AskBio Inc.

Trogenix emerged from stealth on Monday with an undisclosed amount of seed funding to advance its Odysseus platform, which is intended to enable the safe, specific targeting of cancer cells via its synthetic super-enhancers (SSEs) technology.The startup, which was spun out from the University of Edinburgh last year, was built by venture capital firm 4BIO Capital and has also received funding from IQ Capital, Cancer Research Horizons, the US National Brain Tumor Society’s Brain Tumor Investment Fund and AIN Ventures. Trogenix plans to start a Phase I/II trial of its lead programme in patients with glioblastoma next year. According to the firm, the compound was non-toxic and demonstrated "curative responses" in preclinical studies, with evidence of anti-tumour immunity. The company's SSEs, which are engineered DNA elements that act as docking stations for transcription factors uniquely expressed in aggressive cancer cells, are designed to avoid healthy cells thanks to targeted expression of cytotoxic payloads, while also 'waking up' the immune system to provide protection against recurrence. Trogenix expects to deliver its therapeutics as a direct tumour injection for a localised effect. The biotech has set itself the goal of submitting five INDs in five years. In addition to glioblastoma, it's exploring the potential of SSEs in other cancers such as colorectal cancer liver metastases, hepatocellular carcinoma and lung squamous cell carcinoma, as well as regenerative medicine applications. At Trogenix's helm is Ken Macnamara, who previously served as chief operating officer of Asklepios BioPharmaceutical, the gene therapy arm of Bayer. "We founded Trogenix to transform cancer treatment from chronic disease management to potentially curative one-time treatments," Macnamara commented. "Our Synthetic Super-Enhancer technology represents a significant breakthrough in precision cancer therapy, offering unprecedented selectivity in targeting cancer cells and activating the body's own immune system against tumours, all while leaving healthy cells untouched."

The programme utilises Intellia’s CRISPR-based gene editing platform and DNA-writing technology. Credit: Natali _ Mis/Shutterstock. ReCode Therapeutics is set to secure up to $15m in additional funding from the Cystic Fibrosis Foundation to enhance its gene correction research programme. The initiative, conducted in collaboration with Intellia Therapeutics , aims to develop new treatments for cystic fibrosis (CF), including for patients with genotypes that are non-responsive or intolerant to existing cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The programme utilises Intellia’s clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing platform and deoxyribonucleic acid (DNA)-writing technology along with the company’s selective organ targeting (SORT) lipid nanoparticle (LNP) delivery platform aiming at gene correction. The investment will propel the development of up to three gene correction programmes which are designed to address CF mutations not covered by current therapies. It will also enhance ReCode’s existing RCT2100 clinical programme, an investigational inhaled messenger ribonucleic acid (mRNA) therapy, currently in a Phase Ib multi-dose study for CF patients. ReCode Therapeutics CEO Shehnaaz Suliman stated: “This funding will support our gene correction approach, enabling us to significantly accelerate research in collaboration with Intellia and to advance the development of a potentially transformative treatment for CF. “Along with RCT2100, our inhaled mRNA programme, we are building a therapeutic franchise to expand disease-modifying treatment options for all people with CF.” In February 2024, the company announced a strategic collaboration with Intellia Therapeutics to develop gene editing therapies for CF. CF is a debilitating genetic disease leading to persistent lung infections and respiratory failure, stemming from mutations in the CFTR gene. The company’s lead programmes, RCT2100 and RCT1100, target CFTR gene mutations unresponsive to approved modulators and primary ciliary dyskinesia, respectively. Both are inhaled mRNA-based therapies formulated with ReCode’s SORT LNP delivery platform. In January 2023, Asklepios BioPharmaceutical entered a research partnership with ReCode to explore a single-vector gene-editing platform.

