Gene Knockdown Therapies
Gene knockdown therapies represent a groundbreaking approach in the treatment of viral infections, leveraging the power of RNA interference (RNAi) to selectively silence specific genes that viruses exploit to infect host cells. At Nanotreat Inc., we are at the forefront of this innovative field, using Lipid Nanoparticles (LNPs) to deliver small interfering RNA (siRNA) directly to target cells. Our gene knockdown therapies focus on disabling key viral entry points, offering a highly targeted and effective method to prevent infection.
Gene knockdown therapy involves reducing or silencing the expression of specific genes within cells to prevent the production of proteins essential for viral replication or entry. At Nanotreat Inc., our approach centers on using siRNA, which is designed to bind to and degrade the messenger RNA (mRNA) that carries the instructions for making these critical proteins. By doing so, we can significantly reduce the availability of these proteins, making it much harder for viruses to infect host cells.
Key Focus Area
Targeting Viral Entry Receptors:
ACE2 Receptor Knockdown: The ACE2 receptor is a primary entry point for viruses like SARS-CoV-2. By delivering siRNA that specifically targets the mRNA coding for ACE2, our therapies reduce the production of this receptor on the surface of host cells, thereby decreasing the virus’s ability to enter and infect the cells.
TMPRSS2 Knockdown: TMPRSS2 is a protease that facilitates the activation of viral spike proteins, enabling them to fuse with the host cell membrane. Knocking down TMPRSS2 expression through siRNA reduces the virus’s ability to initiate this fusion process, further inhibiting infection.
Precision Delivery via LNPs:
Our siRNA therapies are encapsulated within LNPs, which serve as delivery vehicles that protect the siRNA from degradation and ensure it reaches the target cells efficiently. The LNPs are designed to be cell-permeable, allowing the siRNA to enter the cells and reach the cytoplasm where gene knockdown occurs.
This targeted delivery system enhances the specificity and effectiveness of the gene knockdown, minimizing off-target effects and reducing potential side effects.
Benefits of Gene Knockdown Therapies
High Specificity: Our gene knockdown therapies are designed to target only the specific genes required for viral entry, leaving other cellular functions unaffected. This high level of specificity reduces the likelihood of unintended effects on non-target cells or tissues.
Rapid Response to Viral Variants: Because our therapies target host cell receptors rather than the virus itself, they are less likely to be rendered ineffective by viral mutations or variants. This makes gene knockdown an adaptable strategy in the face of evolving viral threats.
Synergistic Potential: Gene knockdown therapies can be used in combination with other antiviral treatments, such as LNP-based inhibitors, to provide a multi-layered defense against infections. By simultaneously blocking multiple pathways that a virus uses to enter cells, these combined treatments can significantly enhance overall efficacy.
Localized Treatment: Our therapies are administered via nasal sprays, targeting the primary site of infection for many airborne viruses. This localized approach ensures that the treatment acts where it is needed most, with minimal systemic exposure.
Ongoing Research and Development
Nanotreat Inc. is dedicated to advancing the science of gene knockdown through continuous research and development. Our current projects include:
Enhancing Delivery Systems:
Our research focuses on improving the stability and delivery efficiency of LNPs to ensure that the siRNA reaches the target cells intact and in the optimal dose.
Optimizing siRNA Sequences:
We are working to refine the siRNA sequences used in our therapies to maximize their effectiveness in knocking down ACE2 and TMPRSS2 expression, while minimizing any potential off-target effects.
Clinical Validation:
As we move towards clinical trials, we are rigorously testing our therapies in preclinical models to ensure their safety and efficacy. These studies are a crucial step towards bringing our innovative treatments to patients.
Expanding Target Profiles:
Beyond ACE2 and TMPRSS2, we are exploring additional targets that could further reduce the ability of viruses to infect cells, broadening the applicability of our gene knockdown therapies.
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