OrbiMed has joined with New Enterprise Associates and Versant Ventures to funnel $35 million (€31 million) into a French startup that is developing a treatment for a common form of dwarfism. The sizable Series A round will support the advance of the program to clinical proof of concept.
|Therachon founder Elvire Gouze|
Therachon, the Nice, France-based company that raised the cash, has made a positive impression on some notable financiers of rare-disease drugs in its brief life to date. The quick evolution from seed-funded startup--Inserm Transfert Initiative and Versant Ventures helped it get started last year--to well-financed biotech has been underpinned by the strength of its science. Elvire Gouze, the founder of Therachon, has researched the achondroplasia form of dwarfism at the French state research organization Inserm for years, culminating in the publication of encouraging preclinical data in 2013.
"We have discovered a novel protein therapy that has restored normal skeletal growth and reduced co-morbidities in animal models of the disease," Gouze said in a statement. "If we can show the same effects in humans, this could dramatically improve the lives of achondroplasia patients." As with any translational-stage biotech, that is a significant "if", but OrbiMed and its fellow investors have seen enough potential to finance Therachon to the point at which it should have a better idea of whether the approach works in humans.
The treatment is underpinned by understanding of the genetic cause of achondroplasia. Research has linked the stunted bone growth and malformed cartilage that characterizes achondroplasia to a single point mutation in the gene encoding the fibroblast growth factor receptor 3 (FGFR3) protein. In a 2013 paper published in Science Translational Medicine, Gouze and her team showed that mice that received a soluble form of FGFR3 had increased bone length and fewer complications. As mice and humans have the same achondroplasia mutation, the idea could translate to the clinic.
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