ST
Sarepta Therapeutics(SRPT)
CAMBRIDGE, MA
BiotechnologyGene therapy company focused on precision genetic medicine for rare diseases, including Duchenne muscular dystrophy.
Funding Stage
PUBLIC
Employees
1001-5000
Open Jobs
0
Products & Portfolio (3)
AMONDYS 45
casimersen
Peak
RNAINTRAVENOUS · SOLUTION
45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping [see Clinical Studies ()] .
Duchenne muscular dystrophy (DMD) in patients
2021
8
EXONDYS 51
eteplirsen
Peak
RNAINTRAVENOUS · SOLUTION
51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein, which was evaluated in Study 2 and Study 3 [ see Clinical Studies () ].
Duchenne muscular dystrophy (DMD) in patients
2016
0
VYONDYS 53
golodirsen
LOE Approaching
RNAINTRAVENOUS · SOLUTION
2019
15
Open Jobs (0)
No open positions listed yet. Check their careers page directly.
Interview Prep Quick Facts
Portfolio: 3 approved products
Top TAs: Rare Diseases
SEC Filings: 2 available
Portfolio Health
Peak2 (67%)
LOE Approaching1 (33%)
3 total products
Financials (FY2025)
Revenue
$1.2B33%
R&D Spend
$877M(71%)0%
Net Income
-$536MCash
$428M