Immune master regulator orchestrates responses to parasite infection

Medical Xpress | January 31, 2019

A new study has identified the master regulator that maintains a healthy gut and limits damage by parasitic whipworms. Researchers from the Wellcome Sanger Institute and collaborators have revealed that the interleukin 10 receptor (IL-10R) is critical to prevent uncontrolled whipworm infection in mice and a damaging immune response in the gut. The study, published today (31 January) in PLOS Pathogens, helps understand the signaling mechanism that maintains a balance between the host, whipworms and gut bacteria. Unraveling this signaling mechanism will help scientists understand the immune response to other parasites and will shed light on pathways that could be involved in the control of other diseases such as inflammatory bowel disease and allergies. The gut is home to millions of bacteria, known as the microbiota, and also to parasites such as whipworms. The human whipworm - Trichuris trichiura - infects approximately 500 million people globally, causing the neglected tropical disease Trichuriasis, and has evolved over millennia to infect the intestines and reproduce there.

Spotlight

Young patients and their caregivers who enter Children’s Mercy Adele Hall are greeted by displays of colorful characters mounted from the ceiling and walls, whimsical designs and vibrant furnishings. But there’s much more to this facility than its cheerful decor. Children’s Mercy Hospital is consistently ranked among the leading children’s hospitals in the nation by multiple sources including U.S. News & World Report. For more than 120 years, the 367-bed hospital has provided comprehensive care for children in Kansas City, Missouri and beyond.

Spotlight

Young patients and their caregivers who enter Children’s Mercy Adele Hall are greeted by displays of colorful characters mounted from the ceiling and walls, whimsical designs and vibrant furnishings. But there’s much more to this facility than its cheerful decor. Children’s Mercy Hospital is consistently ranked among the leading children’s hospitals in the nation by multiple sources including U.S. News & World Report. For more than 120 years, the 367-bed hospital has provided comprehensive care for children in Kansas City, Missouri and beyond.

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CELL AND GENE THERAPY

Kriya Acquires Redpin Therapeutics, Adding Neurology Pipeline to Gene Therapy Portfolio

Kriya Therapeutics | November 17, 2022

Kriya Therapeutics, Inc., a fully integrated gene therapy company advancing a broad portfolio of innovative therapeutics announced the acquisition of Redpin Therapeutics, Inc., a privately held biotechnology company developing regulatable gene therapies for intractable diseases of the nervous system. The acquisition serves as the foundation for Kriya’s neurology therapeutic area portfolio, with two lead gene therapy programs focused on epilepsy and trigeminal neuralgia. Redpin’s proprietary chemogenetics platform can selectively activate or silence disease-causing neurons, while leaving normal functioning cells unaffected. Currently available treatments for neurological disorders typically rely on systemically administered drugs or surgical interventions to address local neuron dysfunction. However, these approaches can have limited efficacy and off-target side effects. In contrast, chemogenetics leverages gene products that are selectively responsive to a chosen small molecule. Redpin’s technology utilizes gene therapy to express engineered ion channels that are responsive to modulation by the FDA-approved anti-smoking drug varenicline to either inhibit specific overactive neurons or stimulate underactive ones. “Redpin’s innovative chemogenetics platform has the potential to transform the lives of patients suffering from intractable neurological conditions. We are looking forward to integrating Redpin’s platform and pipeline into Kriya’s gene therapy engine as we advance our mission to develop life-changing gene therapies that can address diseases affecting millions of patients around the world.” Shankar Ramaswamy, M.D., Co-Founder and Chief Executive Officer of Kriya “Redpin has a built a breakthrough, validated chemogenetics approach that could have significant potential in the targeted treatment of neurological disorders,” said Dr. Elma Hawkins, Redpin Therapeutics’ President, Chief Executive Officer and Co-founder. “Kriya Therapeutics is the perfect company to take this leading ion channel-based platform forward and deliver an effective treatment option for patients. We look forward to working with the Kriya team to progress these unique targeted therapies to patients in need as rapidly as possible.” Redpin’s investors include 4BIO Capital, Arkin Bio Ventures, Takeda Ventures Inc, New York Ventures and Alexandria Venture Investments. About Epilepsy Epilepsy is a brain disorder that causes recurrent spontaneous seizures. Approximately 3.4 million people in the U.S. suffer from active epilepsy, according to the U.S. Centers for Disease Control and Prevention. Current treatments for epilepsy include medication, neurostimulation and surgery to remove sections of the affected part of the brain. However between 20% to 40% of patients with newly diagnosed epilepsy will become refractory to treatment, and will continue to suffer seizures. Uncontrolled epilepsy can lead to sudden unexpected death in epilepsy (SUDEP). About Trigeminal Neuralgia Trigeminal neuralgia also known as Tic Douloureux, is a severe and chronic disorder that affects the trigeminal, or fifth cranial, nerve. TN patients experience progressive intense stabbing facial pain attacks that can become more severe and last longer over time. Approximately 10,000 to 15,000 new cases of TN occur each year in the U.S., according to the National Organization for Rare Disorders. While currently available treatments include anti-seizure medications and surgical procedures, a significant proportion of patients are refractory to these approaches. About Kriya Therapeutics Kriya is a fully integrated company pioneering novel technologies and therapeutics in gene therapy. The company aims to revolutionize how gene therapies are designed, developed, and manufactured, improving speed to market and delivering significant reductions in cost. The company is advancing a deep and diversified pipeline of innovative gene therapies in multiple therapeutic area divisions, with current pipeline programs in metabolic disorders, neurology and ophthalmology. Kriya is backed by leading life sciences and technology investors, and has core operations in Silicon Valley, California and Research Triangle Park, North Carolina. About Redpin Based in New York City, Redpin Therapeutics is a privately held, preclinical stage gene therapy company developing a proprietary chemogenetics platform for targeted cell therapies to address currently intractable diseases of the central nervous system. Using a powerful and innovative technology that inverts traditional drug development by using gene therapy to target an engineered receptor to any cell type responsible for disease and modulating its function with an already-approved drug. This has the potential to deliver effective treatments for a wide range of currently intractable neurological and psychiatric diseases and disorders. Redpin has a worldwide exclusive license from the Howard Hughes Medical Institute for the therapeutic use of this technology, and the company is working with urgency to apply it to advance important medicines for patients in desperate need of effective therapies.

