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.
MEDICAL, INDUSTRY OUTLOOK
PRNewswire | May 16, 2023
PhenomeX Inc. the functional cell biology company, announced the launch of Beacon Select™ optofluidic system with Opto® B Discovery workflows for antibody discovery.
The Opto B Discovery workflows, with their cutting-edge technologies and features, enable scientists to find lead candidate antibodies tailored to their specific challenges and changing needs. Combined with the value-matched Beacon Select system, more customers may access these propriety antibody discovery applications, but with the optimum level of features that meet their research and budgetary needs. PhenomeX's optofluidic platform is now a standard to deliver optimal antibodies with speed, as highlighted recently in the high-impact journal Cell, where antibodies against COVID-19 variants were discovered on the Beacon system six months before Omicron emergence.
Beacon Select for Antibody Discovery Workflow Feature
Opto B Discovery workflows on the Beacon Select system enable scientists to accelerate antibody screening and hit generation to find lead candidate antibodies and are ideally suited for small- to mid-sized biopharma and CDMOs/CROs. This contrasts with the flagship four-chip Beacon system enabled for antibody discovery, optimized for the highest screening throughputs demanded by large biopharma and established CDMOs/CROs.
The Beacon Select system enables rapid function-first selection of single B cells with the best characteristics for any antibody application, including antibody therapeutics, diagnostics, and reagents. In addition, the Opto B Discovery workflows offer users highly customizable assays to assess antigen and epitope specificity, species or variant cross-reactivity, and functional properties, all in a single-day workflow. By selecting and recovering sequences of only the most desirable antibodies with favorable attributes, users can reduce overall bioprocessing costs, improve the probability of campaign success, and shorten timelines to lead candidates. When combined with PhenomeX's catalog of best-in-class reagents and consumables for mouse, human, and rabbit B cell repertoires, customers can do all this against even the hardest antigen targets, including G coupled-protein receptors (GPCRs) and ion channels, to accelerate antibody discovery beyond traditional methods available today.
PhenomeX participation at 19th Annual PEGS Boston Conference & Expo
PhenomeX is participating at the 19th annual Protein Engineering & Cell Therapy Summit (PEGS) conference and expo being held from May 15-19 at the Hynes Convention Center in Boston, Mass. At the conference, the Company will feature its optofluidic and proteomic platform technologies, including the new Beacon Select system, in booth #517 and present two posters and podium presentations. Attendees can explore demonstrations of the technology and learn more about how PhenomeX's products can advance and accelerate our understanding of cellular function along the continuum of scientific discovery, bioprocessing, clinical research, and immunotherapy development.
About PhenomeX Inc.
PhenomeX is empowering scientists to leverage the full potential of each cell and drive the next era of functional cell biology that will advance human health. We enable scientists to reveal the most complete insights on cell function and obtain a full view of the behavior of each cell. Our unique suite of proven high-throughput tools and services offer unparalleled resolution and speed, accelerating the insights that are key to advancing discoveries that can profoundly improve the prevention and treatment of disease. Our award-winning platforms are used by researchers across the globe, including those at the top 15 global pharmaceutical companies and approximately 85% of leading U.S. comprehensive cancer centers.
INDUSTRIAL IMPACT, MEDICAL
Businesswire | May 05, 2023
Paige, a global leader in end-to-end digital pathology solutions and clinical AI applications to assist in diagnosing cancer, announced it is working with Visiopharm, an artificial intelligence (AI)-driven precision pathology software provider, to bring a wider range of AI products to doctors and patients. Visiopharm’s AI applications will be deployed and natively integrated into the Paige Platform to be accessed through FullFolio™, Paige’s AI-powered clinical worklist, and viewed within FullFocus®, Paige’s whole-slide image viewer. This deep integration offers users greater diagnostic efficiency and quality insights when reviewing IHC-stained slides and creates a seamless user experience for Visiopharm’s image analysis applications, to help pathologists deliver faster, more accurate and more reproducible cancer diagnoses.
“Improving the pathologist experience to ultimately improve patient care is at the heart of our technology. The Paige and Visiopharm integration offers pathologists thorough image analysis and quantitative assistance on whole-slide images within one AI-native integrated platform,” said Dr. David Klimstra, Co-Founder and Chief Medical Officer of Paige. “With this partnership, Paige is furthering our aim to provide the richest portfolio of AI applications delivered through a single platform. In this way, we can increase the impact AI has in helping pathologists diagnose cancer and continue to make the adoption of digital pathology more productive.”
The Paige Platform is interoperable and designed to enable labs to deploy AI-based diagnostic products from AI vendors like Visiopharm, creating one seamless pathology workflow from a single interface. It also integrates with most major scanner vendors and allows bi-directional integration with LIS systems, facilitating a truly open and comprehensive experience.
Digital labs often employ digital technologies from different manufacturers for cancer diagnosis. With AI from Visiopharm and Paige together on a single platform, these labs can have streamlined access to digital images and combined insights together in one place.
Dr. Michael Grunkin, CEO of Visiopharm, stated, "Visiopharm, a global leader in AI-driven precision pathology, delivers standardized decision support for complex tissue biomarkers. Our fully automated (zero-click) applications use a cascade of deep learning networks for slide quality control, tissue detection, tumor identification, and biomarker quantification. Designed to work across various technologies and providers, we join Paige in our commitment to making this technology accessible worldwide on top pathology platforms."
Paige is using the power of AI to drive a new era of cancer discovery and treatment. To improve the lives of patients with cancer, Paige has created a cloud-based platform that transforms pathologists’ workflow and increases diagnostic confidence as well as productivity, all on a global scale. Paige is the first company to receive FDA approval for a clinical AI application in digital pathology. The same Paige technology empowers pharmaceutical companies to evaluate treatment options more effectively for patients and design new biomarkers for drug development, so every patient gets precise treatment options.