A Revolutionary Approach to Gene Therapy

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

Our Mission
To revolutionize healthcare by harnessing the power of cutting-edge RNA and gene therapies to develop and deliver innovative treatments that improve the lives of individuals and families. We are committed to fostering a culture of scientific excellence and collaboration to create life-changing treatments that are safe, effective, and accessible to all.
Our Approach
Our Focus
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
Our gene therapy pipeline explores novel vectors and next-gen combination therapies with a focus on developing transformative treatment platforms for a wide spectrum of metabolic and neurological disorders, and aggressive cancers. 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 Changing Lives
Join us in our mission to revolutionize gene and RNA therapy and transform the lives of individuals affected by the most challenging diseases to treat. 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.
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 gene and RNA therapy to transform healthcare for all.

Raj Reddy

CEO & President, Founder

View Details

Anna Wang

Chief Strategy Officer, Co-Founder

View Details

Dr. Guangping Gao, PhD

Senior Scientific Advisor, Gene Therapy and Rare Diseases

View Details

Dr. Paul Cohen, MD, PhD

Senior Scientific & Medical advisor, Molecular Metabolism

View Details

Dr. Pierre Rosenzweig, MD

Senior Clinical Development advisor, Obesity & Metabolic Disorder

View Details

Dr. Lawrence Kazak, PhD

Senior Scientific Advisor, Obesity & Metabolic Disorders

View Details

Abby Gregor

Legal and Licensing Advisor

View Details

Claire Henchcliffe, MD, D.Phil

Senior Medical Advisor, Neurological Disorders, Parkinson’s Disease

View Details

Dr Christine Duncan

Senior Scientific and Medical advisor , Rare Neurological Disease

View Details

Dr Troy Lund

Senior Scientific and Medical advisor , Rare Neurological Disease

View Details

Dr. Sanjay Gandhi

MD Senior Medical Advisor

View Details
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.

Latest Updates

Our Commitment

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