Main Sector
Pharma (fully integrated)
Subsectors
Gene therapy
Nucleic acid drugs
Indications
Diseases of the nervous system / neurology
Respiratory / pulmonology
Musculoskeletal system and connective tissue / Orthopedics
Congenital malformations, deformations and chromosomal abnormalities / medical genetics
Symptoms, signs and abnormal clinical and laboratory findings, not elsewhere classified
Products
In development: 10
On the market: 4
Description
Sarepta Therapeutics, Inc. is a biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. The company's product SRP-1001 is an investigational siRNA treatment designed to reduce the production of DUX4 protein in skeletal muscle in patients living with FSHD1. The comypany's another product EXONDYS 51 is an antisense oligonucleotide indicated for the treatment of...
