CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Globenewswire | May 02, 2023
Editas Medicine, Inc. a clinical stage genome editing company, announced that the journal Nature Biotechnology published the comprehensive data from a study of the proprietary SLEEK gene editing technology.
Despite major progress in achieving gene disruption with CRISPR-Cas gene editing technologies, efficient knock-in of transgenes continues to be a significant challenge for the gene editing field. To solve this challenge, SLEEK was developed to enable high knock-in efficiency with both viral and non-viral transgene formats while also ensuring robust simultaneous expression of up to four transgene cargos.
The study demonstrated that utilizing SLEEK results in the knock-in of multiple clinically relevant transgenes through a proprietary process that specifically selects only those cells containing the knock-in cargo. This process was developed by leveraging Editas Medicine’s proprietary engineered AsCas12a nuclease, which can achieve very high editing efficiency while maintaining high specificity. More than 90 percent knock-in efficiencies were observed in various clinically relevant target cells, including T cells, B cells, iPSCs, and NK cells. Additionally, SLEEK can be used to fine-tune the expression levels of transgene cargos, an important feature of next-generation cell therapies. As a demonstration of SLEEK’s potential value in clinical applications, the study authors used SLEEK to generate iPSC-derived NK cells capable of high-levels of in vivo persistence and robust tumor clearance in a solid tumor animal model.
“We are thrilled Nature Biotechnology published our paper sharing the comprehensive data on our SLEEK gene editing technology as we believe SLEEK has immense potential for gene editing drug development. As shared in the publication, SLEEK technology enables nearly 100 percent knock-in of functional transgene cargos at specific locations in the genome which may result in highly efficient multi-transgene knock-in for the next generation of cell therapy medicines,” said John A. Zuris, Ph.D., Director of Editing Technologies, Editas Medicine, and senior author on the study.
Editas Medicine believes that SLEEK may enable better product purity as well as shorter manufacturing timelines for the next generation of cell therapy medicines. Earlier this year, the Company announced it licensed its interest in the SLEEK technology to Shoreline Biosciences for specific usage in iPSC-derived NK and iPSC-derived macrophage cell therapies for oncology. The SLEEK technology remains an important Editas capability in iPSC engineering for a wide variety of future applications.
About Editas Medicine
As a clinical stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute and Harvard University’s Cas9 patent estates and Broad Institute’s Cas12a patent estate for human medicines.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Prnewswire | April 12, 2023
Mission Bio, Inc., the pioneer in high-throughput single-cell DNA and multi-omics analysis, announced that it has entered into an agreement with Fulgent Genetics a technology-based company with a well-established clinical diagnostic business and a therapeutic development business. Fulgent Genetics is now able to offer single-cell multi-omics on the Tapestri Platform on its fully customizable menu of services, including whole genome, whole exome, RNA sequencing, tumor profiling, methylation sequencing, liquid biopsy, single cell sequencing, spatial biology, and pathology services to support its growing pharma client demands. Additionally, Fulgent Genetics will evaluate the Tapestri Platform for applications in clinical development to streamline the drug development and approval process.
Over the last ten to fifteen years, there has been a paradigm shift in clinical trials towards precision oncology by using biomarkers to select or enrich trial cohorts. The Tapestri Platform's ability to do single-cell analysis of genotype and phenotype from the same cell can provide the sensitivity and resolution for biomarker discovery unattainable by current bulk methods. Single-cell resolution can reveal treatment resistance mechanisms that may help to better stratify patients in clinical trials. While on treatment, residual levels of disease can be detected with the high sensitivity of single-cell analysis. Characterizing clonal heterogeneity and tracking clonal evolution during treatment can potentially guide adaptive and rational combination therapies for better patient stratification and better clinical trial outcomes.
"The increasing adoption of the Tapestri Platform for translational research and clinical development by many cancer centers and global pharma companies demonstrates the necessity of single-cell multi-omics," said Dr. Hanlin (Harry) Gao M.D., Ph.D., D.A.B.M.G., F.A.C.M.G., Chief Scientific Officer of Fulgent Genetics. "The relationship between Fulgent Genetics and Mission Bio will make it easier and faster for biopharmaceutical customers to generate data for more precise clinical trials."
