First Gene Therapy Clinical Trial to Address Genetic Blindness

PharmaNewsIntelligence | March 11, 2020

Oregon Health & Science University (OHSU) held the first-ever gene therapy clinical trial, BRILLIANCE, to address blindness-causing gene mutation, LCA10, according to a recent press release The clinical trial, sponsored by Allergan plc and Editas Medicine, is one of 14 different clinical trials investigating new genetic treatments for ophthalmic conditions and nearly 50 vision-related clinical trials. The procedure uses gene-editing tool CRISPR to edit human genes, also known as in vivo gene editing. Previously, gene-editing methods have edited genetic material after it was removed from the human body. Being able to edit genes inside the human body is incredibly profound,” said Mark Pennesi, MD, PhD, the Kenneth C. Swan, associate professor of ophthalmology in the OHDU School of Medicine and chief of the OSHU Casey Eye Institute’s Paul H. Casey Ophthalmic Genetics Division.

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Vaccines are among the best tools we have to protect people’s health. And yet, public confidence in vaccines is under threat as never before. So what can we do to stop this dangerous trend and rebuild public trust? A new global policy approach is needed, one that takes public concerns seriously, builds more time for dialogue and patient consultation into increasingly stressed health services, supports public health officials and professionals to build their own confidence and have the answers they need when confronted with questions and concerns. At the same time, we must not shy away from confronting those who undermine the facts behind vaccine safety, real risks and benefits. Otherwise, there is a serious risk that global immunization goals could be undermined and public health compromised.


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insitro Appoints Philip Tagari, Industry-Leading Scientist and Drug Hunter, as Chief Scientific Officer

insitro | December 02, 2022

insitro, a machine learning-powered drug discovery and development company announced that Philip Tagari has been appointed as chief scientific officer. Tagari joins insitro following a 24-year career at Amgen, where he has led the organization's research platforms for over a decade as vice president, research - therapeutic discovery. “I am deeply excited to partner with Philip on insitro’s journey to build a transformative biology platform for the efficient discovery and development of effective medicines for patients in need. Philip brings unparalleled experience in drug development, including building and utilizing cutting-edge platforms, profound scientific expertise across therapeutic areas and modalities, and most importantly, the humility and courage to transcend the drug discovery status quo and do things differently.” Daphne Koller, Ph.D., founder and CEO of insitro Tagari joins a cross-functional team of experts in biology and technology at insitro, part of a uniquely collaborative culture that spans the boundaries of both fields. “Philip’s inspiring track record of fusing science and technology to build innovative platforms and leading therapeutic programs from bench to approval will guide us in the next step of insitro’s journey, as we deploy our expertise to identify exciting new targets and design therapeutic molecules,” said Koller. “insitro is completely redefining our understanding of disease biology and developing novel therapeutic strategies for a variety of grievous illnesses. Using machine learning on genetics and multi-modal phenotypic data at scale from human cohorts and cellular systems, their cutting edge laboratories are rapidly generating differentiated approaches to currently intractable unmet needs,” Tagari said. “I’m thrilled to partner with Daphne and insitro’s world-class scientists and technologists in achieving a patient-driven vision brought to life in a different kind of drug company.” Tagari joins insitro in early 2023 from Amgen, where he has held a variety of roles since 1998, and served as vice president, research - therapeutic discovery, since 2012. During his time at Amgen, Tagari built and led a global organization of over 600 scientists that delivered numerous experimental and marketed therapies in neurology, inflammation, cardiovascular disease and oncology. Throughout his career, which includes a decade at Merck and research positions at McGill University and Oxford University, Tagari made significant contributions toward multiple first-in-class, disease-modifying, life-changing therapies including Lumakras, Repatha, Singulair, Aimovig, Evenity, Tarlatamab, Acapatamab, Efavaleukin alfa, and AMG 133. With more than 30 years of research experience, he brings expertise across neurobiology, metabolic disease, hematology/oncology, immunology and inflammation, cell and molecular biology, antibody discovery and biologics engineering, medicinal, peptide and oligonucleotide chemistry, analytical chemistry and biochemistry, structural biology, pharmacokinetics, safety pharmacology, laboratory automation and information technologies. Tagari has published or contributed to more than 70 peer-reviewed publications. “I had the privilege of working with Philip for more than 15 years, first at Merck, and thereafter at Amgen, and can say unequivocally that he ranks among the most accomplished, and most innovative, scientific leaders in the industry,” said Roger M. Perlmutter, M.D., Ph.D., currently president, chief executive officer, and chairman of Eikon Therapeutics, Inc., (formerly EVP R&D both at Amgen and at Merck), and is a member of insitro’s Board of Directors. “With Philip as chief scientific officer and partner to Daphne, insitro will be well positioned to ensure that insights from its machine learning-enabled discovery platform contribute to the development of important new medicines that will make a meaningful difference for patients.” About insitro insitro is a data-driven drug discovery and development company using machine learning and data at scale to transform the way that drugs are discovered and developed for patients. insitro is developing predictive machine learning models to discover underlying biologic states based on human cohort data and in-house generated cellular data at scale. These predictive models are being brought to bear on key bottlenecks in pharmaceutical R&D to advance novel targets and patient biomarkers, design therapeutics and inform clinical strategy. insitro is advancing a wholly owned and partnered pipeline of biologic insights and molecules in metabolism and neuroscience. Since formation in mid 2018, insitro has raised over $700 million from top tech, biotech, and crossover investors, and from collaborations with pharmaceutical partners.

