CELL AND GENE THERAPY, DIAGNOSTICS
Businesswire | March 28, 2023
Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that they have entered into a new non-exclusive licensing agreement for the use of CRISPR Therapeutics’ gene editing technology, known as CRISPR/Cas9, to accelerate the development of Vertex’s hypoimmune cell therapies for type 1 diabetes (T1D).
“We have multiple programs in our T1D portfolio including VX-880 and VX-264, which are in the clinic, as well as our hypoimmune program, in preclinical development,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. “Having successfully demonstrated clinical proof of concept in T1D in our VX-880 program, we are excited to deepen our relationship with CRISPR Therapeutics with this agreement, which will allow us to further accelerate our goal of generating fully differentiated, insulin-producing hypoimmune islet cells for T1D.”
“We are pleased to expand our long and successful relationship with Vertex with this collaboration which fully leverages our gene editing platform to develop hypoimmune cell therapies for T1D,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “In parallel, we continue to expand our capabilities in regenerative medicine and advance our existing allogeneic gene-edited cell therapy programs.”
Under this agreement, Vertex will pay CRISPR Therapeutics $100 million up-front for non-exclusive rights to CRISPR Therapeutics’ technology for the development of hypoimmune gene-edited cell therapies for T1D. CRISPR Therapeutics will be eligible for up to an additional $230 million in research and development milestones and receive royalties on any future products resulting from this agreement.
CRISPR and ViaCyte, Inc., which was acquired by Vertex in 2022, will continue to collaborate on their existing gene-edited allogeneic stem cell therapies, using ViaCyte cells, for the treatment of diabetes under the terms of their collaboration. A Phase 1/2 study of VCTX211, an allogeneic, gene-edited, stem cell-derived product candidate for T1D, which originated under the CRISPR Therapeutics and ViaCyte collaboration, has been initiated and is ongoing. CRISPR Therapeutics will not obtain any interest in Vertex’s pre-existing pipeline of T1D products, including VX-880 and VX-264.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, and alpha-1 antitrypsin deficiency.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 13 consecutive years on Science magazine's Top Employers list and one of Fortune’s Best Workplaces in Biotechnology and Pharmaceuticals and Best Workplaces for Women.
INDUSTRIAL IMPACT, MEDICAL
Businesswire | April 24, 2023
Guardant Health, Inc. a leading precision oncology company, is pleased to announce that it will present data from two studies highlighting the performance of its blood-based screening technology to detect early-stage colorectal cancer (CRC), including the acceptance of the ECLIPSE study as a late-breaking abstract, at Digestive Disease Week (DDW) taking place in Chicago, Illinois on May 6-9, 2023.
“Despite multiple screening modalities for colorectal cancer, screening rates remain stubbornly low leaving many cancers undetected. A blood test that can accurately detect CRC represents a significant opportunity to overcome some of the known challenges with patient adherence,” said AmirAli Talasaz, Guardant Health co-CEO. “We are thrilled that DDW has accepted the ECLIPSE study, one of the largest studies of its kind, as a late-breaking abstract, and we look forward to sharing this new data with the healthcare community at this year’s meeting.”
“The need for a convenient, less invasive way to screen for CRC is great, and an accurate blood test can play a critical role in improving screening rates, especially for those reluctant to get screened,” said Daniel Chung, MD, gastroenterologist at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School. “I look forward to presenting at DDW and sharing new data from the ECLIPSE Study on the performance and potential of the Shield blood test to help improve adherence to screening."
About Guardant Health
Guardant Health is a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary tests, vast data sets and advanced analytics. The Guardant Health oncology platform leverages capabilities to drive commercial adoption, improve patient clinical outcomes and lower healthcare costs across all stages of the cancer care continuum. Guardant Health has commercially launched Guardant360®, Guardant360 CDx, Guardant360 TissueNext™, Guardant360 Response™, and GuardantINFINITY™ tests for advanced-stage cancer patients, and Guardant Reveal™ for early-stage cancer patients. The Guardant Health screening portfolio, including the commercially launched Shield™ test, aims to address the needs of individuals eligible for cancer screening.
prnewswire | May 08, 2023
Frost & Sullivan announced that it has awarded Genomenon with the 2023 Global Technology Innovation Leadership Award. Each year, Frost & Sullivan presents this award to the company that has developed a product with innovative features and functionality that is gaining rapid acceptance in the market. The award recognizes the quality of the solution and the customer value enhancements it enables. Frost & Sullivan market analysts determined that Genomenon excels in many of the criteria and best practices identified as critical for the global pharma and clinical diagnostics genomics markets.
Genomenon is a genomic intelligence software company that combines AI-powered Genomic Language Processing (GLP) with expert review by genetic scientists to deliver the most comprehensive knowledgebase of genomic evidence available. Their fit-for-purpose combinatorial approach to identifying and indexing published genomic evidence gives them a unique advantage over other open access and commercial genomic data resources in this space.
Clinical diagnostic labs rely on Genomenon's Mastermind Genomic Search Engine to find critical information needed to diagnose rare genetic diseases and identify the most appropriate patient treatment options. Pharmaceutical companies engaged in the development of rare disease therapeutics partner with Genomenon to gain deep insights into the genetic drivers of disease. The Genomenon scientific team leverages information in the Mastermind knowledgebase to provide the genomic intelligence pharmaceutical companies need to better understand disease mechanisms, more accurately estimate disease prevalence, design clinical trials, and prepare documentation for regulatory approvals.
Pavel Zhebrouski, a best practices research analyst at Frost & Sullivan, observed, "Genomenon differentiates itself in the space by providing a unique combination of AI technology and human expertise. Genomenon empowers rare disease pharmaceutical and biotechnology companies to make informed decisions about promising candidates in their pipeline and help propel novel therapeutics to market. Its commitment to innovation and creativity, scientific rigor, and accuracy, while achieving commercial success, differentiates it from competitors," added Tejashri Khadilkar, Frost & Sullivan industry analyst.
Frost & Sullivan Best Practices awards recognize companies in various regional and global markets for demonstrating outstanding achievement and superior performance in leadership, technological innovation, customer service, and strategic product development. Industry analysts compare market participants and measure performance through in-depth interviews, analyses, and extensive secondary research to identify best practices in the industry.
Genomenon is an AI-driven genomics company focused on making genomic information actionable for patients with rare genetic diseases and cancer. Keeping pace with the ever-evolving body of knowledge within genomics, Genomenon connects current research with patient DNA to accelerate clinical decision-making and pharmaceutical drug discovery and development.