When it comes to the data-driven transformation of healthcare enabled through precision medicine “there is perhaps no more poignant example than the response to the COVID-19 pandemic,” NIH Director Francis S. Collins, M.D., Ph.D., and Joshua C. Denny, M.D., CEO of the All of Us Research Program observed in a commentary published March 18 in the journal Cell. “At the same time, COVID-19 has highlighted the need for precision medicine to move further and faster,” they added.
Collins and Denny followed up by suggesting seven areas or “opportunities” to accelerate the promise of precision medicine more equitably: Big data and artificial intelligence; diversity and inclusion; electronic health records; huge longitudinal cohorts; phenomics and environment; privacy, participant trust, and return of value; and routine clinical genomics.
The precision medicine market is expected to nearly double by 2026, rising to $100.168 billion from $60.422 billion in 2020—a compound annual growth rate of 8.69%, according to ResearchandMarkets.com.
Clinical OMICs last presented a “10 to Watch” list of promising startups in 2019. Below are 10 up-and-coming companies to watch for their ambitious and, so far successful, application of omics technologies and clinical genetic insights to deliver on the promise of precision medicine:
“Arc Bio’s mission is to accelerate the metagenomic revolution in infectious disease by offering the first and only turnkey metagenomics solution with its Galileo ONE platform,” CEO Todd Dickinson told Clinical OMICs.
Galileo ONE combines a next-generation sequencing (NGS) lab workflow with an automated bioinformatics pipeline designed to deliver fast, comprehensive, and quantitative microbial profiling. The research use only (RUO) metagenomic sequencing solution leverages the Illumina NGS platform to deliver sample-to-report detection and quantification of >1300 species of bacteria, fungi, DNA and RNA viruses, and parasites from a primary sample—in less than 30 hours.
Galileo ONE is the culmination of 4+ years of development. In 2019, Arc Bio released a proof-of-concept version called Galileo Viral, a 397-strain panel focused on transplant infection research applications. Galileo Viral showed excellent performance compared to qPCR gold standard testing when used by the research groups of Ben Pinksy, M.D., Ph.D. (Stanford University School of Medicine), Angela Caliendo, M.D., Ph.D. (Alpert Medical School of Brown University) and Judith Breuer, Ph.D. (Great Ormond Street Hospital for Children).
“Based on these promising results, we moved into development and optimization of our expanded metagenomic Galileo ONE platform,” Dickinson said. “We are currently working with early access customers and key strategic partners on benchmarking performance of Galileo ONE in the lead up to our commercial launch slated for mid-year 2021.”
With the debut of Galileo ONE, Arc Bio plans to emerge from quasi-stealth mode this year. Established in 2014, Arc Bio has been funded through parent company EdenRoc Sciences, and generated revenue from Galileo Viral and Galileo ONE early access partners that is expected to double this year.
“As part of our efforts to scale rapidly to meet significant product demand in the next two years, Arc Bio will be fundraising in the coming year,” Dickinson added.
After seven years in the Israel Defense Forces, Asaf Zviran, Ph.D., had found success as an electronics engineer in Israel’s civilian defense sector when he was diagnosed with ependymoma, a rare neurological tumor, at age 28.
Zviran’s cancer is in remission today, but frustration over treatment that often resembles a shot in the dark led him to change careers and earn a Ph.D. in molecular biology at the Weizmann Institute of Science.
As a postdoc in the lab of Dan Landau, M.D., Ph.D., of Weill Cornell Medicine and the New York Genome Center, Zviran worked with Landau to develop a DNA-sequencing approach that could detect very low levels of tumor DNA in blood samples as small as 1-2 ml, potentially enabling early detection of cancer recurrence.
At the heart of the minimal residual disease (MRD) platform, detailed in Nature Medicine, is proprietary AI-powered pattern recognition algorithms that sift through the torrents of data generated by whole-genome sequencing to detect residual cancer in the blood. The fully cloud-based platform is GDPR- and HIPAA-compliant, and deployable to any cancer/genomics lab that uses a standard Illumina sequencer.
Zviran spun out the platform in 2018 into C2i Genomics. “C2i” is military lingo for “command, control, and intelligence,” reflecting his desire to bring defense methodologies to oncology.
“C2i is addressing the expensive and painful problem of over and under-treatment of cancer,” Zviran told Clinical OMICs. “By providing 100x more sensitive cancer detection, C2i Genomics is giving both doctors and patients more certainty on which treatment decision is best, which saves costs, and improves outcomes.”
