PacBio’s Precision Health Partnership, NVIDIA’s Open Source SDK, More

December 22, 2021 | Singular Genomics launches new benchtop sequencer, collaborations in neuropsychiatry, lactation research, skin health, chronic pain, and SARS-CoV-2 vaccines, Insilico Medicine announces first-in-human trial of an AI-developed drug. Plus new products from PerkinElmer, Thermo Fisher, Genomenon, and more.

BioLife Solutions has announced a partnership with UNICEF to extend thermal stability of temperature-sensitive COVID-19 vaccines in developing communities in Africa, the Middle East and South America where limited power infrastructure and extreme environmental conditions make it difficult for most ULT freezers to remain operational. As part of this program, UNICEF has approved Stirling's upright SU780XLE freezers and first-of-its-kind, portable UL25NEU freezer models for country specific selection. Press release.

Bruker has released the JPK NanoWizard V BioAFM, a novel system that marks a milestone in automation and ease of use for life science atomic force microscopy research. NanoWizard V is a very fast, automated BioAFM that can optionally be fully integrated with advanced optical microscopes. It enables rapid, quantitative mechanical measurements and the analysis of dynamics on samples ranging in size from sub-molecular to cells and tissues. The automated setup, alignment and re-adjustment of system parameters opens new possibilities for long-term, self-regulating experiments on mechanobiological dynamics. Press release.

AI startup Owkin and Amgen have announced the results of a three-year project to use artificial intelligence to more accurately predict cardiovascular risk. This study demonstrates the ability of AI to improve the way that clinicians predict patients' risk of suffering major cardiovascular events, such as strokes and myocardial infarctions. Using data from 13,756 patients who were part of FOURIER, a large Amgen clinical trial, Owkin trained a machine-learning algorithm to predict those at higher risk of cardiovascular events. Published in the upcoming December issue of the European Heart Journal - Digital Health, the results show that machine learning is more efficient and effective than the statistical models routinely used by clinicians. Press release.

Singular Genomics Systems has launched the G4, what they call the world’s most powerful benchtop sequencer. The NGS platform features novel high-performance chemistry and advanced engineering to deliver accuracy, flexibility, speed and power for a range of applications, including research in oncology and immunology. Orders for the G4 instrument and consumable kits are now being accepted, with shipments expected to begin in the second quarter of 2022. The G4 Sequencing Platform consists of a benchtop NGS instrument and associated consumables powered by a novel sequencing engine to deliver accuracy, proprietary, 4-color sequencing by synthesis (SBS) chemistry with novel enzymes and nucleotides that work together to provide highly accurate paired-read sequencing; flexibility, ability to independently run up to 16 samples or sample pools across four flow cells, the G4 reduces sample pooling requirements and offers flexibility to run multiple experiments in parallel; speed, innovative high-resolution imaging, rapid fluidics and novel high-speed chemistry work in concert to reduce run times from days to hours; and power, with up to three times more data output per hour than any other benchtop instrument, the G4 will sequence up to four human genomes in just 16-19 hours. Press release.

The Forever Healthy Foundation and the Buck Institute for Research on Aging have announced a new partnership to advance early-stage discoveries at the Institute that show promise to reverse physiologic aging in humans. The focus will be on cutting-edge research aimed at repairing age-related damage at the cellular and molecular level, a hallmark of the aging process. Forever Healthy will commit up to $1 million per year for five years to drive this innovation. The funding aims to advance early-stage research with high translational potential in order to speed up the transition from lab to product. Press release.

Ichnos Sciences and Almirall, a Barcelona-based biopharmaceutical company focused on skin health, have entered into an exclusive licensing agreement for the IL-1RAP antagonist ISB 880. Under the agreement, Almirall is granted global rights to develop and commercialize this monoclonal antibody for autoimmune diseases. Ichnos will retain rights for antibodies acting on the IL-1RAP pathway for oncology indications. Within the terms of the agreement, Almirall will assume full cost and responsibility for the global development and commercialization of the compound for all autoimmune disease indications. Ichnos will receive an upfront payment of €20.8 million as well as additional development and commercial milestone payments and tiered royalties based upon future global sales. Press release.

