GenScript, ReForm Biologics, Mission Bio, And More: News From March 2019

April 1, 2019 | March featured exciting new, products, and partnerships from around the bio-IT community from innovating companies, organizations, and universities, including GenScript, ReForm Biologics, Mission Bio, and more.

GenScript launched a single-stranded DNA (ssDNA) service. The new service offers researchers access to high quality, pure ssDNA for CRISPR-based gene insertion, ultimately helping to accelerate the development of gene therapy, cell therapy, as well as transgenic animal models for cancer research and treatment. GenScript utilizes a patent-pending enzymatic approach to develop high purity DNA oligos up to 3000 nucleotides long in large quantities, with an undetectable level of double-stranded DNA (dsDNA) contamination and minimal DNA base damage. GenScript's ssDNA service provides high purity ssDNA up to 3000 nucleotides long at flexible quantities. A unique quality control process involves an additional sequence verification step of the final ssDNA product, ensuring 100% sequence accuracy. "This newly launched ssDNA service, along with GenScript's existing CRISPR plasmids, synthetic sgRNAs, Cas9 proteins, and CRISPR cell line services, aims to make genome editing accessible and easy for all research purposes," Cedric Wu, GenScript Sr. Director of R&D, said in a press release. "GenScript remains committed to expanding our offerings with innovative and high quality services, such as our ultra-pure and sequenced verified ssDNA products, that accelerate research and ultimately make humankind and the environment healthier." Press release

ReForm Biologics announced a collaboration with Astellas to reduce the viscosity of an Astellas clinical-stage biological pipeline program. ReForm’s technology platform transforms the formulation of biotherapeutics in order to enable alternative dosing, improve manufacturing efficiency and extend product life-cycles. Under the terms of the agreement, Astellas will provide funding for the collaboration and the work will be performed at ReForm Biologics’ facilities in Woburn, Massachusetts. “We are excited to initiate this collaboration with Astellas, a global leader in turning innovative science into value for patients. We appreciate the efforts of Astellas Innovation Management team in getting the agreement completed,” John M. Sorvillo, Chief Executive Officer, ReForm Biologics, said in a press release. “This collaboration will demonstrate how new formulation technologies can optimize dosing and support life cycle management in pharmaceutical drug development programs. We look forward to working with Astellas to support the development of this clinical-stage asset.” Press release

Mission Bio announced that it has entered into a collaboration with LabCorp. LabCorp’s Covance Drug Development business will be the first global contract research organization to offer services on the Mission Bio Tapestri Platform, supporting biopharmaceutical customers in their need for faster, more precise clinical trials. Additionally, LabCorp is evaluating the Tapestri Platform for applications in clinical diagnostics and in the development of new companion diagnostics. This collaboration follows LabCorp’s strategic investment in Mission Bio’s $30 million Series B funding round, which was completed in December 2018. “The combination of LabCorp’s diagnostics and drug development capabilities makes it the ideal partner to expand our global ecosystem by opening access to the biopharmaceutical industry,” Charlie Silver, CEO of Mission Bio, said in a press release. “We have had significant interest from global pharma companies that want to access our Tapestri Platform to support their clinical programs. With this partnership, we can now offer complete support from preclinical and biomarker discovery through to clinical trials and commercialization.” Separately, Mission Bio is also announcing an alliance with Argonaut Manufacturing Services, a cGMP contract manufacturing organization (CMO) serving the biopharmaceutical and human diagnostics industries, to serve as its preferred global CMO. With Argonaut’s added expertise in operations and manufacturing, Mission Bio completes its supply chain to provide customers with an end-to-end solution for its biopharmaceutical business and enables turnkey commercialization of its Tapestri Platform. Press release

Biogen announced that it has entered into an agreement to acquire Nightstar Therapeutics, a clinical-stage gene therapy company based in London, United Kingdom, which is focused on adeno-associated virus (AAV) treatments for inherited retinal disorders. Under the terms of the proposed acquisition, Biogen will pay $25.50 in cash for each NST share. This offer represents a total transaction value of approximately $800 million on a fully diluted basis, after taking into account expected transaction expenses and anticipated cash at closing. “Ophthalmology is an emerging growth area for Biogen, and we are excited about the opportunity to work with the talented employees at Nightstar to advance potentially transformative gene therapy programs for rare retinal diseases,” Michel Vounatsos, Biogen’s Chief Executive Officer, said in a press release. “With this proposed acquisition, we are continuing to bolster our pipeline and further execute on our strategy to develop and expand a multi-franchise neuroscience pipeline across complementary modalities. Nightstar would accelerate our entry into ophthalmology by contributing two mid- to late-stage gene therapy assets, with the potential to create long-term shareholder value.” Press release

