CBR1

Breakthrough in Delivering Chimeric Bait Receptors to Immune Cells Accelerates Development LONDON, UK / ACCESSWIRE / December 6, 2024 / Hemogenyx Pharmaceuticals plc (LSE:HEMO), the clinical-stage biopharmaceutical company, is pleased to announce a breakthrough in its development of a novel method for delivering Chimeric Bait Receptors ("CBR") to innate immune cells such as macrophages and monocytes i.e. the white myeloid blood cells that make up the body's first line of defence against infections and responsible for the removal of diseased or damaged cells. This innovative approach utilizes lentiviral vectors (a laboratory constructed and modified retrovirus that can be used to insert genetic material into cells) to achieve highly efficient transduction of macrophages or monocytes, while maintaining durable, long-lasting CBR expression in these cells. In more simple terms, the new methodology will extend the efficacy as well as the durability of CBR within transduced cells.The Company believes that this innovative approach to myeloid immune cell transduction will significantly accelerate the development of CBR-based product candidates, facilitating their progression into IND-enabling studies.The Company's first-class scientific team continues to make good progress with CBR and, once sufficient resources are provided, the Company anticipates moving new CBR-based product candidates into clinical trials, bringing them closer to addressing unmet medical needs.Dr Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented: "We are thrilled to announce this critical advancement in our CBR platform, which represents a significant step forward in our mission to develop cutting-edge therapies for unmet medical needs. Overcoming the challenges of efficiently delivering CBRs to myeloid immune cells has been a key focus for our team, and this breakthrough underscores their dedication and ingenuity We remain committed to leveraging this technology to bring transformative therapies to the clinic and, ultimately, to patients in need. Once sufficient resources are available to CBR, we are confident in our ability to translate these scientific breakthroughs into impactful treatments."Market Abuse Regulation (MAR) DisclosureCertain information contained in this announcement would have been inside information for the purposes of Article 7 of Regulation No 596/2014 (as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018) until the release of this announcement.Enquiries:Hemogenyx Pharmaceuticals plchttps://hemogenyx.comDr Vladislav Sandler, Chief Executive Officer & Co-Founder[email protected]Peter Redmond, Director[email protected] SP Angel Corporate Finance LLPTel: +44 (0)20 3470 0470Matthew Johnson, Vadim Alexandre, Adam Cowl Peterhouse Capital LimitedTel: +44 (0)20 7469 0930Lucy Williams, Duncan Vasey, Charles Goodfellow About Hemogenyx Pharmaceuticals plcHemogenyx Pharmaceuticals is a publicly traded company (LSE: HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility.The Company is a clinical-stage biopharmaceutical group developing new medicines and treatments for life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development.This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact [email protected] or visit www.rns.com.SOURCE: Hemogenyx Pharmaceuticals PLC

Sight loss in certain inherited eye diseases may be caused by gut bacteria, and is potentially treatable by antimicrobials, finds a new study in mice. Sight loss in certain inherited eye diseases may be caused by gut bacteria, and is potentially treatable by antimicrobials, finds a new study in mice co-led by a UCL and Moorfields researcher. The international study observed that in eyes with sight loss caused by a particular genetic mutation, known to cause eye diseases that lead to blindness, gut bacteria were found within the damaged areas of the eye. The authors of the new paper, published in Cell and jointly led by researchers in China, say their findings suggest that the genetic mutation may relax the body's defences, thus allowing harmful bacteria to reach the eye and cause blindness. The gut contains trillions of bacteria, many of which are key to healthy digestion. However, they can also be potentially harmful. The researchers were investigating the impact of the Crumbs homolog 1 (CBR1) gene, which is known to be expressed in the retina (the thin layer of cells at the back of the eye) and is crucial to building the blood-retina barrier to regulate what flows in and out of the eye. The CRB1 gene is associated with inherited eye disease, most commonly forms of Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP); the gene is the cause of 10% of LCA cases and 7% of RP cases worldwide. Using mouse models, the research team discovered the CRB1 gene is key to controlling the integrity of the lower gastrointestinal tract, the first ever such observation. There, it combats pathogens and harmful bacteria by regulating what passes between the contents of the gut and the rest of the body. The team found that when the gene has a particular mutation, dampening its expression (reducing its effect), these barriers in both the retina and the gut can be breached, enabling bacteria in the gut to move through the body and into the eye, leading to lesions in the retina that cause sight loss. Crucially, treating these bacteria with antimicrobials, such as antibiotics, was able to prevent sight loss in the mice even though it did not rebuild the affected cell barriers in the eye. Inherited eye diseases are the UK's leading cause of blindness in working-age people. Onset of disease may vary from very early childhood to adulthood, but deterioration is irreversible and has lifelong implications. To date, the development of treatments has largely focused on gene therapies. The findings of this study suggest that simply using antimicrobials might help prevent deterioration in CRB1-associated inherited eye diseases. Future work will investigate whether this applies in humans. Co-lead author Professor Richard Lee (UCL Institute of Ophthalmology and Moorfields Eye Hospital NHS Foundation Trust) said: "We found an unexpected link between the gut and the eye, which might be the cause of blindness in some patients. "Our findings could have huge implications for transforming treatment for CRB1-associated eye diseases. We hope to continue this research in clinical studies to confirm if this mechanism is indeed the cause of blindness in people, and whether treatments targeting bacteria could prevent blindness. "Additionally, as we have revealed an entirely novel mechanism linking retinal degeneration to the gut, our findings may have implications for a broader spectrum of eye conditions, which we hope to continue to explore with further studies." The study was an international collaboration led by researchers from Zhongshan Ophthalmic Center, Sun Yat-sen University, China, working with UCL Institute of Ophthalmology and Moorfields Eye Hospital NHS Foundation Trust.

