臟炎症，因此需要定期監測。常見的副作用包括注射部位反應、噁心、頭痛、疲勞和發燒。在美國，Tegsedi 僅透過名為 Tegsedi 風險評估和緩解策略 (REMS) 的受限項目提供，以確保安全使用。 Tegsedi 是一種每週一次的自行給藥皮下製劑，已在美國、歐洲、加拿大和巴西獲準用於治療 ATTRv-PN 患者。

寡核苷酸公司及產品簡介（管線治療）

1. 公司概況：諾華製藥

諾華製藥是一家總部位於瑞士的全球領先醫療保健公司，致力於重塑醫學，改善和延長人們的生活。諾華憑藉其強大的創新處方藥產品組合，專注於腫瘤學、免疫學、神經科學和心血管疾病等關鍵治療領域。諾華始終致力於研發，利用先進的科學和數位技術提供高價值療法。諾華業務遍及150多個國家，透過策略合作夥伴關係、豐富的創新療法管線以及對卓越營運和以患者為中心的創新的明確承諾，持續推動永續成長。

產品概述：Pelacarsen

Pelacarsen (TQJ230)，也稱為IONIS-APO(a)-LRx、AKCEA-APO(a)-LRx和TQJ230，是一種在研反義藥物，旨在提供一種直接降低肝臟載脂蛋白(a)和脂蛋白(a) (Lp(a))（一種高度致動脈粥樣硬化、易結的脂蛋白凝結）。脂蛋白a升高被認為是冠狀動脈疾病、心臟病發作、中風和周邊動脈疾病的獨立遺傳原因。 Pelacarsen 是一種旨在降低脂蛋白a的在研反義藥物，由 Ionis 發現，並於 2019 年授權給諾華公司。該藥物目前處於治療高脂蛋白血症的 III 期臨床試驗階段。

2. 公司概況：Oncotelic

Oncotelic Therapeutics 是一家臨床階段的生物製藥公司，專注於開發針對癌症和罕見疾病的創新療法。該公司總部位於加州阿古拉山，利用 RNA 療法和免疫腫瘤學方法治療難治性腫瘤和纖維化疾病。該公司的主要項目包括治療膠質母細胞瘤、黑色素瘤、特發性肺纖維化等。 Oncotelic 結合 TGF-B 抑制和寡核苷酸療法的專業知識，精準調控疾病路徑。 Oncotelic 致力於透過內部研發和合作研究，提供有效的療法，滿足尚未滿足的醫療需求。

產品概述：Travedersen

OT-101（也稱為 travedersen）是由 Oncotelic 開發的一種新型反義寡脫氧核苷酸 (ODN)，用於治療胰腺癌、惡性黑色素瘤、結直腸癌、高級別膠質瘤 (HGG) 以及其他過度表達轉化生長因子 β2 (TGF-B2) 的惡性腫瘤，包括前列腺癌和腎細胞癌。 Travedersen 是一種合成的 18 聚體硫代磷酸酯寡脫氧核苷酸 (S-ODN)，與人類 TGF-B2 基因的信使核糖核酸 (mRNA) 互補。癌症過度表現TGF-B，抑制宿主對癌症的先天免疫反應。 OT-101治療可解鎖TGF-B的掩蔽效應，使先天性或治療性免疫能夠攻擊並消除癌症。 OT-101已完成胰臟癌和黑色素瘤的II期臨床試驗，膠質母細胞瘤的II期臨床試驗也證實其有效性和安全性。

3. 公司概況：Wave Life Sciences

Wave Life Sciences是一家生物技術公司，致力於釋放RNA藥物改變人類健康的廣泛潛力。 Wave的RNA藥物平台PRISM®融合了多種模式、化學創新和對人類遺傳學的深刻洞察，在治療罕見疾病和常見疾病方面取得了科學突破。其RNA標靶工具包包括編輯、剪接、RNA幹擾和反義沉默，為Wave提供了無與倫比的能力，能夠設計並持續交付最能解決疾病生物學問題的候選藥物。 Wave 多元化的研發管線包括杜氏肌肉營養不良症、α-1 抗胰蛋白酶缺乏症和亨丁頓舞蹈症的臨床項目，以及肥胖症的臨床前項目。秉承 "重塑無限可能" 的理念，Wave 正朝著一個讓人類潛能不再受疾病困擾的世界邁進。

產品概述：WVE-N531

WVE-N531 是一種外顯子跳躍寡核苷酸，旨在為適合進行 53 號外顯子跳躍療法的杜氏肌肉營養不良症兒童開發一種疾病修飾療法。 WVE-N531 採用 Wave 一流的寡核苷酸化學修飾技術，包括 PN 骨架化學修飾技術。 WVE-N531 已獲得美國食品藥物管理局 (FDA) 授予的孤兒藥資格和兒科罕見疾病資格。目前，該藥物正處於治療強直性肌肉營養不良症的 II 期臨床試驗階段。

