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.
Iktos | June 22, 2022
Iktos, a company specialized in Artificial Intelligence for new drug design, announced a research collaboration with Zealand Pharma A/S a biotechnology company changing lives with innovative peptide-based medicines to co-develop generative and predictive AI technologies for peptide drug design.
Iktos’ AI technology, based on a comprehensive data-driven chemical structure generation technology, brings new insights into the drug discovery process. This technology automatically designs virtual novel molecules with all the characteristics of a successful drug molecule. Iktos has recently diversified its R&D efforts into the development of an AI technology for peptide-based therapeutics and has developed state-of-the-art generative and predictive models to facilitate design of novel peptide therapeutics with desired properties.
Zealand Pharma A/S has a track record of successfully inventing and developing novel peptide-based drugs. This success is based on the extensive experience in improving the therapeutic characteristics of peptides. Zealand Pharma has a keen interest in expanding its computational chemistry toolbox to include AI and machine learning based approaches for the design of novel therapeutic peptides.
Under the agreement, Zealand will contribute its expertise in peptide drug discovery to Iktos’ generative modelling technologies and expertise in machine learning and AI.
“We are very pleased to join forces with Zealand Pharma and leverage their deep know-how in peptide therapeutics with our state-of-the-art existing technology assets to peptide drug discovery. We look forward to working with Zealand’s experienced R&D team to build leading/state of the art peptide generative and predictive modelling technology in the field of peptides - a new area for Iktos.”
Yann Gaston-Mathé, President and CEO of Iktos
Incorporated in October 2016, Iktos is a French start-up company specialized in the development of artificial intelligence solutions applied to chemical research, more specifically medicinal chemistry and new drug design. Iktos is developing a proprietary and innovative solution based on deep learning generative models, which enables, using existing data, to design molecules that are optimized in silico to meet all the success criteria of a small molecule discovery project. The use of Iktos technology enables major productivity gains in upstream pharmaceutical R&D. Iktos offers its technology both as professional services and as a SaaS software platform, Makya™. Iktos is also developing Spaya™, a synthesis planning software based upon Iktos’s proprietary AI technology for retrosynthesis. Iktos has recently diversified its R&D efforts into the development of an AI technology for peptide-based therapeutics and has developed state-of-the-art generative and predictive models to facilitate design of novel peptide therapeutics with desired properties.
WuXi AppTec | August 17, 2022
WuXi STA, a leading Contract Research, Development, and Manufacturing Organization is pleased to announce the groundbreaking for its new 190-acre pharmaceutical manufacturing campus in Middletown, Delaware. First announced in June 2021, this site will be WuXi STA's second facility in the United States, offering expanded capacity and greater flexibility to meet the needs of customers in the U.S. and around the world.
The WuXi STA Middletown site is located in the Middletown Business Center at 1091 Industrial Drive, and the new state-of-the-art facility will create approximately 500 full-time jobs by 2026. Phase I of the new campus will provide formulation development, clinical and commercial drug product manufacturing services for a variety of oral and injectable dosage forms, as well as packaging, labeling, storage and distribution services for clinical trial materials and commercial drug products.
WuXi STA has 12 sites across the U.S., Europe and Asia that offer a range of services and meet or exceed all regulatory standards. Together with three other drug product manufacturing sites in Couvet Wuxi City (China) and Shanghai this new facility will further enhance the company's global drug production capacity and capabilities.
"The biopharmaceutical industry is part of Delaware's DNA. Over the next five years, WuXi STA plans to build a state-of-the-art pharmaceutical manufacturing campus in one of Delaware's fastest-growing communities, bringing with it good jobs and economic growth. This campus is only possible because of Delaware's world-class, innovative workforce. Thank you to WuXi STA, Middletown Mayor Kenny Branner and everyone who made this project possible."