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Everest Medicines Appoints Rogers Yongqing Luo as Chief Executive Officer to Lead Next Stage of Company Growth

Everest Medicines | September 19, 2022

Everest Medicines a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products to address critical unmet needs in Asia Pacific markets, announced that it has appointed Rogers Yongqing Luo as Chief Executive Officer of the Company, effective immediately. Mr. Luo will also serve as an Executive Director on Everest's Board of Directors. Mr. Luo brings to Everest more than 25 years of experience in the healthcare industry with an extensive leadership track record across clinical development, regulatory affairs and commercialization. Previously, Mr. Luo served as President and General Manager, Greater China at Brii Biosciences and CEO of TSB Therapeutics where he spearheaded the rapid registration approval, GMP certification and successful commercialization of the amubarvimab/romlusevimab combination, the first COVID-19 neutralizing antibody treatment in China. Prior to that, he was Global Vice President and China General Manager at Gilead Sciences where he led the clinical development, regulatory approval and successful commercial launch of eight innovative therapeutics, and efforts that rapidly expanded patient access across China. Mr. Luo previously oversaw the successful launch of several high-profile medicines. He managed sales, marketing, government affairs and market access functions for various leading multinational pharmaceutical companies including Roche and Novartis, and worked two years at Novartis headquarter in Switzerland. "As Everest Medicines continues to evolve our business strategy and expand our novel product pipeline at a rapid pace, we are thrilled to have Rogers join the Company to lead Everest through its next phase of growth in becoming a fully-integrated biopharma company in China. With his robust background as a top executive at both biotech startup and large multinational pharma companies, Rogers brings to Everest a unique set of expertise including comprehensive scientific insight, proven outstanding leadership and excellent commercialization capabilities in patient access, and a demonstrated history of successfully advancing early- and late-stage innovative pipeline to commercial launch in Greater China. In particular, Rogers' leadership will strengthen Everest's new product development and commercial capabilities as we progress toward potential approvals of our late-stage anchor product candidates, including Nefecon, Xerava, etrasimod and mRNA vaccines. We look forward to leveraging Rogers' leadership and industry expertise to generate long-term growth and success across the business." Mr. Wei Fu, Chairman of Everest Medicines and Chief Executive Officer of CBC Group "I'm very excited to join Everest and I look forward to working with this talented, world-class team of scientists and industry professionals to further the Company's industry leadership. We will continue to advance our late-stage pipeline towards commercialization and with our strong balance sheet, we aim to execute synergistic business development deals to accelerate our path to become an integrated biopharma," said Rogers Yongqing Luo, Chief Executive Officer of Everest Medicines. "We will continue to invest into our discovery platform to build an innovative therapeutic pipeline for the benefit of patients with unmet needs in Greater China and other Asia Pacific markets, and to create sustained, long-term value for our shareholders." About Everest Medicines Everest Medicines is a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products that address critical unmet medical needs for patients in Asian markets. The management team of Everest Medicines has deep expertise and an extensive track record of high-quality clinical development, regulatory affairs, CMC, business development and operations both in China and with leading global pharmaceutical companies. Everest Medicines has built a portfolio of potentially global first-in-class or best-in-class molecules, many of which are in late-stage clinical development. The Company's therapeutic areas of interest include oncology, autoimmune disorders, cardio-renal diseases and infectious diseases.

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Kubota Vision Announces Collaboration Agreement with Leading Diabetes Center

