CRISPR-Cas9, IRBM, GenScript, and More: News From September 2019

September 26, 2019

September 26, 2019 | September featured exciting new, products, and partnerships from around the bio-IT community from innovating companies, organizations, and universities, including CRISPR-Cas9, IRBM,GenScript, and more.

The US Patent and Trademark Office (USPTO) awarded a new CRISPR-Cas9 patent to the University of California (UC), University of Vienna, and Dr. Emmanuelle Charpentier covering compositions of single molecule DNA-targeting RNA (sgRNA) and a Cas9 protein or nucleic acid encoding the Cas9 protein. This patent (U.S. 10,400,253) further expands the intellectual property that is protected by UC’s CRISPR-Cas9 portfolio. In addition to the 12 US patents already issued, the USPTO has allowed claims of five other applications which are expected to issue as patents in the coming months. One of these applications will issue as a patent as soon as next week, on September 10, and covers sgRNA’s or DNA molecules encoding sgRNA’s. Altogether, the university will hold 17 total patents in its US CRISPR-Cas9 portfolio covering compositions and methods of targeting and editing genes in any setting, such as within plant, animal, and human cells, as well as patents related to the modulation of transcription. Press release

IRBM announced the signing of a service and development agreement with The University of Texas MD Anderson Cancer Center. The agreement will focus on the development of therapeutic monoclonal antibodies against a novel immune checkpoint target. IRBM will work with MD Anderson’s Oncology Research for Biologics and Immunotherapy Translation (ORBIT) platform, part of the institution’s Therapeutics Discovery division focused on therapeutic antibody development. The parties will collaborate to identify and validate lead antibodies through the pre-clinical proof of concept studies. IRBM’s human antibody discovery platform can rapidly identify and optimize high-quality antibodies, which can be explored and developed as novel therapeutics. IRBM will utilize its proprietary phage-display antibody libraries and immuno-oncology capabilities to generate and optimize fully human antibody candidates against the undisclosed target. Thereafter, MD Anderson has options to continue clinical development of selected antibodies through later clinical stages. Press release

GenScript announced that it will provide research support to Harvard University scientist George Church for his contributions to the Ultra-safe Cell project, the first grand scale community-wide initiative being undertaken by Genome Project-write and The Center for Excellence in Engineering Biology. The project aims to develop a cell line that is completely resistant to all natural viruses, and consequently could pave the way for many biomedical applications, from modeling cell and tissue behaviors to the production of better and safer biologics and therapeutics. Church, a professor of genetics and health sciences and technology at Harvard University and the Massachusetts Institute of Technology (MIT), is working in conjunction with Jeff Boeke, of New York University's Langone Health. To engineer the virus-resistant human cell line, scientists are using a process called DNA recoding to alter about 1 percent of the genome, including the exons of all the genes, and nearby sequences, leaving the vast majority of the non-coding regions untouched. Under the collaboration, GenScript will be providing research support, including DNA synthesis services, for the project. “Recoding every protein in the human genome is a significant and difficult undertaking, requiring, for example, 400,000 changes,” Church said in a press release. “Effective and efficient tools for genomic analysis, DNA synthesis, assembly and testing are crucial to the development of ultra-safe, virus-resistant cells. GenScript’s significant experience in DNA synthesis will be beneficial to this project, and we appreciate the company’s support and continual innovation in the development of new technologies to advance our efforts.” Making large-scale changes to the genome could also help improve cellular survival by making cells less likely to become cancerous and to better resist damage from extreme conditions, such as radiation. Additionally, the project could result in the development of new genomics tools and technologies that are more affordable and broadly available to the scientific community. Press release

