CELL AND GENE THERAPY
Genscript Biotech Corporation | September 23, 2022
GenScript USA Inc., the world's leading life-science research tools and services provider, is collaborating with researchers at the Gladstone-UCSF Institute of Genomic Immunology to advance the development of new, non-viral cell therapies that modify genomic sequences in the pursuit of more effective cell therapy products while limiting the cellular toxicity that is typically associated with previously available methods.
A new study, published in Nature Biotechnology, details methods for achieving highly efficient non-viral knock-in using GenScript's GenExact™ single-strand DNA modified with Cas9-target sequences. This method achieved up to ˜40% knock-in efficiency in primary immune cell types.
One powerful application of CRISPR/Cas genome editing technology involves the precise insertion of DNA sequences via the HDR pathway. Traditionally, researchers have relied on viral vectors to deliver DNA insertion templates used for gene therapy into cells. However, difficulties manufacturing large amounts of clinical-grade viral vectors have delayed getting cell therapies to patients. Additionally, viral vectors can insert genes at any location within the genome, leading to safety concerns.
Previous research by the group at Gladstone and UCSF has shown that synthetic DNA templates can be delivered without the use of viral vectors, but high levels of double-stranded DNA can be toxic to cells, resulting in low efficiency. Efficiency can be improved using a modified version of the DNA templates that can bind to the Cas9 enzyme. However, additional work was required to improve the yield of successfully engineered cells and to make the process compatible with the manufacturing of future cell therapies. Those goals motivated the team's current study.
In this study, the team tested modified GenExact ssDNA designed with Cas9-targeted sequences to determine if the gene-editing efficiency could be improved with lower toxicity. First, synthetic sgRNA was complexed with Cas9 protein to form a RNP complex. Then the RNP and ssDNA template were delivered into the cell via electroporation, enabling efficient gene editing.
The team discovered that this combination of GenExact ssDNA with Cas9-targeted sequence offers up to 40% knock-in editing efficiency with minimal cellular toxicity. This approach can accelerate the development and manufacture of novel, high-yield non-viral gene therapies — and at lower cost.
"We are very proud to have collaborated on this groundbreaking scale-up work by the talented team at Gladstone and UCSF. GenScript is excited for the opportunities that this high-yield cell-line engineering process will provide to our customers," said Ray Chen, Ph.D, president of GenScript Life Science Group.
"We were very happy to partner with Genscript on critical experiments demonstrating high efficiency and yield of CAR knock-in cells at clinical scale. The long ssDNA produced by Genscript exceeded our expectations and helped us clearly demonstrate the potential for future therapeutic applications using these methods.",
Brian Shy, MD, PhD, a former clinical fellow in the lab of Alex Marson, MD, PhD, director of the Gladstone-UCSF Institute of Genomic Immunology
About GenScript Biotech Corporation
GenScript Biotech Corporation is a global biotechnology group. Based on its leading gene synthesis technology, GenScript has developed four major platforms including the global cell therapy platform, the biologics contract development and manufacturing organization platform, the contract research organization platform, and the industrial synthesis product platform.
GenScript was founded in New Jersey, USA in 2002 and listed on the Hong Kong Stock Exchange in 2015. GenScript's business operation spans over 100 countries and regions worldwide, with legal entities located in the USA, mainland China, Hong Kong, Japan, Singapore, the Netherlands, and Ireland. GenScript has provided premium, convenient, and reliable products and services for over 100,000 customers.
Ginkgo Bioworks | October 20, 2022
Today, Ginkgo Bioworks the leading horizontal platform for cell programming, and Zymergen announced that Ginkgo has completed its previously announced acquisition of Zymergen. The acquisition is expected to significantly enhance Ginkgo's platform by integrating strong automation and software capabilities as well as a wealth of experience across diverse biological engineering approaches.
"Today marks an important step in our long-term growth as we complete the Zymergen acquisition and welcome their world-class team to Ginkgo. We are excited to integrate Zymergen's capabilities into our platform and explore new and expanded partnerships and opportunities for their diverse array of product concepts currently under development."
Jason Kelly, CEO and co-founder of Ginkgo Bioworks
Under the terms of the merger agreement entered into on July 24, 2022, Zymergen stockholders received, for each share of Zymergen common stock, 0.9179 shares of Ginkgo Class A common stock. Zymergen shares will no longer be traded on Nasdaq. Ginkgo Class A common stock will continue to trade on NYSE under the ticker symbol DNA.
