Sarepta Therapeutics

SRPTApproved

Cambridge, United StatesFounded 1980sarepta.com

Sarepta Therapeutics is a pioneering, commercial-stage biotech focused on developing and commercializing precision genetic medicines for devastating rare diseases, primarily Duchenne muscular dystrophy. The company has multiple FDA-approved exon-skipping therapies on the market and is advancing a robust pipeline that includes a commercial gene therapy and other candidates for DMD and limb-girdle muscular dystrophy (LGMD). With a hybrid manufacturing strategy and a relentless focus on following the science, Sarepta aims to reclaim futures for patients impacted by rare genetic disorders.

Market Cap

$1.8B

Founded

1980

Focus

Rare DiseaseGenetics & GenomicsRNA Therapeutics

SRPT · Stock Price

USD 16.6960.58 (-78.40%)

Historical price data

AI Company Overview

Technology Platform

Sarepta's core technologies are a proprietary RNA-based Phosphorodiamidate Morpholino Oligomer (PMO) platform for exon-skipping and a gene therapy platform utilizing Adeno-Associated Virus (AAV) vectors to deliver functional transgenes.

Pipeline Snapshot

34 drugs in pipeline, 6 in Phase 3

Drug	Indication	Stage
Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]	Duchenne Muscular Dystrophy	Approved
SRP-9003 + Glucocorticoid	Limb-girdle Muscular Dystrophy	Phase 3
eteplirsen	Duchenne Muscular Dystrophy (DMD)	Phase 3
Eteplirsen	Muscular Dystrophy, Duchenne	Phase 3
SRP-9005 + Corticosteroid	Muscular Dystrophies, Limb-Girdle	Phase 3

Funding History

Total raised: $995M

Debt$550MUndisclosedAug 15, 2020

PIPE$400MRA Capital ManagementDec 15, 2019

IPO$45MUndisclosedJun 15, 1997

FDA Approved Drugs

AMONDYS 45NDAFeb 25, 2021

VYONDYS 53NDADec 12, 2019

EXONDYS 51NDASep 19, 2016

Opportunities

Sarepta's growth opportunities include expanding the label and market share for its approved DMD gene therapy, successfully launching a first-in-class gene therapy for LGMD 2E, advancing its pipeline of PMOs for other DMD mutations, and leveraging its genetic medicine platforms to enter new rare disease indications with high unmet need.

Risk Factors

Key risks include clinical/regulatory setbacks for pipeline candidates, commercial challenges related to pricing and reimbursement of high-cost therapies, manufacturing complexities and scalability issues for gene therapies, and intense competition in the DMD therapeutic landscape.

Competitive Landscape

Sarepta faces competition in DMD from Pfizer's gene therapy and other exon-skipping therapies, and in LGMD from other biotechs developing genetic medicines. Its differentiation lies in its deep dystrophin expertise, first-mover advantage, comprehensive DMD portfolio, and hybrid manufacturing strategy designed for scale.

Publications

Patents

Pipeline

FDA Approvals