透過體內表達DNA或RNA的載體抗體：產業視角下的競爭企業、利害關係人、技術及研發管線分析

本報告對透過體內表達DNA或RNA的載體抗體的利害關係人、研發管線、候選藥物概況及組成以及截至2025年6月的當前產業格局中的商業交易進行了評論和分析。

過去三十年來，被動免疫療法主要利用哺乳動物細胞培養系統中體外產生的單株抗體，已成為一種在臨床和商業上都取得成功的治療方法。儘管治療性抗體在臨床上取得了成功，但它們仍然存在局限性，例如由於生產和管理而導致的成本高昂，以及高劑量重複給藥所需的藥物量。開發生產流程以及在商業規模上進行大批量抗體的GMP生產是一個複雜的過程。此外，這些抗體給藥不方便，需要頻繁給藥，藥物動力學特徵不理想，難以注射至眼部（例如視網膜下注射）和中樞神經系統（例如透過導管置入鞘內注射），且全身給藥副作用大且療效有限，缺乏在癌症治療中至關重要的組織特異性。

載體化抗體有望克服其中的許多局限性，為DNA和RNA技術公司進入比目前基因療法已獲批或正在開發的罕見疾病適應症更大的市場提供絕佳機會。

透過DNA或RNA在體內表現治療性抗體可以克服傳統抗體療法的至少部分限制。抗體轉基因可以透過病毒載體、質粒DNA直接注射到肌肉中、電穿孔或分子製劑（例如脂質奈米顆粒）進行全身給藥。

眼科疾病是透過病毒DNA在體內表現的載體化抗體的主要適應症。多種抗VEGF載體化方案治療濕性AMD的I期和II期臨床試驗結果證明了其安全性和耐受性，在改善視力和視網膜厚度方面表現穩定，並且治療效果可持續長達4年。兩種不同的抗VEGF載體化抗體正在III期臨床試驗中競爭。首個抗VEGF載體化抗體的最終結果預計將於2026年公佈，並可能為一種載體化抗體技術提供臨床驗證。

本報告提供以下方面的最新資訊和分析：

• 利害關係人：病毒DNA、溶瘤病毒DNA、非病毒DNA、RNA技術公司、服務提供者、生物製藥合作夥伴
• 利害關係人概況：技術、地區、成立年份、員工、財務狀況、最高研發階段
• 載體抗體技術公司與生物製藥公司之間的合作關係
• 載體抗體技術：病毒DNA、溶瘤病毒DNA、非病毒DNA（質粒、片段）、載體製劑、細胞轉導、RNA
• 載體抗體候選產品組成：載體抗體候選產品組成：DNA或RNA、遞送方式、給藥途徑
• 載體抗體研發管線：眼科、腫瘤科、神經科、傳染病及其他治療領域領域
• 載體抗體安全性和有效性的臨床經驗
• 載體抗體的分子、臨床前、臨床概況
• 競爭對手分析

方法論：

本報告評估了載體抗體研發領域的產業格局。它全面概述了製藥和技術公司在透過體內表達DNA或RNA進行載體抗體領域的研發和合作活動。本報告是基於對企業利害關係人（包括生物製藥和生物技術公司）的識別和描述。所有已發布資訊均附有完整參考文獻，並透過超連結指向 190 餘篇科學出版物（摘要、海報、簡報、全文）或新聞稿、公司介紹、年度報告、美國證券交易委員會 (SEC) 披露、主頁內容等來源。

相關企業

目錄

頻繁的簡稱

第1章 摘要整理

第2章 簡介

第3章 相關利益者分析

• 概要
• 擁有載體抗體的病毒 DNA 技術公司
• 擁有載體抗體的非病毒 DNA 技術公司抗體
• 擁有載體抗體的RNA技術公司
• 提供載體抗體RNA技術的服務提供者
• 透過許可和合作協議建立的載體抗體合作夥伴關係

