Beyond Achondroplasia

Growing together with Clara

Therachon protein for achondroplasia: TA-46

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A press release from Business Wire stated that Therachon AG, a biotechnology company focused on rare genetic diseases, announced today it has appointed Luca Santarelli, M.D., as Chief Executive Officer and Director, and raised more $5 million for new medicines R&D (research a and development). The new financing will be used to advance the company’s emerging portfolio, including its lead program in achondroplasia, the most common form of short-limbed dwarfism.

I am excited to join Therachon and look forward to continuing to build a world-class pipeline and partnering with the achondroplasia community to develop a truly transformative therapeutic option,” said Dr. Santarelli.

Our lead pipeline candidate TA-46 has demonstrated exceptional preclinical efficacy and holds the promise of fully restoring normal growth and mitigating some of the devastating complications in children suffering from achondroplasia.”

TA-46 is the lab name of the Soluble FGFR3, a protein therapy in development for achondroplasia.

What is preclinical efficacy?

The development of new drugs and the application of drugs originally approved for a different disease almost always involves the need for the drug in question to be tested in animals that have been developed to model the human disease as closely as possible.

Usually it is only if the drug appears to have an effect on the animal model that it is considered worth taking it forward into clinical trial in humans. Such studies, the preclinical phase are key to assessing the efficacy of the drug and predicting success in humans with the disease.

Because the preclinical phase of research is so critical to the decisions made about a possible future therapy, it is very important that experiments done at this stage are based on best practice. This means essentially two things:

  1. Choose the most appropriate animal model.
  2. The experiments must be comparable and reproducible in different labs.

This minimizes the risk of discarding a useful therapy, or conversely, taking a compound into trial in humans that is then proven to have no effect.

Where several animal models exist for one disease, selecting the most appropriate one maximizes the value of the experiments performed. Then, to ensure comparability and reproducibility of results from different labs, as many variables as possible in terms of animal handling and testing need to be controlled for. The variability in phenotype of laboratory animals, which is influenced by housing conditions, litter size, food composition and many other factors, needs to be minimized by the acceptance of common rules in animal handling. Similarly, the use of the same core set of endpoints in all preclinical efficacy studies facilitates the comparison of results.

The soluble FGFR3  was tested in Fgfr3(ach/+) mouse model. There is another model, Fgfr3Y367C/+, in which the mice have a much more severe mutation, that mimics Thanatoforic dysplasia, a lethal form of  bone dysplasia. Even so,  Fgfr3Y367C/+ mice model is considered to present a phenotype between ACH and Thantophoric dysplasia.

 

About Therachon

Therachon is a global biotechnology company focused on developing treatments for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. This rare genetic condition affects about one in 25,000 children and is caused by a genetic mutation of the FGFR3 receptor, which stunts child bone growth.

Therachon´ head researcher Elvire Gouze is committed to translate TA-46 into a new treatment option for patients with achondroplasia.

5 Comments

  1. Thank you Ines for the great work and for keeping us posted

  2. Please let us know if the medicine is available

  3. Please let us know if this drug will available in india

  4. If you done any trials on achondroplasia boy.then i have interested for such trials on my baby.

  5. Vielen Dank auch aus Deutschland, thank you Inês! You give us so much hope. Hugs for Clara and your family 🙂

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