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RCKT 
Rocket Pharmaceuticals Inc (RCKT)
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Upturn Advisory Summary
02/24/2026: RCKT (1-star) is currently NOT-A-BUY. Pass it for now.
Upturn Star Rating
Not Recommended Performance
These Stocks/ETFs, based on Upturn Advisory, consistently fall short of market performance, signaling caution before investing.
Number of Analysts
18 Analysts rated it
Moderately tracked stock, growing coverage, gaining market and investor attention.
1 Year Target Price $7.65
1 Year Target Price $7.65
| 12 | Strong Buy |
| 5 | Buy |
| 1 | Hold |
| 0 | Sell |
| 0 | Strong Sell |
Key Highlights
Company Size Small-Cap Stock | Market Capitalization 459.94M USD | Price to earnings Ratio - | 1Y Target Price 7.65 |
Price to earnings Ratio - | 1Y Target Price 7.65 | ||
Volume (30-day avg) 18 | Beta 0.47 | 52 Weeks Range 2.19 - 9.80 | Updated Date 02/24/2026 |
52 Weeks Range 2.19 - 9.80 | Updated Date 02/24/2026 | ||
Dividends yield (FY) - | Basic EPS (TTM) -2.25 |
Earnings Date
Report Date 2026-03-02 | When - | Estimate -0.4283 | Actual - |
Profitability
Profit Margin - | Operating Margin (TTM) - |
Management Effectiveness
Return on Assets (TTM) -40.56% | Return on Equity (TTM) -74.88% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value 263313691 | Price to Sales(TTM) - |
Enterprise Value 263313691 | Price to Sales(TTM) - | ||
Enterprise Value to Revenue - | Enterprise Value to EBITDA -7.77 | Shares Outstanding 108222228 | Shares Floating 86618907 |
Shares Outstanding 108222228 | Shares Floating 86618907 | ||
Percent Insiders 4.48 | Percent Institutions 85.23 |
Upturn AI SWOT
Rocket Pharmaceuticals Inc
Company Overview
History and Background
Rocket Pharmaceuticals Inc. was founded in 2015. The company is focused on developing gene therapies for rare childhood diseases. Significant milestones include its initial public offering (IPO) and ongoing clinical trials for its lead product candidates.
Core Business Areas
- Gene Therapy for Fanconi Anemia (FA): Rocket Pharma is developing a lentiviral gene therapy (RP-AAV207) for Fanconi Anemia (FA), a rare inherited blood disorder. This program is based on research from the laboratory of Dr. Jean Sullivan at the University of Minnesota.
- Gene Therapy for Pyruvate Kinase Deficiency (PKD): The company is advancing a lentiviral gene therapy (RP-AAV103) for Pyruvate Kinase Deficiency (PKD), another rare genetic disorder affecting red blood cells. This program originates from research conducted at the Children's Hospital of Philadelphia.
- Gene Therapy for X-linked Myotubular Myopathy (XLMTM): Rocket Pharma is also developing a gene therapy (RP-AAVXLM) for X-linked Myotubular Myopathy (XLMTM), a severe neuromuscular disorder. This therapy is based on work done at the University of Geneva and Genethon.
- Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa): The company has a gene therapy program (RP-GSDIA) aimed at treating Glycogen Storage Disease Type Ia (GSDIa), a metabolic disorder that affects the liver and kidneys. This program is based on research from the Nationwide Children's Hospital.
Leadership and Structure
Rocket Pharmaceuticals is led by a management team with experience in biotechnology and drug development. The company operates with a lean structure focused on advancing its gene therapy pipeline through clinical development and regulatory approval.
Top Products and Market Share
Key Offerings
- RP-AAV207 (Fanconi Anemia): A lentiviral gene therapy for Fanconi Anemia. Current market share is effectively 0% as it is in clinical development. Key competitors in the FA space include other gene therapy developers and supportive care providers. Specific market share data is not yet available due to the investigational nature of the product.
- RP-AAV103 (Pyruvate Kinase Deficiency): A lentiviral gene therapy for Pyruvate Kinase Deficiency. Current market share is effectively 0% as it is in clinical development. Competitors include existing treatments and other gene therapy programs in development. Specific market share data is not yet available.
- RP-AAVXLM (X-linked Myotubular Myopathy): A gene therapy for XLMTM. Current market share is effectively 0% as it is in clinical development. Competitors include supportive care and emerging gene therapies from other companies. Specific market share data is not yet available.
