Potential new therapy for Crohn's, colitis identified

Medical Xpress | March 06, 2019

People with inflammatory bowel disease (IBD) live with frequent, miserable episodes of abdominal pain, diarrhea and in severe cases, rectal bleeding. Standard treatments are aimed at directly suppressing inflammation, but many patients find little relief from such an approach. Now, researchers at Washington University School of Medicine in St. Louis have found a compound that may treat IBD without directly targeting inflammation. The compound tamps down the activity of a gene linked to blood clotting. They discovered that the gene was turned on at sites of intestinal inflammation and damage, and blocking its activity reduces IBD symptoms in mice. Notably, the gene is especially active in people with severe disease and in those who don't respond to potent biologic drugs known as TNF blockers that are prescribed to treat severe IBD. The findings, published March 6 in Science Translational Medicine, may be a path to new therapeutic options for people whose IBD can't be controlled effectively with current treatments.

Spotlight

There is growing evidence that telehealth expands access to care, improves health outcomes and healthcare quality, increases patient engagement, and reduces healthcare costs. Yet skeptics are concerned about increased utilization and lack of ROI. This URAC Industry Insight Report offers perspectives from leaders on the front lines of telehealth, who are witnessing the risks and rewards as telehealth adoption accelerates rapidly with the transition from volume to value.

Spotlight

There is growing evidence that telehealth expands access to care, improves health outcomes and healthcare quality, increases patient engagement, and reduces healthcare costs. Yet skeptics are concerned about increased utilization and lack of ROI. This URAC Industry Insight Report offers perspectives from leaders on the front lines of telehealth, who are witnessing the risks and rewards as telehealth adoption accelerates rapidly with the transition from volume to value.

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RESEARCH, INDUSTRY OUTLOOK

BigHat Biosciences Announces Research Collaboration with Merck

BigHat Biosciences, Inc. | November 30, 2022

BigHat Biosciences, Inc., a biotechnology company with a machine learning-guided antibody discovery and development platform, announced a collaboration with Merck, known as MSD outside of the United States and Canada, to apply the company’s technology to design candidates for up to three drug discovery programs. BigHat’s design platform, Milliner, integrates a high-speed characterization with ML technologies to engineer antibodies with more complex functions and better biophysical properties. This approach could help reduce the difficulty of optimizing antibodies and other therapeutic proteins. Under the collaboration, BigHat and Merck will collaborate to optimize up to three proteins by leveraging BigHat’s platform to synthesize, express, purify, and characterize molecules. Mark DePristo, co-founder and CEO of BigHat, continued, "We are thrilled to be collaborating with Merck's world-class drug development teams to design safer, more effective antibodies for these important therapeutic programs.” The teams have initiated work on the first program and are looking forward to using the power of the complementary skills sets within each research team to generate high-quality lead antibodies. “We are excited to begin this collaboration to advance next-generation antibody therapeutics to patients,” said Elizabeth Schwarzbach, BigHat’s Chief Business Officer. "This agreement with Merck brings us a major step closer to our goal of 3-5 deep collaborations with leading biopharmas to complement our internal therapeutic pipeline." “This agreement with BigHat expands Merck’s strategy of applying AI/ML across our drug discovery capabilities. We look forward to working with the team to leverage BigHat’s technology and expertise in enabling molecular design of novel biologic candidates.” Juan Alvarez, vice president of Biologics Discovery, Merck Research Laboratories About BigHat Biosciences, Inc. BigHat Biosciences is designing safer, more effective antibody therapies for patients using machine learning and synthetic biology. BigHat integrates a wet lab for high-speed characterization with machine learning technologies to guide the search for better antibodies. They apply these design capabilities to develop new generations of safer and more effective treatments for patients suffering from today’s most challenging diseases. BigHat is a Series B biotech outside San Francisco with a team-oriented, inclusive, and family-friendly culture. BigHat’s broad pipeline of wholly-owned and partnered therapeutic programs span many disparate indications with high unmet need, such as cancer, inflammation, and infectious disease. BigHat has raised over $100M from top investors, including Section 32, a16z, and 8VC.

