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Upturn AI SWOT - About
RCKT 
Rocket Pharmaceuticals Inc (RCKT)
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Upturn Advisory Summary
10/14/2025: RCKT (2-star) has a low Upturn Star Rating. Not recommended to BUY.
Upturn Star Rating
Below Average Performance
These Stocks/ETFs, based on Upturn Advisory, often underperform the market, warranting careful consideration before investing.
Number of Analysts
18 Analysts rated it
Moderately tracked stock, growing coverage, gaining market and investor attention.
1 Year Target Price $7.73
1 Year Target Price $7.73
| 12 | Strong Buy |
| 5 | Buy |
| 1 | Hold |
| 0 | Sell |
| 0 | Strong Sell |
Analysis of Past Performance
Type Stock | Historic Profit 7.01% | Avg. Invested days 27 | Today’s Advisory Consider higher Upturn Star rating |
Upturn Star Rating | Upturn Advisory Performance  3.0 | Stock Returns Performance  2.0 |
| Profits based on simulation | Last Close 10/14/2025 |
Key Highlights
Company Size Small-Cap Stock | Market Capitalization 460.75M USD | Price to earnings Ratio - | 1Y Target Price 7.73 |
Price to earnings Ratio - | 1Y Target Price 7.73 | ||
Volume (30-day avg) 18 | Beta 0.6 | 52 Weeks Range 2.19 - 18.89 | Updated Date 10/14/2025 |
52 Weeks Range 2.19 - 18.89 | Updated Date 10/14/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -3.09 |
Earnings Date
Report Date - | When - | Estimate - | Actual - |
Profitability
Profit Margin - | Operating Margin (TTM) - |
Management Effectiveness
Return on Assets (TTM) -38.07% | Return on Equity (TTM) -69.65% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value 100079425 | Price to Sales(TTM) - |
Enterprise Value 100079425 | Price to Sales(TTM) - | ||
Enterprise Value to Revenue - | Enterprise Value to EBITDA -7.77 | Shares Outstanding 107903871 | Shares Floating 78240018 |
Shares Outstanding 107903871 | Shares Floating 78240018 | ||
Percent Insiders 3.64 | Percent Institutions 88.47 |
Upturn AI SWOT
Rocket Pharmaceuticals Inc
Company Overview
History and Background
Rocket Pharmaceuticals Inc. was founded in 2015 and focuses on developing gene therapies for rare pediatric diseases. The company has progressed from preclinical research to clinical-stage development of multiple gene therapy programs.
Core Business Areas
- Hematologic Malignancies: Develops gene therapies for hematologic malignancies, including Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD).
- Cardiomyopathies: Develops gene therapies for Danon Disease, a rare X-linked dominant disease.
- RASopathies: Development of gene therapies to treat Noonan Syndrome. This is a less advanced program.
Leadership and Structure
Gaurav Shah, M.D., is the current Chief Executive Officer. The company has a typical biotech organizational structure with departments focused on research, clinical development, manufacturing, and commercialization. It has a Board of Directors providing oversight.
Top Products and Market Share
Key Offerings
- RP-L102 (Fanconi Anemia Group A): A gene therapy for Fanconi Anemia Group A (FA-A). RP-L102 is currently in Phase 2 trials. Given FA-A affects a small population, the market is niche but with significant unmet need. Competitors in gene therapy include Novartis, bluebird bio, and Sarepta.
- RP-L201 (LAD-I): A gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I). RP-L201 is in Phase 1/2 clinical trials. This is a life-threatening disease for which the only treatment is bone marrow transplant so market share is dependent on efficacy/safety profile of RP-L201. Competitors in gene therapy include Novartis, bluebird bio, and Sarepta.
- RP-A501 (Danon Disease): A gene therapy for Danon Disease. RP-A501 is in Phase 1 clinical trials. Danon Disease affects the heart and muscle tissues and RP-A501 is attempting to reverse the course of the disease. Competitors in gene therapy include Novartis, bluebird bio, and Sarepta.
