Consejo Superior de Investigaciones Científicas

SAN DIEGO and RESEARCH TRIANGLE PARK, N.C. and SEATTLE, May 01, 2024 (GLOBE NEWSWIRE) -- Creyon Bio, Inc. ("Creyon"), a drug development company that engineers Oligonucleotide-Based Medicines (OBMs) with industry leading efficiency creating novel, best-in-class gene-centric medicines to treat rare and common diseases, and Cajal Neuroscience, ("Cajal"), a biotechnology company integrating disease-focused genetics and human data with state-of-the-art experimental capabilities to discover novel targets and therapeutics for neurodegeneration, today announced a partnership to develop novel OBMs for neurodegenerative diseases. Under the collaboration, the companies are developing antisense oligonucleotides (ASOs) for multiple neurodegenerative diseases. Cajal is responsible for nominating targets and mechanisms of action based on therapeutic insights from their discovery platform. Creyon is responsible for engineering oligonucleotide therapeutic candidates leveraging the powerful Creyon™ Platform, the first and only OBM platform designed to engineer for safety first, creating lead compounds with optimal pharmacological properties designed to minimize side effects. Cajal will complete validation and IND-enabling studies of the lead therapeutic candidates. Creyon and Cajal have initiated and successfully advanced multiple programs, including one program nearing IND-enabling studies. Chris Hart, Ph.D., Co-Founder, Chief Executive Officer, and President of Creyon Bio, said, “We are well positioned to accelerate the development of much needed therapies for patients with common neurodegenerative disorders by combining Cajal's target identification capabilities with our platform designed to engineer safe, effective, and ultimately best-in-class compounds. This synergy between innovative target discovery capabilities and expert engineering of oligonucleotide-based medicines illustrates the strategic value of collaboration to advance new treatments for a myriad of diseases where there is high unmet need.” “Oligonucleotides offer a promising modality for treating diseases of the central nervous system, including neurodegenerative disease. Using our human genetics platform we have identified novel targets that are well suited to oligonucleotide therapeutics. Creyon’s expertise in developing ASOs has been instrumental in our ability to prosecute on the targets we have identified. We look forward to continuing the collaboration as new targets continue to emerge from our platform,” said Ian Peikon, Ph.D., & Andrew Dervan, M.D., Co-Founders & Co-Chief Executive Officers of Cajal Neuroscience. About Creyon Bio, Inc.Creyon Bio is a clinical stage biotechnology company developing best-in-class precision medicines with industry-leading efficiency through molecular engineering of Oligonucleotide-Based Medicines (OBMs). This includes ASOs, siRNAs, and aptamers. The company is changing how we create novel OBMs, transforming the process from drug discovery to drug engineering. Creyon has built the first and only platform capable of engineering for safety first, translating new target discoveries to OBMs with optimal pharmacological properties. Coupled with our aptamer-based delivery technologies, Creyon Bio is unlocking the full potential of OBMs for common and rare diseases alike. To learn more, visit creyonbio.com. About Cajal NeuroscienceCajal Neuroscience is a biotechnology company committed to developing therapeutics for neurodegenerative diseases. Cajal is focused on identifying genetic modifiers of disease progression and translating them into novel therapeutics for neurodegeneration. Cajal leverages an integrated platform that layers genetics with human multi-omics, longitudinal clinical data, and high-throughput biology. Cajal has built a pipeline of therapeutics based on genetic discoveries into the molecular drivers of disease progression, with an initial focus on Parkinson’s and Alzheimer’s disease. Cajal is headquartered in Seattle, WA. Creyon Bio Media ContactJon YuCreyonPR@westwicke.com Cajal Neuroscience Media Contactmedia@cajalneuro.com

