Ordaōs and NonExomics | September 29, 2022
Ordaōs, a biotechnology company designing novel miniPROTM mini-proteins to help drug hunters deliver life-saving treatments, and NonExomics, a company specializing in the identification of novel targets from non-conventional protein-coding regions, announced a proof-of-concept research collaboration. As part of this endeavor, Ordaōs will use its proprietary design engine to generate and rank novel miniPRO™ candidates that meet specific target product profiles, beginning with three targets. NonExomics will conduct pre-clinical certification of these identified MinPRO™ proteins to demonstrate their promising commercial value.
"This partnership allows us to spotlight the power of our Design Engine to create customized miniPRO™ proteins that are more configurable, penetrable and stable, and easier to manufacture. Our collaboration with NonExomics also allows us the mutual opportunity to underscore the potential of our specialties utilizing machine-driven technology."
David Longo, CEO of Ordaōs
NonExomics identifies whether novel proteins can function as drug targets for therapeutic purposes or can be used for diagnostic processes. The company has developed a technology that effectively combines artificial intelligence, proteomics, transcriptomics, and genomics to identify novel targets for quicker diagnosis and treatment of diseases that are currently considered to be incurable or difficult to manage. NonExomics plans to develop new therapies and diagnostics based on its proprietary nonexomic targets.
"We at NonExomics are thrilled to be embarking on this exciting collaboration between our companies to fuel our shared goal of pursuing and developing new therapies for poorly treated or currently deemed incurable diseases," said Sudhakaran Prabakaran, PhD, CEO and Co-Founder of NonExomics.
To select miniPRO™ candidates, Ordaōs will use its multitask meta-learning Ordaōs Design Engine to generate, appraise, and rank hundreds of thousands of proteins on their structures and drug-like properties for novel miniPRO™ candidates that meet the requirements of the desired specifications of NonExomics' product profile. NonExomics will then conduct a series of pre-clinical demonstrations of the selected Ordaōs miniPRO™ designs to prove they meet the specified product profiles.
More About The Ordaōs Design Engine
Ordaōs uses The Ordaōs Design Engine, to deliver true protein property design - leveraging continuous learning loops and proprietary data sets to translate human-targeted product criteria into machine-designed mini-proteins. Starting with amino acids, the Design Engine generates, appraises, and ranks billions of protein sequences and hundreds of thousands of protein structures and properties to create customized miniPRO™ proteins. These proteins are then rapidly evaluated in vitro to provide intelligent feedback on multiple design objectives including protein structure, binding specificity and affinity, solubility, stability, immunogenicity, and developability. This iterative process delivers optimized mini-proteins to meet the client's specific molecular target product profile (mTPP). They are also less likely to cause adverse side effects and are easier and less expensive to test, develop, and manufacture than traditional proteins. Using this approach, the Ordaōs Design Engine creates more ideal, unseen protein leads than others and can accelerate drug candidate development, increasing the probability of more therapeutically effective candidates. All of this provides clients with a high level of confidence in their investigational new drug (IND) applications.
Ordaōs is a human-enabled, machine-driven drug design company that helps birth novel therapies to reduce patient suffering, improve health, and extend life. Our flagship solution, miniPRO™ mini-proteins, enable drug hunters to deliver safer and more effective treatments in a fraction of the time of traditional discovery methods.
NonExomics science and platform developed over eight years of rigorous academic and industry research, mines novel disease-associated proteins using the dark genome. NonExomics has opened up a new area of science with unlimited possibilities to cure diseases.
CELL AND GENE THERAPY
Q&#178; Solutions and IQVIA, Tasso | October 07, 2022
Q² Solutions, a wholly owned subsidiary of IQVIA and a leading global clinical trial laboratory services organization, today announced the launch of the first self-collection safety lab panel for U.S. clinical trial participants by a leading global clinical trial laboratory.
Developed in collaboration with Tasso Inc, a leader in clinical-grade blood collection solutions, this unique offering combines industry leading laboratory services, patient focused logistics and cutting-edge, self-collection technology.
Participants in clinical trials can now provide a blood specimen for lab testing in the comfort of their own home, without the need to visit an investigator site or have a healthcare professional visit them. The first test panel available through this collaboration is designed to help monitor liver function by measuring the levels of select enzymes and proteins in the blood. The panel includes tests for ALT AST ALP total bilirubin, direct bilirubin, total protein, and albumin.
“The global pandemic has accelerated the use of decentralized approaches in clinical trials. We at Q² Solutions are highly motivated to find ways to reduce patient burden related to laboratory testing, and I’m delighted that, through our collaboration with Tasso, we could make the self-collection liver panel a reality. Soon we will launch more than a dozen safety tests, and we are determined to continue to expand our catalog of tests available via the self-collection of blood.”
Brian O’Dwyer, CEO of Q² Solutions
“We are thrilled to be working with a leader like Q² Solutions on this important initiative to make laboratory testing truly patient-centric,” said Erwin Berthier, PhD, CTO and Co-Founder of Tasso. “Our clinical-grade remote sampling technology enables decentralized laboratory testing without sacrificing sample quality, a key hurdle that home testing has faced in the past. Together, Q² Solutions and Tasso are developing important solutions that make clinical trials better for both patients and sponsors.”
This self-collection liver panel was developed with trial participants at the heart of the design. The offering includes easy to use at-home self-collection kits with instructions and videos, a methodology for direct-to-patient delivery, as well as simplified specimen processing and shipping requirements.
