이 보고서 '세계의 올리고뉴클레오티드 시장 - 경쟁 구도(2025년)'는 올리고뉴클레오티드 경쟁 구도에서 280개 이상의 기업과 320개 이상의 의약품에 대한 종합적인 고찰을 제공합니다.
올리고뉴클레오티드 이해
올리고뉴클레오티드 개요
올리고뉴클레오티드는 데옥시리보스 또는 리보스당, 질소염기, 인산골격으로 이루어지는 반복 뉴클레오티드 단량체로 이루어지는 올리고머입니다. DNA와 RNA 같은 상보체에 특이적으로 결합하는 독특한 능력을 가지고 있으며 이중 가닥이나 더 적은 빈도이지만 고차 하이브리드를 형성합니다. 이 특성으로 올리고 뉴클레오티드는 특정 DNA 또는 RNA 서열을 확인하는 프로브로 사용할 수 있습니다. 올리고뉴클레오티드(ON)는 핵산 중합체의 짧은 사슬이며, 대부분은 13-25개의 뉴클레오티드로 구성되며 표적 DNA 또는 RNA에 혼성화 할 수 있습니다. 안티센스 올리고뉴클레오티드(ASO), 저분자 간섭 RNA(siRNA), 마이크로RNA(miRNA), 압타머 등의 클래스로 분류되어 현재, 신경퇴행성 질환, 암, 나아가 희귀질환에서의 사용이 검토되고 있습니다.
또한, 피부질환, 위장질환, 호르몬질환의 치료에 대해서도 임상시험이 진행되고 있습니다. 다양한 과정을 통해 유전자 발현을 조절하는 데 사용할 수 있습니다. 이러한 분자는 유망함에도 불구하고, 그 임상적 성공은 고유의 생물학적 한계를 극복할 수 있는 효율적인 전달 시스템의 개발과 밀접하게 연결되어 있습니다. 또한, 생체액중의 뉴클레아제에 의한 분해를 받기 쉬운 것, 조직에 침투하는 능력이 한정되어 있는 것, 신장 클리어런스가 빠른 것, 조직 특이적 타겟팅의 필요성 등이, 전신에의 전달을 더욱 복잡하게 하고 있습니다.
올리고뉴클레오티드는 높은 특이성, 프로그램가능성, 합성의 용이성으로 진단학과 치료학에 혁명을 가져왔습니다. 앱 타머는 특정 바이오 마커와 결합하여 표적 이미징을 가능하게하며 화학적 변형으로 생체 내 안정성을 향상시킵니다. 앱타머(예: 페가타니브), 안티센스 올리고뉴클레오티드(예: 누시넬센), siRNA(예: 파티실란) 등이 있으며, 유전자 발현을 조절 또는 침묵시킴으로써 질환을 표적으로 하고 있습니다. RISPR에 근거한 유전자 편집의 기반이 되고 있습니다. 오프 타겟 효과나 신속한 클리어런스등의 과제에도 불구하고, GalNAc 컨쥬게이션이나 AI 유도 설계등의 혁신적 기술에 의해 전달, 특이성, 임상 결과가 개선되어 정밀의료의 진보를 견인하고 있습니다.
올리고뉴클레오티드: 기업 및 제품 프로파일(시판약)
Novartis International AG는 스위스에 본사를 두고 있는 세계 헬스케어 기업이며, 세계 최대의 제약회사입니다. 연구 개발, 제조 및 마케팅을 통해 노바티스는 환경 부하를 줄이고 세계 의약품 액세스를 개선하는 등 지속 가능한 건강 관리 솔루션을 추진하고 있습니다.
제품 개요: LEQVIO
LEQVIO(인크리실란)는 노바티스가 개발한 혁신 의약품 siRNA(small interfering RNA) 기반 치료제로 PCSK9(proprotein convertase subtilisin kexin type) 9) mRNA에 작용합니다. LEQVIO는 다른 치료제와 달리 간에서 표적 단백질의 생산을 억제하고 LDL-C의 간 흡수를 증가시키고 혈류에서 제거합니다. 심혈관 사건 발병 위험이 높은 환자를 적응증으로 후생 노동성에서 승인되었습니다.
Astellas Pharma은 도쿄에 본사를 둔 일본의 다국적 제약기업으로, 헬스케어 솔루션에 대한 혁신적인 접근으로 알려져 있습니다. 미충족 의료 수요에 대응하는 치료법을 추진하는 것으로, 환자의 예후를 개선하는 것에 임하고 있습니다. 또, Astellas Pharma은 신약 개발을 가속해, 북미, 유럽, 아시아 등의 주요 지역에서 시장 프레즌스를 확대하기 위해, 세계적인 파트너십이나 제휴를 적극적으로 추진하고 있습니다.
제품 개요: IZERVAY
IZERVAY의 제품명으로 판매되는 Avacincaptad pegol은 지리적 위축증 치료제로 승인되었습니다. Avacincaptad pegol은 분지된 폴리에틸렌 글리콜(PEG) 분자에 공유 결합된 RNA 압타머입니다.
