Sarepta Therapeutics
Biotechnology company focused on precision genetic medicine for rare diseases. Works across gene therapy and RNA-based modalities, conducts and sponsors clinical trials, pursues manufacturing scale-up for gene therapies, and operates patient-facing support and education programs. Publishes clinical study information and plain-language summaries to support data transparency. Public communications and leadership statements emphasize a patient-centered approach and collaboration with patient communities and advocacy groups.
Industries
Nr. of Employees
Very Large (1000+)
Sarepta Therapeutics
Patents
Processes for preparing phosphorodiamidate morpholino oligomers via fast-flow synthesis
US-12590306-B2
View Details
Processes for preparing phosphorodiamidate morpholino oligomers via fast-flow synthesis
US-12590306-B2
View DetailsProducts
Approved genetic and RNA-based therapeutics for rare diseases
Marketed gene therapy and RNA-based treatments for rare genetic diseases; described as available for specified indications.
SRP-9006
Investigational gene therapy using an AAVrh74 vector and tMCK promoter to target LGMD2L (LGMDR12) via anoctamin-5 gene transfer.
SRP-4053
Golodirsen evaluated in clinical trials for participants with Duchenne muscular dystrophy.
SRP-4045
Casimersen evaluated in clinical trials for participants with Duchenne muscular dystrophy.
SRP-6004
Investigational gene therapy using an AAVrh74 vector and MHCK7 promoter to target LGMD2B (LGMDR2) via dysferlin gene transfer.
SRP-9005
AAV gene therapy candidate for the treatment of Limb-girdle muscular dystrophy type 2C (gamma-sarcoglycan deficiency).
Approved genetic and RNA-based therapeutics for rare diseases
Marketed gene therapy and RNA-based treatments for rare genetic diseases; described as available for specified indications.
SRP-9006
Investigational gene therapy using an AAVrh74 vector and tMCK promoter to target LGMD2L (LGMDR12) via anoctamin-5 gene transfer.
SRP-4053
Golodirsen evaluated in clinical trials for participants with Duchenne muscular dystrophy.
SRP-4045
Casimersen evaluated in clinical trials for participants with Duchenne muscular dystrophy.
SRP-6004
Investigational gene therapy using an AAVrh74 vector and MHCK7 promoter to target LGMD2B (LGMDR2) via dysferlin gene transfer.
SRP-9005
AAV gene therapy candidate for the treatment of Limb-girdle muscular dystrophy type 2C (gamma-sarcoglycan deficiency).
Services
Design and conduct of interventional and observational studies across discovery through post-approval, including participant recruitment, informed consent, site management, and data reporting.
Design and conduct of interventional and observational studies across discovery through post-approval, including participant recruitment, informed consent, site management, and data reporting.
Expertise Areas
- Gene therapy development
- RNA therapeutics (siRNA and antisense exon-skipping)
- Clinical trial management for rare diseases
- Gene therapy manufacturing and scale-up
Key Technologies
- AAV vector-based gene transfer
- siRNA therapeutics
- Antisense exon-skipping oligonucleotides
- Muscle MRI and quantitative imaging (fat fraction)
News & Updates
Series of educational resources and videos explaining gene therapy mechanisms, safety evaluation, eligibility considerations and post-treatment monitoring.
Explainer on use of muscle MRI and fat fraction metrics to track disease progression and evaluate treatment effects.
Overview of a timed functional assessment used to monitor disease progression and guide treatment timing decisions.
Leadership interview highlighting a patient-centered approach to rare disease development, the importance of collaboration with patient communities, and emphasis on innovation alongside safety.
Press release listing recipients of a scholarship program (example of corporate announcements).
Series of educational resources and videos explaining gene therapy mechanisms, safety evaluation, eligibility considerations and post-treatment monitoring.
Explainer on use of muscle MRI and fat fraction metrics to track disease progression and evaluate treatment effects.
Overview of a timed functional assessment used to monitor disease progression and guide treatment timing decisions.
Leadership interview highlighting a patient-centered approach to rare disease development, the importance of collaboration with patient communities, and emphasis on innovation alongside safety.
Press release listing recipients of a scholarship program (example of corporate announcements).