A new study suggests that cognitive loss in patients with brain metastases may be due to the interference that cancer produces in neuronal circuits. When cancer spreads in the brain, it changes the brain's chemistry, interfering with the communication between neurons. This is a very different hypothesis from the one previously accepted, with implications for the diagnosis and treatment of brain metastases. The authors have used artificial intelligence to show that metastasis alters brain activity. Nearly half of all patients with brain metastasis experience cognitive impairment. Until now, it was thought that this was due to the physical presence of the tumour pressing on neural tissue. But this 'mass effect' hypothesis is flawed because there is often no relationship between the size of the tumour and its cognitive impact. Small tumours can cause significant changes, and large tumours can produce mild effects. Why is this? The explanation may lie in the fact that brain metastasis hacks the brain's activity, a study featured on Cancer Cell's cover shows for the first time. The authors, from the Spanish National Research Council (CSIC) and the Spanish National Cancer Research Centre (CNIO), have discovered that when cancer spreads (metastasises) in the brain, it changes the brain's chemistry and disrupts neuronal communication -- neurons communicate through electrical impulses generated and transmitted by biochemical changes in the cells and their surroundings. In this study, the laboratories of Manuel Valiente (CNIO) and Liset Menéndez de La Prida (Cajal Institute CSIC) have collaborated within the EU-funded NanoBRIGHT project, aimed at developing new technologies for the study of the brain, and with the participation of other funding agencies such as MICINN, AECC, ERC, NIH and EMBO. Demonstration with artificial intelligence The researchers measured the electrical activity of the brains of mice with and without metastases and observed that the electrophysiological recordings of the two groups of animals with cancer were different from each other. To be sure that this difference was attributable to metastases, they turned to artificial intelligence. They trained an automatic algorithm with numerous electrophysiological recordings, and the model was indeed able to identify the presence of metastases. The system was even able to distinguish metastases from different primary tumours -- skin, lung and breast cancer. These results show that metastasis does indeed affect the brain's electrical activity in a specific way, leaving clear and recognizable signatures. For the authors, the study represents a "paradigm shift" in the basic understanding of the development of brain metastases and has implications for the prevention, early diagnosis and treatment of this pathology. On the trail of a drug against neurocognitive effects In addition to recording changes in brain electrical activity in the presence of metastasis, the researchers have begun to explore the biochemical changes that might explain this alteration. By analysing the genes expressed in the affected tissues, they have identified a molecule, EGR1, that may play an important role in this process. This finding opens up the possibility of designing a drug to prevent or alleviate the neurocognitive effects of brain metastasis. As Manuel Valiente, head of the CNIO's Brain Metastasis Group explains, "our multidisciplinary study challenges the hitherto accepted assumption that neurological dysfunction, which is very common in patients with brain metastasis, is due solely to the mass effect of the tumour. We suggest that these symptoms are a consequence of changes in brain activity resulting from tumour-induced biochemical and molecular alterations. This is a paradigm shift that could have important implications for diagnosis and therapeutic strategies." Liset Menéndez de la Prida, director of the Laboratory of Neural Circuits the Cajal Institute (CSIC), says: "Using machine learning, we have been able to integrate all the data to create a model that allows us to know whether there is or not metastasis in a brain, just by looking at its electrical activity. This computational approach may even be able to predict subtypes of brain metastases at an early stage. It is a completely pioneering work that opens up an unexplored path." Both authors emphasise the multidisciplinary nature of this complex study that combines neuroscience, oncology and computational analysis, each using a wide range of different techniques. Cognitive study of patients and development of non-invasive techniques The change in focus brought about by this result means that researchers now want to analyse the cognitive status of patients with brain metastasis much more systematically. For Valiente, this is one of the most important next steps. The key to this will be the National Brain Metastasis Network (RENACER) initiated and coordinated by CNIO, which has already served to generate the largest collection of living brain metastasis samples in the world (with prior consent from patients, tissue samples collected during surgical interventions are made available to the international scientific community in the CNIO Biobank), and in which they will now introduce protocols for the neurocognitive assessment of the participating patients. For her part, Liset Menéndez de La Prida will work on integrating the recording of brain activity with the analysis of the molecules involved, "in order to develop new diagnostic probes for brain tumours," she says. This task is in line with the European NanoBRIGHTproject, which aims to develop non-invasive techniques for studying the brain and treating its pathologies, and in which CSIC and CNIO are participating. Another goal is to find drugs that protect the brain from cancer-induced disruptions in neuronal circuits, using the strategies described above. "We will look for molecules involved in metastasis-induced changes in neuronal communication, and evaluate them as possible therapeutic targets," explains Valiente. In addition to the artificial intelligence developed by the CSIC team, they will use the METPlatform technology designed by CNIO to evaluate the potential therapeutic activity of hundreds of compounds simultaneously on brain tissue samples affected by metastasis.

The World Health Organization has secured licensing agreements with Medigen Vaccine Biologics, the University of Chile and the Spanish National Research Council (CSIC) to increase the number of Covid-19 vaccine and assay product options for worldwide use. The licensing agreements fall under WHO’s Covid-19 Technology Access Pool (C-TAP). Medigen is the first private manufacturer, as well as the first vaccine maker, to enter an agreement with C-TAP. The Taiwan-based biotech is licensing its Covid-19 vaccine, MVC COVID-19, and providing staff training to manufacturers. The protein subunit vaccine, which has emergency use authorization in Taiwan, has had three million doses administered in seven countries, according to a WHO press release.