Alnylam Pharmaceuticals는 매사추세츠 주 캠브리지에 본사를 둔 바이오 의약품 회사로, RNA 간섭(RNAi) 치료제의 발견, 개발, 상업화에 주력하고 있습니다. Alnylam의 파이프라인은 다양한 희귀 유전성 질환, 심대사성 질환, 간 감염성 질환을 대상으로 하고 있으며, ONPATTRO(R), GIVLAARI(R), OXLUMO(R), AMVUTTRA(R)를 포함 여러 제품이 승인되었습니다. 이 회사는 과학적 혁신, 전략적 파트너십 및 세계 전개에 중점을두고 치료 옵션이 제한된 환자의 치료 상황을 변경하는 것을 목표로합니다.
제품 개요: AMVUTTRA
AMVUTTRA(R)(부트리실란)는 트랜스사이레틴(TTR)을 신속하게 넉다운하는 RNAi 치료제로, 트랜스사이레틴(ATTR) 아밀로이드증의 근본 원인을 대처합니다. 유전성 트랜스티레틴 중재 아밀로이드 침착의 다발성 신경장애(hATTR-PN)의 치료제으로서 15개국 이상에서 승인 및 판매되고 있으며, 미국에서는 심혈관계 사망률, 심혈관계 입원 및 긴급 심부전 진찰의 감소를 목적으로 한 성인의 야생형 또는 유전성 트랜스사이레틴 개재성 아밀로이드증의 심근증(ATTR-CM)의 치료약으로 승인되어 있습니다.
Ionis Pharmaceuticals는 1989년에 설립되어 캘리포니아주 칼스배드에 본사를 둔 RNA 표적 치료제에 특화된 바이오테크놀러지 기업입니다. 신경질환, 심혈관계 질환, 희소질환 등 폭넓은 질환에 대한 대처를 목표로 하고 있습니다. 바이오 의약품 부문에서 현재의 지속가능성을 지속적으로 확대하고 있습니다.
제품 개요: TEGSEDI
TEGSEDI(이노타젠)는 유전성 트랜스사이레틴 개입성 아밀로이드증(hATTR) 성인 환자를 대상으로 한 안티센스 올리고뉴클레오티드 치료제로 1단계 또는 2단계의 다발성 신경염을 발병합니다. 이 약은 트랜스사이레틴(TTR) 단백질의 생산을 억제하여 신경장애의 진행을 늦춥니다. 일반적인 부작용은 주사 부위 반응, 메스꺼움, 두통, 피로, 발열 등입니다. 미국에서 TEGSEDI는 안전한 사용을 보증하기 위해 TEGSEDI 리스크 평가 및 완화 전략(REMS) 프로그램이라는 제한된 프로그램을 통해서만 이용할 수 있습니다.
올리고뉴클레오티드 기업 및 제품 프로파일(파이프라인 치료제)
Novartis Pharmaceuticals는 스위스에 본사를 둔 세계 유수의 헬스케어 기업이며, 사람들의 삶을 개선하고 확대하기 위해 의료의 방식을 재검토하기 위해 노력하고 있습니다. 강한 헌신을 유지하면서 첨단 과학과 디지털 기술을 활용하여 가치있는 치료를 제공합니다.
제품 개요: Pelacarsen
Pelacarsen(TQJ230)은 IONIS-APO(a)-LRx, AKCEA-APO(a)-LRx, TQJ230으로도 알려진 임상시험 안티센스 의약품으로 간 장의 apolipoprotein(a)를 감소시키고 lipoprotein(a)(Lp(a))(아테롬성이 높고 혈전을 형성하기 쉬운 LDL)을 감소시키는 직접적인 접근법을 제공하도록 설계되었습니다. Lp(a)의 상승은 관상 동맥 질환, 심장 마비, 뇌졸중, 말초 동맥 질환의 독립적인 유전적 원인으로 인식됩니다. Pelacarsen은 Ionis가 발견하여 2019년에 Novartis에 라이선스됐습니다.
Oncotelic Therapeutics는 암 및 희귀질환의 혁신적인 치료법 개발에 주력하는 임상 단계의 바이오 의약품 기업입니다. Oncotelic은 TGF-B 억제와 올리고 뉴클레오티드 치료제의 전문 지식을 통합하고 질병 경로를 정확하게 조절합니다.
제품 개요: Travedelsen
OT-101(Trabedersen이라고도 함)은 Oncotelic가 개발한 신규 안티센스 올리고데옥시뉴클레오티드(ODN)로 췌장암, 악성 흑색종, 대장암, 고악성도 신경교종(HGG), 기타 종양 증식 인자 β2(TGF-B2)를 과발현하는 악성 종양(전립선 암, 신세포 암 등)의 치료제입니다. 8량체 포스포로티오에이트 올리고데옥시뉴클레오티드(S-ODN)입니다. 자연면역 또는 치료면역이 암을 공격하고 배제할 수 있게 합니다.