PBOS | September 30, 2022

Kubota Vision Inc. a clinical-stage specialty ophthalmology company and a wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. announced today that the Company and Joslin Diabetes Center have entered into a material transfer and collaboration agreement for our Patient Based Ophthalmology Suite in-home optical coherence tomography device. Joslin Diabetes Center is conducting two clinical studies to evaluate the ability of PBOS to identify cases of diabetic macular edema that may need treatment compared to a commercially-available OCT device. The studies will be led by Dr. Paolo S. Silva at Joslin Diabetes Center. Dr. Silva is the Co-Chief of Telemedicine at the Beetham Eye Institute of the Joslin Diabetes Center. His work is focused on innovative and investigative work in a field at the intersection of clinical care and technology with the hope of providing an ideal model for the delivery of evidence-based, highly effective, and efficient diabetes eye care to the population that needs it the most. Joslin Diabetes Center, which is affiliated with Harvard Medical School, is a one-of-a-kind institution on the front lines of the world epidemic of diabetes — leading the battle to conquer diabetes in all of its forms through cutting-edge research and innovative approaches to clinical care and education. Dr. Silva stated, “Through these studies, we will determine if home-based in-home optical coherence tomography is useful in the evaluation of diabetic macular edema and how we monitor patients at risk for losing vision from the condition.” “Diabetic retinopathy is one of the most common diseases in the world. The use of our technology brings us great excitement as we continue to focus on the development of medical devices and explore further possibilities.” Ryo Kubota, MD, PhD, Chairman, President, and CEO of Kubota Vision Inc. About PBOS PBOS is a low cost, home-based, ophthalmic self-monitoring OCT device. This small handheld device addresses needs in mobile Health applications for self-monitoring of retina health by patients, in the home and in remote field locations. The PBOS aims to improve ophthalmic treatment outcomes in patients diagnosed with and treated for wet age-related macular degeneration, diabetic macular edema and other neovascular retinal diseases. PBOS is being designed to detect nascent disease progression and support patient re-treatment prior to irreversible vision loss due to disease progression. Key features are low cost and a patient-friendly design, to be used directly by patients at home. PBOS is being designed to capture changes in retinal anatomy. Network connectivity and cloud-based technologies are used to alert the patients and their physicians of disease progression and re-treatment needs. About Kubota Vision Inc. Kubota Vision Inc. is a wholly owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. Kubota Pharmaceutical group’s pipeline includes a wearable device for myopia control using Kubota GlassTM technology and a handheld OCT device for the monitoring of neovascular retinal diseases, to be used directly by patients.

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Cambrex to Acquire Snapdragon Chemistry, a Leader in Continuous Flow API Development Services

Cambrex | November 22, 2022

Cambrex, a leading global contract development and manufacturing organization providing drug substance, drug product, and analytical services across the entire drug lifecycle, today announced that it has entered into a definitive agreement to acquire Snapdragon Chemistry, a leading US-based provider of chemical process development services to a broad range of emerging and established biopharma customers. Snapdragon specializes in active pharmaceutical ingredient batch and continuous flow process development, utilizing state-of-the-art automation technology and proprietary equipment to solve complex process and analytical development challenges. The team of scientists and engineers apply deep process understanding afforded by data-rich experimentation to design and rapidly execute efficient GMP and non-GMP manufacturing processes. With R&D and manufacturing headquartered in Waltham, Massachusetts, Snapdragon's 74 employees come with strong ties to the local scientific community, with 31 PhD scientists on staff. "The acquisition of Snapdragon will accelerate our growth in the area of continuous flow process development and manufacturing, complementing our recent organic investments in our High Point, North Carolina facility. With R&D and manufacturing capabilities in the heart of Boston's biopharma hub, Snapdragon will continue to focus on solving their customers' most difficult process development challenges." Tom Loewald, CEO of Cambrex "We are excited to be joining Cambrex, a company with over 40 years of drug substance development and manufacturing expertise," said Matt Bio, CEO of Snapdragon. "Partnering our best-in-class process development capabilities with Cambrex's larger scale manufacturing facilities in North America and Europe is a natural fit, both for our employees and our customers." Snapdragon recently opened its second facility, a new 51,000-square-foot facility to manufacture experimental pharmaceutical products for human clinical trials. The new facility expanded the company's capacity for supplying clinical intermediates and drug substances. The transaction is expected to close following the completion of customary regulatory approvals. This will be Cambrex's second acquisition within a year along with Q1 Scientific, consistent with its strategy to expand its portfolio of specialized solutions for pharmaceutical development and manufacturing. About Cambrex Cambrex is a leading global contract development and manufacturing organization that provides drug substance, drug product, and analytical services across the entire drug lifecycle. With over 40 years of experience and a growing team of over 2,300 experts servicing global clients from North America and Europe, Cambrex is a trusted partner in branded and generic markets for API and finished dosage form development and manufacturing. Cambrex offers a range of specialized drug substance technologies and capabilities, including biocatalysis, continuous flow, controlled substances, solid-state science, material characterization, and highly potent APIs. In addition, Cambrex can support conventional dosage forms, including oral solids, semi-solids, and liquids, and has the expertise to manufacture specialty dosage forms such as modified-release, fixed-dose combination, pediatric, bi-layer tablets, stick packs, topicals, controlled substances, sterile, and non-sterile ointments.

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Spotlight

Vaccines are among the best tools we have to protect people’s health. And yet, public confidence in vaccines is under threat as never before. So what can we do to stop this dangerous trend and rebuild public trust? A new global policy approach is needed, one that takes public concerns seriously, builds more time for dialogue and patient consultation into increasingly stressed health services, supports public health officials and professionals to build their own confidence and have the answers they need when confronted with questions and concerns. At the same time, we must not shy away from confronting those who undermine the facts behind vaccine safety, real risks and benefits. Otherwise, there is a serious risk that global immunization goals could be undermined and public health compromised.

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