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FROM GENES TO HOPE

Advancing Gene Therapy for Rare Diseases

Exir Gene Pharmed is developing genetic medicines for inherited and rare disorders, bringing together translational science, development, manufacturing, and quality to move promising therapies toward patients.

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NO DISEASE IS TOO RARE FOR HOPE

New Possibilities for Rare Diseases

We develop advanced genetic medicines for rare and serious conditions, translating scientific innovation into real hope for patients and families.

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WHO WE ARE

Turning genetic science into therapeutic possibilities.

Exir Gene Pharmed is a biotechnology company focused on translating advances in gene therapy and genetic medicine into potential treatments for serious inherited and rare diseases.

Science

From molecular insight to therapeutic design.

 

Development

Advancing programs toward clinical translation.

 

Manufacturing & Quality

Building the capabilities to deliver reproducible therapies.

 

PRODUCTS IN DEVELOPMENT

Advancing genetic medicines for serious inherited disorders.

Our development programs bring together genetic science, translational research, manufacturing, and quality with a focus on therapies for rare and serious inherited diseases.

IN DEVELOPMENT

SpinEX (SPX-01)

SpinEX is an investigational AAV-based gene therapy designed to deliver a functional SMN1 gene, targeting the underlying genetic cause of spinal muscular atrophy.

Development stagePreparing for initial clinical study

Explore our pipeline

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