CBR1

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

THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION FOR THE PURPOSES OF ARTICLE 7 OF REGULATION 2014/596/EU AS IT FORMS PART OF LAW IN THE UNITED KINGDOM BY VIRTUE OF THE EUROPEAN UNION (WITHDRAWAL) ACT 2018. UPON THE PUBLICATION OF THIS ANNOUNCEMENT, THIS INSIDE INFORMATION IS NOW CONSIDERED TO BE IN THE PUBLIC DOMAIN. LONDON, UK / ACCESSWIRE / April 28, 2023 / Hemogenyx Pharmaceuticals plc (LSE:HEMO), the biopharmaceutical group developing new therapies and treatments for deadly blood diseases, announces its results for the year ended 31 December 2022. Key Highlights Nearing completion of the IND application to the FDA to enter phase I clinical trials for HEMO-CAR-T for the treatment of R/R AML. CBR platform extended into treatment of multiple viral infections beyond COVID-19 using single CBR-based therapeutic. Initiated IND-enabling activities for CDX bi-specific antibody for treatment of R/R AML. Successfully set up and qualified GMP manufacturing and analytical testing of cell therapies and implemented Quality System in a new purpose-built R&D/manufacturing facility. The full annual report and accounts for 2022 are published on the Company's web site at and will be available from Companies House and the National Storage Mechanism. Cautionary Note Regarding Forward-Looking Statements Certain statements in this news release contain forward-looking information. These statements address future events and conditions and, as such, involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the statements. Such factors include without limitation the completion of planned expenditures, the ability to complete exploration programs on schedule and the success of exploration programs. Readers are cautioned not to place undue reliance on the forward-looking information, which speak only as of the date of this news release. 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 pre-clinical stage biopharmaceutical group developing new medicines and treatments to treat blood and autoimmune disease and to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as platform technologies that it uses as engines for novel product development. Chairman's Statement I am pleased to announce the Company's results for the year ended 31 December 2022. During the year we focussed heavily on bringing our major development project, the key HEMO-CAR-T product candidate, towards its Investigational New Drug ("IND") application to enable us to move into clinical trials. At the same time, we also advanced the development of our other main pipeline assets, our CD3-FLT3 CDX antibody and the Chimeric Bait Receptor ("CBR") platform. After the year end, in January, we successfully raised £4,056,250 in new equity capital at 2.5p per share which will give us the funds to take the Company through the IND process and into the beginning of clinical trials for HEMO-CAR-T and enable us to further advance the CBR project. HEMO-CAR-T Our work during the period under review has primarily focussed on bringing our lead product, HEMO-CAR-T, through the preparatory process to clinical trials, a process which has been more complex and hence longer and more intensive than we had anticipated but which has now reached an advanced stage. We have continued to move other projects forward, in particular our CBR technology, but our main object has been to take HEMO-CAR-T, and with it the Company, to the next critical level of being a clinical-stage company. We have been particularly concerned to cover all aspects in preparing the IND documentation so as to minimise any possible delays and questions that may arise from its review by the US Food and Drug Administration ("FDA"). The IND submission process is very detailed, as it should be to ensure the safety of this key potential treatment for patients suffering from advanced stage relapsed or refractory ("R/R") acute myeloid leukaemia ("AML"). We received constructive early feedback and guidance from a "pre-IND submission" to the FDA which have helped to shape the final submission, along with advice from our Medical Director and a committee of "Key Opinion Leaders" who are experts in the treatment of leukaemias, as well as the design and conduct of clinical trials. We expect to submit the IND application in the very near future. During the last quarter of 2022 and the early months of the current year we successfully carried out the final processes and underwent the internal and third-party tests necessary to complete the detailed IND submission pack. These included Process Development runs of the end-to-end process for the manufacture of HEMO-CAR-T cells and exhaustively documented engineering, or Process Qualification, runs under real-world conditions. These cell manufacturing dry runs were followed by analytical release tests that were conducted both by the Company and a third party to ensure that the manufactured HEMO-CAR-T cells comply with a set of required quality attributes. Among these are the viability, potency and sterility of the resulting cells. CDX CDX, our CD3-FLT3 bispecific antibody, will provide an alternative means of treating AML and of conditioning patients for bone marrow transplants when fully developed. While concentrating our efforts on HEMO-CAR-T as the asset most ready to take the Company to the important clinical trial stage of its maturity, we have taken further steps to develop this important asset during the year. In January 2022, we entered into a service agreement to develop a "master cell line" that will be used to produce CDX antibodies for future clinical trials and patient treatments. We are utilising Selexis' SUREtechnology Platform™, a suite of cell line development tools and technologies that reduces the time, effort and cost in developing high-performance mammalian cell lines. The platform facilitates the rapid, stable, and importantly cost-effective production of recombinant proteins and