Trogenix Unveils Revolutionary Platform to Transform Cancer Treatment Through Precision Viral Immunotherapy Company founded by 4BIO Capital and Professor Steve Pollard as a spinout from the University of Edinburgh to commercialise its breakthrough Synthetic Super-Enhancer platform, Odysseus, to enable unprecedented precision in targeting aggressive cancersIQ Capital, Cancer Research Horizons, National Brain Tumor Society, AIN Ventures and Old College Capital now also investors in Trogenix Dr Ken Macnamara, a veteran gene therapy executive who held leadership roles at Bayer/AskBio and Synpromics, appointed as Chief Executive OfficerTay Salimullah, former executive team member at Novartis Gene Therapies, joins the Board as Trogenix advances towards clinical development Edinburgh, UK, 18 November 2024 -- Trogenix Ltd today emerged from stealth mode to unveil its groundbreaking therapeutic platform for treating aggressive cancers. Built from inception by 4BIO Capital, which specialises in creating breakthrough therapeutic companies, Trogenix spun out from the University of Edinburgh in 2023 and raised a seed financing round earlier this year. Leading life science and deep tech investors IQ Capital, Cancer Research Horizons, the US National Brain Tumor Society’s Brain Tumor Investment Fund, AIN Ventures and the University of Edinburgh’s venture investment fund, Old College Capital, have also invested in Trogenix. "Cancer remains a leading cause of death worldwide, with current therapies often falling short, especially for aggressive solid tumours," said Dr Ken Macnamara, Chief Executive Officer of Trogenix. "We founded Trogenix to transform cancer treatment from chronic disease management to potentially curative one-time treatments. Our Synthetic Super-Enhancer technology represents a significant breakthrough in precision cancer therapy, offering unprecedented selectivity in targeting cancer cells and activating the body's own immune system against tumours, all while leaving healthy cells untouched." The company's lead programme targets glioblastoma (GBM), the most aggressive form of brain cancer, where only 25% of patients survive beyond one year. Preclinical studies have demonstrated curative responses with no toxicity and evidence of persistent anti-tumour immunity, with Phase 1/2 clinical trials planned for 2025. "Trogenix exemplifies our approach to advancing revolutionary therapeutic platforms from concept to reality," said Dmitry (Dima) Kuzmin, Managing Partner at 4BIO Capital and Founding Chairman of Trogenix. "By combining our deep expertise in advanced therapies with the company's breakthrough science, we've helped shape a platform with the potential to fundamentally change cancer treatment. The convergence of synthetic biology, machine learning, gene therapy, and precision targeting in Trogenix's technology represents exactly the kind of innovation needed to deliver next-generation curative treatments. Being able to discern a cancer cell in situ from a healthy cell without relying on a surface antigen is truly revolutionary." The company's proprietary Synthetic Super-Enhancers (SSEs) are engineered DNA elements that act as docking stations for transcription factors uniquely expressed in aggressive cancer cells. This enables highly selective and potent gene control with multiple layers of safety, including direct tumour injection for localised delivery and self-limiting control mechanisms. Through these SSEs, Trogenix's Odysseus platform delivers a comprehensive attack on cancer through controlled cell killing via targeted expression of cytotoxic payloads, immune modulation to overcome tumour-induced immunosuppression, and a unique ‘Trojan Horse’ approach that reawakens the immune system to provide long-term protection against recurrence. World-Class Leadership Team Trogenix emerged from groundbreaking research at the University of Edinburgh - including its UK Centre for Mammalian Synthetic Biology and Institute for Regeneration and Repair - and the Cancer Research UK Scotland Centre. The company was co-founded by Professor Steve Pollard, a renowned authority in regenerative medicine and cancer biology, who serves as Chief Scientific Officer. Under his leadership, the company has rapidly advanced its revolutionary SSE platform from concept to preclinical proof-of-concept. Chief Executive Officer Dr Ken Macnamara brings extensive leadership experience in gene therapy and biotech, including senior roles at AskBio (a Bayer company), and involvement in three successful M&A exits. "Our Odysseus platform represents a significant advancement in cancer treatment," said Professor Steve Pollard, Chief Scientific Officer of Trogenix. "By precisely targeting cancer cell states rather than just cell types, we can achieve unprecedented selectivity while activating the body's own immune system against the tumour. Our preclinical studies in glioblastoma have demonstrated complete responses with no toxicity and evidence of persistent anti-tumour immunity." Beyond GBM, Trogenix is advancing programmes in colorectal cancer liver metastases, hepatocellular carcinoma, lung squamous cell carcinoma, and regenerative medicine, with the goal of submitting five Investigational New Drug (IND) applications in five years. Strategic Board Appointment As Trogenix advances its groundbreaking platform toward clinical development, the company has appointed Tay Salimullah to its Board of Directors, his first Board appointment since leaving Novartis Gene Therapies. Tay has over 20 years of leadership experience in the rare disease and MedTech sectors, with a proven track record in incubating and scaling innovative, high-value technologies, including cell and gene therapies. He spent over a decade at Novartis in a series of leadership roles. As an Executive team member at Novartis Gene Therapies, Tay executed a new commercial model for Zolgensma® securing approvals in more than 55 countries, helping to treat over 4,000 patients, and generating more than $5 billion in revenue. Prior to his roles at Novartis, Tay worked in a variety of investment and healthcare roles, including a decade at Pfizer where he held several strategic and commercial roles. His expertise spans drug development, pricing science, market access, and operational excellence in high-value healthcare solutions. Tay holds a BSc in Management Sciences from the University of Brunel, London. About Trogenix Trogenix unites cutting-edge technologies in genomics, oncology, immunotherapy, and gene therapy to create a revolutionary therapeutic approach through its Odysseus platform. Using proven AAV vectors, our proprietary Synthetic Super-Enhancers (SSEs) are delivered directly to tumour cells without detection. Our SSEs enable unprecedented precision in gene control, effectively revealing cancer to the body's immune system and killing tumour cells. For any cells escaping Trogenix's technology, our 'Trojan horse' reawakens the immune system, countering recurrence and offering potentially curative 'one-and-done' treatments for aggressive tumours. With our lead asset showing curative potential in preclinical glioblastoma studies, we're poised to transform treatment paradigms across multiple cancer types and explore applications beyond oncology, such as regenerative medicine. For more information, visit www.trogenixbio.com. About 4BIO Capital 4BIO Capital (‘4BIO’) is an international venture capital firm headquartered in London, focused solely on the advanced and emerging therapies sector. The 4BIO team, which has an unrivalled network within the advanced therapy sector, comprises leading advanced therapy scientists and experienced life science investors. The firm maintains a global footprint across the US, Europe and Asia with an objective to create, invest in, support, and grow early-stage companies. Its ultimate goal is ensuring access to potentially curative therapies for all patients, with a specific focus on viable, high-quality opportunities in cell and gene therapy, RNA-based therapy, targeted therapies, and the microbiome. 4BIO is currently investing out of its Fund III, having secured support from prominent global institutional investors, including the Children’s Minnesota, UPMC, Development Bank of Japan, Kyowa Kirin, Exor, and many other endowments and foundations. For more information, please visit www.4BioCapital.com For more media enquiries, please contact: trogenix@icrhealthcare.com