CORRECTING DISEASE-CAUSING RNA
WITH A NEW APPROACH TO GENE THERAPY
Genetic diseases are caused by mutations in the genome, which can result in mutant RNA transcripts. Our CORRECTx platform of RNA binding systems can precisely target and correct these defective RNA transcripts. Delivered using AAV gene therapy vectors, this approach is designed to provide durable benefits to patients suffering from rare but devastating disorders—with the convenience of a single administration.
CORRECTING DISEASE-CAUSING RNA
WITH A NEW APPROACH TO GENE THERAPY
Genetic diseases are caused by mutations in the genome, which can result in mutant RNA transcripts. Our CORRECTx platform of RNA binding systems can precisely target and correct these defective RNA transcripts. Delivered using AAV gene therapy vectors, this approach is designed to provide durable benefits to patients suffering from rare but devastating disorders—with the convenience of a single administration.

Our proprietary technology platform, CORRECTx, leverages the power and precision of RNA binding systems and AAV delivery, which provides the durable effect of a one-time gene therapy. The CORRECTx platform is modular and versatile with several different RNA targeting systems that can be used to modify disease-causing RNA via multiple mechanisms, including Destruction, Blocking, Splicing (Exon Skipping), and RNA Editing. Our platform also enables multi-targeting to enhance specificity or expand targeting. CorrectX offers features that address the limitations of DNA-targeted gene therapies, as well as other RNA-targeted approaches, and may lead to new genetic medicines for patients with neurodegenerative and neuromuscular diseases.

PRECISELY
targets and corrects
disease-causing RNA
PRESERVES
the healthy mRNA
transcript
SELF-CONTAINED
with no need for
cellular machinery
DELIVERED
in a one-time
gene therapy
administration

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