top of page
®

Focused pipeline of development candidates where Dualase  has a differentiated mechanism of action 

Initial focus on treating devastating repeat expansion disorders by precisely collapsing the toxic DNA repeats that drive disease - bringing a potential disease-modifying option to patients who currently have none

Pipeline.png
In vivo tolerability and efficacy in disease-relevant cell and animal models demonstrating the differentiated advantage of Dualase  genome editors
®
Anchor 1
CNS.png

Neurodegeneration

Efficient and precise removal of an expanded DNA repeat sequence in a central nervous system (CNS) neurodegenerative disease using dual-guided Dualase  encoded in a single adeno-associated virus (AAV) ​

®

Delivery: 

AAV Light.png

Single AAV

Mechanism: 

Repeat removal

Differentiated benefits:

  • Precise removal of pathogenic DNA repeats leaving non-pathogenic number

  • Single AAV delivery simplifies manufacturing and dosing and has been used in clinical and commercial products 

  • High specificity for target regions and, unlike other genome editors tested, no off-target effects

  • Durable correction of underlying genetic mutation in DNA for potential lasting benefit

Cystic Fibrosis.png

Cystic Fibrosis

Mutation-agnostic correction of multiple CF-causing mutations using RNA formulation encapsulated in a proprietary nebulized lipid nanoparticle (LNP) formulation for targeted delivery to the lung

Delivery: 

LNP-mRNA.png

LNP-mRNA

Repair

Mechanism: 

Differentiated benefits:

  • Seamless repair without insertions, deletions or bystander base changes

  • 'Hotspot' correction of multiple mutations with one Dualase  genome editor

  • Direct delivery to lung to address major source of morbidity and mortality

  • Durable correction of underlying genetic mutation compared to RNA- or protein-targeted therapeutics

®
Anchor 2
Liver.png

Liver

Correction of base change without any detectable nearby base modifications using RNA formulation encapsulated in a lipid nanoparticle

LNP-mRNA.png

Delivery: 

LNP-mRNA

Mechanism: 

Repair

Differentiated benefits:

  • Seamless repair without other detectable bystander base changes

  • Corrects both lung and liver manifestations of the disease with one Dualase  genome editor

  • Small RNAs for simpler manufacturing and decreased potential immunogenicity

  • Durable correction of underlying genetic mutation compared to RNA- or protein-targeted therapeutics

®
Anchor 3
Discovery.png

Discovery

Robust discovery-stage candidates in CNS, skeletal muscle, lung and liver indications with significant pipeline expansion opportunities or further development with partners

​

​​​​Large and growing list of disease-relevant Dualase  targets​, reach out to us to partner:

®
Anchor 4
bottom of page