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MEDTECH

ElevateBio BaseCamp Unveils its LentiPeakTM Lentiviral Vector Platform

ElevateBio, LLC | November 07, 2022

ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering transformative cell and gene therapies, announced the unveiling of ElevateBio BaseCamp’s proprietary LentiPeakTM lentiviral vector platform. LentiPeak is a serum-free, suspension-based, scalable production platform that has demonstrated high volumetric productivity of therapeutically relevant vector yields that meet regulatory guidelines. LentiPeak will enable efficient transition for cell and gene therapies from preclinical stage through clinical development and commercialization with accelerated timelines and reduced manufacturing costs.The LentiPeak platform leverages ElevateBio BaseCamp’s end-to-end process and analytical development expertise and scientific leadership and builds upon the company’s current Good Manufacturing Practice (cGMP) manufacturing capabilities for research, clinical and commercial cell and gene therapies, and regenerative medicines. “As a 30-year veteran of the cell and gene therapy industry, I’ve witnessed the immense advancement of lentiviral vector technology from its infancy to the current landscape with six FDA-approved lentiviral vector-derived cell and gene therapies, The LentiPeak platform is made possible by ElevateBio’s strong scientific foundation, industry-leading expertise, and the investments we’ve made into viral vector production. Our pioneering work continues to be the basis for our continued innovation to drive many more therapies through development to commercialization for our partners.” -Mitchell Finer, Ph.D., Co-founder and President of R&D, ElevateBio ElevateBio BaseCamp’s LentiPeak platform offers a robust and scalable platform process for development and cGMP manufacturing of lentiviral vectors. The LentiPeak platform utilizes a serum-free suspension process capable of producing clinically relevant lentiviral vectors with high titers ranging from 1x10^8 TU/ml to 1x10^9 TU/ml, suitable to meet various therapeutic program needs from preclinical scale to commercial scale supply. The LentiPeak platform can also be seamlessly integrated into cell therapy programs with ElevateBio’s ability to conduct both vector and cell production activities. By leveraging the aligned process unit operations, materials, analytics, and manufacturing expertise at ElevateBio BaseCamp, the LentiPeak platform will simplify the product development and commercialization lifecycle, reducing complexity and ensuring comparability between early-stage and pivotal trials. “Our team of industry-leading experts have developed next-generation platform-based processes that are designed to address lentivirus manufacturing challenges head-on. With our partners’ needs in mind, our proprietary LentiPeak platform has demonstrated leading vector titers with high potency, and process scalability to help our partners de-risk vector production, right from the start, At ElevateBio BaseCamp, we are ever committed to simplifying the complex cell and gene therapy landscape to enable our partners to rapidly progress their therapeutic programs and make these therapies more accessible to patients in need.” -Andrew Sandford, President, ElevateBio BaseCamp About ElevateBio: ElevateBio is a technology-driven company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells (iPSCs), and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. In addition, ElevateBio BaseCamp in Waltham, MA, is a purpose-built facility offering process innovation, process sciences, and current Good Manufacturing Practice (cGMP) manufacturing capabilities. It was designed to support diverse cell and gene therapy products, including autologous, allogeneic, and regenerative medicine cell products and viral vector manufacturing capabilities. Through BaseCamp and its enabling technologies, ElevateBio is focused on growing its collaborations with industry partners while also developing its own portfolio of cellular and genetic medicines. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

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CELL AND GENE THERAPY

Emergent BioSolutions Completes Acquisition of Exclusive Worldwide Rights to TEMBEXA® First FDA-Approved Smallpox Oral Antiviral for All Ages

