Company Spotlight

Cook MyoSite: Advancing Regenerative Medicine 

Cook MyoSite is redefining how the body heals itself—using a person’s own muscle cells to restore function where it's been lost. With late-phase clinical trials underway for its first three indications, the Pittsburgh-based company is staking out a leadership position in regenerative medicine. A subsidiary of Cook Group and a sister company to Cook Medical, it draws on decades of healthcare innovation. 

At the heart of the company’s therapeutic development is iltamiocel, an autologous muscle-derived cell (AMDC) technology that takes a small muscle biopsy—from the thigh—and processes it to isolate and grow muscle progenitor cells. These personalized cells are then reintroduced into smaller, weakened muscles elsewhere in the body to restore their strength and function. 

“We’re a drug developer. Our drug just happens to be a biologic drug that is a patient’s own cells,” said Ron Jankowski, Cook MyoSite’s vice president of regulatory and scientific affairs.

Cook MyoSite currently has three clinical programs in development, each of which has received the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation—an achievement that helps streamline the development path for transformative therapies. 

The company focuses on conditions that severely impact quality of life—particularly for patients who have exhausted existing treatment options. Two of its three clinical indications focus on women’s health conditions that are typically underserved in drug development.

“We aim to help patients who’ve often had severe symptoms for years—patients for whom there are no effective drugs, and surgery hasn’t worked,” said Jankowski. “Our therapies are designed to address the root cause by strengthening the muscles that are no longer functioning properly.”

Cook MyoSite’s lead program targets stress urinary incontinence in women— bladder leakage triggered by physical exertion—a condition typically caused by aging or childbirth. Up to one-third of women experience this condition at some point in their lives. The Phase 3 trial for this indication completed enrollment in mid-2025. 

The company’s second program focuses on post-obstetric fecal incontinence, with its Phase 3 trial expected to finish enrolling this year. The third program targets oropharyngeal dysphagia—difficulty swallowing caused by weak throat muscles—and is being evaluated in a Phase 2 trial.

As with most targeted therapies, the therapeutic agent itself is just part of the solution. The other key component is the delivery mechanism. Cook Myosite pays close attention to the injection procedure to ensure that the cells are delivered and distributed within the precise target area of need.

Beyond its clinical pipeline, Cook MyoSite supports the broader research and drug development fields through its research services arm, which provides tools, consulting and access to its comprehensive muscle cell bank to other drug developers.

Pittsburgh Born and Bred

Privately owned and proudly Pittsburgh-based, Cook MyoSite benefits from the region’s world-class universities, deep talent pool, and exceptional quality of life.

“Cook Myosite was born and raised in Pittsburgh, and we’re proud of that,” said Jankowski. “It’s incredibly rewarding to develop innovative science here at home—science that addresses some of the toughest medical problems—and to contribute to the growth of the region’s thriving biotech ecosystem.”

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