Company profile: Rocket Pharmaceuticals
1.1 - Company Overview
Company description
- Provider of gene therapies for rare childhood disorders, developing lentiviral and AAV vector-based programs including RP-L102 for Fanconi Anemia, RP-L301 for Pyruvate Kinase Deficiency, an AAV program for Danon Disease, and gene therapies for LAD-I, PKP2 arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy.
Products and services
- AAV-based program for Danon Disease: Clinical-stage therapy using an AAV vector to deliver a corrected gene directly into patients, targeting Danon Disease
- RP-L102: Lentiviral-based gene therapy that utilizes a lentiviral vector to deliver a functional gene to patient's cells for the treatment of Fanconi Anemia
- RP-L301: Autologous gene therapy for Pyruvate Kinase Deficiency using hematopoietic stem cells modified with a lentiviral vector containing the PKLR gene
Key contacts
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Financial details
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1.2 - Competitors and similar companies to Rocket Pharmaceuticals
Laronde
HQ: United States
Website
- Description: Provider of an eRNA-based therapeutic platform, offering Endless RNA to program protein expression for therapeutic use; proprietary Natural NanoParticle technology to deliver biomolecules into human cells, enabling diverse administration routes and re-dosing; and AI-driven drug development using proprietary algorithms for rapid RNA medicine design.
- 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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4D Molecular Therapeutics
HQ: United States
Website
- Description: Provider of transformative gene therapeutic products enabled by a Therapeutic Vector Evolution Platform using directed evolution to create customized viral vectors for targeted delivery. Offers R100 for retinal diseases, A101 for aerosol lung delivery, and C102 for intravenous heart muscle delivery, plus a product design & development engine and in-house GMP manufacturing and IND-enabling technical operations.
- 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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Beam Therapeutics
HQ: United States
Website
- Description: Provider of precision genetic medicines using base editing, including BEAM-101 for sickle cell disease; BEAM-201, an anti-CD7 CAR-T for relapsed/refractory T-cell acute lymphoblastic leukemia and lymphoblastic lymphoma; and liver-targeting LNPs BEAM-301 and BEAM-302 to correct the R83C mutation in Glycogen Storage Disease Type 1a and the PiZ allele in severe Alpha-1 antitrypsin deficiency, plus base editing delivery technologies.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
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Generation Bio
HQ: United States
Website
- Description: Provider of genetic medicines including iqDNA, a DNA construct designed to carry large or multiple genes to cells, potentially treating a wide range of diseases; ctLNP, a cell-targeted lipid nanoparticle delivery system for specific tissue targeting enabling personalized, redosable treatments; and RES, a rapid enzymatic synthesis process that efficiently produces highly pure DNA constructs, aiming for global access to genetic medicines.
- 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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AavantiBio
HQ: United States
Website
- Description: Provider of gene transfer and gene editing therapies to treat genetic diseases; a biopharmaceutical company.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
- Acquisitions: ●●
View full AavantiBio company profile →
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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 Rocket Pharmaceuticals
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3. Investors and private equity firms
3.1 - Buyout funds investing in similar companies to Rocket Pharmaceuticals
2.2 - Growth funds investing in similar companies to Rocket Pharmaceuticals
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4 - Top valuation comps for Rocket Pharmaceuticals
4.2 - Public trading comparable groups for Rocket Pharmaceuticals
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