High performance two-plasmid production system achieves E12 vg/mL unconcentrated titers
AI-driven design tools to improve expression precision, efficacy, and manufacturability
Boston, Massachusetts, 18 Sep 2024: Asimov, the synthetic biology company advancing the design and production of therapeutics, today announced the launch of the AAV Edge System, a comprehensive suite of tools for adeno-associated viral (AAV) gene therapy design and manufacturing. The system provides gene therapy developers a single access point to an array of best-in-class tools to supercharge gene therapy development.
While gene therapy holds significant promise for treating otherwise intractable diseases, the field is grappling with challenges in safety, efficacy, manufacturability, and cost. These issues are exacerbated by a fragmented ecosystem where key technologies are siloed across service providers, each offering disparate solutions. This fragmentation leads to suboptimal therapeutic development. Asimov’s AAV Edge System addresses these challenges by providing an end-to-end platform that brings together several essential technologies, allowing developers to select the modules that best meet their design and production needs.
The AAV Edge System offers a comprehensive suite of tools for both payload design and production:
Payload design: The system includes artificial intelligence (AI)-designed, animal-validated tissue-specific promoters to enhance safety and efficacy; advanced DNA sequence optimization capabilities to boost expression levels in vivo; and tools to silence the gene of interest (GOI) during production to improve manufacturing performance by minimizing GOI toxicity. These proprietary genetic parts and design algorithms are accessible via Kernel, Asimov’s computer-aided genetic design software.
Production system: Today's launch introduces Asimov’s transient transfection-based AAV manufacturing system—the first in a planned series of releases for AAV Edge. This platform features a clonal, suspension-adapted, GMP-banked HEK293 host cell line; an optimized two-plasmid system compatible across capsid serotypes; and model-guided process development to improve bioreactor performance, achieving unconcentrated titers up to E12 viral genomes per milliliter (vg/mL).
Alec Nielsen, Co-founder and CEO of Asimov, said: "Our team has been on a roll – AAV Edge is our third launch in cell and gene therapy this year. The cost and safety of gene therapies is top of mind for many in the field, and we're driven to help our partners on both design and production to enable more of these powerful medicines to reach patients. This is Asimov’s latest application in programming biology, made possible by leveraging AI, synthetic biology, and bioprocess engineering. There’s more to come, and we’re excited to keep pushing the envelope.”
Virtual Private Network (VPN): Users connect to the cluster, provide some credentials and are then able to access internal tools.
Single Sign-On: A tool like Kerberos allows you to use the same account across various components.
Home-grown user accounts: You implement an authentication system and users have a separate username/password for your computing infrastructure.
Asimov, the synthetic biology company building a full-stack platform to program living cells, announced today it has been awarded a contract as part of the Defense Advanced Research Projects Agency (DARPA) Automating Scientific Knowledge Extraction (ASKE) opportunity.
Through ASKE, Asimov will work to develop a physics-based artificial intelligence (AI) design engine for biology. The goal of the initiative is to improve the reliability of programming complex cellular behaviors.
“To achieve truly predictive engineering of biology, we require dramatic advances in computer-aided design. Machine learning will be critical to bridge genome-scale experimental data with computational models that accurately capture the underlying biophysics. As genetically engineered systems grow in complexity, they become difficult for humans to design and understand. For simple genetic systems with only a couple of genes, synthetic biologists typically use high-throughput screening and basic optimization algorithms. But to engineer more complex applications in health, materials, and manufacturing, we need radically new algorithms to intelligently design the DNA and simulate cell behavior.”
Alec Nielsen, Phd, Asimov CEO
Asimov’s founders previously built a hybrid genetic engineering and computer-aided design platform called Cello to program logic circuit behaviors in cells. The ASKE opportunity will seek to support an ambitious expansion in the types of biological behaviors that can be engineered. Asimov’s approach will leverage “multi-omics” cellular measurements, structured biological metadata, and novel AI architectures that combine deep learning, reinforcement learning, and mechanistic modeling. Over the past year, the company has ramped up hiring in experimental synthetic biology, machine learning, and data science to accelerate development of their genetic design platform.
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DARPA recently announced a multi-year investment of $2B into innovative artificial intelligence research called the AI Next campaign. A part of this wide-ranging AI strategy is DARPA’s Artificial Intelligence Exploration program, which was developed to help expeditiously move pioneering AI research from idea to exploration in fewer than 90 days. DARPA’s ASKE opportunity is part of this program and is focused on developing AI technologies that can reason over rich models of complex systems.
“Over the past 50 years, DARPA has been a world leader in spurring innovation across the field of AI, including statistical-learning and rule-based approaches. We are proud to work with DARPA to advance the state-of-the-art in AI-assisted genetic engineering.”