Revolutionizing Treatment for Rare Diseases and Conditions
The promise of vectorized gene therapy has ignited a beacon of hope for individuals battling debilitating diseases such as rare neurological diseases and metabolic disorders. Our groundbreaking pipeline, spearheaded by state-of-the-art vectorized gene therapy, is poised to halt disease progression and restore function in patients grappling with some of the most challenging diseases and conditions.
At the forefront of our efforts are two devastating pediatric diseases – severe pediatric obesity and 4H Leukodystrophy. For severe childhood obesity, a rare metabolic condition in young children, we are deploying a multi-targeted approach by activating specific pathways involved in thermogenesis, our bodies natural ability to burn calories and utilize stored fat. For 4H Leukodystrophy, a rare neurological disease, we are developing LNP-mRNA therapies which deliver mRNAs that code for therapeutic functional proteins which instruct cells to produce beneficial proteins.
For aggressive cancers, we are deploying a "smart" gene therapy system that works in tandem with antibody-drug conjugates (ADCs). This dual approach with trispecific nanoantibody drug conjugates uses vectors carrying tumor suppressor genes (TSGs) - the good guys that fight cancer growth to tackle the hardest cancer to treat such as Triple Negative Breast Cancer (TNBC).
Harnessing the power of mRNAs
We leverage mRNA and Lipid Nanoparticles (LNPs) technology, facilitating safe and efficient gene delivery to the targeted cells and tissues. The dose and duration of mRNA expression can be tailored to individual patient needs.
Treating the Root Causes of Obesity
At the forefront of our pipeline is a novel treatment for rare forms of childhood obesity such as Leptin Deficiency Disorder (LDD). Children with LDD lack a hormone that regulates appetite and metabolism, leading to severe obesity from a young age. Using a novel LNP-mRNA therapy to target obesity gene mutations, the goal is to restore leptin signaling to regulate appetite and increase energy expenditure.
Developing World’s First Treatment for 4H Leukodystrophy
Primarily a pediatric disease, this rare genetic disorder affects the nervous system causing developmental delays, motor difficulties, endocrine issues and other abnormalities. Using mRNA encased in LNP that delivers functional gene directly to affected cells. The goals is to enable the production of healthy protein to halt the progression of the disease and potentially restore function.
Companion Diagnostics for Early Detection and Intervention
In conjunction with our therapeutic efforts, we are also developing novel companion diagnostics that leverage miRNA biomarkers sourced from partner human clinical trials. These diagnostics hold the potential to detect complex diseases and cancers at their earliest stages, enabling timely intervention and maximizing the efficacy of our therapeutic approaches.
Partnering for Progress
We are dedicated to fostering collaborative partnerships that accelerate the development of our transformative therapies. We regularly engage with key partners to share pipeline updates and explore new avenues for therapeutic advancement.
Our Vision: Preserving Human Life
At the heart of our endeavors lies an unwavering commitment to preserving human life. Our pipeline of vectorized gene therapies represents a beacon of hope for individuals facing CNS proteinopathies and rare neurological diseases. We are driven by the unwavering belief that these groundbreaking therapies have the potential to halt disease progression, restore function, and transform the lives of countless individuals.
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.