Wave Life Sciences는 RNA 의약품의 폭넓은 가능성을 이끌어 사람들의 건강에 변화를 가져오는 데 주력하고 있는 바이오테크놀러지 기업입니다. 키트에는 편집, 스플라이싱, RNA 간섭, 안티센스 침묵 등이 포함되어 있으며, 질병 생물학에 최적으로 대처하는 후보약을 설계하고 지속적으로 제공하기 위한 탁월한 능력을 Wave에 제공하고 있습니다. Wave의 다양한 파이프라인은 뒤센형 근이영양증, α1 항트립신 결핍증, 헌팅턴병의 임상 프로그램, 비만증의 전임상 프로그램을 포함합니다.
제품 개요: WVE-N531
WVE-N531은 엑손 스키핑 올리고뉴클레오티드이며, 엑손 53의 스키핑이 가능한 뒤센형 근이영양증 소아의 질환 수식 치료제로서 개발되고 있습니다. 최고 수준의 올리고뉴클레오티드 케미스트리를 포함한 Wave의 동급 최고의 올리고 뉴클레오티드 화학 수식을 사용하여 설계되었습니다.
Autotelic Bio는 2015년에 설립된 임상 단계의 한국 바이오테크놀러지 기업으로 차세대 핵산 기반 치료제, 특히 안티센스 올리고뉴클레오티드(ASO)의 개발에 특화하고 있습니다. 안전성 프로파일을 강화하고 표적화되고 최적화된 ASO 요법을 제공하는 것을 목표로 하고 있습니다. Autotelic Bio는 UTC Investment와 Stonebridge Ventures 등 투자자가 주도하는 1,260만 달러의 시리즈 B 라운드로, 1,820만 달러의 자금을 확보하고 있습니다.
제품 개요: ATB 301
ATB-301은 Autotelic Bio가 개발한 트랜스포밍 성장인자 β2(TGF-B2)를 표적으로 하는 임상시험 중 안티센스 올리고뉴클레오티드(ASO)요법입니다. 현재, 췌장암의 치료제으로서 임상시험 제I상 단계에 있습니다.
Tallac Therapeutics는 캘리포니아에 본사를 둔 임상 단계의 바이오 의약품 회사로 암에 대한 새로운 면역 요법의 개발에 주력하고 있습니다. 전신독성을 최소화하면서 항종양면역을 강화하는 것을 목표로, Toll형 수용체 9(TLR9)를 표적으로 한 방법으로 활성화하는 것을 목적으로 하고 있습니다.
제품 개요: TAC001
TAC-001은 새로운 항체 - 올리고 뉴클레오티드 접합체이며 암 면역에서 중요한 역할을하는 B 세포의 표적 면역 활성화와 함께 전신 TLR9 효능을 제공하도록 설계되었습니다. 전임상시험에서 전신투여된 TAC-001은 면역억제나 저항성을 가지는 종양모델을 포함한 많은 가짜 유전자종양에서 단제로 활성을 나타내며 종양의 완전 구제와 면역기억을 가져왔습니다.
ARTHEx Biotech는 유전자 발현의 조절에 의한 혁신적인 의약품의 개발에 중점을 둔 임상 단계의 바이오테크놀러지 기업입니다. ArthemiR는 유전자 질환을 포함한 미충족 의료 수요가 높은 다른 질환에 대한 핵산 기반의 치료법을 동정 및 개발하기 위해, 자사 창약 엔진을 추진하고 있습니다.
제품 개요: ATX-01
ATX-01은 DM1의 병태에 관여하는 마이크로RNA 23b(miR-23b)를 표적으로 하도록 설계된 항-miR 올리고뉴클레오티드입니다. mRNA를 감소시키고 MBNL 단백질의 생산을 증가시키는 독특한 이중 작용기전을 갖는 것으로 입증되었습니다. 조직에 대한 우선적인 전달을 보장하기 위해 구축된 ARTHEx 사내의 디스커버리 엔진에 의해 발견되었습니다.
DelveInsight에 의한 올리고 뉴클레오티드 분석의 전망
본 보고서에서는 제휴, 계약, 라이선싱, 인수 등의 동향을 포함한 의약품의 상세한 상업평가를 제공합니다.
본 보고서에서는 기업(치료법별, 개발 스테이지별, 기술별)의 비교평가를 제공합니다.
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.
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.
Oligonucleotides: Company and Product Profiles (Marketed Therapies)
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%.
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.
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.
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)
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.
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.
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.
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.
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.
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
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.
The report comprises of comparative assessment of Companies (by therapy, development stage, and technology).
Current Treatment Scenario and Emerging Therapies:
Key Players
Key Products
Introduction
Executive Summary
Oligonucleotides: Overview
Oligonucleotides -Analytical Perspective: In-depth Commercial Assessment
Competitive Landscape
Therapeutic Assessment
Oligonucleotides: Company and Product Profiles (Marketed Therapies)
Novartis Pharmaceuticals
LEQVIO
Oligonucleotides: Company and Product Profiles (Pipeline Therapies)
Late Stage Products (Phase III)
Novartis Pharmaceuticals
Pelacarsen
Mid Stage Products (Phase II)
Oncotelic
Trabedersen
Early Stage Products (Phase I)
Autotelic Bio
ATB 301
Preclinical and Discovery Stage Products
Company Name
Product Name
Inactive Products
Oligonucleotides- Unmet needs
Oligonucleotides - Market drivers and barriers