Samsung Biologics | August 02, 2022
GreenLight Biosciences a biotechnology company dedicated to making ribonucleic acid products affordable and accessible for human health and agriculture, and Samsung Biologics a leading global CDMO providing fully integrated end-to-end contract development and manufacturing services, announced the successful completion of the first commercial-scale engineering run for the companies' mRNA production partnership.
Since the announcement of the strategic partnership between GreenLight Biosciences and Samsung Biologics in late 2021, technology transfer and scale-up from the lab bench to Samsung's commercial facility was completed in seven months, demonstrating platform adaptability and scalability.
GreenLight's process—from drug substance and lipid nanoparticle formulation to bulk drug product—can be completed in the same facility, an important capability. GreenLight's mRNA synthesis reaction had a titer of 12g/L at commercial scale and produced 650g of mRNA.
The company's RNA platform allowed GreenLight to move from conceptualizing an mRNA vaccine to delivering released clinical trial material in less than two years. With the demonstration at Samsung, and with GreenLight's Covid booster vaccine clinical trial expected to start in 2022, GreenLight would be capable of supplying mRNA vaccine at a commercial scale.
"This demonstrates a major achievement in our continuing goal to offer one-stop end-to-end mRNA production from Drug Substance to Aseptic Fill Finish to commercial release, all from a single site, as we strive across our biomanufacturing network to fight the pandemic,"
John Rim, CEO and President at Samsung Biologics
Earlier this year, Samsung Biologics completed the expansion of its mRNA drug substance manufacturing suite at its Songdo headquarters, where the company is now fully equipped to provide end-to-end mRNA production for clients.
"One of the greatest challenges when producing quality pharmaceuticals is advancing from a small lab to large-scale commercial production," said GreenLight CEO Andrey Zarur. "We are grateful for the help and support of Samsung in demonstrating that our small mRNA process can scale in a linear fashion to the industrial scale that will be needed to help satisfy the vaccine needs of humanity."
To date, all comparability data conforms with expected outcomes, indicating successful scale up and fit of GreenLight's process to Samsung Biologics' Songdo facility. A second engineering run will start in August, to implement improvements indicated by the first run and to demonstrate repeatability at scale.
About GreenLight Biosciences
GreenLight Biosciences aims to address some of the world's biggest problems by delivering on the full potential of RNA for human health and agriculture. Our RNA platform allows us to research, design, and manufacture for human, animal, and plant health. In human health, this includes messenger RNA vaccines and therapeutics. In agriculture, this includes RNA to protect honeybees and a range of crops. The company's platform is protected by numerous patents. GreenLight's human health product candidates are in the pre-clinical stage, and its product candidates for the agriculture market are in the early stages of development or regulatory review. GreenLight is a public benefit corporation that trades under the ticker GRNA on Nasdaq.
CELL AND GENE THERAPY
Merck and Orna Therapeutics | August 17, 2022
Merck known as MSD outside the United States and Canada, and Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular RNA therapies, today announced a collaboration agreement to discover, develop, and commercialize multiple programs, including vaccines and therapeutics in the areas of infectious disease and oncology.
Under the terms of the agreement, Merck will make an upfront payment to Orna of $150 million, which will be expensed by Merck in the third quarter of 2022 and included in non-GAAP results. In addition, Orna will be eligible to receive up to $3.5 billion in development, regulatory, and sales milestones associated with the progress of the multiple vaccine and therapeutic programs, as well as royalties on any approved products derived from the collaboration. Orna will retain rights to its oRNA-LNP technology platform and will continue to advance other wholly owned programs in areas such as oncology and genetic disease. Merck will also invest $100 million of equity in Orna’s recently completed Series B financing round.
Orna’s proprietary oRNA technology creates circular RNAs from linear RNAs by self-circularization. oRNA molecules have been shown to have greater stability in vivo than linear mRNA and have the potential to produce larger quantities of therapeutic proteins inside the body. Newly synthesized oRNA molecules are more compactly packaged into custom lipid nanoparticles which Orna has engineered to target key tissues in the body. Preclinical data, including presentations at the 2022 American Society of Gene & Cell Therapy Annual Meeting, have demonstrated the potential of oRNA expression and delivery as an approach for further development in multiple areas, including vaccines and oncology therapeutics.
“This broad strategic collaboration brings together Merck’s significant expertise in nucleic acid biology, clinical development, and manufacturing with Orna’s compelling circular RNA technology to explore the opportunity to develop a new generation of potential vaccines and therapeutics. We look forward to working with the talented scientific and technical teams at Orna."
Fiona Marshall, senior vice president and head of discovery, preclinical and translational medicine at Merck Research Laboratories
“We are thrilled to collaborate with Merck, a company committed to breakthrough science, which has recognized the potential our platform can bring to patients. Our oRNA technology plus novel delivery solutions are designed to unlock the full potential of RNA in therapeutics and vaccines,” said Tom Barnes, Chief Executive Officer of Orna. “The combined expertise of Orna and Merck aims to accelerate the development of RNA therapeutics for patients in need of better treatment options.”
