Gene therapy player InnoSkel debuts with €20M for rare skeletal diseases

Gene therapy player InnoSkel debuts with €20M for rare skeletal diseases

Elvire Gouze Ph.D., a serial entrepreneur and expert in skeletal disorders, has formed another company—InnoSkel—after agreeing to sell her first venture Therachon to Pfizer for $340 million upfront last year.

Like Therachon, InnoSkel is focusing on diseases that result in dwarfism, but using a different approach. Therachon’s lead drug TA-46 was a soluble FGFR3 receptor designed to interrupt a molecule pathway linked to dwarfism, but InnoSkel will focus on gene therapies that will target underlying defects as a one-shot therapy.

InnoSkel officially launches with €20 million ($24 million) in first-round financing led by Jeito Capital alongside Vida Ventures and the Turenne Group to advance the platform, which has grown out of work conducted at Gouze’s lab within French institute Inserm.

The Nice, France-based biotech is focusing initially on a group of rare skeletal disorders collectively known as type 2 collagenopathies that affect the structure of the body’s connective tissues. First in the pipe is a gene therapy candidate for spondyloepiphyseal dysplasia congenita (SEDc), the leading cause of dwarfism worldwide. For now, however, details of the program are being kept under wraps.

“We are developing a gene therapy approach that targets the growth plate cartilage,” said Gouze. “We can’t give any specifics at this stage as we have a scientific publication submitted, but the goal is to repair the cartilage allowing for bone growth.”

SEDc is a type 2 collagenopathy caused by a mutation in the COL2A1 gene, which develops in infancy and affects approximately 1 in 100,000 individuals worldwide. It causes very short stature, severe skeletal and joint malformations, and abnormalities affecting the eyes and ears.

InnoSkel has carried out a proof-of-concept study the Institut de Biologie Valrose of the University Cote d’Azur in Nice, which used a mouse model of SEDc.

“In these mice, the gene therapy restores a near normal bone growth that leads to the prevention of the main complications, i.e., neck instability, thorax and spine deformities,” according to Gouze.

“This is highly important because in developing an innovative treatment for skeletal dysplasia, the most important factor is to help the patient with decreasing the complications, pain and need for surgeries,” she added.

At the moment, patients with SEDc have no treatment options and have a very poor quality of life. They tend to reach between 34 to 57 inches tall as adults, and, while their feet, hands and head tend to be regular sized, they have shortened limbs and spine and have degenerative joint disease.

There’s no word yet on when the SEDc candidate will be ready for clinical testing, but InnoSkel reckons the platform underlying the program will be transferable easily to differently type 2 collagenopathies and other skeletal diseases.

“We have been exploring several diseases and are in the process of selecting the best candidates for a modified gene therapy,” said Gouze.

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