"Fulgent Genetics' full scope, end-to-end service and comprehensive genomic portfolio is the perfect one-stop shop for pharma customers," said Todd Druley, M.D., Ph.D., Chief Medical Officer of Mission Bio. "While some pharma customers can and still do partner with us through our internal Pharma Assay Development (PAD) services, this agreement provides another option for them to leverage the single-cell multi-omics expertise and full suite of solutions offered by Fulgent Genetics, particularly as they complete the validation of the Tapestri Platform in their CAP CLIA lab in the near future."
Customers today have access to the Tapestri Platform and its full breadth of single-cell multi-omics and scMRD assays through Fulgent Genetics' labs.
About Mission Bio
Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others.
The company's Tapestri Platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously from the same sample at single-cell resolution.
MEDTECH, INDUSTRIAL IMPACT
Prnewswire | April 06, 2023
ConcertAI, LLC a leader in AI Software-as-a-Service (SaaS) technology and Real-world Evidence solutions for life sciences and healthcare, announced the release of its next generation of Clinical Trial Optimization (CTO 2.0).
"Trial sponsors have a range of new imperatives they're trying to meet: assuring that the trial population is reflective of the ultimate population that would have access to the drug; to set and meet diversity goals for those ethnic, racial and economic groups that are uniquely, negatively impacted by a disease; and meeting ever higher productivity imperatives for identifying and activating sites faster and working with those sites most likely to accrue to the pre-activation feasibilities," said Jeff Elton, Ph.D. CEO of ConcertAI. "Meeting these goals requires working with sites and investigators not historically preferred, designing trials for a minimum of burden on research sites and patients and, perhaps most importantly, moving more studies into community-based research settings."
CTO 2.0 now includes a range of features: (1) clinical depth and tools, such as line of therapy assessments, Kaplan-Meier survival curves, and cox-proportional hazard assessments that guide alternative designs with the largest underlying research dataset for oncology; (2) data integrating electronic medical-derived, medical claims, and social determinants of health; (3) site- and investigator-level information on current and previous clinical trials derived from public and private sources; and (4) operational trial metrics and site profile information to highlight performance and capabilities.
The solution's foundation is a study digitization layer that can handle any depth of inclusion and exclusion (I/E) criteria. Protocols can be assessed relative to standard-of-care in different settings and in different time periods, all the way down to the individual event or clinical activity level. There are APIs for integrating past study protocols from any legacy systems of records. Optimizer and recommendations engines can provide study-level optimizations and semi-automate site selection. Additional APIs can integrate past site survey data and investigator databases as a means of simplifying and unifying what are often non-integrated solutions within the clinical analytics and clinical operations organization. The SaaS technology will support researchers and clinicians in selecting the most appropriate sites for clinical trials, balancing potential patients available and previous trial performances at those sites to give the highest likelihood of success for a clinical trial. In assurance that trials can be conducted across settings and utilize the latest digital trial solutions that support direct EMR to EDC, CTO 2.0 leverages the latest clinical informatics integrations and data standards to ensure research can be conducted at scale and meeting, or exceeding, enhanced standards of care.
"In the past, clinicians used previously published protocols and recently approved studies in the same disease to inform trial designs for new studies," said Ronan Brown, ConcertAI's Chief Operating Officer. "Now that is changing. We'll now see greater reliance on large scale, multi-sourced, clinical datasets that have low latency, reflecting the latest treatments, supporting new designs, lower site burden, greater, diversity, and expanded views of relevant sites. It's a significant step forward in clinical trial planning and operations."
ConcertAI is a leader in Real-World Evidence (RWE) and AI technology solutions for life sciences and healthcare. Our mission is to accelerate insights and outcomes for patients through leading real-world data, AI technologies, and scientific expertise in partnership with the leading biomedical innovators, healthcare providers, and medical societies.