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INDUSTRIAL IMPACT

Naveris Inc. Raises $51 Million to Advance Commercialization of NavDx

Naveris, Inc. | September 20, 2022

Naveris, Inc., a commercial-stage life sciences company dedicated to improving patient care through earlier detection of viral-driven cancers, today announced a $33.4 million expansion of its Series A financing, bringing the total investment in Naveris to $51 million. The financing was led by Gurnet Point Capital, joined by TechU Ventures and BrightEdge, the impact and venture capital arm of the American Cancer Society. Naveris’ blood tests for earlier cancer detection use proprietary patented DNA fragmentomics technology to distinguish between viral DNA arising from cancers versus infection. Proceeds from this financing will be used to advance commercialization of NavDx, Naveris’ flagship blood test for the early detection of cancers caused by the human papillomavirus (HPV), and to generate the clinical data needed to expand into other cancer types and indications. “We are delighted to have the continued support of Gurnet Point Capital and TechU Ventures, and to welcome Alice Pomponio and the rest of the BrightEdge team, who collectively bring a deep understanding of both the oncology space and commercial-stage businesses. We founded Naveris on the belief that advances in molecular diagnostics will play a vital role in improving cancer outcomes. This new investment underscores NavDx’s commercial success and potential, and our experienced team’s ability to transform this vision into a reality for patients and oncologists.” Piyush B. Gupta, Ph.D., Naveris founder and Chief Executive Officer "Since our initial investment in 2020, we have been impressed by the Naveris team's vision and their ability to commercially execute on a differentiated strategy in the rapidly developing field of blood-based cancer detection,” said Travis Wilson, Partner at Gurnet Point Capital. “We are pleased to be providing additional capital to ensure that their transformative technology for early cancer detection will benefit as many patients as possible." “We could not be more excited to back Piyush and his team as Naveris works to increase access to diagnose, detect, and monitor viral cancers” said Alice Pomponio, BrightEdge Managing Director. “We understand the burden of HPV related cancers on patients. And we seek to build on the years of research and advocacy of the American Cancer Society to support the next generation of mission aligned companies reducing patient burden.” Along with Naveris' commercial activities, more than 25 medical institutions have partnered with the company to conduct clinical studies and trials utilizing its technology. The company has also partnered with biotechnology companies developing immunotherapies for HPV-related cancers. About Naveris, Inc. Founded in 2017, Naveris is a privately held biotechnology company with facilities in Massachusetts and North Carolina. The company’s mission is to improve outcomes for the millions of people at risk of developing viral cancers through novel molecular diagnostics that enable earlier cancer detection. Naveris’ clinical laboratory is certified for high complexity testing under CLIA, and is accredited by the College of American Pathologists and the New York State Department of Health Wadsworth Center. About Gurnet Point Capital Gurnet Point Capital is a leading healthcare fund that invests in de-risked life sciences companies. Gurnet Point primarily focuses on businesses that have high growth potential in the late product development and commercialization stages of their evolution. These companies become partners not just because of their capacity to generate economic value, but also because of their potential to deliver social impact. Gurnet Point’s team of highly experienced industry executives work closely with its portfolio companies, with an active approach driving operational transformation and outsized returns.

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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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