About Ginkgo Bioworks
Ginkgo is building a platform to enable customers to program cells as easily as we can program computers. The company's platform is enabling biotechnology applications across diverse markets, from food and agriculture to industrial chemicals to pharmaceuticals. Ginkgo has also actively supported a number of COVID-19 response efforts, including K-12 pooled testing, vaccine manufacturing optimization and therapeutics discovery.
Zymergen is a biotech company that designs and produces molecules, microbes and materials for diverse end markets. Zymergen partners with nature to make better products, a better way, for a better world.
Neumora | October 12, 2022
Neumora Therapeutics, Inc. a clinical-stage biotechnology company pioneering precision medicines for brain diseases through the integration of data science and neuroscience, today announced the close of a $112 million Series B financing. The Series B syndicate includes both new and existing investors, such as Abu Dhabi Growth Fund Altitude Life Science Ventures, Amgen, ARCH Venture Partners, Exor Ventures, F-Prime Capital, Invus, Mubadala Capital, Newpath Partners, Polaris Partners and other undisclosed investors. The funding will support the advancement of a broad clinical and preclinical pipeline of novel precision medicine candidates for neuropsychiatric disorders and neurodegenerative diseases and the development of the company's precision neuroscience platform.
“This Series B financing reflects Neumora’s continued progress in building a best-in-class neuroscience company, including assembling a world-class team, scaling up an industry-leading data science and translational neuroscience platform, and advancing a broad and growing pipeline of seven development programs, including our internal discovery efforts and business development activities. This considerable amount of progress in such a short period of time reflects our urgency to address the relative lack of progress and innovation in neuroscience with our data-driven, precision medicine approach.”
Paul L. Berns, co-founder, chairman and chief executive officer of Neumora
Neumora continues to advance a broad and growing pipeline of clinical and preclinical programs, including a balance of both clinically validated and novel approaches targeting a broad range of underserved neuropsychiatric disorders and neurodegenerative diseases. The company recently completed enrollment in a Phase 2a clinical trial for its most advanced product candidate, NMRA-140, a kappa opioid receptor antagonist in development for the treatment of major depressive disorder. Neumora is also progressing NMRA-511, a clinical-stage vasopressin 1a receptor antagonist in development for neuropsychiatric disorders, and NMRA-266, an M4 muscarinic receptor positive allosteric modulator being advanced toward initiation of Phase 1 development for schizophrenia in 2023. The company’s earlier-stage pipeline includes multiple first-in-class opportunities in preclinical development, including neurodegeneration programs focused on Parkinson’s disease and amyotrophic lateral sclerosis (ALS).
“We are pleased to have the support of this leading group of both new and existing investors that share our vision and excitement with our progress to date,” said Joshua Pinto, Ph.D., chief financial officer of Neumora. “With this Series B financing, we have successfully raised approximately $650 million in capital, resulting in a very strong financial position to continue advancing our broad pipeline through multiple near-term value-creating milestones.”
Neumora was founded to pioneer a new era of precision neuroscience medicines by using patient enrichment strategies to help potentially improve clinical development success rates. The company’s proprietary approach leverages recent advances in data science and translational neuroscience to cut through the heterogeneity inherent in brain disease. By approaching patient enrichment and clinical development strategies through this lens, Neumora aims to match the right patient populations to the right targeted therapeutics, thereby driving innovation and the potential for clinical development success in this field and, ultimately, providing better therapies to patients.
In connection with the completion of the Series B financing, Alaa Halawa, partner and head of U.S. Ventures at Mubadala Capital, joined Neumora’s board of directors. Mr. Halawa said, “We are proud to partner with Neumora in helping advance the company’s efforts to build the precision neuroscience company of the future. The integration of technology, namely AI/ML, and proprietary clinical data is helping us develop a deeper understanding of the complex drivers of brain disease to develop novel treatments for neuropsychiatric disorders and neurodegenerative diseases - we are humbled to be part of their journey.”
Neumora Therapeutics, Inc. is a clinical-stage biotechnology company pioneering precision medicines for brain diseases through the integration of data science and neuroscience. Neumora is redefining neuroscience research and development with a data-driven precision neuroscience platform to cut through brain disease heterogeneity to match the right patient populations to targeted therapeutics. Neumora is relentless in its commitment to discovering, developing and commercializing targeted therapies for people living with brain diseases.