Innoskel Announces First Patient Enrolled in Observational Study to Evaluate Children Living with Type II Collagen Disorders

Valbonne, FRANCE

Innoskel Announces First Patient Enrolled in Observational Study to Evaluate Children Living with Type II Collagen Disorders

  • Study will build a type II collagen disorder observational data set to inform the design of clinical trials of novel therapies for patients with these debilitating disorders

Nice, France, 13 July 2022 – Innoskel (the “Company”), a pioneering bioscience platform company developing transformative therapies for the unmet needs of individuals with rare bone disorders, today announced the enrollment of the first patient in the ROCKET observational study. The study will evaluate the course of type II collagen disorders in children with short stature.

Elvire Gouze, Chief Executive Officer and Founder of Innoskel, 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.

ROCKET is an international multicenter study enrolling children aged from 0-12 years, with a confirmed diagnosis of a type II collagen disorder. Data will be prospectively collected on relevant endpoints ranging from clinical manifestations, including growth, physical and respiratory function, to laboratory values and patient-reported quality of life. 

Innoskel intends to leverage the data from ROCKET to inform the design of its planned interventional clinical trial for INS-101, the Company’s preclinical gene therapy candidate in development for the treatment of patients with type II collagen disorders. INS-101 is currently being investigated to restore normal cartilage growth in bone growth plates and to prevent the skeletal deformities that cause severe clinical complications in type II collagen disorders, such as cervical instability, hip deformity, early onset osteoarthritis, vision and breathing difficulties, and to date has demonstrated strong efficacy in preclinical studies.

Samantha Parker, Chief Patient Access Officer of Innoskel, said:It is our mission to pioneer novel treatments for people living with rare skeletal disorders. 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.


About Innoskel
Innoskel is a pioneering bioscience platform company developing transformative therapies for the unmet needs of individuals with rare bone disorders. Initially the Company is developing a gene therapy for a group of diseases known as type II collagen disorders, whilst also expanding the use of its technology and know-how to target and treat other rare bone conditions. The Company’s goal is to make a meaningful difference to the lives of underserved patient populations suffering from severe bone disorders. Innoskel’s main offices and laboratories are in Sophia-Antipolis Biotechnology Park, Nice, France.

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About type II collagen disorders
Type II collagen disorders are skeletal dysplasia resulting from mutations in the COL2A1 gene. Clinical presentations range from perinatal lethal disease to disproportionate short stature with progressive scoliosis/kyphosis, joint degeneration and pain or average stature children with high myopia, retinal detachment and early onset osteoarthritis. There is no disease modifying treatment available. Currently, treatment or standard of care consists of surgical procedures to reduce the risk of paralysis (e.g., cervicomedullary decompression or spinal fusion), surgery to treat malformation of the hips, and utilization of long-term modalities of care (e.g., brace therapy) to reduce pain and restore function.

About the ROCKET study
The ROCKET study aims to build a natural history data set through collection of a number of clinical, imaging, and laboratory assessments that may be specific predictors of type II collagen disorder progression and clinical outcome. Having a type II collagen disorder natural history data set can inform potential efficacy endpoints and biomarkers for future clinical trials. Further information on the ROCKET observational study can be found on the website (study identification number: NCT05408715) here.

For more information please contact:

Elvire Gouze, Founder and CEO

Consilium Strategic Communications
Mary-Jane Elliott / Melissa Gardiner / Lucie Foster
Tel: +44 (0) 20 3709 5700