第4章 媒介化抗體技術的利害關係者的簡介

• 病毒載體DNA企業
  • 4D Molecular Therapeutics (4DMT)
  • Adverum Biotechnologies
  • Avirmax Biopharma
  • Capsida Therapeutics
  • Chengdu Origen Biotechnology
  • Cirrus Biotherapeutics
  • Frontera Therapeutics
  • Ikarovec
  • Kriya Therapeutics
  • MeiraGTx Holdings
  • Neuracle Genetics
  • Regeneron Pharmaceuticals
  • REGENXBIO
  • Ring Therapeutics
  • Scout Bio (now: Ceva)
  • Shape Therapeutics
  • Skyline Therapeutics
  • VectorY
  • Vironexis Biotherapeutics
  • Voyager Therapeutics
• 腫瘤溶解性病毒DNA企業
  • Accession Therapeutics
  • Akamis Bio
  • ImmVira
  • Transgene
• 非病毒DNA企業
  • Inovio Pharmaceuticals
  • PharmAbs
  • PulseSight Therapeutics
  • RenBio
  • Entos Pharmaceuticals
  • Nanite
  • Rampart Bioscience
  • Be Biopharma
  • BiomEdit
• RNA企業
  • BioNTech
  • De novo Biotherapeutics
  • Hopewell Therapeutics
  • METiS Pharmaceuticals
  • RNAimmune
  • Shattuck Labs
  • Suzhou Abogen Bioscience
  • Immorna
  • Nuclix Bio
  • Sail Biomedicine
• RNA服務公司
  • Charles River
  • Nutcracker Therapeutics
  • ProBio
  • ProMab Biotechnologies
  • ST Pharm
  • WuXi AppTec

第5章 媒介化抗體技術的分析

• 抗體的生物內表現的病毒載體DNA技術
• 抗體的生物內表現的腫瘤溶解性病毒DNA技術
• 抗體的生物內表現的非病毒DNA技術
• 抗體的生物內表現的RNA技術

第6章 媒介化抗體技術的簡介

• 抗體的生物內表現的病毒載體DNA技術
• 抗體的生物內表現的腫瘤溶解性病毒DNA技術
• 抗體的生物內表現的非病毒DNA技術
• 抗體的生物內表現的RNA技術

第7章 抗體的生物內表現的開發平台(管線)及產品候補的分析

• 概要
• 眼科領域載體抗體管線分析
• 腫瘤領域載體抗體管線分析
• 傳染病領域載體抗體管線分析
• 神經學領域載體抗體管線分析
• 自體免疫、代謝和罕見疾病領域載體抗體管線分析

第8章 媒介化抗體產品候補的簡介

• 用於抗體體內表現的病毒載體DNA候選產品
• 用於抗體體內表現的Onolitic病毒DNA候選產品
• 非病毒DNA產品抗體體內表現候選物
• 抗體體內表現的RNA候選物

第9章 參考文獻

This report provides you with a landscape description and analysis of Vectorized antibodies by in vivo expression of DNA or RNA regarding stakeholders, R&D pipeline, profile & composition of drug candidates and business deals from an industry perspective as of June 2025.

Passive immunotherapy with monoclonal antibodies produced ex vivo mostly in mammalian cell culture systems has become a clinically and commercially successful treatment modality during the last three decades. Despite the clinical success of therapeutic antibodies, they still have limitations including the high cost, caused in great part by manufacturing and control, and the amount of drug needed for repeated administrations at high doses. Development of the manufacturing process as well as commercial scale GMP manufacturing of antibodies in great amounts is a complex process. Other limitiations refer to the inconvenience of frequent administrations associated with an unsatisfactory pharmacokinetic profile, challenging administration procedures to the eye (e.g. subretinal injection) or the central nervous system (e.g. intrathecal infusion with indwelling catheter) or side effects and limited efficacy upon systemic administration without tissue specificity, an important aspect in cancer therapy.

Vectorized antibodies hold promise to overcome many of these limitations and provide a great opportunity for DNA and RNA technology companies to enter larger markets compared with the rare disease indications for which current gene therapies are approved or in development.

In vivo expression of therapeutics antibodies by DNA or RNA may overcome at least some of the limitations of conventional antibody therapy. The antibody transgene may be delivered by viral vectors, by direct injection of plasmid DNA into the muscle followed by electroporation or by molecular formulations, such as lipid nanoparticles, for systemic administration.

Ophthalmic diseases are the lead indication for vectorized antibodies expressed in vivo by viral DNA. Clinical results from phase I and II clinical studies of various anti-VEGF vectorized programs for treatment of wet AMD demonstrated safety and tolerability and stable to improved vision and retinal thickness as well as long-term, durable treatment effects up to 4 years. Three distinct anti-VEGF vectorized antibodies are competing in clinical phase III. Topline results from the first anti-VEGF vectorized antibody are expected in 2026 and may provide clinical validation of one vectorized antibody technology.

