INNOSKEL

Innoskel is a pioneering bioscience platform company developing transformative therapies for the unmet needs of individuals with rare bone disorders.¹ In December 2020, it was announced that Innoskel had raised €20 million in a Series A financing round. The fundraise was co-led by Jeito Capital and Vida Ventures, with additional support from Région Sud Investissement and The Turenne Group. Proceeds from the financing will be used to advance the company’s lead program clinical stage operations. Innoskel was founded by Elvire Gouze, PhD, a scientific expert in skeletal disorders with over 20 years working in molecular pharmacology.²

Innoskel is developing treatment options for a group of rare skeletal disorders collectively known as type 2 collagenopathies that affect the structure of the body’s connective tissues (collagen). Innoskel’s lead asset, and initial point of focus, is a gene therapy for spondyloepiphyseal dysplasia congenita (SEDc), a type 2 collagenopathy that develops in infancy and affects approximately 1 in 100,000 individuals worldwide . SEDc is the second leading cause of dwarfism worldwide and is characterized by very short stature, severe skeletal and joint malformations, and abnormalities affecting the eyes and ears. The asset has demonstrated compelling efficacy in proof-of-concept studies conducted at the Institut de Biologie Valrose (iBV) of the University Cote d’Azur, Nice, France.²

In 2022, The Chandler Project expanded its focus at the 4th Annual Achondroplasia Research Conference to highlight research surrounding non-FGFR skeletal dysplasias such as SEDc. At the conference, Samantha Parker, Innoskel’s chief patient access officer, shared Innoskel’s commitment to advancing novel therapies to provide hope for those affected by rare bone disorders. In their pipeline, their lead asset is INS101, a gene therapy for type II collagen disorders, which has demonstrated strong efficacy in preclinical studies.

innoskel’s scientific approach

Innoskel is developing a robust pipeline of gene therapies that have the ability to target the cells responsible for cartilage formation and bone growth (chondrocytes) in the growth plate cartilage.³

Gene therapy for type II collagen disorders

Type II collagen disorders are classified into several phenotypes depending on severity and skeletal involvement. All forms of type II collagen disorder are caused by mutations (e.g. changes) in the COL2A1 gene. Typically the COL2A1 gene instructs cells in the growth plates (called chondrocytes) to make type II collagen. Type II collagen is necessary for the chondrocytes to line up, mature and convert into bone. With a faulty COL2A1 gene, chondrocytes cannot make type II collagen correctly and this affects bone formation and skeletal growth. Type II collagen is also present in the cornea, the vitreous of the eye and the inner ear, thus people with type II collagen disorders may also have affected vision and hearing.³

innoskel’s pipeline

Their lead asset is INS-101, a gene therapy for type II collagen disorders, which has demonstrated strong efficacy in preclinical studies.³
They are leveraging this technology and their know-how to develop other therapies to treat other serious rare bone disorders.³
As they progress their platform, they will be working closely with the patient community and their families.³
Innoskel is committed to advancing its novel therapies to provide hope for the rare bone disorder community.³

The most common type II collagen disorder causing disproportionate short stature is Spondyloepiphyseal Dysplasia congenita (SEDC). SEDC 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. Other complications may include respiratory insufficiency, joint pain, hip and spine deformities and early onset of osteoarthritis. INS-101 is designed to be administered systemically and restore COL2A1 function in growth plates. INS-101 has demonstrated strong efficacy in a mouse model, showing restoration of bone growth and prevention of disease complications.³