Replica Analytics | June 30, 2022
Critical Path Institute and Replica Analytics, an Aetion company, announced a new partnership that involves leveraging synthetic data to further catalyze the generation of actionable solutions to accelerate drug development for rare diseases.
Replica Analytics will help generate synthetic datasets across rare and orphan indications in which patient-level datasets are often quite small, which heightens considerations regarding data privacy and accessibility.
Together with real data, these synthetic data will help maximize the utility of C-Path's Rare Disease Cures Accelerator-Data and Analytics Platform which provides a centralized and standardized infrastructure to support and accelerate rare disease characterization targeted to accelerate drug development. Additionally, the platform includes a framework that supports the rigorous conduct of natural history studies, with attention to established data quality standards, to be most useful to clinical trial design and regulatory review. It includes a robust, integrated database and analytics hub that allows for the aggregation of rare disease data from various sources and the efficient and effective interrogation of that data. Once added to RDCA-DAP, the synthetic data will also represent valuable real-world, electronic health record (EHR) based data available on the platform, opening doors to modeling and research based on longitudinal focused analyses.
"Our collaboration with Replica Analytics is important because synthetic data can add value to real-world data while observing data privacy considerations, which will help accelerate overall rare disease drug development. The goal of RDCA-DAP is to provide a centralized and standardized infrastructure to support and accelerate rare disease characterization and therapy development, this collaboration is in line with our efforts."
RDCA-DAP Scientific Director Alexandre Betourne, Pharm.D., Ph.D
Synthetic data generation is a privacy enhancing technology that has been gaining rapid adoption, particularly in the life sciences sector. SDG uses AI to create machine learning models that learn the statistical patterns and properties of real datasets to generate data that retain the same characteristics as the original dataset, but with no one-to-one mapping back to an identifiable person. SDG can help amplify small datasets, simulate virtual patients to augment patients in existing datasets, and optimize the design of small sample clinical trials.
"We are certainly seeing a growing opportunity to partner with organizations like C-Path for generating datasets that are fit-for-purpose," says Khaled El Emam, Ph.D., SVP and GM of Replica Analytics. "Synthetic data, which preserves the integrity and utility of source data, as well as being privacy-protective, can be a very valuable tool to enable this collaboration."
In May 2022, C-Path hosted a webinar in which the Replica Analytics team introduced SDG and discussed the role the technology can play in researching and treating rare diseases. The event was recorded and can be viewed on C-Path's YouTube channel.
Critical Path Institute is an independent, non-profit organization established in 2005 as a public and private partnership. C-Path's mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations.
Critical Path Institute is supported by the Food and Drug Administration of the U.S. Department of Health and Human Services and is 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government sources, totaling $11,196,634. The contents are those by the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.
About Replica Analytics, an Aetion company
Replica Analytics is the premier science-based SDG technology provider to the healthcare industry. The company a pioneer in the development of unique technologies for generating privacy-protective synthetic data that maintain the statistical properties of real-world data. The company was acquired in late 2021 by Aetion, the leading regulatory-grade real-world evidence technology provider. Replica Synthesis software provides a full suite of synthetic data generation and evaluation capabilities that can solve multiple grand challenges facing the life sciences industry, and health research in general.
Aetion is a healthcare analytics company that delivers real-world evidence for the manufacturers, purchasers, and regulators of medical treatments and technologies. The Aetion Evidence Platform® analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on safety, effectiveness, and value. Founded by Harvard Medical School faculty members with decades of experience in epidemiology and health outcomes research, Aetion informs healthcare's most critical decisions—what works best, for whom, and when—to guide product development, commercialization, and payment innovation.
Cosán and Genomind | July 20, 2022
Cosán, a provider of technology-driven preventative care services for older adults and the physicians that support them, and Genomind, a leading precision healthcare company, have partnered to fill an existing gap in aging adult care management by providing Cosán’s network of physicians with Genomind’s integrated precision medication management platform. The platform will provide insights that will help Cosán to achieve the best outcomes for their patient population, prevent adverse drug events and reduce avoidable costs of medication mismanagement.
With over 40% of U.S. adults 65 years of age and older taking five or more prescription medicines, aging populations are at high-risk for adverse drug events. Through this partnership, Genomind’s analytic capabilities will provide Cosán with risk stratification insights, enabling Cosán clinicians to target individuals that are most likely to benefit from medication reviews and pharmacogenetictesting, also known as drug-gene testing. Genomind’s PGx test adds a layer of precision medicine that leverages genetics to help providers select appropriate medications and dosing. Equipped with advanced pharmacogenetic screening, clinical concierge services and access to expert pharmacists, Cosán’s care teams can then incorporate individualized insights into each patient’s chronic care management plan so medications are administered safely.
“This partnership brings a new level of pharmacy and genetic expertise to the thousands of providers and the patients they support, marking yet another stride in the delivery of effective preventative care management services. These unique drug analytic and genetic testing capabilities provide Cosán’s network of physicians and care coordinators with another tool designed to help eliminate avoidable drug risks and improve overall care quality for vulnerable patients receiving care from their homes.”
David Hunt, Founder & President at Cosán
According to the CDC, adverse drug events cause approximately 1.3 million emergency department visits each year, and older adults are nearly seven times more likely to end up hospitalized after such events than younger persons. Many of these adverse events are caused by just a few drugs. This new platform will help physicians develop personalized therapeutic approaches for patients based on their individual genetic makeup and are designed to improve patient and physician satisfaction and reduce medical expenses. With the recent expansions by Medicare to cover PGx testing in certain circumstances, this population is particularly suited to dramatically benefit from this platform.
