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For people living with rare genetic bone disorders, everyday life can be unpredictable. A minor injury, a routine dental visit, even normal growth can trigger permanent bone formation that limits movement, causes chronic pain, and shortens life expectancy.

A Queen’s-founded biotech startup is now working to change that reality.

, a company rooted in years of Queen’s-led research is developing a potential treatment that is currently in preclinical research for two rare conditions that cause abnormal bone formation outside the skeleton. The goal is early pharmaceutical intervention to help preserve mobility, independence, and quality of life.
 

Improving outcomes for rare genetic conditions

Queen’s researcher (Biomedical and Molecular Sciences) is the scientific founder of Ocythera. His research spans fundamental biology, with current therapeutic applications focused on fibrodysplasia ossificans progressiva (FOP), and hereditary multiple osteochondromas (HMO).

FOP is one of the rarest genetic disorders in the world, affecting fewer than one in two million people. In individuals with the condition, normal wound healing goes wrong. Instead of repairing soft tissue, the body forms bone inside muscles and connective tissue. The disease causes progressive and irreversible loss of mobility, often leading to chronic pain and severe physical impairment. 

“In FOP, something as simple as a bruise can lead to permanent bone formation,” says Dr. Petkovich. “Over time, those injuries accumulate and progressively limit a person’s ability to move, eat, and live independently.”

HMO is not as rare but still affects a small population. It causes multiple bone tumours that often begin forming in childhood, interfering with growth and movement. The condition can result in skeletal deformities, short stature, scoliosis, and early-onset osteoarthritis, and in rare cases the tumours can become malignant. 
 

Limits of current treatments

There is only one treatment approved for FOP which works by exposing the entire body to high levels of retinoids, and nothing has yet been approved for HMO, leaving multiple surgical interventions as the only means to improve the lives of these patients. That can be difficult to manage, especially in children whose bones are still growing.

Ocythera’s strategy takes a different route. Rather than adding retinoids, the company’s therapy aims to control how retinoic acid is metabolized in specific tissues.

“We’re focused on understanding whether intervening earlier can change how these conditions progress,” says Dr. Petkovich. “That question is especially important for children whose bones are still developing.”
 

Path of discovery 

Dr. Petkovich’s path to addressing these conditions began long before Ocythera existed. He has spent much of his career studying how Vitamin A influences growth and development in the body. That work helped reveal how Vitamin A, through a molecule called retinoic acid, regulates gene activity. Retinoic acid plays a key role in bone and cartilage development, but only when its levels are tightly controlled.

His team identified an enzyme, produced by the gene CYP26B1, that breaks down retinoic acid and helps guide how tissue repairs itself after injury. Years later, that enzyme became the central drug target behind Ocythera’s approach.

Much of that insight came from studying tissue regeneration in zebrafish, a model organism widely used in research to understand how tissues grow and repair themselves. By observing how zebrafish regenerate fins after injury, Petkovich’s team was able to track how retinoic acid levels rise and fall during normal healing.

“Retinoic acid is essential, but it has to be turned on and off at the right time,” says Dr. Petkovich. “What we uncovered was a critical mechanism the body uses to regulate that process.”

Petkovich has founded three therapeutic companies over the course of his career, giving him experience in moving discoveries from academic research toward clinical development.
 

Research shaped by collaboration

Advancing the research required contributions from experts in chemistry and disease biology. Dr. Petkovich’s colleague, and Queen’s research associate, Donald Cameron refined compounds that block the enzyme identified by Petkovich’s lab, while researcher Tracie Pennimpede (Biomedical & Molecular Sciences) developed disease models that closely reflect how abnormal bone forms in FOP, and (Ophthalmology) provided guidance as a clinical advisor.

From left to right: lab group members Dr. Tracie Pennimpede, Sarina Lee, and Dr. Don Cameron.

Taking the work beyond the laboratory required support that extended beyond academic research alone. As the project advanced, issues such as intellectual property protection, research agreements, and external partnerships were coordinated through Queen’s Partnerships and Innovation.

“They helped us navigate the early stages and made critical introductions,” says Dr. Petkovich. “That support was essential in getting Ocythera off the ground.”

One of those introductions led to , a pan-Canadian organization that helps innovators overcome early-hurdles,  and translate health research into strong, investable, Canadian life sciences companies. adMare provided early funding and scientific expertise, including undertaking critical biology and chemistry studies, that helped move the research toward clinical application. 

Queen’s research infrastructure also supported this phase of the work. Preclinical studies drew on advanced imaging and technical expertise through the , allowing the team to examine how abnormal bone formed and how it responded to treatment.
 

Preparing for clinical trials 

adMare has now re-invested in the company, alongside Investissement Quebec, to support the next phases of development. Ocythera is now completing the studies required before applying to Health Canada and the U.S. Food and Drug Administration to begin human clinical trials, including safety testing, manufacturing planning, and regulatory review.

“For people living with these conditions, there are very few options,” says Dr. Petkovich. “That reality is what drives the work forward and makes it important to keep pushing the science.”