Beyond Achondroplasia

Growing together with Clara

Soluble FGFR3 going forward with Therachon


Soluble FGFR3 was first mentioned here in September 2013. This protein is able to restore growth by acting has a decoy receptor and prevents FGFs from binding to mutant FGFR3 and was able to increase bone length and reduce complications associated with achondroplasia in transgenic mice with achondroplasia. Here you can find an explanatory scheme of the action of this protein in the chondrocytes with achondroplasia mutation in the growth plate.

Therachon was mentioned here in October 2014, and holds the investigation on the soluble FGFR3, the therapy  that aims to restore normal bone growth and proportions in children with the achondroplasia.

I wrote an article about soluble FGFR3 and Prof. Elvire Gouze, head researcher on FGFR3 soluble for 6 years now, who founded TherAchon (meaning Therapeutics for Achondroplasia). At that point, this project had investments from Versant Ventures and Inserm Transfert Initiative (ITI) and this meant that Therachon had some funding to proceed with the studies on sFGFR3, refusing a pharmaceutical company to take over this project.

One day ago, on the 30th September 2015, Therachon did an incredibly important press release:

“Therachon is a developer of innovative therapies for achondroplasia, announced today that it closed a $35 million Series A round. The funds will be used to advance Therachon’s lead program, a soluble form of human fibroblast growth factor receptor 3, through clinical proof of concept.” 

Now, other two major investors joined this venture: OrbiMed and New Enterprise Associates.


As Elvire Gouze said: “This financing validates the work we have been doing at Inserm for the last six years. We have discovered a novel protein therapy that has restored normal skeletal growth and reduced co-morbidities in animal models of the disease. If we can show the same effects in humans, this could dramatically improve the lives of achondroplasia patients.”

As we met Elvire and learned about her innovative approach, we became very excited at the prospect of working with her to develop a life-altering therapy for children with achondroplasia,” said Tom Woiwode, Ph.D., Therachon’s chairman and managing director at Versant Ventures, which seeded Therachon along with ITI in 2014. “We’re thrilled to have brought together an exceptional international syndicate to support the company as we advance the program towards clinical trials.” This is the next big step for Soluble FGFR3.

“Stephen Squinto, Ph.D., venture partner at OrbiMed, added, “As there are no current therapies available for achondroplasia, Therachon is in a unique position to fill a significant need. Therachon is a great example of the type of high-impact investments we look to support at OrbiMed.”

It is quite curious to see how big companies don’t mention the existence of any healthy competition such as BioMarin with Vosoritide (BMN-111), soon heading to phase 3.

This will be for sure a “race” between pharmaceutical companies and venture capital companies to see who “gets there first” and who has the most efficient product in terms of reducing or even eliminating complications/morbidities and rescuing growth with less side effects and the best price.

I have full confidance that FGFR3 soluble has a huge potential to have the best results in achondroplasia.

Above all, Congratulation to Elvire Gouze! For her extraordinary efforts and excellent work and research for achondroplasia. This news is a huge step forward for new studies for achondroplasia.



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