“The prevalence and cost of medication mismanagement is unacceptable, costing thousands of lives and billions of dollars annually. We’re thrilled to join Cosán in helping to improve care management for older adults, who are at high risk for these adverse drug events,” said Cynthia Kilroy, Co-CEO, Genomind.
About Cosán Group
Cosán Group, established in 2015, is an industry-leading healthcare organization creating new pathways to modern aging with technology-driven preventative care services, offering concierge home care for older adults. Early market exposure in the delivery of technology and services to support the Chronic Care Management (CCM) program, and Behavioral Health Integration (BHI) programs with Remote Patient Monitoring (RPM) has propelled Cosán to deliver a practice and patient-centric approach to remote care coordination.
Genomind is a leading precision health company focused on optimizing the treatment experience for individuals and healthcare providers through actionable genetic insights and innovative health technology. Genomind’s platform integrates precision health technology and pharmacogenetic testing on an individual and population level beyond mental health. Built on the foundation of compassionate care and clinical expertise, Genomind is empowering a more targeted and personalized approach to medication management and supporting providers in a new standard of care.
Septerna | July 29, 2022
Septerna, a biotechnology company discovering and advancing novel small molecule medicines targeting G protein-coupled receptors announced the addition of Liz Bhatt, MS, MBA, as Chief Operating Officer, and Ran Xiao as Vice President of Finance and Business Operations to its leadership team. Septerna launched in January 2022 and is growing a pipeline of differentiated products using its GPCR Native Complex™ platform to unlock the vast untapped potential of GPCR drug targets for a wide range of diseases.
We are thrilled to welcome Liz and Ran as we continue to build our exceptional team. Together, they bring deep experience in corporate, commercial, and business strategy. They also have significant operational expertise building and supporting both private and public companies,” said Jeffrey Finer, MD, PhD, Chief Executive Officer and Co-founder of Septerna and Venture Partner at Third Rock Ventures. “Since our launch in January, we’ve made rapid progress advancing our platform and early discovery programs, and now expanding our focus to strategic growth and operational excellence is absolutely critical so that we can fully realize the untapped opportunity space for new GPCR medicines.”
“Septerna launched with the ambitious plan to expand the GPCR target landscape by developing and industrializing new drug discovery technologies and processes that can convert this target-rich superfamily of proteins into impactful new therapies for patients across a range of therapeutic areas. I’m excited to be working with a team of talented GPCR scientists and drug hunters and look forward to using my experience leading teams and developing corporate and product strategies to build Septerna into a robust company poised for long-term growth.”
Liz Bhatt brings more than 25 years of strategy, deal-making and company-building experience across a range of biotech and pharmaceutical companies. Liz was previously Chief Business & Strategy Officer at Applied Molecular Transport where she oversaw corporate development, portfolio and project management, and commercial strategy. Prior to AMT, Liz was Chief Operating Officer at Achaogen where she held various strategic and operational roles. Liz spent over a decade at Gilead Sciences as VP, Corporate Development leading deals across all therapeutic areas and as Senior Director, Commercial Strategy leading long-term global commercial and strategic planning, and supporting the launch of several products. Liz also held management roles at Eli Lilly and Maxygen and started her career in research at Affymax. She currently serves as an independent director on the board of eFFECTOR Therapeutics. Liz earned a BA in chemistry from Pomona College, an MS in biomedical sciences from the University of California San Diego, and an MBA from the Kellogg School of Management at Northwestern University.
Ran Xiao has more than 20 years of experience in corporate accounting, finance, and business operations in the biotech industry. Prior to Septerna, Ran was the VP of Finance and Corporate Controller at Ambys Medicines, Inc, where she supported the company launch and managed various general and administrative functions through a rapid growth period. Before joining Ambys, Ran was the Corporate Controller at Corvus Pharmaceuticals, where she played a key role in the company’s initial public offering. Prior to Corvus, Ran held various managerial positions at Intermune, Natus Medical, Celera Genomics and CV Therapeutics. Ran has a BS in accounting from Shanghai University of Finance and Economics and an MBA from the Illinois Institute of Technology. Ran is a Chartered Financial Analyst.
G protein-coupled receptors are the largest and most diverse family of cell membrane receptors, and humans have hundreds of different GPCRs, each involved in controlling specific biological functions. GPCRs on the surface of each cell bind a wide range of external signaling molecules from throughout the body, and the GPCR transmits the signal across the cell membrane to drive internal cellular mechanisms. GPCRs have been widely studied as drug targets and are the largest family of proteins targeted by approved drug products. An estimated 700 approved drugs target GPCRs, representing approximately one-third of all currently approved drugs. Despite the pharmacological success of GPCRs as a drug class to date, the large majority of potential therapeutic GPCR targets remain undrugged.
Septerna, Inc., is a biotechnology company creating broad new drug discovery opportunities across many disease areas for the abundant drug target class of G protein-coupled receptors. The company’s Native Complex™ Platform recapitulates GPCRs with their native structure, function, and dynamics outside of the cellular environment to enable new technologies for industrial-scale drug discovery for the entire GPCR target class for the first time. Septerna has an emerging pipeline of GPCR-targeted small molecule drug discovery programs, along with growth potential to reach many GPCRs that have been undruggable and unexploited to date. Septerna was launched in 2022 by scientific founders who have made groundbreaking GPCR discoveries and by founding investor Third Rock Ventures.