The report brings you up-to-date with information about and analysis of:

• Stakeholders: companies with technologies in viral DNA, oncolytic virus DNA, non-viral DNA and RNA; service providers and biopharmaceutical partners;
• Coroporate profiles of stakeholders: technology, territory, year of foundation, employees, financial situation and highest R&D stage
• Partnerships o f vectorized antibody technology companies and biopharmaceutical companies;
• Vectorized antibody technologies; viral DNA, oncolytic virus DNA, non-viral DNA (plasmid, molecular formulation, cellular delivery) and RNA;
• Compositions of vectorized antibody product candidates: DNA or RNA, delivery method and route of administration;
• Pipeline of vectorized antibodies: in ophthalmology, oncology, neurology, infectious disease and other therapeutic areas;
• Clinical experience in safety and efficacy with vectorized antibodies;
• Molecular, preclinical and clinical profile of vectorized antibodies;
• Competitor analysis.

Methodology:

This report evaluates the industry landscape of vectorized antibodies in research and development. The report provides a comprehensive overview of the R&D and partnering activities of pharmaceutical and technology companies in the field of vectorized antibodies by in vivo expression of DNA or RNA. This report is based on the identification and description of corporate stakeholders including biopharmaceutical companies and biotechnology companies. All publicly available information is fully referenced, either with more than 190 scientific references (abstracts, posters, presentations, full paper) or hyperlinks leading to the source of information, such as press releases, corporate presentations, annual reports, SEC disclosures and homepage content.

Who will benefit from the report?

• Business development and licensing (BDL) specialists;
• Venture capital, private equity and investment managers;
• Managers of Big Pharma venture capital firms;
• Financial analysts;
• CEO, COO and managing directors;
• Corporate strategy analysts and managers;
• Chief Technology Officer;
• R&D Portfolio, Technology and Strategy Management;
• Clinical and preclinical development specialists.

Related Companies:

Table of Contents

Frequent Abbreviations

1. Executive Summary

2. Introduction

3. Stakeholder Analysis

• 3.1. Overview
• 3.2. Viral DNA Technology Companies with Vectorized Antibodies
• 3.3. Non-Viral DNA Technology Companies with Vectorized Antibodies
• 3.4. RNA Technology Companies with Vectorized Antibodies
• 3.5. Service Providers with RNA Technology for Vectorized Antibodies
• 3.6. Partnerships with Licensing and Collaboration Agreements for Vectorized Antibodies

4. Profiles of Stakeholders in Vectorized Antibody Technologies

• 4.1. Viral Vector DNA Companies
  • 4.1.1. 4D Molecular Therapeutics (4DMT)
  • 4.1.2. Adverum Biotechnologies
  • 4.1.3. Avirmax Biopharma
  • 4.1.4. Capsida Therapeutics
  • 4.1.5. Chengdu Origen Biotechnology
  • 4.1.6. Cirrus Biotherapeutics
  • 4.1.7. Frontera Therapeutics
  • 4.1.8. Ikarovec
  • 4.1.9. Kriya Therapeutics
  • 4.1.10. MeiraGTx Holdings
  • 4.1.11. Neuracle Genetics
  • 4.1.12. Regeneron Pharmaceuticals
  • 4.1.13. REGENXBIO
  • 4.1.14. Ring Therapeutics
  • 4.1.15. Scout Bio (now: Ceva)
  • 4.1.16. Shape Therapeutics
  • 4.1.17. Skyline Therapeutics
  • 4.1.18. VectorY
  • 4.1.19. Vironexis Biotherapeutics
  • 4.1.20. Voyager Therapeutics
• 4.2. Oncolytic Virus DNA Companies
  • 4.2.1. Accession Therapeutics
  • 4.2.2. Akamis Bio
  • 4.2.3. ImmVira
  • 4.2.4. Transgene
• 4.3. Non-Viral DNA Companies
  • 4.3.1. Inovio Pharmaceuticals
  • 4.3.2. PharmAbs
  • 4.3.3. PulseSight Therapeutics
  • 4.3.4. RenBio
  • 4.3.5. Entos Pharmaceuticals
  • 4.3.6. Nanite
  • 4.3.7. Rampart Bioscience
  • 4.3.8. Be Biopharma
  • 4.3.9. BiomEdit
• 4.4. RNA Companies
  • 4.4.1. BioNTech
  • 4.4.2. De novo Biotherapeutics
  • 4.4.3. Hopewell Therapeutics
  • 4.4.4. METiS Pharmaceuticals
  • 4.4.5. RNAimmune
  • 4.4.6. Shattuck Labs
  • 4.4.7. Suzhou Abogen Bioscience
  • 4.4.8. Immorna
  • 4.4.9. Nuclix Bio
  • 4.4.10. Sail Biomedicine
• 4.5. RNA Service Companies
  • 4.5.1. Charles River
  • 4.5.2. Nutcracker Therapeutics
  • 4.5.3. ProBio
  • 4.5.4. ProMab Biotechnologies
  • 4.5.5. ST Pharm
  • 4.5.6. WuXi AppTec

