Scientists discover new type of immune cells that are essential for forming heart valves

Medical Xpress | February 21, 2019

UCLA researchers have identified for the first time the origin of an immune cell that plays a critical role in the formation of healthy heart valves. The findings could pave the way for new treatments for heart valve disorders, which can be caused by congenital defects, aging or disease. Their study, led by Dr. Atsushi "Austin" Nakano, a UCLA associate professor of molecular, cell and developmental biology and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, was published in the journal Developmental Cell.
Building on previous research by Nakano, which showed that the embryonic heart tube produces blood progenitor cells, the new study found that those cells, in turn, generate specialized immune cells called macrophages. The research also revealed that these heart-derived macrophages are particularly adept at consuming excess tissue, a skill that makes them indispensable to the formation and maintenance of heart valves.

Spotlight

Structured soft materials have the potential to revolutionize regenerative medicine. By imparting shear during processing, it is possible to take the small number of materials approved by medical regulators and create self‐healing polymeric structures. How so‐called fluid gels can facilitate the delivery of cells and proteins and enable additive layer manufacturing of complex biological structures is discussed.

Spotlight

Structured soft materials have the potential to revolutionize regenerative medicine. By imparting shear during processing, it is possible to take the small number of materials approved by medical regulators and create self‐healing polymeric structures. How so‐called fluid gels can facilitate the delivery of cells and proteins and enable additive layer manufacturing of complex biological structures is discussed.

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INDUSTRIAL IMPACT

FogPharma Announces $178 Million Series D Financing to Advance Pipeline of First-in-Class Helicon Polypeptide Therapeutics Targeting

