DM1 circRNA therapy
Category
Drugs National Companies
Register type
Biotech Sanitaria
Company name
Product / Service
DM1 circRNA therapy
Type
Gene Therapy
Therapeutic area
Enfermedades genéticas y raras
Properties/Indication
NoctuRNA develops 1st-in-class therapies based on circRNAs for the effective treatment of currently incurable diseases, including infectious and genetic disorders. Our circRNAs specifically target secondary RNA structures critical to the progression of several diseases. The therapeutic strategy of NoctuRNA possesses several compelling competitive advantages:
1. It involves a disruptive and innovative mechanism of action that holds tremendous potential, particularly for rare diseases such as Steinert, ALS or FXTAS, where the future drug could qualify for orphan drug designation.
2. circRNAs are naturally occurring within human cells, providing noctuRNA’s therapies with a more robust safety profile when compared to exogenous alternatives such as small molecules.
3. circRNAs demonstrate exceptional stability due to the absence of free ends vulnerable to exonucleases. This unique feature eliminates the highly-cost need of the nucleotide modifications typically required in other RNA-based therapies like siRNAs.
We identified NTX-DM1 as our lead circRNA therapeutic for DM1 (Myotonic Dystrophy Type 1, Steinert disease), showing strong efficacy in vitro by correcting splicing defects in patient-derived cells and in vivo by improving phenotypes in 83% of treated Drosophila. In vivo efficacy studies in mouse models are currently ongoing.
1. It involves a disruptive and innovative mechanism of action that holds tremendous potential, particularly for rare diseases such as Steinert, ALS or FXTAS, where the future drug could qualify for orphan drug designation.
2. circRNAs are naturally occurring within human cells, providing noctuRNA’s therapies with a more robust safety profile when compared to exogenous alternatives such as small molecules.
3. circRNAs demonstrate exceptional stability due to the absence of free ends vulnerable to exonucleases. This unique feature eliminates the highly-cost need of the nucleotide modifications typically required in other RNA-based therapies like siRNAs.
We identified NTX-DM1 as our lead circRNA therapeutic for DM1 (Myotonic Dystrophy Type 1, Steinert disease), showing strong efficacy in vitro by correcting splicing defects in patient-derived cells and in vivo by improving phenotypes in 83% of treated Drosophila. In vivo efficacy studies in mouse models are currently ongoing.
Development phase
Preclinical
Phases developed in Spain
R&D
Research location
España
Patent
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