Science & Technology

Our Technology

Unleashing the Power of RNAi Therapy
Canary is pioneering transformative therapy by harnessing RNAi technology, combined with large scale human data analysis and AI, to precisely reprogram metabolic function and engineer lasting health.

Key Differentiators:

There is a pressing need for next gen therapies that target the underlying causes of obesity, MASH and other metabolic disorders. Canary developing novel therapies that offers improved efficacy, safety and patient convenience.

Limitations of Traditional Gene Therapy Approaches:

What Makes Canary Unique

Superior Specificity: Our vectorized nanoantibody platform delivers LNP-encapsulated mRNA directly to target cells in the CNS and other targeted tissues, minimizing off-target effects and maximizing therapeutic impact.
Tailored LNP Design: We utilize proprietary LNP engineering to optimize delivery, with the potential to cross the blood-brain barrier and ensure stable, efficient mRNA release within targeted cells.
Personalized Medicine: The dose and duration of mRNA expression can be tailored to individual patient needs.
Disease-Specific Solutions: We apply our platform to develop individualized gene therapies for diverse diseases, including rare conditions with limited or no treatment options.
Rapid Prototyping and Development: Our innovative platform, supported by extensive in silico research, and preclinical and safety studies, enables rapid scalable and cost-effective development of gene therapies, accelerating time to market.

Revolutionizing Neurological Disease Treatment with Dual-Combination Gene Therapy

Addressing the Root Cause of Proteinopathies

Neurological disorders often arise from proteinopathies, characterized by the accumulation of misfolded proteins in the brain. These misfolded proteins disrupt cellular processes, leading to neuronal death and the progressive decline in cognitive and motor functions.

Canary’s dual-combination therapy targets the root cause of proteinopathies by employing two distinct yet synergistic mechanisms:

Vectorized Antibodies

One Target

Our proprietary vectorized antibodies are designed to specifically bind and neutralize misfolded proteins, preventing their aggregation and toxicity. These antibodies are engineered to cross the blood-brain barrier, ensuring effective delivery to the affected areas of the brain.

Functional Transgene Replacement

Multiple Target

We utilize gene therapy to replace defective genes with functional copies, correcting the genetic abnormalities that underlie protein misfolding. This approach restores the production of normal proteins, halting the progression of disease and promoting cellular repair.

Why is Canary Targeting the Endocannabinoid System

The endocannabinoid system plays a vital role in maintaining our metabolic homeostasis.

CB1 Receptors: Key to Metabolic Balance

CB1 receptors in our body affect appetite, glycemic control, lipid metabolism and overall energy balance. Their dysregulation can lead to metabolic disorders like type 1 & type 2 diabetes and obesity.

Power of RNAi

By using RNA interference (RNAi) to directly target the genetic cause of CB1R dysfunction, we can silence the gene responsible for CB1R expression in peripheral tissues, to achieve a more targeted and effective therapeutic outcome.

Precision & Safety

siRNA CB1R Therapy selectively targets CB1R in peripheral tissues, minimizing central nervous system side effects and reducing risk of events associated with global CB1R blockade

Metabolic Improvements

Addresses the root cause of CB1R dysfunction can lead to long-lasting metabolic improvements including insulin sensitivity, glucose tolerance, and lipid profiles.

Canary Cure’s Reprogram Platform

The REPROGRAM integrates three core technologies:

large-scale human data analysis + AI + siRNA technology for targeted gene silencing

Human Data Analysis

Map subcutaneous fat distribution & metabolic characteristics, identifying genetic and epigenetic markers that influence fat storage, browning, and metabolic activity.
Correlate subcutaneous fat composition with key metabolic health markers
Using this data-driven approach ensures that our targets have strong translational relevance and allows us to identify patient populations most likely to benefit from our therapies.

Artificial Intelligence

AI algorithms identify complex patterns in subcutaneous fat remodeling and systemic metabolic impact that might not be apparent through conventional analysis methods.
Our machine learning models optimize siRNA target selection by analyzing the complex interplay of genes regulating adipocyte identity and thermogenesis.
AI also helps predict patient-specific responses to fat browning therapies

Targeted Gene Silencing

Winning a Nobel Prize, siRNA technology is a powerful way to selectively 'turn off' disease-causing genes.
Unlike conventional drugs that might partially block a protein’s function, siRNA stops the protein from being made in the first place.
CCT-217’s dual silencing therapy triggers a complete reprogramming of fat cells from storage mode to energy-burning mode, and in turn further triggering many other metabolic benefits.