C2i raised $12 million in Series A financing last year, followed on April 15 with a $100 million Series B led by Casdin Capital, bringing the company’s total financing to $113.5 million.
Deepcell is pioneering an artificial intelligence (AI)-powered platform designed to bring cell morphology into a new era. The platform applies AI with advanced microfluidics and high-throughput imaging to identify, sort, and classify viable cells in any biological sample based on morphological distinctions for basic and translational research.
CEO and co-founder Maddison Masaeli, Ph.D., told Clinical OMICs Deepcell’s platform addresses three cell morphology challenges: A lack of adequate markers for specific applications, the pressing need to improve the cost and complexity of single cell analysis, and new discoveries in cell biology.
“Deepcell has the potential to identify infinitesimal morphological differences and patterns that are hardly accessible by the human eye,” Masaeli said. “Using unsupervised deep learning, Deepcell’s platform cannot only identify unusual patterns or cells within samples, but it also continually improves as results from each analysis are fed back into the brain.”
Using microfluidics-based technology, Deepcell’s platform precisely images single cells in cell suspension and presents them to an imaging device. The imaging system extracts detailed features and feeds them in real time to the Deep Neural Network, which analyzes the images and can profile and classify cells based on morphology features alone.
Deepcell’s platform also addresses technical issues associated with staining and labeling cells, as well as errors in human judgment: “Deepcell is able to accurately isolate cells in a way that does not perturb the cell,” Masaeli said. “This will make cell analysis workflows more efficient and allow researchers to understand the cell at a higher resolution.”
Deepcell plans to offer a cloud service enabling users to analyze their data and train new AI models, while continuously improving existing ones.
To date, Deepcell has raised $25 million, including a $20 million Series A completed in December 2020 and led by Bow Capital, with substantial participation from Andreessen-Horowitz—which led the $5 million seed round.
Mogrify’s immuno-oncology and ophthalmology programs include induced pluripotent stem cell (iPSC)-derived allogeneic cell therapies targeting hematological and solid malignancies, and in vivo reprogramming therapies for retinal degeneration.
“Mogrify’s mission is to transform the development of ex vivo cell therapies and pioneer a new class of in vivo reprogramming therapies for immuno-oncology, ophthalmology and other disease areas,” Pierre-Louis Joffrin, corporate development officer with Mogrify, told Clinical OMICs.
Its MOGRIFY platform enhances existing stem-cell forward reprogramming methods or bypass development pathways altogether, affecting a direct trans-differentiation between a mature cell type to another mature cell type. The epiMOGRIFY extension, enhances directed differentiation or cell conversion to support development of scalable off-the-shelf therapies for diseases with high unmet clinical needs.
Both platforms deploy NGS, gene regulatory, and epigenetic network data to enable prediction of the transcription factors (or small molecules) and optimal culture conditions required to produce any target human cell type from any source human cell type.
Since launching in 2019, it has raised over $37 million from Parkwalk, Ahren Innovation Capital, 24Haymarket, Darrin M. Disley, Ph.D., Jonathan Milner, Ph.D., the University of Bristol Enterprise Fund III, and strategic investors Astellas Venture Management.
Last year, Sangamo Therapeutics became Mogrify’s second U.S. biopharma partner by applying Mogrify’s proprietary iPSCs and embryonic stem cells to accelerate the development of scalable and accessible zinc finger protein gene-engineered chimeric antigen receptor regulatory T cell (CAR-Treg) therapies for patients with inflammatory and autoimmune diseases.
A cancer diagnosis can overwhelm patients and their families with information that may be unclear and even inaccurate. Despite rapid advances in cancer care, 25% of patients said they were not getting the most advanced treatments, according to the 2016 CancerCare Patient Access and Engagement Report, and 80% were not informed about clinical trials options. Also, less than 30% of advanced breast cancer patients said they were undergoing genomic testing that could improve their treatment outcomes, according to study findings by Outcomes4Me Inc., a Cambridge, MA, health tech company.
Outcomes4Me seeks to address the cancer knowledge gap and improve health outcomes by empowering patients with understandable, relevant, and evidence-based information. The company has developed a direct-to-consumer artificial intelligence (AI)-powered patient empowerment platform for shared decision-making between patients and providers.
The platform harnesses regulatory-grade, real-world data and patient experiences to generate deeper insights that improve care, accelerate research, and achieve better outcomes.