FogPharma | November 22, 2022

FogPharma®, a biopharmaceutical company pioneering a new class of precision medicines that could ultimately prove applicable to the vast majority of therapeutic targets, including those previously considered “undruggable,” today announced a $178 Million Series D financing. The financing round includes new investors ARCH Venture Partners, Milky Way Investments and Fidelity Management & Research Company and existing investors VenBio Partners, Deerfield Management, GV, Cormorant Asset Management, funds and accounts advised by T. Rowe Price Associates, Inc., Invus, Farallon Capital Management, HBM Healthcare Investments, Casdin Capital, and PagsGroup, also participated. Proceeds from the Series D financing will be used to advance and accelerate FogPharma’s growing pipeline of hyperstabilized α-helical polypeptide therapeutics, a proprietary new class of drugs designed to overcome the limitations of today’s precision medicines with broad applicability to the vast majority of disease targets and therapeutic areas. FogPharma’s lead Helicon polypeptide development candidate, FOG-001, a first-and-only-in-class direct TCF-blocking β-catenin inhibitor with potential applicability to significant cancer patient populations, is expected to enter clinical development in mid-2023. In addition, FogPharma is advancing other first-in-class programs against important, biologically validated cancer targets that have remained elusive to other approaches including TEAD, NRAS, Pan-KRAS, ERG and Cyclin E1. “FogPharma continues to make rapid progress on our moonshot mission to achieve universal druggability – a world where no targets are off-limits to medicine. We believe that Helicon polypeptides, a compelling new therapeutic modality, represent the future of precision medicine. We are thrilled by the support of our investors and will continue to build our platform capabilities, product pipeline which aims to address a significant percentage of cancer patient populations, and our phenomenal team across all levels as we aim to create one of the most impactful new classes of drugs in history.” Gregory Verdine, Ph.D., founder, chairman and chief executive officer of FogPharma In connection with the Series D Financing, Rick Klausner, M.D., has been appointed to FogPharma’s board of directors. In addition, Dr. Verdine has been appointed as chairman of the board. “The team at FogPharma is building an unprecedented new therapeutic modality and robust pipeline with the potential to make a meaningful difference in the lives of cancer patients,” said Dr. Klausner. “I am excited to join the board of directors and be part of something special – particularly at this important time as FogPharma continues to impressively scale its science, team, operations and infrastructure, with the goal of advancing its first Helicon polypeptide therapeutic into the clinic.” Dr. Klausner is currently the founder and chief scientist of Altos Labs and founder and chairman of Lyell Immunopharma. Dr. Klausner was founder and director of Juno Therapeutics and founder and director of GRAIL. He is also the chairman of Sonoma Biotherapeutics and co-founder and chairman of Lifemine Therapeutics. Previously, Dr. Klausner served as senior vice president, chief medical officer and chief opportunity officer of Illumina Corporation and as executive director for global health for the Bill and Melinda Gates Foundation. Dr. Klausner was appointed by Presidents Clinton and Bush as the eleventh director of the U.S. National Cancer Institute (NCI) between 1995 and 2001. Dr. Klausner served as chief of the Cell Biology and Metabolism Branch of the National Institute of Child Health and Human Development as well as a past president of the American Society of Clinical Investigation. He has served in senior advisory roles to the U.S., Norwegian, Qatari and Indian governments. About FogPharma’s Universal Druggability Platform and Helicon™ Polypeptide Therapeutics Existing drug classes are limited in both reach and applicability, with more than 80% of known human protein disease targets considered “undruggable” because they are beyond the reach of both antibodies and small molecules. FogPharma’s Helicon peptide drug discovery engine integrates directed evolution, proprietary α-helix conformational hyperstabilization chemistry, highly multiplexed drug optimization technology, artificial intelligence including deep learning and machine learning, structure-based drug discovery, cancer genomics and biology, and multiscale manufacturing to rapidly discover Helicon polypeptide therapeutics. This novel therapeutic modality combines the targeting strength and specificity of antibodies with the broad tissue distribution, intracellular target engagement and oral dosing optionality of small molecules to address the limitations of today’s precision medicines and reach the most difficult targets – achieving universal druggability. About FOG-001 FogPharma’s lead Helicon polypeptide development candidate, FOG-001, a first-and-only-in-class direct TCF-blocking β-catenin inhibitor. Dysregulation of the Wnt/β-catenin signaling pathway has been shown to occur in at least 20% of all human cancers. In the U.S. alone, FOG-001 has the potential to become a new treatment option for >1 million patients suffering from a broad range of intractable cancers. In biochemical and cellular studies, FOG-001 has been shown to potently, precisely and selectively disrupt the interaction of β-catenin with its obligate downstream transcription factor, TCF. Preclinical studies have demonstrated the ability of FOG-001 to cause tumor growth inhibition and regression by disrupting β-catenin-dependent signaling. FOG-001 is the inaugural member of FogPharma’s TCF-Catenix family of direct-acting β-catenin antagonists and combines key features that distinguish it from previously reported Wnt/β-catenin pathway modulators: FOG-001 acts inside the cell, where it directly binds the key oncogenic driver β-catenin; and FOG-001 blocks TCF-β-catenin engagement at the most downstream node in the canonical Wnt pathway, thus abrogating the signal transmission mechanism by which most, if not all, known Wnt pathway mutations are believed to drive oncogenesis. About FogPharma FogPharma is a biopharmaceutical company pioneering the discovery and development of Helicon™ polypeptides. Through this novel therapeutic modality, FogPharma aims to address the limitations of existing drug classes and achieve universal druggability – a world where no targets are off-limits to new medicines. Spun out of Harvard University by pioneering academic scientist and successful biotech company builder Dr. Gregory Verdine, FogPharma is advancing a broad pipeline of Helicon polypeptide therapeutics against important and biologically validated cancer targets that have remained elusive to other approaches, with the goal of providing new targeted treatment options for significant cancer patient populations. FogPharma is headquartered in Cambridge, Mass., and has raised more than $360 million to date from leading life sciences investors.