The Power of LNP mRNA Precision Medicine

Leveraging Proven Technology to Transform Gene and RNA Therapy for Rare Metabolic Disorders & Rare Diseases

What is LNP & how does it work:

Lipid nanoparticles (LNPs) are delivery vehicles composed of biocompatible lipids (fats) that encapsulate therapeutic agents. They offer important therapy advantages:

Biocompatibility: Their lipid composition closely resembles biological membranes, minimizing immune system reactions and toxicity.
Versatility: They can be customized to encapsulate various types of therapeutic agents for diverse applications.
Targeted Delivery: They can be functionalized with targeting ligands to specifically bind to receptors on cells in the central nervous system (CNS), enhancing drug delivery and reducing side effects.
Controlled Release: They can be designed to release their cargo gradually over time, providing sustained therapeutic effects.

What is mRNA & how does it work:

mRNA (messenger ribonucleic acid) is a molecule that carries instructions from DNA in the nucleus to ribosomes in the cytoplasm, telling them which proteins to build.
The mRNA is encased in lipid particles that protect it and help it enter cells. Once inside the targeted cell, the mRNA instructs the cell's machinery to make copies of specific proteins.
mRNA was first used in COVID-19 vaccines with great success.

Key Advantages:

How is Canary using AI to support R&D

Target Identification and Validation: AI can help analyze large datasets of genetic and molecular information. This has helped Canary find and prioritize promising targets for gene therapy development saving time and resources.
Vector Design and Optimization: AI is being used to help Canary design and optimize vectors used to deliver gene therapy payloads. Machine learning can analyze simulations of vector interactions with cells to predict potential problems and guide iterative design improvements.
Trial Design and Analysis: Canary is using AI for high-throughput analysis of pre-clinical data, significantly accelerating the analysis. AI will be used to analyze clinical trial data to identify early signs of efficacy or need for dosing changes, allowing for faster adjustments to trial design.
Regulatory Approval: Canary will use AI I to help analyze large datasets of pre-clinical and clinical data to prepare regulatory submissions, streamlining the approval process.

Canary’s Chorus-siRNA Technology Platform

The key differentiator of our Chorus-siRNA platform is its intentional exploitation of synthetic gene regulatory networks.We've engineered our dual siRNA components to leverage a novel natural cellular crosstalk, creating amplified effects. This synergistic approach allows for more potent gene modulation at lower doses, potentially reducing side effects while enhancing therapeutic efficacy.

The therapeutic advantages are substantial:

Lower effective doses translate to reduced potential for off-target effects and improved safety profiles.
Effectively addressing diseases characterized by compensatory mechanisms that have historically limited single-target approaches.
By targeting gene regulatory networks rather than individual genes, we can modulate entire disease pathways, addressing the root causes of complex conditions rather than just treating symptoms.
This technology creates a "resistance firewall." If a cell begins to develop resistance to one siRNA component through mutation or pathway rewiring, the second siRNA continues to exert pressure from a different angle, maintaining therapeutic efficacy.

RNAi drug development complemented by AI guided novel biomarker discovery and diagnostics.

Leveraging the power of AI-guided advanced diagnostics

The company uses AI to power its advanced diagnostic platforms, which allows it to identify novel biomarkers and track and monitor responses to therapies and medications.

Developing novel biomarkers

The company uses its advanced diagnostic platforms to develop novel biomarkers that can be used to predict disease risk, identify and characterize disease presence, and track and monitor responses to therapies and medications.

Using miRNA technologies

The company uses a combination of miRNA technologies, including SRM technology, to develop new drugs that can treat cancer and other life-threatening conditions.

Micro RNA mechanism of Action

The Approach

Limitations and solutions

Limitations

One Target

Our Solution

Multiple Target

The Science of RNAi

Leveraging the body’s naturally occurring ribonucleic acid interference (RNAi) response, Canary is working to develop therapies that target key disease proteins.