At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities.
About Orna Therapeutics
Orna Therapeutics was founded on groundbreaking research by Alex Wesselhoeft, Ph.D., and Daniel G. Anderson, Ph.D. from MIT and built by MPM Capital and BioImpact Capital, an investment management affiliate of MPM. Orna’s proprietary circular RNA (oRNA) is engineered as linear RNA that self-circularizes. By taking a line and turning it into a circle, oRNA exhibits numerous advantages over traditional linear mRNA therapies such as simplified production, increased protein expression, and a superior immunogenicity profile. With proprietary lipid nanoparticles including those from Orna’s joint venture with ReNAgade Therapeutics, an RNA delivery company, our technologies expand the possibilities of what RNA therapeutics can achieve.
SYNTHEGO | August 03, 2022
Synthego, the genome engineering company, announced the launch of Engineered Cell Libraries, a novel offering that further enables access to CRISPR by providing arrayed CRISPR-edited cells for direct use in functional screening assays. The innovative solution leverages Synthego’s Eclipse™ Platform. This high-throughput cell engineering platform delivers cell-based models for disease research by providing highly predictable CRISPR-engineered cells at scale through the integration of engineering, bioinformatics, and proprietary science. Synthego’s Engineered Cell Libraries provide unparalleled speed, scalability, and efficiency to accelerate the drug discovery process by enabling a faster path between experimental design and execution.
As the newest addition to Synthego's Engineered Cells product line, the offering is a custom arrayed library of multi-guide™ knockout cell pools (immortalized or iPSC) delivered ready-made to researchers, efficiently enabling them to bypass the need for equipment and reagent sourcing, transfection, generation of an extensive CRISPR library, and hiring and training of staff. This allows researchers to overcome common hurdles associated with CRISPR-based target discovery and focus specifically on the science.
“Arrayed screening approaches are more sensitive, compatible with a broader assortment of downstream assays, and can yield data that is more readily interpretable than the more commonly used pooled approach but are underutilized due to lack of infrastructure and limited bandwidth. With Engineered Cell Libraries, Synthego continues to provide transformative solutions for accelerated drug discovery which ultimately can bring a wider range of therapeutics to market faster.”
Travis Maures, Synthego’s Chief Technology Officer
With Engineered Cell Libraries, scientists specify the human or mouse cell type (Immortalized or iPSC available at launch) and gene targets they desire to knockout to generate a custom “Knockout Cell Library.” The cells are then edited on Synthego’s Eclipse Platform, which handles guide design, cell line optimization, editing through transfection, and assessment of editing efficiencies, so cells are ready to screen upon arrival.
Engineered Cell Libraries on the Eclipse Platform additionally benefit customers with
Scalability and flexibility - Engineered Cell Libraries allow researchers to maximize their screening power and identify more targets earlier in the screening process.
Predictability and transparency - Synthego’s multi-guide™ technology achieves reliably high knockout efficiencies so researchers can confidently proceed with their screens.
Cell engineering expertise and support - Synthego uses its expertise to bring automation innovation that provides greater consistency in outcomes and scalability.
Synthego’s Eclipse Platform and Engineered Cell Libraries enable a wide range of applications in research and development across various disease areas and research disciplines such as oncology and neurology. Engineered Cell Libraries were employed in a recent study that used genetic screening to identify host factors that either facilitate or inhibit infection by SARS-CoV-2 and that could potentially be targeted with existing drugs that have been approved for other indications.
“We were able to quickly combine our proteomic expertise with Synthego's genome engineering capabilities in a matter of weeks,” said Nevan J. Krogan, Director, Quantitative Biosciences Institute, University of California, San Francisco. “Normally, work such as this would take many years. We were able to quickly pinpoint which human genes are important for infection, and that allowed us to jump to which ones if we were able to drug them, could have a positive pharmacological effect on SARS-CoV-2 infection. That whole pipeline allowed us to identify several potential drug candidates, several of which we're still looking at.”
Ultimately, Synthego’s goal is to enable scientists to spend less time thinking about method development and more time running their functional assays. The addition of Engineered Cell Libraries is driving impact in biopharma research and development.
Synthego was founded to revolutionize genome engineering technology, helping translate genomics into the clinic and ultimately making engineered biological therapies accessible to all patients. The company leverages machine learning, automation, and gene editing to build platforms for science at scale. With its foundations in engineering disciplines, the company’s platforms vertically integrate proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance both basic research and therapeutic development programs. With its technologies cited in more than a thousand peer-reviewed publications and utilized by thousands of commercial and academic researchers and therapeutic drug developers, Synthego is at the forefront of innovation enabling the next generation of medicines by delivering genome editing at an unprecedented scale.