InnoSkel Awarded Innovation Passport by the UK MHRA for the Treatment of a Rare Bone Disorder

INS-101, InnoSkel’s lead gene therapy program, is being developed for the treatment of SEDc in pediatric patients, for whom there is currently no disease-modifying therapy available. SEDc is a rare genetic bone disorder that is present from birth and is associated with skeletal deformities that worsen with age, some of which can be life-threatening requiring repeat surgical procedures. Patients with SEDc also experience a number of other severe complications, including irregular bone growth, respiratory insufficiency, joint pain, hip and spine deformities and early onset of osteoarthritis. INS-101 is designed to be administered by a systemic delivery route to restore COL2A1 function in growth plates, making it an ideal candidate to treat SEDc. INS-101 has demonstrated strong efficacy in a mouse model, showing restoration of bone growth and prevention of disease complications. Read More