“In order to truly improve cancer outcomes, we need to transition from an episodic approach to patient management to a continuum experience that anticipates patients’ needs and provides timely, personally specific, and actionable information. This is exactly what the Outcomes4Me platform does,” Maya R. Said, Sc.D., the company’s founder and CEO, told Clinical OMICs.
“The platform’s AI-powered personalization unlocks an adaptive experience that empowers patients to stay informed, understand their treatment options, find clinical trials, manage their quality of life, and ultimately advocate for themselves,” Said added.
The company’s key collaborations include the National Comprehensive Cancer Network (NCCN), Wolters Kluwer, Massachusetts General Hospital, Vanderbilt Ingram Cancer Center, and Foundation Medicine. Outcomes4Me completed a $12 million Series A round in April, bringing its total financing raised to $16.7 million.
Phosphorus says its mission is to extend and improve lives by making genomics a foundational part of everyone’s health and wellness journey.
“While much is known about the power of genomics to positively affect health and medical management decisions, testing is still underutilized,” said Gabriel Lazarin, VP of Medical Affairs at Phosphorus. “And, when testing is done, it is often performed after a diagnosis, which misses the opportunity for earlier interventions.”
To address this, Phosphorus created GeneCompass, a genomic screen that offers what it calls the most comprehensive and clinically actionable assessment of genetic health for identifying and preventing risk for genetic conditions, as well as a pharmacogenetic drug-response assessment.
GeneCompass Plus analyzes 440 genes covering 301 monogenic conditions and disease susceptibility areas and 126 different drug reactions in conditions that include cancer, cardiology, infertility, lipidology/emias, and metabolic disorders.
GeneCompass also enables hospitals, health systems, and physician-owned laboratories to insource the technology via its Managed Lab Service. Lazarin said Phosphorus’ approach incentivizes and empowers healthcare providers to fully own the patient care continuum.
“We provide the expertise to organizations so that they can build the same accurate and cost-efficient test for their practice, solving the last barrier to routine integration of genomics into everyday care,” Lazarin said.
Phosphorus is based in New York City with a CLIA and CAP-certified laboratory in Secaucus, NJ.
“At Proscia, we’re accelerating pathology’s shift from analog to digital, enabling life sciences organizations, health systems, and laboratories around the world to make this transition,” Mike Bonham, M.D., Ph.D., Proscia’s CMO, told Clinical OMICs.”
Proscia says its Concentriq platform sits at the intersection of digital and computational pathology, marrying enterprise scalability with powerful artificial intelligence (AI) applications. It is designed to allow organizations to manage their pathology practice, including ingesting, viewing, managing, analyzing, and sharing images from any scanner. Concentriq can also serve as an AI launchpad, enabling laboratories to seamlessly deploy computational applications at scale.
Proscia partners with leading academic and commercial laboratories to develop and validate AI applications across laboratory settings—including Johns Hopkins University; University of California, San Francisco; UMC Utrecht; LabPON; Unilabs; University of Florida; and Thomas Jefferson University Hospitals.
With key industry partners, Proscia offers a joint solution with Visiopharm uniting image analysis and image management, enabling research organizations to better leverage computational data at scale. Proscia is also integrating Ibex’s Galen Prostate application into Concentriq to deliver AI-powered triaging, cancer detection, and grading of prostate core needle biopsies into routine workflows to drive efficiency and quality improvements.
The Joint Pathology Center selected Concentriq to modernize the world’s largest repository of human tissue data dating back to the Civil War. Proscia’s other marquee customers include the University of Pennsylvania, NASA’s Jet Propulsion Laboratory, and a consortium of the NIH’s National Cancer Institute (NCI) researching cancer overdiagnosis.
Jonathan Rothberg, Ph.D., disrupted next-generation sequencing in 2000 when he founded 454 Life Sciences, and later invented semiconductor chip-based sequencing, and founded companies that include CuraGen, Clarifi, RainDance Technologies, LAM Therapeutics, Hyperfine Research, and Butterfly Network.
Another Rothberg-founded company created through his 4Catalyzer startup accelerator, Quantum-Si, has created the first next-generation protein sequencing platform, designed to provide a full solution spanning from sample preparation to sequencing and data analysis with the goal of revolutionizing proteomics.
