2020-07-003 - HEPATEX - Cell-based extra-hepatic support system

HEPATEX

2020-07-003

ONCOBOTS is a patented technology based on enzyme-powered nanomotors, which are capable to navigate in fluids due to the biocatalytic decomposition of fuel contained in body fluids. ONCOBOTS core can be loaded with anticancer drugs and their surface can also include additional moieties, such as antibodies for cancer targeting, PEG for immune system camouflaging, or markers for fluorescence detection.

By offerign an alternative to enhancing cell therapies and organ transplantation, the technology would open the path to new cell-based solutions and also will reducen the reliance on cadaveric organs, shortening the waiting lists and directly improving patient outcomes and quality of life, repsectively. Environmentally, it will minimize the need for resource-intensive transplant procedures. The project promotes the innovation in healthcare (SDG 3), while the team's expertise in synthesis, scaling and technological improvements ensures ethical, transparent governance and continuos advacements in this life-saving technology.

This technology has the potential to create a significant impact on both, citizens and global sustainability. By providing a viable alternative to traditional organ and tissue transplantation and leveling-up the traditional approach of cell therapies infusions which are currently, without encapsulation. This will impact majorly in reducing the dependency on donor organs, alleviating the pressure on healthcare systems and improving therapeutic solutions for the broader population. On a global scale, having an improved solution will impact public health by addressing solutions as the Type 1 diabetes, which has a severe impact on patients lifestyle and is an incresing condition among juvenile population in EU (295,000). This will not only be benefitial economically for the healthcare systems but also environmentally by contributing with a more sustainable solution while conserving medical resources (organ transplantation) and overall supporting a more resilient and efficient healthcare system.

Cell therapy for tissue replacement is limited due to the difficulty in achieving adequate cell survival, integration and funcitonality within the host tissue.Challenges include ensuring a sufficient supply of viable cells, managing the immune response to prevent rejection, and promoting proper vascularization to support cell nutriton and waste removal. Here, we offer a solution by employing a cell encapsulation technology. The encapsulation allows: cell protection from the host immune system; cell confinement and optimal diffusion of nutrients and oxygen within the body.

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Business-to-business (B2B) model. Envisioned business model focuses on a platform approach, specifically targeting co-development partnerships for a spin-off related to an encapsulation method. The new company will offer encapsulation solutions (through licensing agreements) that will improve cell viability, minimizing cell rejection and ensuring delivery of tissue subproducts, leveraging expertise in encapsulation and cell biology. The goal is to provide value to companies and hospitals that already have cell therapies products in their pipeline by enhancing the delivery of these therapies. Clients will be companies and hospitals with in-house production of specialized cell therapies for therapeutic objectives, such as tissue rejeneration or tissue transplantation, interested in improving the viability of their transplanted cells.

The main needs of the market of cell therapy for tissue transplantation are: reducing graft rejection, limited amount of available organs for transplantation and problems on organ functionality caused by negligent maintenance of cadaveric organs and the challeging conditions needed to obtain scalable cell therapies production.

Primary market: Type 1 Diabetes Mellitus (T1DM), with focus on the severe hypoglycemic population (Latindra indication, the only one FDA-approved and marketed cell therapy for T1DM in the US). Latindra cell infusion do not encapsulate the cells, this could limit the efficiency of the therapy. The price of this therapy is around 300,000$ per patient (~291,448.89€). To reflect a realistic calculation of the market, the price and indication of Latindra was used as a mirror of our target market and a realistic representation for the TAM,SAM,SOM. Additional markets are being also explored to offer our technology as a cell encapsulation platform aimed for other indications.

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Investments for funding a spin-off to perform the required activities (regulatory preclinical, early clinical validation) to accelerate market readiness of the technology, including the development of a GMP facility for cell encapsultion production.

Optimitzation of in-vivo viability of cell encapsulates. Optimum number of encapsulated cells to reach a therapeutic effect. Main figures on time-to replacement of the spheroids. A regulatory roadmap for the therapeutics. Investment for 2 years.

HEPATEX technology would offer the first temporary alternative to cell and organ transplantation, reducing costs and increasing the number of patients that could benefit from organ replacement therapies. The therapy can be autogenic (cells from the same patient), allogenic (cells from another individual) or for future applications xenogenic (cells from animals). The allogenic path is the one currently being tested.

Further pre-clinical in-vivo experiments are required to assess the behaviour of the therapy. The product would be considered an advanced therapy, and requires further studies on the regulatory steps to be performed. Bottlenecks in cell therapy manufacturing (automation, scalability, shortages) should be also taken into account. High cost of cell therapies makes hard their HTA. High quality clinical studies are required for regulatory approval.

(2024): Preclinical studies, in vivo test on diabetic mice (2025): Assessment of the spheroids (immune response and quality after cryopreservation). Commercial and regulatory classification, market analysis and development of a business plan. In-vivo testing in T1DM mice models (6-12M period). Collab agreement with VHIR. (2026): Continuation of in-vivo testing in T1DM mice models. Projected start of EIC Transition with VHIR as affiliated entity. Engagement with EMA for regulatory advice (according to classification provided by AliraHealth) (2027): Start of First-in-human testing, as an early assessment of behaviour in the human body. Engamenet with KOLs, potential investors, patient and physician groups. Regulatory advisory. Spin-off foundation. (2028): Finalisation of EIC Transition grant. Envisioned start of Phase 1 clinical trial. Fundraising seed or series A rounds. (2029): upfront licensing agreement. (2030): clinical trial phase 2. (2031): Clinical trial phase 3. Completion of regulatroy pre-commercial requirements. (2032): EMA Approval

The patent application is already in national phases in Europe, USA, China, Japan (PCT priority date 19/09/2020). Granted in Japan, under examination in the other territories.

The project is available for investors interested in supporting an early-stage spin-off developing cell therapy products.

The intangible assets related to this technology are highly valuable and include proprietary knowledge in the synthesis, scaling, and optimization of the collagen cell encapsulation process. This expertise, developed by a specialized research team, forms the core intellectual property that sets the technology apart in the field of tissue replacement therapies. Additionally, the long-standing know-how in regulatory compliance, quality control, and the development of medical-grade materials further strengthens the competitive edge. Relationships with key stakeholders in the healthcare and biotechnology sectors, as well as a robust research and innovation pipeline.

The Quality Management System for the fabrication and medical use of the collagen cell encapsulation spheres will be designed to comply with regulatory standards, including ISO 13485 for medical devices. This will ensure that the manufacturing process meets the highest standards of safety, performance, and traceability. The system will integrate risk management (ISO 14971) to identify and mitigate potential hazards, while adhering to Good Manufacturing Practices (GMP). Regular audits, quality checks, and validations will be conducted throughout production to ensure the spheres consistently meet regulatory requirements and provide reliable, safe outcomes for patients.

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