CCT-217

A New Category of Metabolic Therapy: Thermogenic Adipose Reprogramming

Our Approach

• RNAi Therapeutics: We leverage the power RNAi therapeutics to modulate gene expression and correct underlying genetic defects. These technologies offer precise and customizable treatment options for a wide range of diseases.
• Reprogram Platform: Integrating three core technologies: large-scale human data analysis to ensure that our targets have strong translational relevance, AI to optimize siRNA target selection and siRNA technology to selectively 'turn off' disease-causing genes.
• Act Locally, Treat Systemically: Where the therapeutic agent remains localized at the treatment site while the biological effects spread throughout the body through bioactive signaling pathways, treating the root causes of disease beyond the symptoms.
• Speed to Market: Our R&D strategy which incorporates AI supported research and accelerated regulatory pathways, prioritizes speed and efficiency to deliver life-saving treatments to patients as quickly as possible.

Our Focus

Three Disease Areas. One Platform.

• Obesity: Unlike conventional approaches, our therapies prioritize fat loss, preservation of lean body mass and body composition improvement. Our novel dual siRNA therapeutics target the energy expenditure systems - promoting the browning of white fat to increase metabolism and facilitate sustainable healthy weight loss. With the convenience of less frequent dosing and better safety profile, this dual gene silencing therapy offers a sustainable biological solution where current therapies only offer temporary suppression.
• MASH (Metabolic Dysfunction-Associated Steatohepatitis): By activating the liver’s own fat-burning signals through the adipose endocrine system, our therapies address the underlying metabolic dysfunction driving fatty liver disease and inflammation — without targeting the liver directly.
• Rare Metabolic Disease: In conditions where disordered energy metabolism drives pathology, Canary’s ability to re-establish healthy metabolic balance offers a pioneering therapeutic approach for patients with Prader-Willi Syndrome, Hypothalamic Obesity, Bardet-Biedl Syndrome, and Congenital Lipodystrophy. We are developing a dual RNA metabolic restoration platform that directly modulates the gene expression pathways driving these conditions, offering a fundamentally different approach from the symptomatic strategies that families and clinicians currently rely on.

CCT-217’s Remarkable Ability to Beige Human White Fat

Harnessing the Power of Thermogenesis to Combat Obesity

• By activating specific pathways involved in thermogenesis, our therapy aims to boost the body’s ability to burn calories and utilize stored fat.
• Our innovative technology utilizes lipid nanoparticles (LNPs) to deliver therapeutic molecules directly to the tissues responsible for generating heat. This targeted approach aims to increase energy expenditure and promote fat burning.
• This approach promotes thermogenesis (fat burning) and lipolysis (breakdown of fats), leading to more energy expenditure and broader metabolic health.

RNA Interference (RNAi) for Therapeutic Breakthroughs

• RNA interference (RNAi) is a natural cellular process that acts as a defense mechanism against viruses. It uses small interfering RNAs (siRNAs) to target and silence specific genes, effectively preventing the production of harmful proteins.
• Unlike most conventional drugs, which target proteins, RNAi therapies target the root cause of disease by addressing the genetic instructions that lead to the production of faulty proteins.
• This "upstream" approach allows us to tackle conditions previously considered "undruggable," opening new possibilities for treatment.
• With a proven track record of safety and efficacy, RNAi represents a promising avenue for developing the next generation of targeted therapies.

Next Generation Gene Therapy with Novel Vectors

• Our innovative approach combines vectorized antibodies with functional transgene replacement to mutant genes to provide comprehensive and lasting solutions to metabolic and neurodegenerative disorders and rare diseases.
• Our proprietary vectorized antibodies are designed to specifically bind and neutralize misfolded proteins, preventing their aggregation and toxicity.
• Our approach aims to restore lost neurological function by correcting the underlying genetic and proteinopathies.
• Our goal is to arrest disease progression with a single administration of our dual-combination therapy, eliminating the need for lifelong medication and its associated side effects.

LNP-mRNA Therapies Guided by Precision Nanoantibodies

• We aim to treat existing diseases by instructing cells to produce beneficial proteins and/or correcting abnormal protein production.
• We leverage the precision of antibodies to specifically target diseased cells, ensuring highly selective delivery of therapeutic mRNA.
• Our proprietary LNP formulations enhance stability and intracellular mRNA release, maximizing therapeutic efficacy with potential for re-dosing.
• LNP-mRNA technology is a robust, well-proven, and transformative platform that is adaptable to diverse mRNA payloads and target cell populations, enabling rapid development of therapies for various diseases.

Revolutionizing Gene Therapy with Powerful Novel Vectors