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Preventing the onset of
genetic neurological diseases

Pioneering a new era of innovative treatments for neurological conditions targeting axonal transport defect


Located in Grenoble, France, HuntX Pharma

is a biotech company created in December 2022 to develop game-changing therapies for neurological disorders with unmet medical needs.

At the genesis of the project sits a paradigm shift in the field of neuroscience: impaired axonal transport is as a key factor in the development of many neurological diseases.

As a result of this groundbreaking discovery initiated by Pr. Frédéric Saudou (co-founder and CSO of HuntXPharma), this pathological mechanism is identified in over 50 neurological diseases. This discovery paves the way for the development of new innovative drugs targeting axonal transport, and these survival molecules can protect the brain from neuronal death.

HuntX Pharma intends to be a pioneer and l
eader in the treatment of diseases related to axonal transport defects (ATD), starting with Huntington's disease.


Every day at HuntX Pharma, we work towards developing disease modifiers which will provide relief and improve life for patients with neurological disorders, who are otherwise faced with the progressive and inevitable degeneration of their neurons, leading to premature death.

Our ambition is grounded in high-quality, patient driven scientific research, supported by world renowned experts in the field.


"Developing small molecule,

Changing people lives"

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Laure Jamot, PhD,

President of HuntX Pharma


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Dr. Laure Jamot is a neuroscientist with 20 years of experience in Preclinical and Clinical development with 7 years on HD. She is the inventor of several granted patent families. She previously co-founded and was CSO / COO of THAC (The Healthy Aging Company). She has launched 16 clinical trials on 50 rare diseases with 300 “Centres de Maladies Rares” and carried out 2 interventional clinical trials with first in man, on a rare hepatic disease (Fulminant hepatitis, phase 1 & 2).

Laure Jamot is a scientific graduate of Université René Descartes and a business graduate of Haute Ecole de Commerce (HEC Challenge+). She worked in an academic environment at Mount Sinai Hospital (Toronto, Canada) and then at Hôpital de la Pitié-Salpétrière (Paris, France) prior to dedicating her career to research and development of therapies for patients affected by rare diseases.

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Prof. Frédéric Saudou, PhD,
Chief Scientific Officer of HuntX Pharma


Frédéric Saudou is Professor at the Grenoble Institut of Neuroscience (GIN) since 2013. After studying molecular pharmacology and human genetics at the University of Strasbourg with R. Hen, and with J.L. Mandel, Frédéric Saudou did a second postdoc at Harvard Medical School with M.E. Greenberg.

In 2000, he moved back to France as group leader at Institut Curie then was director of the GIN from 2013 to 2023.

His work focuses on the mechanisms by which Huntingtin - the protein that when mutated causes Huntington’s disease - leads to neuronal dysfunction. His main contribution is the unraveling of huntingtin function in the control of axonal transport of trophic factors.  In 2014, he received the Richard Lounsbery prize for medicine and biology from the French and US National Academies of Science. He is author of more than 100 publications in high impact journals.


What is
Disease ?

HD is a rare and inherited neurodegenerative disease caused by a triplet expansion mutation in one gene : Huntingtin (HTT). The consequence of the mutation is the breakdown of neurons in specific region of the brain (cortex and striatum) affecting movement, intellect and emotion. The triad of Psychiatric, Cognitive and Motor symptoms appears mainly around the third and fourth decades of life. The aggravation of the symptoms with age leads to a loss of autonomy for patients who progressively need assistance for every single daily task.
Some 300,000 people worldwide are concerned by Huntington Disease (global data, HD 2021; Delveinsight, HD 2022)

HD - a crucial need

for therapies

If a better caring of the patient has undoubtedly improved their lives, we are still waiting for a cure for HD. There are treatments that reduce anxiety, depression and motor symptoms, but they do not slow the progression of the disease. Because HD is caused by a well-characterized mutation in a single gene (HTT), the efforts to develop therapies have been focused on correcting the mutation or eliminating the mutant protein.

However, these strategies have not yet translated into therapies for patients. Thus, there are great expectations for new therapeutic options.

Innovating to save lives

After demonstrating that axonal transport defect (ATD) is directly responsible for HD, we identified a new class of molecules (''first in class'') that can rescue axonal transport.

Science and Innovation
Our how-know

We demonstrated during preclinical in vivo studies that HX127, our first compound, protects the brain from disease-associated degeneration and reverses the triad of symptoms.

Science and innovation

After 25 years of experience studying Huntingtin biology, we were the first to demonstrate that defects in the transport of “brain-derived neurotrophic factor (BDNF)”, a trophic factor essential for brain function, is responsible for the dysfunction and degeneration of striatal and cortical neurons, the most impacted neurons in HD patients (Gauthier et al., 2004).

Based on this sound knowledge of the huntingtin's function and dysfunction, we identified a new class of molecules that rescues ATD of BDNF and circuit function in HD affected long before neurodegeneration occurs (Vitet et al., 2020).

Our technology

We developed state of the art microfluidic devices also known as brain-on-a-chip which reconstitutes specific brain circuits to study neuronal transport between two structures of interest. This allows us to identify therapeutic molecules using high temporal and spatial resolution imaging (Virlogeux et al., 2018). As a proof of concept, we used this tool to select our first candidate drug, HX127, which has been evaluated and validated in vivo on relevant HD models (Virlogeux et al., 2021). This device, which recreates the neuronal circuitry, offers a wide range of therapeutic applications for many neurological diseases related to ATD.

Key publications
from the team

Bruyère, J., Abada, Y.-S., Vitet, H., Fontaine, G., Deloulme, J.-C., Cès, A., Denarier, E., Pernet-Gallay, K., Andrieux, A., Humbert, S., Potier, M.-C., Delatour, B., Saudou, F., 2020. Presynaptic APP levels and synaptic homeostasis are regulated by Akt phosphorylation of huntingtin. eLife 9, e56371.

Ehinger, Y., Bruyère, J., Panayotis, N., Abada, Y.-S., Borloz, E., Matagne, V., Scaramuzzino, C., Vitet, H., Delatour, B., Saidi, L., Villard, L., Saudou, F., Roux, J.-C., 2020. Huntingtin phosphorylation governs BDNF homeostasis and improves the phenotype of Mecp2 knockout mice. EMBO Mol Med 12, e10889.

Gauthier, L.R., Charrin, B.C., Borrell-Pagès, M., Dompierre, J.P., Rangone, H., Cordelières, F.P., De Mey, J., MacDonald, M.E., Lessmann, V., Humbert, S., Saudou, F., 2004. Huntingtin controls neurotrophic support and survival of neurons by enhancing BDNF vesicular transport along microtubules. Cell 118, 127–138.

Saudou, F., Humbert, S., 2016. The Biology of Huntingtin. Neuron 89, 910–926.

Virlogeux, A., Moutaux, E., Christaller, W., Genoux, A., Bruyère, J., Fino, E., Charlot, B., Cazorla, M., Saudou, F., 2018. Reconstituting Corticostriatal Network on-a-Chip Reveals the Contribution of the Presynaptic Compartment to Huntington’s Disease. Cell Reports 22, 110–122.

Virlogeux, A., Scaramuzzino, C., Lenoir, S., Carpentier, R., Louessard, M., Genoux, A., Lino, P., Hinckelmann, M.-V., Perrier, A.L., Humbert, S., Saudou, F., 2021. Increasing brain palmitoylation rescues behavior and neuropathology in Huntington disease mice. Sci. Adv. 7, eabb0799.

Vitet, H., Brandt, V., Saudou, F., 2020. Traffic signaling: new functions of huntingtin and axonal transport in neurological disease. Curr Opin Neurobiol 63, 122–130.



They support us !
Planting a Tree
HuntX Pharma launches its first fundraising:€0,75M for 2024 to achieve value-creation milestones


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