Market Dynamics
Industry Overview
The gene therapy market is rapidly growing with significant investment and regulatory interest. The industry is characterized by high risk, high reward, and long development timelines. Regulatory hurdles and manufacturing complexities are significant challenges. There are several big pharmaceutical companies attempting to enter this space or purchase the smaller biotechnology companies.
Positioning
Rocket Pharmaceuticals Inc. is a clinical-stage biotech company specializing in gene therapies for rare diseases. Its competitive advantage lies in its focused approach to rare pediatric diseases and its pipeline of potentially transformative gene therapies.
Total Addressable Market (TAM)
The total addressable market for gene therapies is estimated to be in the billions of dollars, with specific rare disease markets ranging from $100 million to $1 billion each. Rocket is positioned to capture a significant portion of these rare disease markets with successful clinical trials and regulatory approvals.
Upturn SWOT Analysis
Strengths
- Strong pipeline of gene therapy candidates
- Focus on rare pediatric diseases with high unmet need
- Experienced management team
- Proprietary gene therapy platform
Weaknesses
- High cash burn rate
- Clinical trial risks
- Manufacturing complexities
- Dependence on regulatory approvals
Opportunities
- Potential for breakthrough therapies
- Expansion into new rare disease indications
- Partnerships with larger pharmaceutical companies
- Positive regulatory outcomes
Threats
- Clinical trial failures
- Competition from other gene therapy companies
- Regulatory setbacks
- Adverse safety events
Competitors and Market Share
Key Competitors
- BLUE
- SRPT
- CRSP
Competitive Landscape
Rocket Pharmaceuticals competes with other gene therapy companies and larger pharmaceutical companies exploring gene therapy. Its advantage lies in its focus and innovative platform, but it faces challenges from larger competitors with greater resources.
Growth Trajectory and Initiatives
Historical Growth: Growth has been driven by pipeline expansion and clinical trial progress. The company has consistently raised capital to fund its operations.
Future Projections: Future growth is contingent on successful clinical trial outcomes and regulatory approvals. Analyst estimates vary, but project significant revenue potential if therapies are approved. The company has a market cap of ~$3 billion USD.
Recent Initiatives: Recent initiatives include advancing clinical trials for RP-L102, RP-L201, and RP-A501, and expanding manufacturing capabilities.
Summary
Rocket Pharmaceuticals is a clinical-stage biotechnology company focused on rare disease gene therapies. Its strength lies in its focused pipeline and potential to address significant unmet needs. However, clinical trial risks and high cash burn are significant challenges. Successful clinical development and eventual commercialization will determine its long-term success.
Peer Comparison
Sources and Disclaimers
Data Sources:
- Company Website
- SEC Filings
- Analyst Reports
- ClinicalTrials.gov
Disclaimers:
This analysis is for informational purposes only and does not constitute financial advice. Investment decisions should be based on individual due diligence.
AI Summarization is directionally correct and might not be accurate.
Summarized information shown could be a few years old and not current.
Fundamental Rating based on AI could be based on old data.
AI-generated summaries may have inaccuracies (hallucinations). Please verify the information before taking action.
About Rocket Pharmaceuticals Inc
Exchange NASDAQ | Headquaters Cranbury, NJ, United States | ||
IPO Launch date 2015-02-18 | CEO & Director Dr. Gaurav D. Shah M.D. | ||
Sector Healthcare | Industry Biotechnology | Full time employees 299 | Website https://www.rocketpharma.com |
Full time employees 299 | Website https://www.rocketpharma.com | ||
Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases in the United States. It develops vivo adeno-associated viral (AAV) programs, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure (RP-A501), which is in Phase 2 trial; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), which is in phase 1; and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is a form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart, which is in preclinical stage. It also develops vivo lentiviral (LV) programs, which include programs for Leukocyte Adhesion Deficiency-I (LAD-I), a genetic disorder that causes the immune system to malfunction (RP-L201); Fanconi Anemia (FA), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells (RP-L102); and Pyruvate Kinase Deficiency (PKD), a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia (RP-L301). It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; REGENXBIO, Inc.; and Temple University. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.
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