Metabolon will collaborate with Brigham and Women’s Hospital and Beth Israel Deaconess Medical Center (BIDMC) in a first-of-its-kind Lactation Lab, providing comprehensive metabolomic profiles to enhance understanding of the biology underlying lactation to identify solutions to improve maternal and infant health. The value of this work is two-fold. In the short-term, the findings will generate fundamental knowledge required for the discovery of interventions to improve maternal and infant health outcomes. In the long-term, the development of the Lactation Lab will provide a model for discovery in the field of lactation science, bringing much-needed scientific rigor to investigations during this critical period that influences the lifelong health of two generations. Press release.

Cerevel Therapeutics and Herophilus have announced a phased multi-year, joint research collaboration in neuropsychiatry. The collaboration integrates the deep expertise in neuroscience drug discovery at Cerevel Therapeutics with state-of-the-art disease models and the AI-enabled deep phenotyping from Herophilus. Herophilus’ proprietary neuroimmune patient-derived brain organoids and neuroimmune genetically engineered brain organoids are designed to overcome the limitations of traditional animal models by incorporating human-specific genes with known relevance to schizophrenia. Leveraging these advanced in vitro humanized systems enables the potential discovery, evaluation, and prioritization of novel therapeutic strategies that may be missed through standard approaches. Press release.

ArisGlobal has announced the go-live of the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS II), an electronic safety reporting platform, powered by ArisGlobal’s LifeSphere MultiVigilance. The successful go-live marks a key milestone in the FDA's Technology Modernization Action Plan, and the culmination of its efforts to adopt a cutting-edge platform to automate the intake and processing of adverse event reports. Designated a modernization priority by the United States Department of Health and Human Services, FAERS II is a mission critical platform for the FDA's Centers of Drug and Biologic Evaluation and Research (CDER/CBER), providing a modern, all-in-one solution for collection, triage and processing of adverse event reports. With safety case volumes growing amid a global public health crisis, the platform will leverage the flagship safety product of ArisGlobal's LifeSphere R&D technology suite, LifeSphere MultiVigilance, to increase efficiency and improve data quality. A proven and scalable solution already adopted by many of the largest global biopharmaceutical companies, LifeSphere MultiVigilance is the only safety system in production that uses AI and machine learning to automate the end-to-end processing of adverse event reports in the industry for market authorization holders. Press release.

CEPI, the Coalition for Epidemic Preparedness Innovations, and Affinivax have announced a partnership to advance the development of a novel vaccine that could provide broad protection against SARS-CoV-2 variants, and potentially other betacoronaviruses. CEPI will provide funding of up to $4.5 million to support the initial development of a vaccine candidate based on Affinivax’s innovative Multiple Antigen Presenting System (MAPS) technology platform. CEPI’s investment in Affinivax will fund vaccine candidate design and selection, manufacturing process development and preclinical testing against specific SARS-CoV-2 variants. The collaborative program aims to establish preclinical proof of concept for a novel vaccine candidate designed to protect against new COVID-19 variants and that is also suitable for use in low- and middle-income countries. CEPI will evaluate the potential for further investment in the development of this vaccine candidate based on the data derived from this initial partnership with Affinivax. Press release.

IDBS and Synthace have entered into a strategic partnership to provide IDBS Polar users with state-of-the-art data foundations for experimentation in high-throughput process development (HTPD). Synthace’s cloud-based automation platform augments Polar’s HTPD capabilities by automating the capture, structuring and enrichment of the millions of data points generated per experiment across disconnected bioreactors and analytical instruments in the bioprocessing laboratory. Polar HTPD, powered by Synthace, helps bioprocess development teams adopt faster throughput approaches and significantly shorten timelines by supporting better decision making with more reliable data sets, rich data analysis and modern enterprise data management. This combined solution enables users to confidently scale up to hundreds of automated, parallelized experiments per month. Press release.

Optibrium has announced the publication of a peer-reviewed study in the Journal of Computer-Aided Molecular Design, “Imputation of Sensory Properties Using Deep Learning”. In collaboration with International Flavors & Fragrances (IFF), a global leader in co-creating sensorial experiences, and Intellegens, an AI company, the team applied Optibrium’s Augmented Chemistry platform to predict human panel-based assessments, including sensory properties, such as odor intensity and odor detection threshold. The study demonstrated the platform’s high predictive reliability and the opportunity to reduce the need for human testing when developing new flavor and fragrance ingredients, opening the potential for cost and time savings. The study also reinforced the translational ability of the method for related applications, including offering new insights into drug discovery data to identify opportunities for new drugs and streamline the drug discovery process. Press release.

