Asimov Launches a Suite of AI Models, Host Cells & Genetic Tools for End-to-End Gene Therapy Development

AAV Edge

Asimov recently 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.”

Asimov’s mission is to advance humanity’s ability to design living systems, enabling biotechnologies with outsized societal benefit. The company is developing a synthetic biology platform – from cells to software – to design and manufacture next-generation therapeutics, including biologics, cell/gene therapies, and RNA through a combination of products, services, and collaborations. Founded by bioengineers from MIT and Boston University and headquartered in Boston, the company has raised over $200 million from top institutional investors including Andreessen Horowitz, CPP Investments, Horizons Ventures, and Fidelity Management & Research Company. For more information, visit www.asimov.com.