Periodic Reporting for period 1 - TOP-EVICARE (Technology Of Protein delivery in Extracellular Vesicle-induced Cardiac Repair)
Okres sprawozdawczy: 2023-10-01 do 2025-03-31
Heart failure is a major global health burden. Current therapies fail to deliver therapeutic proteins directly into heart cells due to limitations in nanoparticle delivery systems. The ERC-funded TOP-EVICARE project (Sept 2023–Mar 2025) developed an innovative solution using extracellular vesicles (EVs) to deliver proteins into cardiac cells.
EVs are naturally derived, low-immunogenic particles capable of targeting tissues and crossing biological barriers. The project aimed to:
1. Validate a novel EV-loading platform.
2. Protect intellectual property (IP).
3. Analyze the market for EV-based therapies.
4. Develop a commercialization plan.
Key outcomes included successful delivery of therapeutic proteins in vivo, a patent filing, identification of a $1.5B global market by 2030, and a business plan to launch a spinout company.
This technology enables precise intracellular protein delivery, addressing previously untreatable targets and offering new hope for heart failure and other diseases. Continued studies, funding, and regulatory navigation are essential for clinical translation.
EVs are naturally derived, low-immunogenic particles capable of targeting tissues and crossing biological barriers. The project aimed to:
1. Validate a novel EV-loading platform.
2. Protect intellectual property (IP).
3. Analyze the market for EV-based therapies.
4. Develop a commercialization plan.
Key outcomes included successful delivery of therapeutic proteins in vivo, a patent filing, identification of a $1.5B global market by 2030, and a business plan to launch a spinout company.
This technology enables precise intracellular protein delivery, addressing previously untreatable targets and offering new hope for heart failure and other diseases. Continued studies, funding, and regulatory navigation are essential for clinical translation.
The project was structured around four core tasks:
Task 1: EV-Loading Validation & GMP Optimization
Performed:
- In vitro testing of a proprietary linker for protein delivery.
- In vivo rodent studies for biodistribution and functionality.
- EV production optimized under GMP-like conditions.
Achievements:
- 95% protein recombination in Cre-loxP cells.
- Efficient heart-targeted delivery via intramyocardial injection.
- Functional delivery of Cre recombinase to cardiac tissue.
- Consistent, scalable EV production achieved.
Task 2: Intellectual Property
Performed:
- Patent prepared and filed with Utrecht Holdings.
- FTO analysis underway for specific cardiac uses.
Achievements:
- Patent secured: "Extracellular vesicles for drug delivery and uses thereof."
- Licensing discussions ongoing with spin-off JAMA Tx.
Task 3: Market and Stakeholder Analysis
Performed:
- Market potential and competition assessed.
- Stakeholder workshops and events organized.
Achievements:
- $1.5B projected EV therapeutics market by 2030.
- EV platform shown to outperform other delivery methods.
Partnerships formed with ThermoFisher, ESC, and others.
Task 4: Business Plan Development
Performed:
- Participated in Faculty of Impact & Academic Startup Competition.
- Business model targeting protein/peptide-based therapies developed.
Achievements:
- Commercial roadmap created for spinout JAMA Tx.
Summary:
TOP-EVICARE validated a scalable, high-efficiency EV-based delivery platform, filed IP, confirmed market viability, and set a clear path to commercialization.
Task 1: EV-Loading Validation & GMP Optimization
Performed:
- In vitro testing of a proprietary linker for protein delivery.
- In vivo rodent studies for biodistribution and functionality.
- EV production optimized under GMP-like conditions.
Achievements:
- 95% protein recombination in Cre-loxP cells.
- Efficient heart-targeted delivery via intramyocardial injection.
- Functional delivery of Cre recombinase to cardiac tissue.
- Consistent, scalable EV production achieved.
Task 2: Intellectual Property
Performed:
- Patent prepared and filed with Utrecht Holdings.
- FTO analysis underway for specific cardiac uses.
Achievements:
- Patent secured: "Extracellular vesicles for drug delivery and uses thereof."
- Licensing discussions ongoing with spin-off JAMA Tx.
Task 3: Market and Stakeholder Analysis
Performed:
- Market potential and competition assessed.
- Stakeholder workshops and events organized.
Achievements:
- $1.5B projected EV therapeutics market by 2030.
- EV platform shown to outperform other delivery methods.
Partnerships formed with ThermoFisher, ESC, and others.
Task 4: Business Plan Development
Performed:
- Participated in Faculty of Impact & Academic Startup Competition.