Chimeric Bait Receptors for Combatting Airborne Viral Infections Can Be Delivered Intranasally LONDON, UK / ACCESSWIRE / February 14, 2024 / Hemogenyx Pharmaceuticals plc (LSE:HEMO), the clinical stage biopharmaceutical group, announces that it has demonstrated in vivo that its proprietary Chimeric Bait Receptor ("CBR") can be delivered intranasally in the form of messenger RNA ("mRNA") for the potential treatment of airborne viral infections. CBR-based countermeasures against viral infections that are include a novel method of their delivery. To satisfy real-world requirements for the deployment of such countermeasures, they must be both easy to administer and have a long shelf life at ambient temperatures. CBR is a platform technology under development that aims to program the immune cells that are responsible for innate immunity (e.g., macrophages) to eliminate viruses and certain malignant cells. Our team of scientists has demonstrated in vivo that mRNA encoding a CBR-based therapeutic can be mixed with a polymer and a solvent at room temperature to form polymer nanoparticles encapsulating mRNA. Such nanoparticles can be used to deliver CBR-coding mRNA into the upper respiratory tract ("URT") and lungs of small animals. Once administered, nanoparticles were shown to successfully deliver mRNA into the immune cells of the URT and lungs. The immune cells in turn begin making CBR molecules within hours post-application, potentially becoming active "defenders" against airborne infections. As previously reported, the Company has been able to dramatically accelerate its research and development efforts by usingArtificial Intelligence ("AI") and in-house synthesis of mRNA. AI tools allow the Company's scientists to triage variants of CBR constructs efficiently to select those with the greatest likelihood of success, while in-house mRNA synthesis facilitates the rapid building of CBR constructs. Dr Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented: "This major breakthrough in the method of delivery of our CBR demonstrates that it could potentially be used as an off-the-shelf prevention and/or treatment for viral infections. Intranasal delivery of CBR would be both cost-efficient and easy to administer, making it ideal for the protection of both the civilian population and in biodefense." Market Abuse Regulation (MAR) Disclosure Certain information contained in this announcement would have been inside information for the purposes of Article 7 of Regulation No 596/2014 (as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018) until the release of this announcement. Enquiries: Hemogenyx Pharmaceuticals plc Dr Vladislav Sandler, Chief Executive Officer & Co-Founder headquarters@hemogenyx.com Peter Redmond, Director peter.redmond@hemogenyx.com SP Angel Corporate Finance LLP Tel: +44 (0)20 3470 0470 Matthew Johnson, Vadim Alexandre, Adam Cowl Peterhouse Capital Limited Tel: +44 (0)20 7469 0930 Lucy Williams, Duncan Vasey, Charles Goodfellow About Hemogenyx Pharmaceuticals plc Hemogenyx Pharmaceuticals is a publicly traded company (LSE: HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility. The Company is a clinical-stage biopharmaceutical group developing new medicines and treatments for life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development. This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit . SOURCE: Hemogenyx Pharmaceuticals PLC View the original press release on accesswire.com