PerkinElmer announced the research use only (RUO) launch of the NEXTFLEX Variant-Seq SARS-CoV-2 Kit v2 to accelerate the detection of SARS-CoV-2 variants. This complete next generation sequencing (NGS) solution will allow laboratories to increase sequencing throughput and make reliable variant identifications. Using positive SARS-CoV-2 samples, the NEXTFLEX Variant-Seq SARS-CoV-2 Kit v2 detects mutations associated with all SARS-CoV-2 variants, including the latest Variant of Concern (VOC) identified by the World Health Organization, Omicron (B.1.1.529). This kit offers substantial benefits to laboratories in terms of speed, scalability and cost savings by incorporating newly developed NEXTFLEX normalization beads, which reduces turnaround time by three hours on average for 96 samples (when compared to other standard library prep kits that require quantification and normalization of individual samples before pooling). Additionally, the NEXTFLEX Variant-Seq SARS-CoV-2 Kit v2 uses 1,536 Unique Dual Index (UDI) barcodes to enable the sequencing of 6,000 SARS-CoV-2 libraries in one flow cell. This provides extremely high throughput and minimizes per sample costs associated with sequencing. Press release.

PacBio and the UCLA Institute for Precision Health and David Geffen School of Medicine at UCLA have formed a research collaboration to further identify the causes of rare diseases. The study will leverage PacBio’s HiFi long-read sequencing technology for whole genome sequencing (WGS) to look at undiagnosed pediatric rare disease patients who have already been sequenced with short-read technology. Dr. Stanley Nelson, Director, California Center for Rare Diseases, and professor, pathology and laboratory medicine and human genetics, David Geffen School of Medicine at UCLA, will be pioneering the combined use of full-length isoform sequencing (Iso-Seq) and long-read WGS in an effort to investigate the effect on diagnostic yield in these unresolved cases. Press release.

Thermo Fisher Scientific has introduced a new protein to complement its growing portfolio of CRISPR genome editing solutions. For research applications requiring highly precise genome editing, including engineering CAR T cells and creating cellular models for disease discovery, the Invitrogen TrueCut HiFi Cas9 Protein significantly minimizes off-target events while retaining maximum on-target editing efficiency. CRISPR genome editing is inherently messy, often resulting in edits in unintended locations. Limiting off-target effects is critical for applications such as translational research, drug screening and drug modeling as researchers look down the road to future therapeutic development. The Invitrogen TrueCut HiFi Cas9 Protein demonstrates near complete elimination of off-target effects compared to wild-type Cas9 in several cell types, including T cells and induced pluripotent stem cells (iPSCs), and demonstrates improved off-target profiles relative to other products on the market. Press release.

Genomenon has announced the new Mastermind Search Companion, a Google Chrome browser plug-in for use with 14 variant databases, Google, and Google Scholar. The free extension, available through the Chrome Web Store to all Google Chrome users enables deeper insight into genetic evidence related to searches across a wide range of variant databases used by geneticists, genetic counselors, variant scientists, and researchers. Mastermind Search Companion seamlessly integrates Mastermind’s insight into the scientific literature with the online resources variant analysts already use. Because Mastermind has 100X more genomic evidence than any other source, the extension provides in-platform access to additional genetic data for genes and variants searched on any of the following platforms: ClinVar, HGMD, dbSNP, old dbSNP, OMIM, COSMIC, CIViC, UCSC Browser, Google Scholar, Google, VarSome, GeneCards, NCBI, Gnomad, SNPedia, and OncoKB. Users of the extension will find an optimized visual display with an interactive sliding drawer tab which contains helpful information and direct links to the prioritized genomic evidence within Mastermind. This allows analysts to quickly navigate their standard search sites and expand them with Mastermind search results. Mastermind Search Companion delivers the ability to get more insight within any existing process and set of databases. Press release.