- Business model targeting protein/peptide-based therapies developed.
Achievements:
- Commercial roadmap created for spinout JAMA Tx.
Summary:
TOP-EVICARE validated a scalable, high-efficiency EV-based delivery platform, filed IP, confirmed market viability, and set a clear path to commercialization.
The EV platform surpasses nanoparticle systems by offering high biocompatibility, low immunogenicity, and efficient intracellular delivery. The proprietary linker enables >95% protein recombination in vitro—vastly outperforming traditional EV-loading methods (<5%).
In vivo data confirm heart-specific delivery and functionality, showcasing the platform’s therapeutic potential for diseases previously limited by delivery barriers.
Additional Results
1. Validation & Scalability:
In vitro/in vivo efficacy confirmed; GMP-like production achieved.
2. IP Protection:
Patent filed; commercialization via JAMA Tx underway.
3. Market Insights:
Cardiovascular applications key to EV market growth. Competitor benchmarking and stakeholder backing support strategic direction.
4. Commercial Strategy:
Clear roadmap for spinout formation and market entry.
Impacts
- Scientific: Enables access to “undruggable” targets; sets a new standard for non-viral delivery in HF, cancer, and neurodegenerative diseases.
- Societal: Improved HF therapies may reduce the 18M annual deaths and €210B EU cost burden.
- Economic: JAMA Tx poised to create jobs and generate biotech growth through licensing and partnerships.
- Innovative: Shifts delivery paradigm toward EV-based systems, encouraging new therapeutic pipelines and academic-industry collaboration.
Key Needs
To realize clinical and market impact, the following are essential:
1. Further Research:
Proof-of-concept in pig models, payload specificity, and endosomal escape studies. ATMP dossier preparation for PRIME/EMA approval.
2. Funding & Market Access:
Additional capital and grants needed for clinical development. Engagement with regulators and partners is key.
3. IP Support:
FTO rights and patents for new applications must be secured with legal backing.
4. International Expansion:
Global partnerships, especially in North America and Asia, are crucial.
5. Regulatory Framework:
Collaboration with EMA/FDA and standardization of EV production for compliance.
Overview
TOP-EVICARE achieved all major goals:
- 95% EV loading efficiency; in vivo delivery validated.
- Patent filed, licensing under negotiation.
- Market demand identified; EV platform outperforms alternatives.
- Commercialization roadmap defined.
With further validation and funding, this platform could redefine intracellular therapeutic delivery, particularly in cardiovascular care.
In vivo data confirm heart-specific delivery and functionality, showcasing the platform’s therapeutic potential for diseases previously limited by delivery barriers.
Additional Results
1. Validation & Scalability:
In vitro/in vivo efficacy confirmed; GMP-like production achieved.
2. IP Protection:
Patent filed; commercialization via JAMA Tx underway.
3. Market Insights:
Cardiovascular applications key to EV market growth. Competitor benchmarking and stakeholder backing support strategic direction.
4. Commercial Strategy:
Clear roadmap for spinout formation and market entry.
Impacts
- Scientific: Enables access to “undruggable” targets; sets a new standard for non-viral delivery in HF, cancer, and neurodegenerative diseases.
- Societal: Improved HF therapies may reduce the 18M annual deaths and €210B EU cost burden.
- Economic: JAMA Tx poised to create jobs and generate biotech growth through licensing and partnerships.
- Innovative: Shifts delivery paradigm toward EV-based systems, encouraging new therapeutic pipelines and academic-industry collaboration.
Key Needs
To realize clinical and market impact, the following are essential:
1. Further Research:
Proof-of-concept in pig models, payload specificity, and endosomal escape studies. ATMP dossier preparation for PRIME/EMA approval.
2. Funding & Market Access:
Additional capital and grants needed for clinical development. Engagement with regulators and partners is key.
3. IP Support:
FTO rights and patents for new applications must be secured with legal backing.
4. International Expansion:
Global partnerships, especially in North America and Asia, are crucial.
5. Regulatory Framework:
Collaboration with EMA/FDA and standardization of EV production for compliance.
Overview
TOP-EVICARE achieved all major goals:
- 95% EV loading efficiency; in vivo delivery validated.
- Patent filed, licensing under negotiation.
- Market demand identified; EV platform outperforms alternatives.
- Commercialization roadmap defined.
With further validation and funding, this platform could redefine intracellular therapeutic delivery, particularly in cardiovascular care.