- RP-GSDIA (Glycogen Storage Disease Type Ia): A gene therapy for GSDIa. Current market share is effectively 0% as it is in clinical development. Competitors include dietary management and other therapeutic approaches. Specific market share data is not yet available.
Market Dynamics
Industry Overview
The gene therapy market is a rapidly evolving and high-growth sector within the biotechnology industry, driven by advances in genetic engineering and increasing understanding of rare genetic diseases. It is characterized by high research and development costs, lengthy clinical trial processes, and significant regulatory hurdles.
Positioning
Rocket Pharmaceuticals is positioned as a developer of gene therapies for rare childhood diseases, focusing on unmet medical needs. Its competitive advantage lies in its diversified pipeline of gene therapy candidates targeting distinct rare genetic disorders, leveraging well-established scientific platforms.
Total Addressable Market (TAM)
The TAM for rare disease gene therapies is substantial and growing, with each specific indication representing a niche but significant market. For example, the market for FA, PKD, XLMTM, and GSDIa collectively represents a multi-billion dollar opportunity. Rocket Pharmaceuticals is positioned to capture a share of these markets upon successful development and commercialization of its therapies.
Upturn SWOT Analysis
Strengths
- Diversified pipeline of gene therapy candidates for rare diseases.
- Experienced management team with a focus on gene therapy development.
- Leveraging established scientific platforms and academic collaborations.
- Potential for first-in-class or best-in-class therapies.
Weaknesses
- Clinical and regulatory risks inherent in gene therapy development.
- Long development timelines and significant capital requirements.
- Reliance on the success of multiple product candidates.
- Limited commercialization experience as a relatively young company.
Opportunities
- Significant unmet medical needs in the rare disease space.
- Advancements in gene editing and delivery technologies.
- Potential for accelerated regulatory pathways for rare disease treatments.
- Partnership and collaboration opportunities with larger pharmaceutical companies.
Threats
- Competition from other gene therapy developers and novel therapeutic approaches.
- Unforeseen safety or efficacy issues during clinical trials.
- Changes in regulatory landscape or reimbursement policies.
- Failure to secure adequate funding for ongoing development.
Competitors and Market Share
Key Competitors
- AVROBIO, Inc. (AVRO)
- uniQure N.V. (QURE)
- BioMarin Pharmaceutical Inc. (BMRN)
- CSL Limited (CSL:AU)
Competitive Landscape
Rocket Pharmaceuticals Inc. faces intense competition from established players and emerging biotechs in the gene therapy space. Its advantages lie in its focused strategy on rare childhood diseases and its diversified pipeline. However, it must overcome challenges related to clinical trial success, regulatory approval, and manufacturing scale-up to compete effectively against companies with larger market share and established commercial infrastructure.
Growth Trajectory and Initiatives
Historical Growth: Rocket Pharmaceuticals' historical growth has been characterized by pipeline expansion through acquisitions and licensing, progression of its gene therapy candidates through preclinical and early-stage clinical trials, and securing significant funding rounds.
Future Projections: Future growth projections are highly dependent on the successful clinical development and regulatory approval of its lead product candidates. Analyst estimates would focus on potential peak sales for each indication, adjusted for probability of success. Successful clinical trial results and regulatory approvals are key drivers of future revenue and profitability.
Recent Initiatives: Recent initiatives likely include advancing its lead gene therapy programs into later-stage clinical trials, expanding its manufacturing capabilities, and potentially engaging in strategic partnerships or collaborations to support further development and commercialization.
Summary
Rocket Pharmaceuticals Inc. is a clinical-stage gene therapy company focused on rare childhood diseases. Its strengths lie in a diversified pipeline and experienced management, while weaknesses include high development costs and clinical risks. Opportunities abound in the unmet needs of rare diseases, but threats from competition and regulatory hurdles are significant. The company's success hinges on the successful progression of its gene therapy candidates through clinical trials and to market approval.
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Sources and Disclaimers
Data Sources:
- Company investor relations websites and SEC filings.
- Biotechnology industry analysis reports.
- Financial news and market data providers.
Disclaimers:
This analysis is based on publicly available information and is intended for informational purposes only. It does not constitute investment advice. Investors should conduct their own due diligence before making any investment decisions. Market share data for clinical-stage companies is estimated and subject to change.
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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