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Rare Focus,
Real Impact.

THX Pharma (Theranexus) is an innovative biopharmaceutical company. A CEA spinoff, we specialize in the treatment of rare neurological diseases.

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Advancing Therapeutic Innovation for Patients with Rare Diseases

We develop innovative therapies designed to address the significant needs of patients living with rare neurological conditions, especially when options are limited or inconvenient.

 

Grounded in scientific excellence and driven by innovation, we transform research into practical, patient-centered treatments –with the ambition to make a lasting difference in patients’ lives.

Therapeutic Focus

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Batten Disease

Juvenile Batten disease (CLN3) is a rare, inherited neurodegenerative disorder in children, characterized by the build-up of toxic substances in the brain and the eye, leading to progressive loss of visual, cognitive, and motor functions.

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Niemann-Pick C Disease

Niemann-Pick type C is a rare, progressive genetic disease marked by considerable clinical variability, yet severe progression, leading to significant therapeutic needs.

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Gaucher Disease

Gaucher disease is a rare genetic disorder with diverse manifestations and a variable course; a challenging pathology that calls for new therapeutic approaches, tailored to patient needs.

From Execution to Vision

Year of Creation

2013

Drug Candidates
in Development

4

Patent Families

3

Targeted Diseases

5

Our Research Department

Our Research efforts focus on unlocking the potential of antisense oligonucleotides (ASOs) to address the underlying causes of rare neurological diseases. Through a flexible and evolving platform, we design targeted therapies aimed at delivering meaningful clinical outcomes –  bridging the gap between scientific innovation and real-world patient benefit.

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Laboratoire

Shareholders

We are thankful for our strong shareholder base who support our long-term vision and commitment to advancing care for patients with rare diseases. Their trust enables us to accelerate the development of new solutions in areas with acute medical needs.

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