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CELL AND GENE THERAPY

Q² Solutions Launches Innovative Self-Collection Safety Lab Panel in Collaboration With Tasso

Q² Solutions and IQVIA, Tasso | October 07, 2022

Q² Solutions, a wholly owned subsidiary of IQVIA and a leading global clinical trial laboratory services organization, today announced the launch of the first self-collection safety lab panel for U.S. clinical trial participants by a leading global clinical trial laboratory. Developed in collaboration with Tasso Inc, a leader in clinical-grade blood collection solutions, this unique offering combines industry leading laboratory services, patient focused logistics and cutting-edge, self-collection technology. Participants in clinical trials can now provide a blood specimen for lab testing in the comfort of their own home, without the need to visit an investigator site or have a healthcare professional visit them. The first test panel available through this collaboration is designed to help monitor liver function by measuring the levels of select enzymes and proteins in the blood. The panel includes tests for ALT AST ALP total bilirubin, direct bilirubin, total protein, and albumin. “The global pandemic has accelerated the use of decentralized approaches in clinical trials. We at Q² Solutions are highly motivated to find ways to reduce patient burden related to laboratory testing, and I’m delighted that, through our collaboration with Tasso, we could make the self-collection liver panel a reality. Soon we will launch more than a dozen safety tests, and we are determined to continue to expand our catalog of tests available via the self-collection of blood.” Brian O’Dwyer, CEO of Q² Solutions “We are thrilled to be working with a leader like Q² Solutions on this important initiative to make laboratory testing truly patient-centric,” said Erwin Berthier, PhD, CTO and Co-Founder of Tasso. “Our clinical-grade remote sampling technology enables decentralized laboratory testing without sacrificing sample quality, a key hurdle that home testing has faced in the past. Together, Q² Solutions and Tasso are developing important solutions that make clinical trials better for both patients and sponsors.” This self-collection liver panel was developed with trial participants at the heart of the design. The offering includes easy to use at-home self-collection kits with instructions and videos, a methodology for direct-to-patient delivery, as well as simplified specimen processing and shipping requirements. About Q² Solutions Q² Solutions is a leading global clinical trial laboratory services organization that provides comprehensive testing, project management, supply chain, biorepository, biospecimen, and consent tracking solutions. Leveraging our next generation technologies, we deliver agile and precise services designed to meet the diverse needs of our clients. We provide scientific expertise and innovative solutions for ADME, bioanalytical, genomics, vaccines, and central laboratory services, including flow cytometry, anatomic pathology, immunoassay, molecular, companion diagnostics and translational science, with meticulous regional and global clinical trial implementation support and high-quality data delivery. At Q² Solutions, our work is rooted in research, grounded in collaboration, and guided by our passion to turn the hope of patients and caregivers around the world into the help they need. About IQVIA IQVIA is a leading global provider of advanced analytics, technology solutions, and clinical research services to the life sciences industry. IQVIA creates intelligent connections across all aspects of healthcare through its analytics, transformative technology, big data resources and extensive domain expertise. IQVIA Connected Intelligence™ delivers powerful insights with speed and agility — enabling customers to accelerate the clinical development and commercialization of innovative medical treatments that improve healthcare outcomes for patients. With approximately 82,000 employees, IQVIA conducts operations in more than 100 countries. IQVIA is a global leader in protecting individual patient privacy. The company uses a wide variety of privacy-enhancing technologies and safeguards to protect individual privacy while generating and analyzing information on a scale that helps healthcare stakeholders identify disease patterns and correlate with the precise treatment path and therapy needed for better outcomes. IQVIA’s insights and execution capabilities help biotech, medical device and pharmaceutical companies, medical researchers, government agencies, payers and other healthcare stakeholders tap into a deeper understanding of diseases, human behaviors and scientific advances, in an effort to advance their path toward cures. About Tasso Trusted diagnostics made easy. Tasso is an emerging healthcare technology company that is transforming the traditional blood collection paradigm with a patient-centric approach. The company’s devices enable simple, convenient, and virtually painless blood collection for users. Tasso technology has the power to bring healthcare anywhere, any time. Headquartered in Seattle, Washington, Tasso is privately held and funded by grants, investments, and co-development deals with various industry leaders.