About Q² Solutions
Q² Solutions is a leading global clinical trial laboratory services organization that provides comprehensive testing, project management, supply chain, biorepository, biospecimen, and consent tracking solutions. Leveraging our next generation technologies, we deliver agile and precise services designed to meet the diverse needs of our clients. We provide scientific expertise and innovative solutions for ADME, bioanalytical, genomics, vaccines, and central laboratory services, including flow cytometry, anatomic pathology, immunoassay, molecular, companion diagnostics and translational science, with meticulous regional and global clinical trial implementation support and high-quality data delivery. At Q² Solutions, our work is rooted in research, grounded in collaboration, and guided by our passion to turn the hope of patients and caregivers around the world into the help they need.
IQVIA is a leading global provider of advanced analytics, technology solutions, and clinical research services to the life sciences industry. IQVIA creates intelligent connections across all aspects of healthcare through its analytics, transformative technology, big data resources and extensive domain expertise. IQVIA Connected Intelligence™ delivers powerful insights with speed and agility — enabling customers to accelerate the clinical development and commercialization of innovative medical treatments that improve healthcare outcomes for patients. With approximately 82,000 employees, IQVIA conducts operations in more than 100 countries.
IQVIA is a global leader in protecting individual patient privacy. The company uses a wide variety of privacy-enhancing technologies and safeguards to protect individual privacy while generating and analyzing information on a scale that helps healthcare stakeholders identify disease patterns and correlate with the precise treatment path and therapy needed for better outcomes. IQVIA’s insights and execution capabilities help biotech, medical device and pharmaceutical companies, medical researchers, government agencies, payers and other healthcare stakeholders tap into a deeper understanding of diseases, human behaviors and scientific advances, in an effort to advance their path toward cures.
Trusted diagnostics made easy. Tasso is an emerging healthcare technology company that is transforming the traditional blood collection paradigm with a patient-centric approach. The company’s devices enable simple, convenient, and virtually painless blood collection for users. Tasso technology has the power to bring healthcare anywhere, any time. Headquartered in Seattle, Washington, Tasso is privately held and funded by grants, investments, and co-development deals with various industry leaders.
CELL AND GENE THERAPY
Genscript Biotech Corporation | September 23, 2022
GenScript USA Inc., the world's leading life-science research tools and services provider, is collaborating with researchers at the Gladstone-UCSF Institute of Genomic Immunology to advance the development of new, non-viral cell therapies that modify genomic sequences in the pursuit of more effective cell therapy products while limiting the cellular toxicity that is typically associated with previously available methods.
A new study, published in Nature Biotechnology, details methods for achieving highly efficient non-viral knock-in using GenScript's GenExact™ single-strand DNA modified with Cas9-target sequences. This method achieved up to ˜40% knock-in efficiency in primary immune cell types.
One powerful application of CRISPR/Cas genome editing technology involves the precise insertion of DNA sequences via the HDR pathway. Traditionally, researchers have relied on viral vectors to deliver DNA insertion templates used for gene therapy into cells. However, difficulties manufacturing large amounts of clinical-grade viral vectors have delayed getting cell therapies to patients. Additionally, viral vectors can insert genes at any location within the genome, leading to safety concerns.
Previous research by the group at Gladstone and UCSF has shown that synthetic DNA templates can be delivered without the use of viral vectors, but high levels of double-stranded DNA can be toxic to cells, resulting in low efficiency. Efficiency can be improved using a modified version of the DNA templates that can bind to the Cas9 enzyme. However, additional work was required to improve the yield of successfully engineered cells and to make the process compatible with the manufacturing of future cell therapies. Those goals motivated the team's current study.
In this study, the team tested modified GenExact ssDNA designed with Cas9-targeted sequences to determine if the gene-editing efficiency could be improved with lower toxicity. First, synthetic sgRNA was complexed with Cas9 protein to form a RNP complex. Then the RNP and ssDNA template were delivered into the cell via electroporation, enabling efficient gene editing.
The team discovered that this combination of GenExact ssDNA with Cas9-targeted sequence offers up to 40% knock-in editing efficiency with minimal cellular toxicity. This approach can accelerate the development and manufacture of novel, high-yield non-viral gene therapies — and at lower cost.
"We are very proud to have collaborated on this groundbreaking scale-up work by the talented team at Gladstone and UCSF. GenScript is excited for the opportunities that this high-yield cell-line engineering process will provide to our customers," said Ray Chen, Ph.D, president of GenScript Life Science Group.
"We were very happy to partner with Genscript on critical experiments demonstrating high efficiency and yield of CAR knock-in cells at clinical scale. The long ssDNA produced by Genscript exceeded our expectations and helped us clearly demonstrate the potential for future therapeutic applications using these methods.",
Brian Shy, MD, PhD, a former clinical fellow in the lab of Alex Marson, MD, PhD, director of the Gladstone-UCSF Institute of Genomic Immunology
About GenScript Biotech Corporation
GenScript Biotech Corporation is a global biotechnology group. Based on its leading gene synthesis technology, GenScript has developed four major platforms including the global cell therapy platform, the biologics contract development and manufacturing organization platform, the contract research organization platform, and the industrial synthesis product platform.
GenScript was founded in New Jersey, USA in 2002 and listed on the Hong Kong Stock Exchange in 2015. GenScript's business operation spans over 100 countries and regions worldwide, with legal entities located in the USA, mainland China, Hong Kong, Japan, Singapore, the Netherlands, and Ireland. GenScript has provided premium, convenient, and reliable products and services for over 100,000 customers.