TEMBEXA and Smallpox | September 27, 2022

Emergent BioSolutions Inc. announced that it has completed its acquisition of exclusive worldwide rights to TEMBEXA® the first oral antiviral approved by the U.S. Food and Drug Administration for all age groups for the treatment of smallpox, from Chimerix. TEMBEXA was approved in June 2021 and is indicated for the treatment of human smallpox disease in adult and pediatric patients, including neonates. The completion of the acquisition follows the satisfaction or waiver by the parties, as applicable, of all closing conditions, including expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act), as amended, and receipt of consent from the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response within the U.S. Department of Health and Human Services, for a sub-contract agreement between Chimerix and Emergent. “The addition of TEMBEXA to our smallpox medical countermeasure franchise, which consists of our smallpox vaccine and therapeutic for smallpox vaccine complications, creates a more comprehensive offering to combat this deadly public health threat. We look forward to supporting the U.S. government’s smallpox preparedness strategy on a broader scale by executing on this BARDA contract.” Paul Williams, SVP government/MCM business at Emergent The 10-year contract valued at up to $680 million, to supply up to 1.7 million treatment courses of tablet and suspension formulations of TEMBEXA® to the U.S. government, was awarded to Chimerix on August 29, 2022. The contract includes an initial product procurement valued at approximately $115 million, with optional future procurement, valued at up to approximately $551 million, exercisable at the sole discretion of BARDA. In addition to product procurement, the contract includes reimbursed post marketing activities of approximately $12 million. Financial Terms Based on the terms of the final BARDA agreement, Emergent is expected to pay Chimerix An upfront payment of $238 million; Potential milestone payments of up to $124 million contingent on the potential exercise by the U.S. government of procurement options following the base period; 15% royalty on gross profit from sales of TEMBEXA outside the U.S.; 20% royalty on gross profit from sales of TEMBEXA in the U.S. that are in excess of the 1.7 million treatment courses as contemplated in the existing BARDA contract; and Up to an additional $12.5 million upon achievement of certain development-based milestones. ABOUT TEMBEXA TEMBEXA is an oral antiviral approved by the FDA in June 2021 for the treatment of human smallpox disease caused by variola virus in adult and pediatric patients, including neonates. TEMBEXA is formulated as 100 mg tablets and 10 mg/mL oral suspension dosed once weekly for two weeks. The oral suspension formulation is particularly important for patients who have difficulty swallowing due to age or medical status. TEMBEXA is not indicated for the treatment of diseases other than human smallpox disease. The effectiveness of TEMBEXA for the treatment of smallpox disease has not been determined in humans because adequate and well-controlled field trials have not been feasible and inducing smallpox disease in humans to study the drug’s efficacy is not ethical. TEMBEXA efficacy may be reduced in immunocompromised patients based on studies in immune deficient animals. TEMBEXA has a BOXED WARNING for increased risk for mortality when used for longer duration. About Smallpox Smallpox is a highly contagious disease caused by the variola virus. Historically, smallpox was one of the deadliest diseases in history with a case fatality rate of approximately 30%. Despite successful eradication of smallpox in the 1970s, there is considerable concern that variola virus could reappear, either through accidental release or as a weapon of bioterrorism. According to the U.S. Centers for Disease Control and Prevention variola virus is ranked in the highest risk category for bioterrorism agents due to its ease of transmission, high mortality rate, and potential to cause public panic and social disruption. Based on a recent report – The Department of Health and Human Services Fiscal Year 2023 Public Health and Social Services Emergency Fund Justification of Estimates for Appropriations Committee – smallpox remains a threat of high concern to both the domestic and international community. About Emergent BioSolutions At Emergent, our mission is to protect and enhance life. For over 20 years, we’ve been at work defending people from things we hope will never happen—so we are prepared just in case they ever do. We provide solutions for complex and urgent public health threats through a portfolio of vaccines and therapeutics that we develop and manufacture for governments and consumers. We also offer a range of integrated contract development and manufacturing services for pharmaceutical and biotechnology customers.

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