Illumina announced the appointment of Joydeep Goswami as Senior Vice President of Corporate Development and Strategic Planning. Mr. Goswami will be responsible for driving planning, strategic partnerships and acquisitions, and will report to President and Chief Executive Officer, Francis deSouza. With more than 20 years of global experience, Mr. Goswami has a strong track record of developing strategy and leading new product development, partnerships, licensing, and mergers and acquisitions. Most recently, he served as the President of Thermo Fisher Scientific’s Clinical Next-Generation Sequencing (NGS) and Oncology business unit, where he oversaw efforts that drove the adoption of NGS in clinical oncology, research and reproductive health. “We are very pleased to welcome Joydeep to Illumina,” deSouza said in a press release. “Joydeep’s global experience, entrepreneurial spirit and deep background in research and clinical businesses, will serve Illumina well as we accelerate the adoption of sequencing in existing markets and enable the most impactful, new opportunities for genomics.” "I am honored to be joining Illumina and look forward to helping harness the power of the genome to shift the paradigm of healthcare and life science research across the globe," Goswami said in a statement. Press release

iSpecimen announced it has partnered with new providers of diseased and healthy hematologic tissue to offer one of the industry’s largest, most integrated networks of donors and collection sites available while addressing proliferating demand for hematopoietic stem and immune cell products. Now, researchers can readily access cells with highly specific characteristics through the iSpecimen Marketplace, an online platform that increases access to human biospecimens from specific patients and healthy donors that can provide them. iSpecimen continues to expand its partner network around the world, enabling researchers to efficiently procure bone marrow aspirate, peripheral blood mononuclear cells (normal and mobilized), and whole blood—all with corresponding donor profile/clinical data from healthy and diseased donors. Researchers today increasingly use these types of tissue and cell products in their work to develop stem cell therapies, immunotherapies, vaccines, diagnostics, new treatments for infectious and autoimmune diseases, and in cell-based assays to advance drug discovery and preclinical development. Press release

Ginkgo Bioworks announced a Series E financing round of $290 million with participation from all existing major investors and other new investors including funds and accounts advised by T. Rowe Price Associates, bringing the company’s total funding to $719 million. The latest round of funding will be used to further expand the reach of Ginkgo’s platform for cell programming, enabling greater access to the expertise, scale, resources, and technology needed to effectively harness the power of biology. Companies across industries and of all sizes are starting to understand the potential of synthetic biology to replace costly or unsustainable manufacturing processes and solve pressing global challenges. The funding will propel Ginkgo’s platform and enable its team of experienced organism engineers to unlock the full potential of biological engineering and deliver transformative technologies with far-reaching impact. Most recently, Ginkgo established itself as a home for early-stage startups and entrepreneurs to build their biotech businesses. Through two new partnerships with Y Combinator and Petri, startups can access Ginkgo's platform and mentorship in exchange for equity. With access to Ginkgo, startups will now have a pathway to bring bold, world-changing ideas to fruition. Press release

The iQ Group Global announced it has added a novel anticancer drug platform to its portfolio that has the ability to develop a range of cancer therapeutics. TEX-Core is an anticancer drug platform that has the ability to develop a range of well-tolerated, MRI-detectable cancer therapeutics that target drug-sensitive and drug-resistant solid tumors. ­The first cancer therapeutic to be commercialized from the platform is OxaliTEX, a novel chemical entity (WO 2015/191797) that targets only solid tumor cells, activates within the tumor and overcomes drug-resistance mechanisms with minimal side effects. Press release

Procarta Biosystems announced a CARB-X award of up to $9.2 million to progress its pipeline of antibiotic precision medicines. The award includes initial non-dilutive funding up to $2.2 million with a further $7.0 million available if certain project milestones are met. The new antibiotics are based on Procarta’s proprietary oligonucleotide-based antimicrobial platform and will target potentially life-threatening infections caused by Gram-negative ESKAPE pathogens. “This new award from CARB-X recognizes the value of the novel modalities in our pipeline and their potential to precipitate a paradigm shift in antimicrobial treatments,” said Andrew Lightfoot, Chief Executive Officer of Procarta. “We are delighted to have support from CARB-X recognizing the importance of developing new antibiotics to combat antimicrobial resistance. This award from CARB-X follows closely on a €1.5m investment from the Novo Holdings REPAIR Impact Fund. Together, these new funds will be used to progress our lead asset, PRO-202 and to develop our proprietary drug discovery platform to build a pipeline of antimicrobial agents to cover the ESKAPE pathogens.” Press release

