Beyond Treatment:
Gene Silencing for Metabolic Transformation

The Future of Genomic Medicine and Precision Diagnostics

LATEST STUDY RESULTS:

CCT-217 Demonstrates Significant Weight Loss and Preserves Lean Body Mass with Potential for Single or Twice-Yearly Dosing

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On Feb 29
Rare Disease Day
Canary is Committed to Developing Next Gen Treatments for Diseases that Impact 300 Million People Globally.
Our Mission
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.
Our Approach
Our Focus
Energy-storing human white adipocytes before treatment ​

Energy-storing human white adipocytes before treatment ​

Transformation towards fat burning human beige adipocyte

Transformation towards fat burning human beige adipocyte

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

This reprogramming of human fat cell to create metabolically active cells represents a significant breakthrough in translational adipose tissue research with profound implications for obesity therapeutics.
The first microscopic field reveals a dense population of human adipocytes which stores energy. The second picture reveals adipocytes that have undergone remarkable morphological transformation, following CCT-217 siRNA treatment into smaller, more numerous lipid droplets, in just 72 hours. The increased multilocularity (multiple small lipid droplets within cells) is characteristic of enhanced thermogenic capacity.
Beige adipose tissues predominantly functions to dissipate energy surplus, increasing resting energy expenditure and counteracting obesity. This is precisely the mechanism of action in Canary’s CCT-217 which treats obesity through the transformation of our white fat into metabolic active, fat burning beige fat.
Therapeutics

Harnessing the Power of Thermogenesis to Combat Obesity

Therapeutics

RNA Interference (RNAi) for Therapeutic Breakthroughs

Therapeutics

Next Generation Gene Therapy with Novel Vectors

LNP-mRNA Therapies Guided by Precision Nanoantibodies

Revolutionizing Gene Therapy with Powerful Novel Vectors

Revolutionizing Gene Therapy with Powerful Novel Vectors

Neurological disorders affect nearly 1 billion people worldwide, and many of these conditions are currently untreatable. Traditional therapies often fall short in addressing the underlying causes of these debilitating diseases, leaving patients and their families with limited options and dwindling hope.
At Canary, we are pioneering a transformative approach to gene therapy, harnessing the power of next-gen non-viral vectors and adeno-associated viruses (AAVs) to deliver therapeutic genes directly to the central nervous system (CNS) with remarkable efficacy and tolerability. Our innovative gene therapies hold the promise of not only halting disease progression but also restoring neurological function, offering hope to countless individuals and families affected by neurological disorders.
Restoring Myelin Integrity
Adrenoleukodystrophy (ALD), a devastating neurological disorder that affects both children and adults, is a prime target for our gene therapy approach. ALD is characterized by the progressive destruction of myelin, the protective sheath that surrounds nerve fibers, leading to severe neurological impairment. Our gene therapies aim to restore myelin integrity, halting disease progression and potentially reversing neurological deficits.
Harnessing the Power of AAVs
AAVs, nature's own delivery vehicles, have emerged as a powerful tool in gene therapy, offering a safe and efficient means to introduce therapeutic genes into target cells. Our proprietary AAV vectors are meticulously designed to navigate the complexities of the CNS, delivering the therapeutic cargo with precision and minimal disruption.
Revolutionizing Gene Therapy with Powerful Novel Vectors

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 is a devastating pediatric disease – 4H Leukodystrophy. We are developing LNP-mRNA therapies which deliver mRNAs that code for therapeutic functional proteins which instruct cells to produce beneficial proteins. We leverage the precision of antibodies to specifically target diseased cells in the CNS, ensuring highly selective delivery of therapeutic mRNA.
Our Pipeline
Leveraging the power of RNA therapeutics to revolutionize the treatment landscape metabolic disorders, Canary’s cutting-edge pipeline harnesses the potential of gene silencing technology to precisely target the underlying genetic causes of obesity, MASH as well as metabolic dysfunction that impact other diseases.
Transforming Obesity Therapy
Unlike conventional approaches, our therapies prioritize fat loss, preservation of lean body mass and body composition improvement. With the convenience of less frequent dosing and better safety profile, this siRNA offers a promising solution for individuals battling obesity and its associated metabolic complications.
CNS Proteinopathy

CNS Proteinopathy

Neurological Disease

Neurological Disease

Our Team

With global industry experience, our team values collaboration, shares a passion for finding solutions to life’s biggest challenges and a desire to unlock the power of RNA therapy to transform healthcare for all.

Raj Reddy

President and CEO, Canary Cure Therapeutics

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Anna Wang

Chief Strategy Officer, Co-Founder

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Dr. Paul Cohen, MD, PhD

Senior Scientific & Medical advisor, Molecular Metabolism

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Dr. Pierre Rosenzweig, MD

Senior Clinical Development advisor, Obesity & Metabolic Disorder

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Dr. Lawrence Kazak, PhD

Senior Scientific Advisor, Obesity & Metabolic Disorders

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Abby Gregor

Legal and Licensing Advisor

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Revolutionizing Treatment for a Rare Pediatric Disease
Caused by POLR3A mutations leading to insufficient RNA polymerase III enzyme impacting protein production and causing severe neurological degeneration, 4H Leukodystrophy is primarily a pediatric disease with no cure. This devastating rare genetic disorder affects the nervous system causing developmental delays, motor difficulties, endocrine issues and other abnormalities.
Canary is developing a first-ever treatment for 4H Leukodystrophy using mRNA encased in LNP that aims to deliver functional POLR3A mRNA directly to affected cells, bypassing the faulty gene, enabling the production of healthy protein. We leverage the precision of antibodies to specifically target diseased cells in the central nervous system with the goal of a highly selective and targeted delivery of therapeutic mRNA. Our proprietary LNP is being formulated to enhance stability and intracellular mRNA release, maximizing therapeutic efficacy.

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Our Commitment

We are committed to advancing the field of RNAi therapy and improving the lives of patients worldwide. Our team of scientists, engineers, and clinicians is dedicated to: