Developments in Biotechnology Platforms Leading to Increasing Immuno-oncology Applications
This research service investigates technologies that propel the development of new targeted immunotherapies, particularly those that deliver therapeutic DNA and proteins. The top 3 growth opportunities focus on enhanced gene delivery systems for greater precision, the support of such systems through novel biotech platforms, and the affordability of precision medicine/targeted immunotherapies through modular, decentralized manufacturing systems.
Regarding technology, nonviral gene delivery and nucleic acid delivery offer significant advantages in terms of safety, particularly from immunogenicity and carcinogenicity. Not using viral vectors also makes the regulatory pathway and the biomanufacturing process more affordable because no additional precautions are needed; this, in fact, makes the price tag lower.
The study touches on the advantages and limitations of viral and nonviral gene delivery systems for targeted immunotherapies. Safety of administration without immunogenicity remains as the most relevant advantage. Liposomes have no replication risk and are less immunogenic than viruses.
Other benefits are:
- a practically unlimited transgene size;
- the possibility of repeated administration;
- a cost-affordable model; and
- an easy manner to produce them in large amounts.
The plurality of gene delivery systems is broadly benchmarked in terms of transfection efficiency, precision in cancer immunotherapy, and capability to pass the membrane barrier.
As for remarkable innovation in biotech platforms, the research highlights gene addition as a novel approach that uses a delivery system to insert new genes directly into cells.
The addition of a functional gene can take place either outside (ex vivo) or inside (in vivo) the body and can be used in cancer immunotherapy through CAR T-cell technology.
Gene delivery strategies are based on a comprehensive suite of clinically validated technologies, including electroporation, liposomes, nanoparticles, and nonviral and viral delivery modalities. Electroporation is best suited for efficient delivery to blood cells and immune cells ex vivo. Lipid nanoparticles (LNP) are better indicated for in vivo delivery to the liver and potentially other organs. Adenoassociated vectors are typically used for in vivo delivery to the eye and central nervous system (CNS).
As for optimal manufacturing process, closed-ended DNA (ceDNA) vectors exhibit a linear and continuous structure, which can be used for insertion of a transgene into a gene safe harbor (GSH) in the genome. For gene delivery, cell-targeted lipid nanoparticle (ctLNP) designed to avoid activation of the immune system upon initial dose can be used, while capsid-free approaches can be utilized to facilitate manufacturing.
This research profiles a remarkable number of companies succeeding in the targeted immunotherapy space. A competitive analysis of the top three innovators, based on their IP activity and patent innovation focus, is included. Funding and investment, including leading deals and investors focused on the technologies empowering targeted immunotherapies, are also covered.
Key Points Discussed:
1. Top innovations in biotechnology platforms focused on targeted immunotherapies
2. Emerging technologies in gene and nucleic acids direct delivery systems
3. Outstanding companies succeeding the targeted immunotherapy space
4. Industry landscape and patent filing trends in the industry
5. Major deals and investors focused on developing novel technologies for targeted immunotherapies
6. Growth opportunities in the targeted immunotherapy area
7. Best practices for risk management during the development of new technologies that enhance targeted immunotherapies
Table of Contents
1.2 The Strategic Imperative 8™
1.3 The Impact of the Top Three Strategic Imperatives on Targeted Immunotherapies
1.4 About The Growth Pipeline Engine™
1.5 Growth Opportunities Fuel the Growth Pipeline Engine™
1.6 Research Methodology
2.2 Framework for Personalized Targeted Immunotherapy
2.3 Groundbreaking Immunotherapy Technology Platforms
2.4 Fit-for-Purpose Immunotherapy Approaches
2.5 Regulatory Environment
2.6 Research Context
2.7 Research Scope
3.2 Drivers and Accelerators
3.3 Challenges and Restraints
4.2 Technologies Empowering Targeted Immunotherapies
4.3 Germline Gene Editing Tools and Stem Cell Reprogramming
4.4 Next-Generation Targeted Immunotherapy Requirements
4.5 Viral and Nonviral Vector Delivery in Gene and Cell Therapies
4.6 Process Opportunity Plays for Biopolymers & Bioprocessing in Gene Therapy
4.7 Innovation Ecosystem and Collaboration Hubs
4.8 Key Partnerships and Alliances in Gene Delivery
4.9 Companies Focused on Viral and Nonviral Gene Delivery Systems in Targeted Immunotherapy
4.10 Novel Synergies in Gene Delivery Systems Driving Targeted Immunotherapy Success
4.11 Companies Focused on Gene Editing and Stem Cell Reprogramming Technologies
4.12 Companies Focused on Medicinal Chemistry and Platform Chemistry Technologies
5.2 Clinical Validation Status of Nonviral Gene Delivery Systems
5.3 Competitive Landscape
5.4 State-of-the-Art Therapeutics Pipeline Dashboard: Inhibrx, Inc.
5.5 State-of-the-Art Therapeutics Pipeline Dashboard: BioNTech SE
5.6 State-of-the-Art Therapeutics Pipeline Dashboard: Adaptimmune Therapeutics llc.
5.7 State-of-the-Art Therapeutics Pipeline Dashboard: Asher Bio
5.8 State-of-the-Art Therapeutics Pipeline Dashboard: Gritstone bio
5.9 State-of-the-Art Therapeutics Pipeline Dashboard: Jounce Therapeutics, Inc.
6.2 Next-Generation AAV Capsids, Abeona Therapeutics, US
6.3 CRISPR Gene Editing, Editas Medicine, US
6.4 AAV, Zinc Finger Nucleases, Sangamo Therapeutics, US
6.5 Electroporation, Nonviral (LNP), Viral (AAV), Beam Therapeutics, US
6.6 Customized AAV Vectors, 4D Molecular Therapeutics, US
6.7 T-Cell Immunotherapy - Microfluidic Vortex Shedding, Indee Labs, US
6.8 Precise Gene Editing Technology, Poseida Therapeutics, US
6.9 Lentiviral Gene Delivery, Avrobio, US
7.2 AAV Gene Delivery Technology, Spark Therapeutics (Roche), US
7.3 Gene Replacement and Gene Knockdown, Voyager Therapeutics, US
7.4 AAV Gene Delivery Technology, AGTC, US
7.5 LV Gene Delivery and CAR T-cell Technology, BlueBird Bio, US
7.6 DNA Nanoparticles, Copernicus Therapeutics, US
8.2 Leading R&D Investments in Targeted Immunotherapy Platforms
8.3 Principal Deals in Targeted Immunotherapy Platforms
8.4 Leading Investors in Targeted Immunotherapy Platforms
8.5 Investor Movements in Targeted Immunotherapy Platforms
8.6 Government Funding in Targeted Immunotherapy Platforms
8.7 Prime Contract Transactions in Targeted Immunotherapy Platforms
8.8 Assistance Prime Transactions in Targeted Immunotherapy Platforms
8.9 Venture Capital Funding Assessment in Targeted Immunogene Therapy
8.10 Venture Capital Funding Deals in Targeted Immunogene Therapy
9.2 Competitive Intelligence, BlueBird Bio
9.3 Competitive Intelligence, Generation Bio
10.2 Growth Opportunity 2: Innovative Biotech Platforms for Precision Immunotherapy
10.3 Growth Opportunity 3: Modular, Decentralized Manufacturing for Affordable Targeted Gene Immunotherapy
10.4 Best Practices for Risk Management in Targeted Immunotherapy
A selection of companies mentioned in this report includes:
- 4D Molecular Therapeutics, US
- Abeona Therapeutics, US
- Adaptimmune Therapeutics llc.
- AGTC, US
- Asher Bio
- Avrobio, US
- Beam Therapeutics, US
- BioNTech SE
- BlueBird Bio, US
- Copernicus Therapeutics, US
- Editas Medicine, US
- Generation Bio
- Gritstone bio
- Homology Medicines, US
- Indee Labs, US
- Inhibrx, Inc.
- Jounce Therapeutics, Inc.
- Poseida Therapeutics, US
- Sangamo Therapeutics, US
- Spark Therapeutics (Roche), US
- Ultragenyx Pharmaceutical, US
- Voyager Therapeutics, US