Sarepta Therapeutics, Inc.
Sarepta is a global biotechnology company dedicated to engineering precision genetic medicine for rare diseases. Their mission is to transform genetic understanding into genetic medicine, aiming to shorten drug development times, build extensive gene therapy manufacturing capacity, and rethink access and reimbursement models. They focus on diseases like Duchenne muscular dystrophy, limb-girdle muscular dystrophies, and Charcot-Marie-Tooth disease, utilizing platforms such as gene therapy, RNA technologies, and gene editing. The company emphasizes supporting the rare disease communities through patient support programs, educational resources, and grants, with a commitment to innovation, patient-centricity, and diversity.
Industries
Nr. of Employees
Very Large (1000+)
Sarepta Therapeutics, Inc.
Cambridge, Massachusetts, United States, North America
Products
Approved therapies for rare genetic diseases
Commercially approved therapies (four FDA-approved treatments stated) for rare neuromuscular genetic diseases.
Investigational therapy pipeline (multi-modality)
A development portfolio of more than 40 investigational programs spanning gene therapy, RNA-targeted therapies, and gene editing across disease areas including Duchenne muscular dystrophy, limb-girdle muscular dystrophies, and Charcot–Marie–Tooth disease.
Approved therapies for rare genetic diseases
Commercially approved therapies (four FDA-approved treatments stated) for rare neuromuscular genetic diseases.
Investigational therapy pipeline (multi-modality)
A development portfolio of more than 40 investigational programs spanning gene therapy, RNA-targeted therapies, and gene editing across disease areas including Duchenne muscular dystrophy, limb-girdle muscular dystrophies, and Charcot–Marie–Tooth disease.
Services
Personalized patient support and access program (case management)
One-on-one case management to assist patients and caregivers with benefits investigation, enrollment coordination, treatment logistics, financial-assistance navigation, and ongoing education.
Compassionate use and managed access support
Administration and coordination of compassionate use/managed access pathways to facilitate access to investigational therapies for eligible patients outside of clinical trials.
Clinical trial operations and patient-safety coordination
Operational support for clinical-trial execution including site coordination, safety monitoring, and application of regulatory guidance to maintain trial integrity during disruptions.
Personalized patient support and access program (case management)
One-on-one case management to assist patients and caregivers with benefits investigation, enrollment coordination, treatment logistics, financial-assistance navigation, and ongoing education.
Compassionate use and managed access support
Administration and coordination of compassionate use/managed access pathways to facilitate access to investigational therapies for eligible patients outside of clinical trials.
Clinical trial operations and patient-safety coordination
Operational support for clinical-trial execution including site coordination, safety monitoring, and application of regulatory guidance to maintain trial integrity during disruptions.
Expertise Areas
- Gene therapy development
- RNA therapeutics and exon skipping
- Gene editing applications for rare diseases
- Clinical trial management for rare genetic diseases
Key Technologies
- Gene therapy (viral vector-based delivery)
- Viral vector platforms
- RNA exon-skipping technologies
- CRISPR/Cas9 gene editing