vaccines, providing seamless integration of the development continuum from discovery to commercialisation. This is an important step in moving CDX towards clinical trials. The Company's existing intellectual property protection for CDX was further strengthened by the China National Intellectual Property Administration granting a patent to it, which joins patents previously granted in the US for CDX and monoclonal antibodies used for the development of both CDX and HEMO-CAR-T. Exploration of ways to finance and further the pre-clinical and clinical testing of CDX continued with early-stage conversations with potential development partners. CBR Work has also continued in an encouraging manner on the development of our CBR platform. As shareholders are aware, the essence of the CBR-based approach is programming immune cells using a novel type of modifiable synthetic receptor to destroy viral pathogens and potentially to programme immune cells to destroy certain malignant cancer cells. The Company has also developed an associated derivative technology, the Bait Macrophage Engager ("BME"), whose constructs act like antibodies, directing immune cells to neutralise them. We believe this novel approach holds great promise and the invention is the subject of a seminal provisional patent application that was filed in March 2022. This project was initiated prior to the COVID-19 pandemic as a new way to combat emerging viral diseases and potentially as-yet unknown infections (referred to as "Disease X"). The platform has been successfully tested in the laboratory against variants of the SARS-CoV-2 virus that causes COVID-19 as they have emerged. Detailed subsequent work suggests that its use could be expanded to certain cancers, and has provided evidence that the CBR platform is applicable in principle to almost any known form of virus. The Company has successfully demonstrated in vitro that immune cells programmed with a CBR-based construct against SARS-CoV-2 selectively consume a live synthetic virus. Importantly, the function of the CBR construct was not affected by known mutations of the spike protein that endows the virus with the ability to infect cells. The Company has now begun in vivo tests with a partner in a biosafety level 3 ("BSL3") facility to demonstrate that CBR could be used against infectious replicating SARS-CoV-2 virus. Work also continues in relation to CBR's applicability to certain cancers. In recent months, further progress has been made. As announced in January 2023, our scientists have identified a target protein that can be incorporated into a single multipurpose CBR-based therapeutic capable of treating multiple viruses that belong to different viral families, instead of having to make a separate CBR construct for every virus. Among them are Dengue, Ebola, Marburg, Zika and Chikungunya. These viruses are among the most dangerous to humans, causing serious and often fatal diseases, and for which few effective treatment options exist. The Company's technology utilises synthetic biology and artificial intelligence approaches to advance medicine to protect society from future pandemics that may challenge the global economy, health, and national defence. When fully developed, we would be able to create front-line treatments that may prevent the development of the next pandemic. Moreover, these new therapeutic tools can be used to protect against bio-terrorism, potentially rendering a universe of viral bio-weapons ineffective. We continue to believe that this platform has the capacity to be extremely valuable. New Custom R&D Facility In July 2022, we officially opened our new custom-designed laboratory in the Mink Building in the Manhattanville area of New York City, a state-of-the-art research facility of some 10,000 square feet that includes two clean rooms for cell therapy manufacturing. We can now manufacture cells in-house, accelerating and simplifying the commercialisation of our cell therapy product candidates. The facility is near to world-class educational institutions that play a leading role in the rich local life sciences ecosystem, including Columbia University and City College. New Appointments We made two important appointments during the year: Dr Koen van Besien was appointed as our Medical Director, and we also welcomed a Director of Quality, Stuart Tinch. Dr van Besien, who is Chief of the Division of Hematology and head of the Wesley Center for Immunotherapy at University Hospitals Seidman Cancer Center, has been associated with the Company since its founding as a member of our Scientific Advisory Board. Now that we are moving closer to clinical trials, he has stepped up to a position in which he is engaged in refining the protocol for those trials and their implementation. Stuart Tinch brings over seven years of Good Manufacturing Practice ("GMP") expertise to Hemogenyx Pharmaceuticals. He will be instrumental in creating a culture and system of quality to ensure that the Company's therapies are held to the standards of current GMP regulations. Financial Results Overall, the Group made a loss of £3,986,982 (2021: £5,108,310 loss) during the period under review. The increased operating loss of £3,997,548 (31 December 2021: £2,702,754) marks the increasing volume of work and need to engage external service providers as our assets are taken towards the crucial clinical trial stage of their development. It only remains for me to thank our CEO Dr Vladislav Sandler and his scientific team for their excellent and highly productive work under a tight budget, as well as my fellow directors, and to look forward with confidence to the achievement of important milestones during the present financial year. Prof Sir Marc Feldmann AC, FRS MB BS, PhD, FRCP, FRCPath, FAA, F Med Sci Chairman 27 April 2023 Click on, or paste the following link into your web browser, to view the full announcement. 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 source version on accesswire.com: