NOPASS
Understanding NoPass™
A Game-Changer in RNA-Based Therapies
What is NoPass Design?
NoPass Design is Speratum Biopharma’s innovative approach to overcoming the limitations of traditional RNA-based therapies. It is a re-engineering of natural sequences that induce RNA interference (RNAi), to generate therapeutic molecules with improved efficacy and safety.
Key Highlights:
- Chemical-Free Design: Engineered structural elements remove the need for chemical modifications.
- Precision Pathways: Activation of non-canonical pathways boosts specificity and minimizes risks.
- Enhanced mRNA Targeting: Prioritizes cleavage over translation repression for improved functionality.
How NoPass Works?
NoPass replicates natural miRNA/siRNA functions but is optimized for therapy by:
- Conserving “seed” sequences to preserve target profiles.
- Optimizing complementary regions for more precise gene targeting.
- Modifying sequence elements to activate non-canonical pathways, circumventing competition with endogenous miRNAs.
- Adjusting structural elements to enhance target mRNA destruction over translation repression.
Challenges and Opportunities in RNA-Based Drug Development
Challenges
- Delivery Mechanisms: Safely delivering RNA molecules to target cells while avoiding degradation is a major hurdle.
- Stability: RNA is inherently unstable and prone to enzymatic breakdown, requiring innovative stabilization techniques.
- Off-Target Effects: Ensuring specificity to avoid unintended interactions with non-target genes or proteins.
- Manufacturing Complexity: Producing RNA at scale with consistent quality remains a challenge.
Opportunities
- Broad Therapeutic Potential: RNA-based drugs can target diseases previously considered untreatable, such as genetic disorders and rare diseases.
- Rapid Development: RNA therapies can be designed and tested faster than traditional small molecules or biologics.
- Customizable Treatments: Personalized RNA-based solutions offer hope for precision medicine tailored to individual patients.
- Expanding Platforms: Advances in lipid nanoparticles (LNPs) and other delivery systems are enhancing efficacy and safety.
Specific Cleavage Motifs
Enable precise cleavage and trimming of the passenger strand, preventing its incorporation into the RISC complex and minimizing off-target effects
Why NoPass is Special?
Revolutionary Mimic Technology: NoPass mimics any miRNA or siRNA sequence, making it versatile for a wide range of therapeutic targets. Its unique design improves efficacy, specificity, and safety.
miR-198 Correlation: Speratum leverages NoPass technology to optimize miR-198 delivery, targeting cancer cells with precision. This synergy enhances the therapeutic potential of miR-198, combating tumor growth, drug resistance, and metastasis.
Patent coverage:
Secures Speratum’s innovative edge in RNAi technology, ensuring long-term market exclusivity and scientific leadership.
US20220145290A1 Patent Pending
Substrate sequence design for RNAi-mediated multi-site regulation of genomic and non-genomic RNAs.
WO2022040594A1 Patent Pending
Novel RNA scaffolding for combinatorial RNA interference therapies.
The Science Behind NoPass
Combines the strengths of both miRNA and siRNA with enhanced specificity and reduced side effects.
miRNA vs. siRNA: miRNA (microRNA): Endogenous small RNAs that regulate gene expression by silencing mRNAs. They are versatile but sometimes lack precision.
siRNA (small interfering RNA): Synthetic or naturally occurring RNAs designed for specific gene silencing. They offer high precision but can have off-target effects.
Thank you TED Ed for allowing us to use this video
The Impact of NoPass
Results Achieved with NoPass:
- Multiple mRNA targets silenced simultaneously with minimal off-target effects.
- Broad applicability across various disease areas, especially oncology.
- Energy-efficient and stable RNA designs tailored for specific therapeutic needs.