RNAi therapies utilize two short segments of RNA, known as short interfering, or silencing RNA (siRNA), to defeat proteins involved in the progression of certain diseases. The siRNA segments are encapsulated within a histidine-lysine polypeptide (HKP) that ensures their intact delivery to target cells within the body. Once taken up at a cellular level, the siRNA strands are released to activate an enzymatic process driven by the RNA-Induced Silencing Complex (RISC). Through this process, the messenger RNA strands of the disease proteins are targeted and cleaved, ultimately preventing their function to express proteins within a cell.

In addition to the synergistic effect of dual gene/protein targeting, RNAi therapeutics work to prevent the reproduction of harmful proteins within the body, rather than neutralizing existing disease. Additionally, this new class of drugs offers speedier development timelines compared to traditional methods of development involving the screening of small molecules or generating antibodies against a protein. This speedier development along with the potential for earlier treatment offers hope for improving the lives of patients who have been diagnosed with conditions ranging from cancer to fibrotic diseases.

Canary DigiGENE miRNA Diagnostic overview

Our point of care diagnostic platform uses Al to read miRNA expression patterns in complex diseases at the earliest stages.

Studies have shown a clear distinction in miRNA expression patterns between disease sample and normal healthy samples. This is a heat map of four breast cancer patients showing separation of miRNA expression profiles in tumors and normal tissues.

WHAT IS THE SCIENCE BEHIND RNA interference?

RNA interference (RNAi) is a natural process that cells use to regulate gene expression. It works by silencing the expression of genes that are involved in disease.

How does RNAi work?

RNAi is a two-step process. In the first step, a double-stranded RNA (dsRNA) molecule is cleaved by an enzyme called Dicer. This produces two single-stranded RNA (ssRNA) molecules, which are called siRNAs (short interfering RNAs).

In the second step, the siRNAs bind to a protein complex called the RNA-induced silencing complex (RISC). The RISC then binds to the target mRNA and cleaves it, preventing it from being translated into protein.

AntiMirs

AntiMirs are short RNA molecules that are designed to bind to and inhibit the function of miRNAs. They can be used to treat diseases that are caused by the overexpression of miRNAs.

AntiMirs can be delivered to cells in a variety of ways, including

Intravenous injection
This is the most common method of delivering AntiMirs
Intramuscular injection
This method is used when the target cells are located in the muscle tissue
Intranasal delivery
This method is used to deliver AntiMirs to the cells in the nose and throat
Inhalation
This method is used to deliver AntiMirs to the lungs
Topical delivery
This method is used to deliver AntiMirs to the skin

Canary's AlloMir gene-silencing platform can be leveraged to investigate a variety of diseases and conditions

Cancer

Viral infections

Autoimmune diseases

Genetic diseases

Neurological diseases

Advantages of AntiMir technology

Stability

AntiMirs are more stable than siRNAs. This is because they are single-stranded RNA molecules, while siRNAs are double-stranded RNA molecules. Double-stranded RNA molecules are more easily degraded by enzymes in the bloodstream and tissues.

Specificity

AntiMirs can be designed to be more specific than siRNAs. This is because they can be designed to bind to a specific miRNA, while siRNAs can bind to a range of miRNAs.

Safety

AntiMirs may be safer than siRNAs. This is because they are less likely to trigger an immune response.

AntimicroRNA (AntiMir) therapeutics have several potential advantages over siRNA therapeutics:

Broader therapeutic application

AntiMIR's can target a wider range of genes than siRNAs, as they can be designed to mimic the binding sequences of endogenous miRNAs, which regulate the expression of hundreds or even thousands of genes. This makes AntiMIR's more attractive for the treatment of complex diseases with multiple genetic components.

More potent gene silencing

AntiMIR's are more potent gene silencing agents than siRNAs, as they can trigger the degradation of multiple target mRNAs simultaneously. This is because AntiMIR's can bind to multiple miRNA-binding sites on a target mRNA, while siRNAs can only bind to one site.

Reduced off-target effects

AntiMIR's are less likely to have off-target effects than siRNAs, as they are designed to mimic the binding sequences of endogenous miRNAs. Endogenous miRNAs are highly conserved, which means that their binding sequences are very similar between different cell types and tissues. This reduces the risk of AntiMIR's binding to and silencing unintended genes.

More stable in vivo

AntiMIR's are more stable in vivo than siRNAs, as they are less likely to be degraded by nucleases. This is because AntiMIR's are more resistant to cleavage by Dicer, the enzyme that cleaves double-stranded RNA into siRNAs.

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