Periodic Reporting for period 1 - i-THYMUS (The i-Thymus: wielding the potential of gene therapy, cell therapy and induced pluripotent stem cells for the regeneration of the thymus and the adaptive immune system)
Período documentado: 2023-11-01 hasta 2024-10-31
The thymus is a small but vital organ essential for immune development. In certain rare diseases, there are children who are born without a thymus. These children are unable to develop an effective immune system and they therefore face profound vulnerability to infections and rarely survive past the age of three without intervention. Currently, the only treatment option, thymus tissue transplantation, is dependent on donor tissue, carries a significant mortality rate and morbidity due to the risk of immune complications or graft rejection, and commercial products based on this therapy are only available in the US and costs exceed €2.5 million per patient.
Our solution, i-Thymus, aims to change this reality. Leveraging advanced gene-modified cell therapy, i-Thymus uses the patient’s own cells to create implantable thymus tissue, offering a potentially safer, more accessible, and cost-effective alternative to transplantation. In vivo proof-of-concept data is already established, and with the support of the EIC Accelerator, we are accelerating pre-clinical development to prepare for clinical trials and market entry.
Our pathway includes rapid market entry through Orphan Drug Designation, ensuring that the therapy reaches those most urgently in need. Building on early clinical success and revenue, we will expand the application of i-Thymus to address additional immune deficiencies. i-Thymus has the potential to meet significant unaddressed needs within the healthcare system, advancing strategic goals for improved public health and patient outcomes across Europe.
Our solution, i-Thymus, aims to change this reality. Leveraging advanced gene-modified cell therapy, i-Thymus uses the patient’s own cells to create implantable thymus tissue, offering a potentially safer, more accessible, and cost-effective alternative to transplantation. In vivo proof-of-concept data is already established, and with the support of the EIC Accelerator, we are accelerating pre-clinical development to prepare for clinical trials and market entry.
Our pathway includes rapid market entry through Orphan Drug Designation, ensuring that the therapy reaches those most urgently in need. Building on early clinical success and revenue, we will expand the application of i-Thymus to address additional immune deficiencies. i-Thymus has the potential to meet significant unaddressed needs within the healthcare system, advancing strategic goals for improved public health and patient outcomes across Europe.
To create patient-specific i-Thymus organoids, we leverage stem cell technology, differentiating induced pluripotent stem cells (iPSCs) through a controlled series of stages to produce functional thymus tissue. This process mimics the natural embryonic formation of the thymus. Using selected combinations of growth factors, we guide these cells to adopt the characteristics needed to form thymus tissue. The complex sequence of steps establishes the foundation necessary for guiding the development of immune cells that protect against infections – a function missing in patients born without a thymus.
During the first year of the EIC-backed project, we have refined our protocols, enhancing the differentiation of stem cells into thymic tissue. Building on prior research and newly acquired insights, we evaluated various growth factors, media formulations, and reagents, not only for their role in efficiently guiding stem cells but also to ensure their suitability for clinical-grade manufacturing. These adjustments have brought us closer to a protocol that meets stringent Good Manufacturing Practice (GMP) standards, which are essential for producing reliable, high-quality thymus organoids that can be safely used in patients.
Furthermore, our team has made key improvements to the gene therapy vector that supports this process, enhancing its ability to drive cell differentiation and promote thymus functionality. This optimised design is critical in helping to secure consistent outcomes in future clinical applications.
Last, we have established a commercial athymic (i.e. born without thymus) mouse model in our lab with human immune progenitor cells. This humanised mouse model will be used to study the efficacy of i-Thymus in developing human T-cells in the next stage of the EIC Accelerator project.
During the first year of the EIC-backed project, we have refined our protocols, enhancing the differentiation of stem cells into thymic tissue. Building on prior research and newly acquired insights, we evaluated various growth factors, media formulations, and reagents, not only for their role in efficiently guiding stem cells but also to ensure their suitability for clinical-grade manufacturing. These adjustments have brought us closer to a protocol that meets stringent Good Manufacturing Practice (GMP) standards, which are essential for producing reliable, high-quality thymus organoids that can be safely used in patients.
Furthermore, our team has made key improvements to the gene therapy vector that supports this process, enhancing its ability to drive cell differentiation and promote thymus functionality. This optimised design is critical in helping to secure consistent outcomes in future clinical applications.