A Dundee University spin-out firm which uses artificial intelligence (AI) to speed up drug discovery has entered a multi-million pound partnership with a US biopharma company. Under the deal, Exscientia will work on three therapeutic programs for Celgene in the areas of oncology and autoimmunity. Exscientia will receive an initial $25m (£19m) upfront payment from Celgene. It will also receive royalties on sales that result from the collaboration. Exscientia said a key goal of the partnership was to apply AI to improve the speed of delivery of new treatments for patients. The collaboration will see Exscientia use its Centaur Chemist AI drug discovery platform, which it says can greatly reduce the time it takes to discover pre-clinical drug candidates. The deal extends Exscientia's list of partnerships with blue-chip pharma and biotech companies, with existing collaborations involving Roche, GSK, Sanofi, and Evotec. Full Article

BioIVT announced the launch of its XPRESSWAY Profile Safety Package, a compendium of 46 gene expression profiles for genes known to play pivotal roles in drug safety assessments. Those 46 genes are included in the initial screening panel used by pharmaceutical companies to evaluate new chemical entities (NCEs) for potential off-target interactions and toxic effects. Pharmaceutical companies routinely evaluate the binding and/or function of NCEs on these targets to determine their drug safety risk and make go/no-go decisions in their lead selection programs. "BioIVT is always looking for ways to support our customers and help inform strategic decisions in the drug discovery process. We developed the XPRESSWAY Profile Safety Package to provide additional context for pharmacology screening data and allow sponsors to make go/no-go decisions regarding NCEs more efficiently. We also partner with customers to conduct custom gene expression studies that further support lead selection and optimization," Amanda Woodrooffe, vice president and general manager, PHASEZERO Research Services at BioIVT, said in a press release. Customers use BioIVT's database profiles to interpret target expression and support target validation, and also to identify novel or unexpected expression that may indicate either safety liability or an opportunity to repurpose a drug acting on that target. Press release

Novogene announced that the Tianjin Novogene Med Lab – NGS Clinical Lab for genomic testing has successfully renewed its CAP accreditation, passing rigorous on-site inspection by experts from the College of American Pathologists (CAP) in recognition of the high standards of excellence established since its first accreditation in February 2017. The College of American Pathologists is a US non-governmental organization that is widely considered the leader in laboratory quality assurance. Based on standards and guidance from the Clinical & Laboratory Standards Institute (CLSI) and the Clinical Laboratory Improvement Amendments (CLIA) of 1988, the CAP has created detailed checklists containing a core set of requirements which apply to all areas performing laboratory tests and procedures. Through its laboratory quality solutions programs, the CAP improves patient safety and advances the quality of pathology and laboratory service through education, standard setting and ensuring laboratories meet or exceed regulatory requirements. Founded on June 23rd, 2014, the Tianjin Novogene Med Lab – NGS Clinical Lab is a modern, large-scale and independent clinical laboratory established in accordance with the standards of provincial medical inspection institutes issued by the Ministry of Health in China. It was approved by the Municipal Health and Family Planning Commission in December 2014 for the application of clinical gene amplification technology. Since March 7th, 2016, the Med Lab has been operating according to the international authoritative clinical laboratory quality certification system-the CAP -and passed the professional on-site inspection by CAP experts on February 10th, 2017, making it the first CAP-accredited NGS clinical lab in mainland China. After two years of effective operation and continuous improvement of its quality system, the Tianjin Novogene Med Lab – NGS Clinical Lab has once again passed the on-site inspection and successfully maintained its CAP accreditation. Press release