4. 公司概況：Autotelic Bio

Autotelic Bio 是一家成立於 2015 年的韓國臨床階段生物技術公司，專注於開發下一代核酸療法，尤其是反義寡核苷酸 (ASO)。該公司利用其專有的 ASODE 和 CAT 平台，旨在提供具有更高療效和安全性的標靶優化 ASO 療法。該公司多元化的研發管線包括用於治療癌症、特發性肺纖維化 (IPF) 和代謝性疾病的聯合用藥候選藥物。截至 2022 年 3 月，Autotelic Bio 已獲得 1,820 萬美元融資，包括由 UTC Investment 和 Stonebridge Ventures 等投資者領投的 1,260 萬美元 B 輪融資。該公司旨在透過攻克先前無法治癒的癌症靶點，成為癌症治療的全球領導者。

產品概述：ATB 301

ATB-301 是由 Autotelic Bio 公司開發的一種針對轉化生長因子 β2 (TGF-β2) 的在研反義寡核苷酸 (ASO) 療法。目前正在評估該療法與重組白細胞介素 2（阿地白介素）聯合用於治療晚期或轉移性實體瘤，包括胰腺癌和腎細胞癌。該療法旨在抑制 TGF-β2，從而調節腫瘤微環境並增強免疫反應。在胰臟癌模型中，該藥物已顯示出透過抑制 TGF-β2 來抑制腫瘤生長並增加癌組織中 CD8 T 細胞的功能。目前，該藥物正處於胰臟癌治療的 I 期臨床試驗階段。

5.公司概況：Tallac Therapeutics

Tallac Therapeutics 是一家總部位於加州的臨床階段生物製藥公司，專注於開發新型癌症免疫療法。該公司專有的 Toll 樣受體激動劑抗體偶聯物 (TAAC) 平台旨在直接在腫瘤微環境中刺激先天性和適應性免疫反應。 Tallac 的主導計畫旨在靶向激活 Toll 樣受體 9 (TLR9​​)，以增強抗腫瘤免疫力，同時最大限度地降低全身毒性。 Tallac 利用其平台開發下一代免疫腫瘤療法，正在推進一系列候選藥物研發管線，旨在治療各種實體瘤並改善患者預後。

產品概述：TAC001

TAC-001 是一種新型抗體-寡核苷酸偶聯物，可發揮全身性 TLR9 激動作用，並靶向激活 B 細胞的免疫功能，而 B 細胞在癌症免疫中發揮關鍵作用。在臨床前研究中，全身給藥的TAC-001作為單一藥物在多種假基因腫瘤模型（包括免疫抑製或抗藥性模型）中顯示出活性，可完全清除腫瘤並產生免疫記憶。

6. 公司概況：ARTHEx Biotech

ARTHEx Biotech是一家臨床階段的生物技術公司，專注於透過調節基因表現開發創新藥物。我們的先導化合物ATX-01正在I-IIa期ArthemiR™研究中進行評估，用於治療一種罕見的神經肌肉疾病－1型強直性肌肉營養不良症(DM1)。 ArtemiR研究由EIC加速器計畫共同資助，資助協議編號為N0 190181217。 Artemis正在推進其內部藥物發現引擎，以識別和開發基於核酸的療法，用於治療其他存在巨大未滿足醫療需求的疾病，包括遺傳性疾病。公司總部位於西班牙瓦倫西亞。

產品概述：ATX-01

ATX-01 是一種抗 miR 寡核苷酸，旨在靶向參與 DM1 病理的 microRNA 23b (miR-23b)。已證明 ATX-01 在人類 DM1 成肌細胞系和兩種小鼠模型中具有獨特的雙重作用機制，可降低毒性 DMPK mRNA 並增加 MBNL 蛋白的產生。 ATX-01 由 ARTHEx 的內部發現引擎發現，該引擎旨在識別、設計和優化新型基因表現調節劑，並確保優先遞送至受疾病影響的目標組織。該藥物目前正處於 I/II 期臨床開發階段，用於治療第 1 型強直性肌肉營養不良症。

DelveInsight 寡核苷酸分析展望

• 各公司寡核苷酸合作詳細商業評估分析

本報告對該藥物進行了詳細的商業評估，包括合作、協議、許可和收購趨勢。報告還包含細分領域，以表格形式對企業合作（許可/夥伴關係）、企業學術合作和收購進行了分析。

• 寡核甘酸競爭情形

本報告對各公司（按療法、開發階段和技術）進行了比較評估。

目錄

• 簡介
• 函數
• 作用機制
• 結構
• 用途
• 寡核苷酸合作研究公司分析
• 公司比較評估（依療法、開發階段和技術）
• 依產品類型評估
• 依階段和產品類型評估
• 依給藥途徑評估
• 以分期和給藥途徑評估
• 依分子類型評估
• 依階段和分子類型評估
• 公司概況
• 產品描述
• 研發活動
• 產品開發活動
• 比較分析

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