Vectorized antibodies by in vivo expression of DNA or RNA: a competitive business, stakeholder, technology and pipeline analysis from an industry perspective
Vectorized antibodies by in vivo expression of DNA or RNA: a competitive business, stakeholder, technology and pipeline analysis from an industry perspective
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 of 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.
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-TNFα 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® 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