OnlyBoth has launched at new population-health engines that provide detailed comparative insights on specific city neighborhoods across the United States. The 52 Artificial Intelligence-powered engines – one for each state plus D.C. and New England – automatically compare census tracts across 500 cities and discover unique insights spotlighting neighborhood health disparities and specific drivers that impact health outcomes, all for improved community engagement. Population-health comparisons are essential for healthcare providers, payers, and government and local health officials seeking to identify both challenges and achievements in the neighborhoods they serve. As healthcare stakeholders make population health a priority, they need to deeply understand their communities to successfully engage with them and meet their unique demands. Organizations can thus break down the complex goal of improving population health by identifying key interventions in each area and implementing them neighborhood by neighborhood. OnlyBoth has previously launched healthcare engines for comparative analytics and reporting that benchmark thousands of hospitals, nursing homes, dialysis facilities, long-term-care hospitals, inpatient rehabilitation facilities, home health agencies, and hospice agencies nationwide to help achieve greater transparency in healthcare environments. Press release

BioTuring announced its upcoming release of a curated single-cell RNA sequencing database of nearly 5,500,000 cells from 114 publications and projects this September onto BioTuring Single-cell Browser (BBrowser), an interactive and intelligent platform to browse and explore single-cell data. With this release, the company will double the number of publications indexed in its single-cell browser, currently reaching over 60 studies (making up 4,400,000 cells), crossing the number of cells hosted on available public repositories like Human Cell Atlas (HCA) and Broad Institute Single-cell Portal. Out of 5.5 million cells, the database covers a broad range of tissues, diseases, and research fields. Among the indexed datasets are valuable comprehensive atlases such as the Mouse Organogenesis Cell Atlas (MOCA) by Cao et al. (2019) and the Tabula Muris dataset (Tabula Muris Consortium, 2017). Notably, the database includes a list of the most comprehensive sets of data in the immune system, especially in the tumor microenvironment, says the atlas of infiltrating T cells in liver cancer (Zheng et al., 2017), breast cancer (Azizi et al. 2018), basal and squamous cell carcinoma (Yost et al., 2019), making a valuable source for reference and meta-analysis in immuno-oncology research. Press release

Mission Bio announced the launch of Copy Number Analysis, extending Copy Number Variation (CNV) capabilities to its Tapestri Platform with no changes to the current workflow. With this latest capability, Mission Bio’s Tapestri Platform now stands as the first and only commercial platform for single-cell DNA analysis, with comprehensive insight into mutations and CNVs from the same cell on a single platform, expanding the company’s leadership in single-cell DNA analysis for oncology. CNVs, where portions of the chromosome are amplified or deleted, are the cause of many genetic disorders and are present in 45% of cancers. When determining the clonal heterogeneity across a cancer sample, it’s important to comprehensively measure both DNA mutations and copy number alterations in every cell because disease progression and therapy response are based on differences in genetic alterations across cells. This includes both single nucleotide variations (SNVs), which are prognostic and provide insight into the disease development and progression, and CNVs, which tend to predict the severity of the disease. “Access to both SNVs and CNVs at the single-cell level, from the same cell, gives us higher resolution to explore tumor evolution and disease progression,” Samra Turajlic of Francis Crick Institute said in a press release. “We look forward to analyzing these novel data types to help us better resolve the genetic and cellular heterogeneity in cancer.” Mission Bio’s high-throughput single-cell DNA analysis on the Tapestri Platform yields unprecedented insight into the diversity of genomic changes found in individual cancer cells. Moreover, for the first time, CNVs and SNVs can be measured from the same single cell across thousands of cells. With the Tapestri Platform, researchers and clinicians can now leverage a single platform to measure nearly every type of DNA change in every diseased cell. This results in significant cost savings and improved workflow, all while enabling even deeper insight into patterns of tumor evolution, therapy response, and resistance. Press release

The Wyss Institute for Biologically Inspired Engineering at Harvard University and Miraki Innovation have unveiled BOA Biomedical, a company that aims to solve the global healthcare demand for a device that rapidly diagnoses and treats infectious diseases, focusing specifically on sepsis and antibiotic-resistant infections. BOA is a portfolio company founded by Miraki that plans to commercialize technology developed at the Wyss Institute. Earlier this year, BOA obtained investigational device exemption (IDE) from the US Food & Drug Administration to allow the device to be used in a first-in-human clinical study to collect safety and effectiveness data. This approval follows BOA’s exclusive worldwide licensing agreement with the Harvard Office of Technology Development. Press release

