2023-12-019 - BrainTAG - Delivering Therapies to the Brain by Generating Artificial Brain-Specific Targets

BrainTAG

2023-12-019

New brain-delivery platform exploiting the physiology of the brain vasculature. Retain molecular tags exclusively on its surface, thereby generating ‘artificial’ targets to specifically deliver therapies to the brain, 'Brain Targets Artificially Generated' (BrainTAG)

The institution has a clear commitment towards the inclusion and diversity, employee engagement, human rights and wellbeing. Furthermore, the project has a commitment towards reducing environmental impact, engaging citizenship and overseed ethical aspects and gender equality, regarding the widespread of the technology.

By precisely directing therapies to the brain, the technology aims to enhance treatment efficacy while significantly reducing systemic exposure to medications. This targeted approach can lead to fewer side effects, improving the overall quality of life for patients. As a result, patients may experience better adherence to treatment regimens, reduced hospital visits, and a diminished burden on caregivers. Ultimately, this innovation has the potential to transform Alzheimer's care, making therapies safer and more effective while promoting better health outcomes.

Brain disorders are the leading cause of disability and the second leading cause of death worldwide, affecting 1 in 3 people during their lifetime. These disorders, ranging from migraines to Alzheimer's and brain cancer, not only have a profound health impact but also cost over €700 billion annually in Europe. With an aging population, the prevalence of brain disorders is expected to rise, posing a significant health and economic burden. The Brain-TAG delivery platform aims to improve treatment by enhancing the delivery of medicines to the brain, maximizing therapeutic effects and reducing side effects. This innovation could improve quality of life and alleviate the economic strain on society.

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An effective therapy-delivery platform will de-risk the development of treatments against brain disorders by increasing the currently inadequately low rate of clinical translation. We envision a B2B business model, we will improve biodistribution profiles of brain directed drugs by combinating the drugs with our platform. Portfolio would be increased via co-development contracts.

There is a demand for more effective treatments for neurological disorders, such as Alzheimer's, Parkinson's and brain tumors. The market requires enhanced delivery methods, improving treatment outcomes, and reducing side effects, ultimately providing a solution to a significant unmet medical need in neurology and neuro-oncology.

Market of Alzheimer treatment

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Financement for the in-vivo validation of the therapy-targeting to the brain in mouse models of Alzheimer. Financement for a market study, product classification and regulatory strategy.

1.Optimize the Brain-TAG NP to maximize the delivery to brain endothelial cells. 2.Validate the identified peptides in vivo applying the Brain-TAG platform to deliver therapy-loaded NP to the brain to treat Alzheimer Disease 3.Validate business opportunities of creating a spin-off to commercialize the asset.

Brain-TAG creates exclusive artificial targets on brain vessels, allowing precise delivery of therapies to the brain without pertubation of the endothelial receptors, offering a more physiological solution to reach the brain.

Regulatory hurdles (EMA, FDA approvals). Scientific evidence against standards, high development costs, competition from established pharmaceutical companies.

(2024): in-vitro optimization. Engagement of functionalized nanoparticles to maximize NP delivery to brain endothelial cells. (2025): In vitro optimizatin and validation. Expected enter of National Phase of BrainTAG PCT application. (2026): in-vivo PoC, delivering NP to Alzheimer's disease mouse models. Business validation for spin off foundation. (2027): Assesment of the results obtained. Spin-off launch and in-vivo validation (2028): Gathering robust data on preclinical development. Fostering colllaboration, partnerships or co-development to advance to testing in non-rodent models. Seed or Series A funding. (2029): Regulatory assessment with EMA. Phase 1 clinical trials (partnered or co-developed) (2030): projected license agreement (2031): Phase 2 clinical trials (2032): Phase 3 clinical trials (2033): EMA approval (2034): envisioned EU market entry (2035): product commercialization.

PCT patent application with priority date 7th June 2023

The project align with advancing global health and sustainability. Socially, it addresses a critical unmet need by offering a more effective, targeted treatment for Alzheimer's, improving patient outcomes and reducing the burden on caregivers and healthcare systems. Environmentally, the precision of the delivery system minimized drug wastage and reduces the environmental impact of widespread medication use. In terms of governance, the project is committed to ethical research, regulatory compliance and transparency in clinical trials, enusring safety and efficacy while maintaining high standards of patient care and data integrity.

Firstly, propietary knowledge and expertise in peptide design and formulation are vital intellectual properties that differentiate the technology in a competitive market. Additionally, strong relationships with academic institutions, regulatory bodies, and industry partners contribute to collaborative innovation and facilitate smoother pathways to market. The project is also committed to sustainability by minimizing drug wastage and optimizing therapeutic delivery, which aligns with growing environmental concerns in healthcare. Collectively, these intangible assets not only booster the project's competitive advantage edge but also its long-term viability in an increasingly eco-conscious landscape.

Quality management will be crucial in adhering to regulatory standards that ensure safety and efficacy. Compliance with regulations and correct product classification will allow encompasing design, production, and post-market surveillance. Additionally, adherence to Good Manufacturing Practices (GMP) will be required to ensure consistent product quality during manufacturing. Regulatory bodies like the FDA and EMA will require extensive documentation and validation of clinical trial results, including Phase I and II studies, to support an Investigational New Drug (IND) application. Failure to meet these quality and regulatory requirements could delay market entry and hinder patient access to potentially transformative brain-targeted therapies.

BrainTAG has the potential to revolutionize brain drug delivery by increasing brain specificity, delivery efficiency and safety profile. Hence, our platform will have a clear beneficial impact on brain disorders and strong potential for market success. This platform differs from current delivery strategies employing ‘natural’ targets with inherent brain-specificity and safety limitations having the potential to achieve truly brain-specific therapy delivery against a wide range of brain disorders.