Last, we have established a commercial athymic (i.e. born without thymus) mouse model in our lab with human immune progenitor cells. This humanised mouse model will be used to study the efficacy of i-Thymus in developing human T-cells in the next stage of the EIC Accelerator project.
Results and potential impact
Securing Orphan Drug Designation (ODD) from both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) represents a major breakthrough for the i-Thymus project. The early recognition by two regulatory authorities not only highlights the therapeutic promise and unique impact of our innovation but also facilitates a streamlined path to market. With ODD status, i-Thymus will benefit from regulatory support, financial incentives, and market exclusivity once approved, all of which accelerate our timeline and enhance the project’s impact. This ODD underscores our commitment to addressing an urgent unmet medical need for patients born without a thymus and strengthens our position as a leader in pioneering therapies for rare immune disorders.
To protect the intellectual property underpinning this innovation, we have filed a patent application and registered trademarks that secure both our proprietary technology and brand identity. This IP strategy is vital as it ensures long-term competitive advantage, supports future licensing opportunities, and enables us to attract and retain investment to further develop and expand our platform.
Another pivotal result has been the optimisation of our gene therapy vector. Our vector is also suitable for Good Manufacturing Practice (GMP) production, a critical step for ensuring consistent quality and safety at clinical scale.
Key needs to ensure further uptake and success
A key need is now the establishment of a GMP network for transitioning from pre-clinical to clinical stage upon the finalisation of GMP processes; discussions with suitable partners are currently ongoing.
Another key need is the ongoing Series A financing, for which we have commitments and several planned follow-on discussions with potential investors. In the light of outreach, we have elevated our company’s online presence with a professionalised brand and website that now includes expanded technology content, the entire team, and a dedicated news section, providing a more informative and engaging experience for our audiences (www.genewity.tech).
With our recent additions of an experienced COO and a CFO with background and network in venture capital, our team is further strengthened in operations and finance. For this, they are supported by our advisors as seasoned experts from science, clinic and industry. This, alongside our active capital-raising efforts, underscores our growth trajectory and commitment to our mission. In total, the team has grown as a direct result of the project in the past year with the additional hiring of a third senior researcher, an experienced research technician, and a dedicated project manager.
Securing Orphan Drug Designation (ODD) from both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) represents a major breakthrough for the i-Thymus project. The early recognition by two regulatory authorities not only highlights the therapeutic promise and unique impact of our innovation but also facilitates a streamlined path to market. With ODD status, i-Thymus will benefit from regulatory support, financial incentives, and market exclusivity once approved, all of which accelerate our timeline and enhance the project’s impact. This ODD underscores our commitment to addressing an urgent unmet medical need for patients born without a thymus and strengthens our position as a leader in pioneering therapies for rare immune disorders.
To protect the intellectual property underpinning this innovation, we have filed a patent application and registered trademarks that secure both our proprietary technology and brand identity. This IP strategy is vital as it ensures long-term competitive advantage, supports future licensing opportunities, and enables us to attract and retain investment to further develop and expand our platform.
Another pivotal result has been the optimisation of our gene therapy vector. Our vector is also suitable for Good Manufacturing Practice (GMP) production, a critical step for ensuring consistent quality and safety at clinical scale.
Key needs to ensure further uptake and success
A key need is now the establishment of a GMP network for transitioning from pre-clinical to clinical stage upon the finalisation of GMP processes; discussions with suitable partners are currently ongoing.
Another key need is the ongoing Series A financing, for which we have commitments and several planned follow-on discussions with potential investors. In the light of outreach, we have elevated our company’s online presence with a professionalised brand and website that now includes expanded technology content, the entire team, and a dedicated news section, providing a more informative and engaging experience for our audiences (www.genewity.tech).
With our recent additions of an experienced COO and a CFO with background and network in venture capital, our team is further strengthened in operations and finance. For this, they are supported by our advisors as seasoned experts from science, clinic and industry. This, alongside our active capital-raising efforts, underscores our growth trajectory and commitment to our mission. In total, the team has grown as a direct result of the project in the past year with the additional hiring of a third senior researcher, an experienced research technician, and a dedicated project manager.