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DirectoryBusiness & Technical ServicesResearch & DevelopmentZugSarepta International Holdings GmbH
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Sarepta International Holdings GmbH

Summary

About the company

The core activity of Sarepta Therapeutics is the development and manufacturing of gene therapies for the treatment of rare diseases, particularly muscular dystrophies such as Duchenne muscular dystrophy, limb-girdle muscular dystrophy (LGMD), and Charcot-Marie-Tooth disease. The company focuses on researching and developing precision gene medicine and aims to increase efficiency in drug development and shorten the time from laboratory to patient.

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Precision genetic medicine for rare diseases
Precision genetic medicine for rare diseases, developed through Gene Therapy, RNA technologies and Gene Editing; focus on neuromuscular and other rare genetic disorders.
Gene therapy development
Gene therapy development to supplement missing or faulty genes; includes the Gene Therapy Engine and the development of therapies for rare genetic disorders.
RNA technologies
RNA technologies using an Exon-Skipping approach for the treatment of genetic disorders; part of the Precision-Genetic-Medicine platform and focused on rare diseases.
Gene editing
Gene editing to remove or cut out mutated exons, including with CRISPR/Cas9; part of the development work for rare genetic diseases.
Clinical studies
Clinical studies for investigational therapies in various stages of development; carefully designed and conducted to develop treatments for rare diseases.
Pipeline development
Development pipeline with more than 40 investigational therapies across different stages; focus on genetic disorders, including Duchenne, LGMD and Charcot-Marie-Tooth.
Grants & Giving
Grants, donations, medical education support and sponsorships for advocacy groups, non-profits, academic institutions and medical professional organisations in the field of rare diseases.
Research collaborations
Strategic partnerships and investigator-initiated studies to advance research in rare genetic disorders and to support development programmes.
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Location

Baarerstrasse 14
6300 Zug

At a glance

Legal form
Limited Liability Company
UID
CHE-319.605.632
Founded
2017 · 9 years
Last commercial register change
October 17, 2023
Employees
1 to 10

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