5. Analysis of Vectorized Antibody Technologies

• 5.1. Viral Vector DNA Technologies for In Vivo Expression of Antibodies
• 5.2. Oncolytic Virus DNA Technologies for in vivo Expression of Antibodies
• 5.3. Non-Viral DNA Technologies for in vivo Expression of Antibodies
• 5.4. RNA Technologies for in vivo Expression of Antibodies

6. Profiles of Vectorized Antibody Technologies

• 6.1. Viral Vector DNA Technologies for in vivo Expression of Antibodies
  • 6.1.1. Therapeutic Vector Evolution (4DMT)
  • 6.1.2. AAV.7m8 VectorPlatform (Adverum)
  • 6.1.3. Macular Retina-Targeting AAV2 Capsid (Avirmax)
  • 6.1.4. CNS-Selective AAV Gene Therapy (Capsida)
  • 6.1.5. AAV8 and Variant Gene Therapy with Vectorized Antibodies (Chengdu)
  • 6.1.6. APEX Technology & Manufacturing platform (Frontera)
  • 6.1.7. AAV Gene Therapy & Riboswitch Technology (MeiraGTx)
  • 6.1.8. NAV Technology Platform (REGENXBIO)
  • 6.1.9. Anellogy platform (Ring Tx)
  • 6.1.10. AAV.ai Capsid Discovery Platform (Shape)
  • 6.1.11. Advanced adeno-associated virus (AAV) based platform (Skyline)
  • 6.1.12. VecTab, VecTron & VeCap (VectorY)
  • 6.1.13. TRACER Capsid Discovery Platform (Voyager)
• 6.2. Oncolytic Virus DNA Technologies for in vivo Expression of Antibodies
  • 6.2.1. TROCEPT Technology Platform (Accession)
  • 6.2.2. Tumor-Specific Immuno-Gene Therapy (T-SIGn) Platform (Akamis)
  • 6.2.3. Invir.IO (Transgene)
• 6.3. Non-Viral DNA Technologies for in vivo Expression of Antibodies
  • 6.3.1. DNA-encoded Monoclonal Antibodies: DMAbs (Inovio)
  • 6.3.2. Ciliary Electro-Transfection of Plasmid (PulseSight)
  • 6.3.3. MYO Technology (RenBio)
  • 6.3.4. Fusogenix PLV Technology (Entos)
  • 6.3.5. SAYER Technology (Nanite)
  • 6.3.6. HALO DNA-LNP (Rampart)
  • 6.3.7. B Cell Engineering Platform (Be Biopharma)
  • 6.3.8. Probiotic Vectored Antibody: pvAb (BiomEdit)
• 6.4. RNA Technologies for in vivo Expression of Antibodies
  • 6.4.1. RiboMab Technology (BioNTech)
  • 6.4.2. AiLNP & AiRNA (METiS)
  • 6.4.3. Linear mRNA and Circular RNA (Abogen)
  • 6.4.4. mRNA-Encoded Antibody Platform (Charles River)
  • 6.4.5. RNA Precision Manufacturing Platform (Nutcracker)
  • 6.4.6. ringRNA & Ribo-grAb ("RNA-generated recombinant Antibodies") (Ring Therapeutics)
  • 6.4.7. Endless RNA (eRNA) (Sail)

7. Analysis of Pipeline and Product Candidates for in vivo Expression of Antibodies

• 7.1. Overview
• 7.2. Analysis of the Pipeline of Vectorized Antibodies in Ophthalmology
  • 7.2.1. Clinical Experience on the Safety and Efficacy of Vectorized Antibodies in Ophthalmology
• 7.3. Analysis of the Pipeline of Vectorized Antibodies in Oncology
  • 7.3.1. Clinical Experience with the Safety and Efficacy of Vectorized Antibodies in Oncology
• 7.4. Analysis of the Pipeline of Vectorized Antibodies in Infectious Diseases
• 7.5. Analysis of the Pipeline of Vectorized Antibodies in Neurology
• 7.6. Pipeline of Vectorized Antibodies in Autoimmune, Metabolic and Rare Diseases