Strata Oncology announced the launch of version 3.0 of StrataNGS, its pan-cancer assay for solid tumors. The updated 500-gene assay utilizes DNA and RNA from tumor samples to detect all clinically actionable biomarkers — including microsatellite instability (MSI), tumor mutational burden (TMB), and PD-L1 — recommended by leading guidelines, in a single test. Test results are provided in a streamlined report that facilitates rapid, confident interpretation and identification of potential treatment options and clinical trials. StrataNGS is provided at no cost to advanced cancer patients enrolled in the Strata Trial at the company's partner health systems. Results are returned in less than 10 business days using industry-low sample input requirements. The test employs RNA sequencing and DNA sequencing to simultaneously detect mutations, small frame-preserving insertions/deletions (indels), amplifications, deep deletions, de novo deleterious mutations, gene fusion events, MSI, TMB, and PD-L1. In addition, the updated assay includes analytical validation of 36 gene expression markers that represent emerging immuno-oncology and antibody-drug conjugate targets. Although these biomarkers are not yet reported clinically, the data is available to Strata Oncology and investigators at partner cancer centers for correlative analyses and new trial design. Press release

Petra announced it has secured a global license from Takeda to develop, manufacture, and commercialize serabelisib and two additional PI3Kα-specific inhibitors. The agreement grants Petra a license for all human therapeutic uses, except for a subset of undisclosed rare-disease indications, which Takeda had previously out-licensed. Petra plans to initiate a Phase 1b/2 study with serabelisib in late 2019 to advance pioneering research and insights in PI signaling pathways with a focus on PIK3CA-mutated solid tumors. The PI3K signaling pathway is a frequently mutated pathway in human cancer. "Today marks a transformational moment for Petra and a new era in the therapeutic use of PI3K inhibitors. We are pioneering new approaches that have the potential to increase tumor responses with PI3K inhibitors and minimize side effects, with profound clinical implications for patients diagnosed with a PIK3CA-mutated tumor," wrote President and CEO, Brian O'Callaghan. Press release

A team of engineers at the UC Berkeley and the Keck Graduate Institute (KGI) of The Claremont Colleges combined CRISPR with electronic transistors made from graphene to create a new hand-held device that can detect specific genetic mutations in a matter of minutes. The device, dubbed CRISPR-Chip, could be used to rapidly diagnose genetic diseases or to evaluate the accuracy of gene-editing techniques. The team used the device to identify genetic mutations in DNA samples from Duchenne muscular dystrophy patients. Doctors and geneticists can now sequence DNA to pinpoint genetic mutations underlying a host of traits and conditions, and companies like 23andMe and AncestryDNA even make these tests available to curious consumers. But unlike most forms of genetic testing, including recently developed CRISPR-based diagnostic techniques, CRISPR-Chip uses nanoelectronics to detect genetic mutations in DNA samples without first "amplifying" or replicating the DNA segment of interest millions of times over through a time- and equipment-intensive process called polymerase chain reaction, or PCR. This means it could be used to perform genetic testing in a doctor’s office or field work setting without having to send a sample off to a lab. "CRISPR-Chip has the benefit that it is really point of care, it is one of the few things where you could really do it at the bedside if you had a good DNA sample," Niren Murthy, professor of bioengineering at UC Berkeley and co-author of the paper, told Berkley News. "Ultimately, you just need to take a person’s cells, extract the DNA and mix it with the CRISPR-Chip and you will be able to tell if a certain DNA sequence is there or not. That could potentially lead to a true bedside assay for DNA." Full Article

Standigm announced it has raised $11.5 million in a Series B funding round led by Mirae Asset Venture Investment and Mirae Asset Capital. Standigm will use the funds to scale the AI technology platforms and advance its drug discovery pipelines toward license-out. The new round brings Standigm’s total financing to $15 million. The round included participation from Kakao Ventures, Atinum Investment, DSC Investment, LB Investment, Wonik Investment Partners, as well as Mirae Asset Venture Investment and Mirae Asset Capital. Kakao Ventures, one of the leading early-stage VCs in Asia, continued to invest Standigm since its seed round. “We have been very impressed with the progress Standigm has made over the past years in global drug discovery market,” Kijun Kim, Vice President in Kakao Ventures, said in a press release. “The feasibility of Standigm AI is already acknowledged among the leading pharmaceutical companies. We’re pleased to continue our support. This will allow standigm to broaden its own pipelines.” Press release

Scientists at The University of Southern Denmark will be using an app created by Datacubed Health to conduct the first crowdsourcing study of the impact of smart phone use on working memory, mental focus and attention. The scientists will also be using the app to study the effects of mindfulness techniques and music listening patterns on concentration, stress, and working memory. Both studies will begin in April. To engage users, the studies will use the app both for electronic consent and data collection. The app employs an interface that take participants on journeys though numerous interactive maps and virtual worlds as they “journey” through the research study. While the impact of smart phone use on cognition has been the subject of much popular discussion in recent years, few objective neuropsychological studies have been conducted with large numbers of participants, said Ulrich Kirk, Associate Professor at the Institute of Psychology at the University of Southern Denmark, who will be leading the study. “These studies will allow us to bring cognitive neuroscience to people’s everyday lives,” Professor Kirk said in a press release. “With Datacubed’s involvement in creating an app for iPhone and Android devices, thousands of people will be able to participate using their own phones.” Press release

Vineti and OutcomeRx, a new division of CareMetx, announced a new alliance to build patient-centered digital and services solutions to streamline cell and gene therapies. “Gene and cell therapies are creating miracles for patients and their families,” said Bob Dresing, CareMetx Chairman and Co-founder, in a press release. “To bring these treatments to patients, OutcomeRx and Vineti will help to overcome unprecedented challenges, including the complex processes around the supply chain, getting patients treated, paying for the therapy and evaluating the patient’s health over time.” Personalized therapeutics, such as cell and gene therapies and personalized cancer vaccines, are highly individualized medical innovations that require specialized technology solutions to ensure each patient receives and benefits from individualized drug therapy. In January, the US Food and Drug Administration announced that it expects to receive more than 200 new cell and gene therapy applications per year by 2025, and to approve 10 to 20 new cell and gene treatments each year. “Personalized therapies need to be supported by technology solutions as innovative as their science,” Amy DuRoss, CEO and Co-founder of Vineti, wrote in a statement. “Providing large numbers of patients with personalized therapies requires a new digital foundation that supports the patient journey. Vineti is excited to be working with CareMetx’s new division, OutcomeRx, and its distinctive digital and services platform. Together, we can make a real commitment to helping patients.” Press release

Agenus has developed a proprietary AI platform—Adaptive Learning Platform Systems (ALPS)—to provide insights into cancer biology and help identify new targets for drug development. ALPS is also designed to help predict responses to treatment and with the development of precision combinations. Agenus takes data from genetic analysis of millions of immune cells in their laboratory and from collaborators, and they match them with tumor profiles of patients from their clinical trials. These capabilities hold the answers to some of the most important questions in cancer immunotherapy. Including, why do current immunotherapy medicines work so well in some patients but not others? What determines whether a treatment will be effective in a particular patient? Using ALPS, Agenus is finding answers to some of these questions. In doing so, they expect to develop effective treatment strategies for wider numbers of cancer patients who are not benefiting from today’s medicines. Newsletter

DataDirect Networks (DDN) showcased game-changing storage solutions and applications that empower users to accomplish their most challenging AI objectives at the NVIDIA GPU Technology Conference (GTC) in San Jose, California. DDN featured and demonstrated innovations in its A³I architecture and new customer use cases in autonomous driving, life sciences and healthcare, retail and financial services. The company showcased the next generation of A³I reference architectures, which include NVIDIA’s DGX POD, DGX-2, and DDN’s AI400 parallel storage appliance. Through industry standard benchmarks and customer use cases using the new reference architectures, DDN demonstrated how it establishes higher levels of application acceleration when combining the power of GPUs with parallel storage systems. Featured use cases included testing results from the Max Delbrück Center (MDC) for Molecular Medicine in conjunction with Zuse Institute Berlin (ZIB). MDC is a leading molecular biology and clinical research center in Germany, and ZIB is an interdisciplinary research institute for applied mathematics and data-intensive high-performance computing. MDC originally partnered with DDN in 2018 to greatly accelerate its applications and to efficiently manage large volumes of data. Using an A³I architecture with an NVIDIA DGX-1 system and an AI200 from DDN, MDC was able to improve accuracy and precision within analyzed images while simultaneously accelerating training performance by 240%. More recent testing with an A³I architecture comprised of NVIDIA’s DGX-2 system and DDN’s AI400 revealed results that more than doubled previous improvements. DDN also announced a partnership with Robovision. Press release