Delaware Governor John Carney
"The First State continues to be a first-rate destination for businesses looking to innovate and grow," said Senator Tom Carper. "This investment by WuXi STA helps position Delaware as a continued global leader in biopharmaceuticals, further cementing the manufacturing might of one of our fastest growing communities. Delaware's workforce is ready to help build the future of our life-saving pharmaceuticals. This state-of-the-art campus will create good jobs and help fortify our supply chains here at home."
"This biopharmaceutical facility is one most significant private sector investment ever in the Middletown region," said Senator Chris Coons. "I am excited about WuXi STA's decision to choose Middletown for one of their new manufacturing facilities. This isn't just the largest private-sector investment in Middletown's history – it's a defining moment that will bring many more good-paying jobs to Delaware and will further cement the First State as a world leader in biopharmaceutical research and manufacturing. Thanks are due to everyone from Middletown to Dover to Washington who helped us get to this point, whether by making continued investments in NIIMBL, improving our education system from kindergarten to our universities, or ensuring that Delaware workers have the skills manufacturers like WuXi AppTec are looking for."
"As the First State, Delaware has always had a spirit of innovation – constantly seeking new opportunities and ventures that will strengthen our economy and our state as a whole," said Rep. Lisa Blunt Rochester. "Today's groundbreaking of the WuXi STA pharmaceutical manufacturing plant represents another step in that spirit of innovation – bringing good-paying jobs to Middletown and strengthening communities throughout Delaware. The state-of-the-art facility will bring 500 full-time jobs over the next several years, growing our state's workforce and allowing us to manufacture more goods domestically - enhancing our economic competitiveness on the global stage."
"We are truly looking forward to our partnership with WuXi STA, as the groundbreaking today marks the kick-off to them making their new home in Middletown, Delaware. WuXi STA will be joining our bustling industrial area, just steps away from Clarios, Datwyler, Amazon and Breakthru Beverage, just some of the companies that have invested in Middletown. When asked by WuXi STA about our business-friendly attitude, these businesses expressed their wholehearted support for how we do business and we couldn't be more excited about WuXi STA wanting to join them, and the Town of Middletown, in our journey together," said Middletown Mayor Ken Branner. Branner added he was thankful for the support of Gov. John Carney and the Delaware Prosperity Partnership.
"I would like to thank our federal, state and local partners in Delaware for their continued support in the establishment of this site," said Dr. Minzhang Chen, co-CEO of WuXi AppTec and CEO of WuXi STA. "WuXi STA continues to increase our capabilities and capacities to better serve our customers through a more robust and reliable supply chain. With our integrated Contract Research, Development, and Manufacturing Organization platform and proven quality system, we look forward to working with our customers to swiftly deliver their innovative therapies to market in order to save lives and improve healthcare for patients."
About WuXi STA
WuXi STA a subsidiary of WuXi AppTec is a leading pharmaceutical development and manufacturing capability and technology platform company serving the life sciences industry, with global operations. As a premier Contract Research, Development, and Manufacturing Organization WuXi STA offers its worldwide partners efficient, flexible and high-quality solutions for integrated chemical, manufacturing and controls from preclinical to commercial uses, including the development and manufacturing of small molecule, oligonucleotide, peptide and various complex chemical conjugate.
About WuXi AppTec
As a global company with operations across Asia, Europe, and North America, WuXi AppTec provides a broad portfolio of R&D and manufacturing services that enable the global pharmaceutical and healthcare industry to advance discoveries and deliver groundbreaking treatments to patients. Through its unique business models, WuXi AppTec's integrated, end-to-end services include chemistry drug CRDMO biology discovery, preclinical testing and clinical research services, cell and gene therapies CTDMO helping customers improve the productivity of advancing healthcare products through cost-effective and efficient solutions. WuXi AppTec received AA ESG rating from MSCI in 2021 and its open-access platform is enabling more than 5,850 collaborators from over 30 countries to improve the health of those in need – and to realize the vision that "every drug can be made and every disease can be treated."