Optimized gene editing for treating ALS through compact RNA-guided nucleases
Acrobat Genomics is advancing CRISPR/Cas technology to treat ALS by developing compact RNA-guided nucleases. This approach targets the genetic driver C9ORF72, aiming to enhance gene editing efficiency and reduce neurotoxicity with a high-throughput screening platform and AAV delivery.
Overview
Acrobat Genomics is pioneering the development of next-generation gene editing technologies to tackle Amyotrophic lateral sclerosis (ALS), a debilitating neurodegenerative disease. The solution focuses on optimizing CRISPR/Cas-based systems to target mutations in the C9ORF72 gene, which are known to contribute to ALS. By engineering compact RNA-guided nucleases (ARGNs), Acrobat Genomics aims to improve gene editing precision and efficacy, addressing the challenge of off-target effects and in vivo delivery limitations associated with first-generation technologies.
Technical specifications
- High-throughput screening platform (AcrobaTx): Utilizes protein language models to create and screen ARGN variants in human cells, aiming for a four-fold increase in editing efficiency compared to wild type proteins.
- Single vector AAV delivery system: Leverages the compactness of ARGN to deliver gene editing tools specifically to motor neurons derived from ALS patient iPSCs, with the goal of achieving a significant reduction in neurotoxicity.
- Targeted C9ORF72 correction: Focuses on reducing aberrant peptide production and RNA aggregation, which are key contributors to neurotoxicity in ALS.
Technology readiness level
This innovative gene editing solution is currently at Technology Readiness Level 4, indicating that it has been validated in a laboratory setting with therapeutically relevant human cell lines. The platform is progressing towards further therapeutic development and in vivo applications.
About Acrobat Genomics
Acrobat Genomics is a biotechnology company that develops AI-driven protein and genome engineering solutions. Their technology platform integrates high-throughput functional screening within living human cells with machine learning to train AI models for the design and optimization of novel therapeutic molecules. By learning from millions of measurements taken directly in human cells, the company aims to move beyond traditional in silico discovery methods, enabling the rapid design of therapeutics that function effectively in complex biological environments.
The company serves the biotechnology and healthcare sectors by applying its platform to biosecurity and the development of gene editing-based therapeutics. As a prime contractor for programs such as the DARPA Rapid Inhibitor Discovery and Development pipeLine (RIDDL), Acrobat Genomics works to create rapid responses to emerging threats, including the development of novel inhibitors for gene editing technologies. Founded in 2022, the company has secured funding from the National Institutes of Health, private investors, and biopharma collaborators to advance its research in therapeutic applications and protein design.
delivered to your inbox.