Our research delves into the complexities of misfolded protein aggregation in the central nervous system and dysregulated microRNA expression, which are implicated in a wide range of diseases including neurodegeneration, metabolic disorders, and aggressive cancers. By addressing these underlying causes, we strive to develop groundbreaking therapies that can truly transform the lives of patients with rare diseases and hard to treat conditions.
A Revolutionary Approach to RNA & Gene Therapy
1. Vectorized Antibodies:
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.
2. Targeted LNP mRNA Therapy:
We harness the power of lipid nanoparticle (LNP) delivery systems to introduce mRNA precisely into the cells most affected by disease. This mRNA carries genetic instructions for therapeutic proteins. This strategy maximizes therapeutic benefit while minimizing potential side effects, offering a new frontier in personalized disease treatment
A Multi-Pathway Approach to Treating Metabolic Disorders
Gene mutations disrupt the body's normal regulation of appetite and energy expenditure, leading to excessive weight gain. Often appearing at an early age, this type of rare metabolic disorders including Leptin Deficiency Disorder (LDD) can be debilitating leading to life-long health problems. The current solutions which largely focus on suppressing appetite are not designed for childhood obesity and don't address the underlying genetic cause of the condition.
Children with LDD lack a critical hormone that regulates appetite and metabolism. At Canary, we are developing a new multi-target approach using mRNAs encapsulated in LNPs to restore leptin signaling to regulate appetite and increase energy expenditure. Our focus is to restore the body’s natural way of regulating energy balance.
Harnessing the power of thermogenesis
Thermogenesis, the conversion of energy into heat is our body’s fat burning and energy expenditure mechanism. By activating specific pathways involved in thermogenesis, our therapy aims to boost the body's ability to burn calories, utilize stored fat and regulate energy balance.
Restoring leptin signaling
Children with Leptin Deficiency Disorder (LDD) lack a hormone that regulates appetite and metabolism leading to severe obesity from a very young age. Using lipid nanoparticles (LNPs) to deliver mRNA to target obesity gene mutations, our goal is to restore leptin signaling to regulate appetite, increase energy expenditure and boost the body’s own ability to burn fat while ensuring energy needs are met.
A Paradigm Shift in Neurological Disease Treatment
Canary’s dual-combination therapy represents a paradigm shift in neurological disease treatment. Our approach offers several key advantages over conventional therapies:
Revolutionizing Gene Therapy with Powerful Novel Vectors
The promise of vectorized gene therapy has ignited a beacon of hope for people battling debilitating central nervous system (CNS) proteinopathies and rare neurological diseases. Our groundbreaking pipeline spearheaded by novel vectorized gene therapy is poised to halt disease progression and restore function.
At the forefront of our efforts are two devastating CNS diseases – 4H Leukodystrophy and Early Onset Parkinson’s Disease (EOPD). For 4H Leukodystrophy, a rare pediatric disease, we are developing LNP-mRNA therapies which deliver mRNAs that code for therapeutic functional proteins which instruct cells to produce beneficial proteins. Upregulating expression of the PINK1 and/or Parkin genes and clearing toxic protein are the goals of our EOPD therapy which leverages our proprietary vectorized nanoantibody platform.
Our Pipeline
Our gene therapy pipeline is expanding rapidly, with a focus on developing transformative treatments for a wide spectrum of neurological disorders. We are committed to translating our cutting-edge research into clinical applications, bringing hope to patients and their families who have long awaited effective treatments.
Join us in our mission to revolutionize gene therapy and transform the lives of individuals affected by neurological disorders and rare diseases. Your support will fuel our research and development efforts, bringing us closer to a future where these debilitating conditions can be cured and lives are restored.Together, we can make a difference.