8. Profiles of Vectorized Antibody Product Candidates

• 8.1. Viral Vector DNA Product Candidates for in vivo Expression of Antibodies
  • 8.1.1. 4D-150
  • 8.1.2. AAV8.2-anti-C5 scFv
  • 8.1.3. AAV8.CAT311 Gene Therapy with Vectorized Antibodies
  • 8.1.4. AAV-anti-TNFalpha- scFv
  • 8.1.5. ABI-110
  • 8.1.6. FT-003
  • 8.1.7. IKC151V
  • 8.1.8. Ixoberogene soroparvovec; ixo-vec
  • 8.1.9. KH631
  • 8.1.10. KH658
  • 8.1.11. KRIYA-586
  • 8.1.12. NG101; RY104
  • 8.1.13. SKG0106
  • 8.1.14. Surabgene lomparvovec; sura-vec; ABBV-RGX-314
  • 8.1.15. Vectorized Anti-Amyloid Antibody
  • 8.1.16. VNX-101
  • 8.1.17. VNX-202
  • 8.1.18. VTX-001
  • 8.1.19. VTX-002
  • 8.1.20. VTX-003
• 8.2. Onolytic Virus DNA Product Candidates for in vivo Expression of Antibodies
  • 8.2.1. BT-001
  • 8.2.2. MVR-C5252
  • 8.2.3. MVR-T3011
  • 8.2.4. NG-350A
  • 8.2.5. TG6050
  • 8.2.6. TROCEPT-01; ATTR-01
• 8.3. Non-Viral DNA Product Candidates for in vivo Expression of Antibodies
  • 8.3.1. DNA-based Anti-Zika Antibody
  • 8.3.2. DNA-based Incretin Receptor Agonists
  • 8.3.3. EYS606
  • 8.3.4. Optimized dMAb AZD5396 and dMAb AZD8076 with Hylenex-R Recombinant
  • 8.3.5. PST-809 (EYS809)
  • 8.3.6. DNA-Encoded PGT121 Antibody
  • 8.3.7. HALO BTE-LNP
  • 8.3.8. HALO TNALP-LNP
  • 8.3.9. BE-102
  • 8.3.10. BiTE BCM
  • 8.3.11. RMP100-HSPC-TNALP
• 8.4. RNA Product Candidates for in vivo Expression of Antibodies
  • 8.4.1. ABO2203
  • 8.4.2. BNT141
  • 8.4.3. BNT142
  • 8.4.4. LNP-mRNA BiTE
  • 8.4.5. MTS105
  • 8.4.6. NTX-470
  • 8.4.7. NTX-0471
  • 8.4.8. NTX-472
  • 8.4.9. RV-525

9. References

Figures & Tables

• Table 1: Corporate Stakeholders in Vectorized Antibody R&D
• Table 2: Product Categories Pursued by Viral DNA Technology Companies
• Table 3: Profiles of Viral DNA Companies with Vectorized Antibody Technologies
• Table 4: Product Categories Pursued by Non-Viral DNA Technology Companies
• Table 5: Profiles of Non-Viral DNA Companies with Vectorized Antibody Technologies
• Table 6: Product Categories Pursued by RNA Technology Companies
• Table 7: Profiles of RNA Companies with Vectorized Antibody Technologies
• Table 8: Partnerships of Pharma/Biotech and Companies with Vectorized Antibody Technologies
• Table 9: Overview of Kriya Therapeutics' AAV Gene Therapy R&D Pipeline
• Table 10: REGENXBIO's Pipeline of Vectorized Antibodies
• Table 11: Aegis Life's Pipeline of Vectorized Antibodies for Infectious Diseases
• Table 12: Overview of Viral Vectors Used for in vivo Expression of Antibodies by DNA
• Table 13: Oncolytic Virus Technologies with DNA for in vivo Expression of Antibodies
• Table 14: Overview of Non-Viral DNA Technologies for in vivo Expression of Antibodies
• Table 15: Overview of RNA Technologies for in vivo Expression of Antibodies
• Table 16: Clinical and Non-Clinical Stage Vectorized Antibodies per Technology Modality and per Therapeutic Area
• Table 17: Preclinical and Research Stage Vectorized Antibodies per Technology Modality and per Therapeutic Area
• Table 18: Pipeline of Vectorized Antibodies in Ophthalmology
• Table 19: Pipeline of Vectorized Antibodies in Oncology
• Table 20: Pipeline of Vectorized Antibodies in Infectious Diseases
• Table 21: Pipeline of Vectorized Antibodies in Neurology
• Table 22: Pipeline of Vectorized Antibodies in Autoimmune, Metabolic & Rare Diseases
• Table 23: Overview of Clinical Development Program of ABBV-RGX-314