Innoskel has enrolled the first patient in the global, observational ROCKET study of its preclinical gene therapy candidate INS-101 to analyse the course of type II collagen disorders in children living with short stature.

The multi-centre study is enrolling children of the age zero to 12 years, with a confirmed diagnosis of a type II collagen disorder.

The company plans to collect data prospectively on relevant endpoints that range from clinical manifestations, including physical and respiratory function, growth, to laboratory values and quality of life reported by the patient.

Innoskel CEO and founder Elvire Gouze said: “The significant lack of data available on type II collagen disorders highlights the important need to collect this type of data to not only inform clinical development but also to help with clinical care including establishing best practices.

“We look forward to partnering with our sites to execute this study and increase our collective knowledge so that we may efficiently advance INS-101 with a goal of delivering a new and much needed treatment for these children.”

By leveraging the ROCKET study results, the company will inform the design of its planned interventional clinical trial for INS-101.

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This therapy is being developed to treat patients with type II collagen disorders.

At present, INS-101 is being investigated for restoring normal cartilage growth in bone growth plates and prevention of the skeletal deformities that lead to severe clinical complications in collagen disorders.

These complications include hip deformity, early onset osteoarthritis, cervical instability, vision and breathing difficulties.

Innoskel chief patient access officer Samantha Parker said: “With INS-101, we have the potential to fundamentally change the treatment paradigm for children with type II collagen disorders and provide them with a meaningful difference in their quality of life.

“The findings from this observational study will help us accelerate the development of INS-101 and bring this transformative therapy to individuals who have been waiting for new options.”

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