Company profile: Sarepta Therapeutics
1.1 - Company Overview
Company description
- Provider of precision genetic medicines for rare diseases, including gene therapies for Duchenne muscular dystrophy, limb-girdle muscular dystrophy, and Charcot-Marie-Tooth disease; RNA-targeted exon-skipping therapies for Duchenne muscular dystrophy using PMO and PPMO chemistries; and SareptAssist, a U.S. patient support program for initiating and maintaining therapy.
Products and services
- RNA-targeted Exon Skipping: PMO/PPMO-based therapy that targets RNA for exon-skipping in Duchenne muscular dystrophy, leveraging these chemistries to enhance treatment efficacy
- Gene Therapy for Duchenne Muscular Dystrophy: Precision-medicine gene therapy delivering functional copies of the dystrophin gene to muscle cells to treat Duchenne muscular dystrophy
- Gene Therapy for Limb-Girdle Muscular Dystrophy: Genotype-specific gene therapies targeting specific genetic defects in various subtypes of limb-girdle muscular dystrophy to restore muscle function
Key contacts
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Financial details
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1.2 - Competitors and similar companies to Sarepta Therapeutics
Atsena Therapeutics
HQ: United States
Website
- Description: Provider of clinical-stage gene therapies and AAV delivery technologies for inherited retinal diseases: ATSN-101 for GUCY2D-associated LCA1, ATSN-201 leveraging AAV.SPR for XLRS, and ATSN-301 dual AAV vectors for MYO7A-associated USH1B; plus laterally spreading capsids for broad retinal delivery, dual-vector methods for large genes, and intravitreal capsids that evade neutralizing antibodies.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
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- Total funding raised: $●●●m
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CRISPR Therapeutics
HQ: United States
Website
- Description: Provider of a proprietary CRISPR/Cas9 gene-editing platform and therapies to treat serious diseases, including CASGEVY™ (exagamglogene autotemcel), approved in some countries for sickle cell disease and transfusion-dependent beta thalassemia; CTX112, an investigational allogeneic CD19 CAR T therapy; and ex vivo and in vivo gene-editing and cell therapy approaches using viral and non-viral delivery.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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Akouos
HQ: United States
Website
- Description: Provider of AAV vector-based gene therapies to restore and preserve hearing in genetically defined patient populations, addressing causes from single gene mutations to ototoxic drug exposure and aging. Offers AK-OTOF, a gene therapy targeting sensorineural hearing loss due to otoferlin mutations, and the Resonate Program, no-cost genetic testing with optional counseling for auditory neuropathy.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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Egen
HQ: United States
Website
- Description: Provider of safe, efficient delivery systems for the manufacture of nucleic acid and anti-cancer drugs and for creating products to treat human diseases, offering synthetic biocompatible delivery vehicles to deliver therapeutic genes, inhibitory RNA (siRNA & shRNA), and small molecules.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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Replicor
HQ: Canada
Website
- Description: Provider of nucleic acid polymer (NAP) antivirals targeting hepatitis B and delta (HBV/HDV), including REP 2139. These oligonucleotides interact with viral proteins to block replication, are used in combination therapies, deliver broad-spectrum antiviral effects, and promote functional cure.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
- Acquisitions: ●●
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2.M&A buyers
2.1 Potential strategic acquirers in the sector
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2.2 - Strategic buyer groups for Sarepta Therapeutics
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3. Investors and private equity firms
3.1 - Buyout funds investing in similar companies to Sarepta Therapeutics
2.2 - Growth funds investing in similar companies to Sarepta Therapeutics
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4 - Top valuation comps for Sarepta Therapeutics
4.2 - Public trading comparable groups for Sarepta Therapeutics
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