“Our platform has the potential to enable users to study the proteome in an unbiased and scalable way, similar to the manner in which next-generation DNA sequencing technologies transformed genomics analysis,” Quantum-Si CEO John Stark told Clinical OMICs. “Our goal is to provide broad access to proteomics tools across academic research labs, core labs, and biopharma R&D labs through our end-to-end workflow solution and proprietary semiconductor chip.”
Quantum-Si’s platform consists of the Carbon automated sample preparation instrument, the Platinum next-generation protein sequencing instrument, the Quantum-Si Cloud software service, and reagent kits and chips for use with their instruments. The company’s proprietary semiconductor chip is designed to read proteins at the single molecule level—specifically amino acids.
Quantum-Si expects its benchtop Carbon and Platinum instruments to cost approximately $50,000 combined—compared with legacy instruments like mass spectrometers, which can cost $250,000 to over $1 million per new instrument and require specialized training.
“We believe that the affordability and simplicity of our single molecule detection platform will provide users the opportunity to perform proteomics studies at scale,” Stark added.
Sense Biodetection more than tripled its total capital raised in April, when it completed a $50 million Series B financing. The molecular diagnostics developer, which has now raised more than $70 million in financing, plans to use the proceeds to accelerate the launch of its Veros™ COVID-19 test, and further develop a portfolio of instrument-free, rapid molecular tests.
“There is an enormous cost to healthcare due to inaccurate or untimely diagnostic testing,” Ryan Roberts, chief commercial officer with Sense Biodetection, told Clinical OMICs. “While it has been true for years, COVID-19 has brought it to light in a dramatic way: rapid testing can be unreliable, especially for infected but asymptomatic patients. Conversely, the gold-standard PCR lab-run test requires centralized machine processing which can take days. We see a significant opportunity for a highly accurate, rapid test that is easy to use, instrument-free and disposable.”
The Veros COVID-19 test will be based on Sense’s Veros platform, designed to detect a variety of deadly and costly diseases using nucleic acid amplification and non-fluorescent color detection of amplified analytes.
The platform is intended to underpin a new class of rapid molecular diagnostic tests that emulate the performance of central laboratory PCR testing, but are disposable and easy to use since they do not need an accompanying instrument or reader. As a result, Sense says, Veros diagnostics can be used beyond traditional healthcare settings—enabling better access, outcomes, and value for patients and providers.
Koch Disruptive Technologies, a subsidiary of Koch Industries, led the Series B financing, with participation from Sense’s existing investors Cambridge Innovation Capital, Earlybird Health, Jonathan Milner, and Mercia Asset Management.
Based in Abingdon, U.K., Sense was founded in 2014. Five years later, it raised £12.3 million ($17.2 million) in Series A financing, with the proceeds intended toward test development.
StrideBio is developing next-generation genetic medicines, initially targeting rare central nervous system and cardiovascular disorders in patients whose underlying disease cause is not addressable by traditional approaches.
The company’s adeno-associated viral (AAV) vectors deliver a genetic payload that results in expression of a healthy version of a specific gene that is not functioning correctly in patients, providing a one-time treatment with life-changing or curative potential.
Its pipeline of independent and partnered programs includes treatments for Friedreich’s ataxia, Niemann-Pick disease type C, Rett syndrome, Dravet syndrome, Angelman syndrome and alternating hemiplegia of childhood.
“Our platform technology is focused on rational engineering of the AAV capsid or shell that carries the genetic payload being delivered to target tissues and cells in a patient,” StrideBio CEO Sapan Shah, Ph.D., told Clinical OMICs. “StrideBio’s STRIVE platform utilizes the 3D structure of the AAV capsid to identify regions that can be modified to incorporate novel properties including evasion of neutralizing antibodies, specific tissue targeting or de-targeting, enhanced potency and manufacturability at scale.”
In Research Triangle Park, NC, StrideBio is building an integrated gene therapy product engine including capsid and genetic construct design, in-house manufacturing at 1000L scale, and clinical development capabilities.
StrideBio has raised $97.2 million in two equity financings, most recently a $81.5 million Series B round. It has collaborations with key partners including CRISPR Therapeutics, Takeda Pharmaceuticals, and Sarepta Therapeutics which have brought in approximately $70 million in upfront non-dilutive funding—with potential for more than $2 billion in milestone-based payments.
“We are inspired by the dramatic progress the field of gene therapy has made in recent years and believe our STRIVE platform is uniquely positioned to enable improved next-generation AAV based therapies for patients who desperately need them,” Shah added.