Rare Metabolic Disorders

Complex Genetic Disorders Require Multidisciplinary Approaches

Pioneering RNA Therapeutics for Metabolic Restoration

Rare metabolic genetic disorders are devastating problems for children and their families. Obesity caused by gene mutations, such as in Prader-Willi Syndrome (PWS), causes children to have an insatiable appetite and rapid weight gain. The early-onset obesity can cause cardiovascular and metabolic comorbidities and is associated with increased mortality.

PWS, a rare, complex genetic disorder requiring multi-disciplinary care currently has no cure. Our lead product, CCT-987 is designed to directly correct the peripheral adipose biology by delivering a payload of CB1R siRNA and Leptin mRNA directly to subcutaneous adipose tissue. The dual RNA therapy has the potential to achieve true metabolic restoration – driving profound improvements in the fat-to-muscle ratio and systemic metabolic parameters that far exceed the capabilities of centrally acting agents.

"While the field has made vital strides in managing the behavioral symptoms of PWS, the structural and metabolic deficits have remained largely out of reach. CCT-987 changes that paradigm. A treatment that promotes weight loss while actively enhancing lean mass isn't just an improvement—it's a fundamentally different therapeutic proposition.

For these patients, achieving metabolic restoration and reversing profound muscle weakness could be absolutely life-changing.”

- Dr. Jennifer Miller, MD
Renowned Clinician and KOL in Pediatric Endocrinology and PWS

No approved disease-modifying therapy exists for PWS

Canary Cure’s Solution:

Effective Prader-Willi Syndrome treatment requires a dual-organ approach for full metabolic restoration. Obesity and metabolic dysfunction in PWS is not solely brain-driven appetite disorder, the patient’s fat is also “broken”. Driven by two parallel, independent genetic lesions, PWS requires a comprehensive metabolic solution.
What CCT-987 Does A dual RNA therapy that reaches the diseased fat cells directly — not the brain — and reprograms that ‘broken fat’ so it matures into healthy, working tissue again.

Repairs the SNORD116 fat defect — the true root cause of PWS, left untreated until now
Targets the muscle weakness (hypotonia) that existing therapies cannot treat.
Works alongside current therapies — different mechanism, no overlap
Directly and physically corrects the congenital peripheral adipose defect — ideally suited to establish a baseline of metabolic health early in life.

Mechanism of Action

While silencing the CB1R gene removes the β-arrestin1 brake, allowing physiological leptin to trigger a potent metabolic reset signal. This transitions the treatment paradigm from “behavioral muting” to “molecular repair.

Component	Target Action	Function Result
CB1R siRNA	Silences the CB1R receptor gene locally in the adipocyte	Removes the Brake: Prevents β-arrestin1/TC-PTP recruitment, restores stat3 phosphorylation, restores leptin signaling pathway
Leptin mRNA	Reprograms fat cells to secrete high-fidelity leptin signaling	Restores the Signal: Replaces the missing message via a pulsatile, physiological rhythm.

CCT-987’s RESCUE MECHANISM

CCT-987 is delivered as a dual-cargo lipid nanoparticle (LNP) using Canary’s proprietary Hyper-15™ formulation, administered via subcutaneous injection directly into adipose tissue.

Dosing every 3 weeks.

Offloading Stress

Preventing Glucotoxicity

Adipose Sink

Pioneering RNA Therapy

We are revolutionizing healthcare by harnessing the power of cutting-edge RNA therapies to develop and deliver innovative treatments that address the root causes of diseases. 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.

Disclaimer: Product candidates discussed on this website are in preclinical and/or investigational stages of development. They have not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. The safety and efficacy of these investigational products have not been established, and they are not currently available for commercial.