Deep Science Ventures (DSV) announced a partnership with Cancer Research UK. Deep Science Ventures was founded to create a better method for launching science companies – one that takes a systems-level view of the opportunity from the start, both at the industry and biological level. Through their partnership with Cancer Research UK, DSV will seek to build at least three companies over the next year. DSV will principally work with Cancer Research UK’s network of early academics while also bringing in researchers and industry experts from related disciplines across the world. Press release

Platelet BioGenesis (PBG) announced that it has raised more than $26 million in Series A-1 financing. This round – in addition to Series A financing and numerous grants – brings PBG’s total funding to approximately $45 million. Ziff Capital Partners (ZCP) and Qiming Venture Partners USA co-led the Series A-1 round, marking the former’s first investment in PBG. Existing investors Nest.Bio Ventures and eCoast Angels also participated in the round, among others. As a part of ZCP’s investment, Steven Altschuler, the company’s managing director of healthcare ventures, will join PBG’s board of directors. Dr. Altschuler is a recognized expert in gene and cell therapy, and chairman of the board of Spark Therapeutics, a company he co-founded while serving as the CEO of Children’s Hospital of Philadelphia. Spark Therapeutics’ advancements have garnered attention across the industry and the company is currently in the process of being acquired by Roche for $4.3 billion. Dr. Altschuler also sits on the board of several other highly innovative biotech companies, including AsclepiX Therapeutics Inc. and ImVaX Inc. PBG will use the funds from the Series A-1 round to accelerate the development of its donor-independent platelet manufacturing capabilities and advance its proof of concept work around platelet-based therapeutics. The company plans to double its staff to approximately 50 employees in 2020. Press release

ThinkParQ announced its global partnership with NetApp. The new partnership will provide an easy to deploy, cost effective and easy-to-manage high performance turnkey solution, that incorporates the NetApp’s E-Series storage (including the new EF600 system) powered by BeeGFS, with enhanced technical support from NetApp. The NetApp E-Series storage with BeeGFS will accelerate workloads and provide customers with consistent, near-real-time access to their data whilst lowering TCO. “We have been working closely with the NetApp E-Series team for some time now to ensure that what we promise is delivered. An innovative, turn-key solution that delivers where performance matters, that is easy to deploy and use, and most importantly, delivers fasters results enabling customers to maximize the value of their data,” Frank Herold CEO of ThinkParQ, said in a press release. BeeGFS transparently spreads your data across multiple servers and their back-end storage to prevent bottlenecks and to enable continuous high performance. The NetApp E-Series storage systems can support a number of workloads simultaneously and can deliver up to 2 million random read IOPS and 24GBps sustained (maximum burst) write bandwidth per scalable building block. Optimized for both flash and spinning media, E-Series systems include built-in technology that monitors workloads and automatically adjusts configurations to maximize performance. As certified and fully trained BeeGFS systems engineers, the NetApp support team will help assess and troubleshoot the overarching BeeGFS deployment, not just the file system. The NetApp experts use proven best practices, tools, and methodologies to guide customers to get optimal performance out of your NetApp and BeeGFS investment.  Press release

The Life Science division of Philip Morris International (PMI) has launched a computational challenge to identify and test new strategies for the diagnosis of Inflammatory Bowel Disease (IBD). The competition, which comes with a prize pool of $12,000, aims to find the best machine learning algorithm that can reliably diagnose IBD using data obtained from non-invasive clinical samples. As well as winning a cash prize, successful participants have the opportunity to collaborate with leading biologists and data scientists, and to publish their findings in peer-reviewed journals. The Metagenomics Diagnosis for Inflammatory Bowel Disease Challenge (MEDIC) challenge is launched through sbv IMPROVER, PMI’s international crowdsourcing platform. Now open for submissions, the challenge asks participants to propose computational methods that can be applied to metagenomics data collected from stool samples to produce accurate IBD diagnoses. Participants train their algorithms using two large, publicly available datasets while a third dataset, obtained from an independent study sponsored by PMI, is used to test the algorithms. Press release