Insilico Medicine has announced that the first healthy volunteer has been dosed in a first-in-human microdose trial of ISM001-055. ISM001-055 is a potentially first-in-class small molecule inhibitor of a novel biological target discovered by Pharma.AI, Insilico Medicine’s end-to-end AI-powered drug discovery platform. It is being developed for the treatment of idiopathic pulmonary fibrosis (IPF), a chronic lung disease that results in progressive and irreversible decline in lung function. After completing IND-enabling studies, Insilico Medicine has initiated the microdose trial to begin characterizing the pharmacokinetic profile in humans. The trial, administering ISM001-055 intravenously in healthy volunteers, is being conducted in Australia. ISM001-055 demonstrated highly promising results in multiple preclinical studies including in vitro biological studies, pharmacokinetic and safety studies. The compound significantly improved myofibroblast activation which contributes to the development of fibrosis. ISM001-055’s novel target is potentially relevant to a broad range of fibrotic indications. Press release.

A new company spawned by McMaster University innovation in the arena of vaccine manufacturing has received a grant from the Bill & Melinda Gates Foundation. Elarex Inc., based in Burlington, has been awarded a $1.2M Canadian grant from the non-profit humanitarian foundation to develop a new technology for keeping liquid mRNA vaccines safe and viable without the deep-freeze that is necessary today for storing and transporting such vaccines. Maintaining the “cold chain” from the point of manufacture through the administration of single doses is necessary, but it presents a cumbersome, resource-intense and expensive barrier to the equitable global distribution of vaccines, particularly in developing countries. A technology breakthrough in improving the thermostability of mRNA liquid nanoparticles in liquid form would enable better use and distribution of mRNA vaccines in low- and middle-income countries. Press release.

NVIDIA is making it easier than ever for researchers to harness federated learning by open-sourcing NVIDIA FLARE, a software development kit that helps distributed parties collaborate to develop more generalizable AI models. Federated learning is a privacy-preserving technique that’s particularly beneficial in cases where data is sparse, confidential or lacks diversity. But it’s also useful for large datasets, which can be biased by an organization’s data collection methods, or by patient or customer demographics. NVIDIA FLARE—short for Federated Learning Application Runtime Environment—is the engine underlying NVIDIA Clara Train’s federated learning software, which has been used for AI applications in medical imaging, genetic analysis, oncology and COVID-19 research. The SDK allows researchers and data scientists to adapt their existing machine learning and deep learning workflows to a distributed paradigm. Making NVIDIA FLARE open source will better empower cutting-edge AI in almost any industry by giving researchers and platform developers more tools to customize their federated learning solutions. Press release.

Standigm has signed a Memorandum of Understanding ("MOU") with Institut Pasteur Korea ("IPK"), the infectious disease-focused research institute contributing to public health, for AI-based drug discovery research for infectious disease. The MOU signing took place at the headquarters of Standigm, attended by Jinhan Kim, CEO of Standigm, and Youngmee Jee, CEO of IPK. The endeavor combines Standigm's AI drug discovery platform and IPK's research expertise in infectious disease. Under the MOU, the two parties will actively conduct joint research and academic exchanges to derive innovative drug candidates for infectious disease, setting the research priority on discovering novel anti-tubercular drug candidates. Previously, the two parties conducted research collaboration on anti-tubercular drug discovery, applying Standigm's deep learning technology to the data generated through IPK's cell-based screening of one hundred thousand compounds against tuberculosis. As a result, hit compounds with several key scaffolds effective in multidrug-resistant and extensively drug-resistant tuberculosis (MDR/XDR-TB) were identified. Press release.

Albert Einstein College of Medicine and Montefiore Health System have been awarded a National Institutes of Health (NIH) grant to establish a multidisciplinary research center focused on treatments for people with both chronic pain and opioid use disorder (OUD). The Integrative Management of Chronic Pain and OUD for Whole Recovery Research Center at Montefiore Einstein (IMPOWR-ME) will receive $5.1 million for its first two years; additional funding at the same level is expected for the following three years. The grant is part of the NIH's Helping to End Addiction Long-term Initiative, or HEAL Initiative, which aims to accelerate the pace of scientific solutions to stem the national opioid public health crisis. Press release.