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MEDTECH

Neumora Therapeutics Announces $112 Million Series B Financing

Neumora | October 12, 2022

Neumora Therapeutics, Inc. a clinical-stage biotechnology company pioneering precision medicines for brain diseases through the integration of data science and neuroscience, today announced the close of a $112 million Series B financing. The Series B syndicate includes both new and existing investors, such as Abu Dhabi Growth Fund Altitude Life Science Ventures, Amgen, ARCH Venture Partners, Exor Ventures, F-Prime Capital, Invus, Mubadala Capital, Newpath Partners, Polaris Partners and other undisclosed investors. The funding will support the advancement of a broad clinical and preclinical pipeline of novel precision medicine candidates for neuropsychiatric disorders and neurodegenerative diseases and the development of the company's precision neuroscience platform. “This Series B financing reflects Neumora’s continued progress in building a best-in-class neuroscience company, including assembling a world-class team, scaling up an industry-leading data science and translational neuroscience platform, and advancing a broad and growing pipeline of seven development programs, including our internal discovery efforts and business development activities. This considerable amount of progress in such a short period of time reflects our urgency to address the relative lack of progress and innovation in neuroscience with our data-driven, precision medicine approach.” Paul L. Berns, co-founder, chairman and chief executive officer of Neumora Neumora continues to advance a broad and growing pipeline of clinical and preclinical programs, including a balance of both clinically validated and novel approaches targeting a broad range of underserved neuropsychiatric disorders and neurodegenerative diseases. The company recently completed enrollment in a Phase 2a clinical trial for its most advanced product candidate, NMRA-140, a kappa opioid receptor antagonist in development for the treatment of major depressive disorder. Neumora is also progressing NMRA-511, a clinical-stage vasopressin 1a receptor antagonist in development for neuropsychiatric disorders, and NMRA-266, an M4 muscarinic receptor positive allosteric modulator being advanced toward initiation of Phase 1 development for schizophrenia in 2023. The company’s earlier-stage pipeline includes multiple first-in-class opportunities in preclinical development, including neurodegeneration programs focused on Parkinson’s disease and amyotrophic lateral sclerosis (ALS). “We are pleased to have the support of this leading group of both new and existing investors that share our vision and excitement with our progress to date,” said Joshua Pinto, Ph.D., chief financial officer of Neumora. “With this Series B financing, we have successfully raised approximately $650 million in capital, resulting in a very strong financial position to continue advancing our broad pipeline through multiple near-term value-creating milestones.” Neumora was founded to pioneer a new era of precision neuroscience medicines by using patient enrichment strategies to help potentially improve clinical development success rates. The company’s proprietary approach leverages recent advances in data science and translational neuroscience to cut through the heterogeneity inherent in brain disease. By approaching patient enrichment and clinical development strategies through this lens, Neumora aims to match the right patient populations to the right targeted therapeutics, thereby driving innovation and the potential for clinical development success in this field and, ultimately, providing better therapies to patients. In connection with the completion of the Series B financing, Alaa Halawa, partner and head of U.S. Ventures at Mubadala Capital, joined Neumora’s board of directors. Mr. Halawa said, “We are proud to partner with Neumora in helping advance the company’s efforts to build the precision neuroscience company of the future. The integration of technology, namely AI/ML, and proprietary clinical data is helping us develop a deeper understanding of the complex drivers of brain disease to develop novel treatments for neuropsychiatric disorders and neurodegenerative diseases - we are humbled to be part of their journey.” About Neumora Neumora Therapeutics, Inc. is a clinical-stage biotechnology company pioneering precision medicines for brain diseases through the integration of data science and neuroscience. Neumora is redefining neuroscience research and development with a data-driven precision neuroscience platform to cut through brain disease heterogeneity to match the right patient populations to targeted therapeutics. Neumora is relentless in its commitment to discovering, developing and commercializing targeted therapies for people living with brain diseases.

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