Company | 2020 Revenue | Recent Revenue (2024) | Established Year |
Novartis | $48.66B | $54.47B | 1996 |
Gilead Sciences / Kite | $22.09B | $27.31B | 1987 (Gilead) / 2009 (Kite) |
Bristol Myers Squibb | $42.52B | $49.45B | 1856 |
Roche | $63.11B | $70.72B | 1896 |
bluebird bio | $0.48B | $1.23B | 1992 |
REGENXBIO | $0.12B | $0.46B | 2009 |
uniQure | $0.14B | $0.48B | 1998 |
Headquarter: Basel, Switzerland
Established: 1996
Novartis is a global healthcare company that provides solutions to address the evolving needs of patients worldwide. They focus on developing treatments in areas like oncology, immunology, dermatology, and neuroscience.
Research, development, and marketing of innovative pharmaceutical products
Addressing unmet medical needs with groundbreaking therapies
Developing and providing patient support programs for access to treatments
Strong portfolio in oncology and immunology
Robust pipeline of gene therapies and cell-based treatments
Strategic acquisitions such as the purchase of AveXis for gene therapies
Facing patent expirations on key drugs – Solved through diversification and pipeline expansion.
Regulatory delays – Streamlined approval processes with regulatory bodies.
High competition in generic medicines – Solved by innovating with new drug formulations.
Partnered with the Bill & Melinda Gates Foundation to develop new treatments for malaria.
Entered into an agreement with Microsoft to enhance R&D capabilities through artificial intelligence.
Collaborated with the University of Pennsylvania for cell and gene therapies in oncology.
Headquarter: Foster City, California, USA
Established: 1987 (Gilead Sciences), 2009 (Kite Pharma)
Gilead Sciences is a biopharmaceutical company focusing on the development of antiviral drugs, especially for HIV, hepatitis, and cancer. Kite Pharma specializes in cell therapy treatments for cancer.
Development of antiviral drugs and cell therapies
Advanced research in oncology, particularly immuno-oncology
Improving patient access to therapies through collaborations
Industry leader in HIV and HCV therapies
Leading position in cell therapy with Kite Pharma
Innovative approaches in CAR-T therapies for cancer
Declining sales of older antiviral treatments – Solved by investing in next-generation HIV and oncology therapies.
High costs of CAR-T therapies – Solved by implementing patient access programs and cost-reduction strategies.
Supply chain constraints for cell-based therapies – Solved through partnerships with global manufacturers.
Collaboration with Bristol Myers Squibb for expanding oncology research
Agreement with the National Cancer Institute for advancing CAR-T cell therapies
Partnership with the Gates Foundation for global HIV prevention and treatment
Headquarter: New York, USA
Established: 1856 (BMS), 2016 (Juno Therapeutics)
BMS is a leading global biopharmaceutical company, and Juno Therapeutics is known for its innovative cell and gene therapies for cancer.
Advancing research in oncology, immunology, and cardiovascular treatments
Pioneering the development of CAR-T therapies through Juno Therapeutics
Building a robust pipeline for personalized medicine in cancer
Strong oncology pipeline, particularly in immuno-oncology
Advanced expertise in CAR-T cell therapies
Strategic acquisitions and collaborations, including the purchase of Celgene
Integration challenges after acquiring Celgene – Solved by streamlining operational systems and talent integration.
Competition in immuno-oncology – Solved by enhancing personalized medicine approaches.
Complex manufacturing for CAR-T therapies – Solved by improving production facilities.
Partnership with bluebird bio for the development of gene therapies
Collaboration with the University of California for cutting-edge immuno-oncology research
Joint venture with CureVac for developing mRNA vaccines
Headquarter: Basel, Switzerland (Roche), Philadelphia, USA (Spark)
Established: 1896 (Roche), 2013 (Spark Therapeutics)
Roche is a global leader in diagnostics and pharmaceuticals, while Spark Therapeutics specializes in gene therapy for genetic diseases.
Focused on precision medicine, diagnostics, and oncology
Leading innovations in gene therapies through Spark Therapeutics
Expanding personalized treatment options in oncology and rare genetic diseases
Robust presence in oncology and diagnostics
Pioneering in gene therapies with Spark for diseases like hemophilia and retinal diseases
Strong global market reach with a diversified portfolio
Regulatory hurdles for new therapies – Overcome by engaging with regulators early in the development process.
Competition from other gene therapy companies – Solved by enhancing unique value propositions like single-dose therapies.
Pricing and reimbursement challenges – Solved by forming partnerships to ensure accessibility.
Partnership with Chugai Pharmaceuticals for advanced cancer treatments in Asia
Collaborating with the FDA to fast-track gene therapies for genetic diseases
Research alliance with the Broad Institute for innovative cancer therapies
Headquarter: Cambridge, Massachusetts, USA
Established: 1992
Bluebird bio is a biotechnology company specializing in developing gene therapies for rare genetic diseases and cancer.
Development of gene therapies for genetic disorders such as sickle cell anemia and beta-thalassemia
Pioneering in the development of lentiviral gene therapies
Collaborating with global healthcare providers to make gene therapies more accessible
Leader in gene therapy innovation
Strong pipeline targeting hematological disorders
Deep commitment to rare disease research and patient outcomes
Manufacturing challenges for gene therapies – Addressed by expanding production capabilities.
Regulatory scrutiny of new gene therapies – Handled by strengthening collaborations with regulators.
High cost of treatments – Resolved by implementing patient assistance programs.
Partnered with Celgene for oncology research
Collaborated with Regeneron Pharmaceuticals for new gene-editing techniques
Joined forces with the Bill & Melinda Gates Foundation to develop affordable therapies for low-income countries
Headquarter: Rockville, Maryland, USA
Established: 2009
REGENXBIO is a biotechnology company focused on developing gene therapies for neurological, ophthalmic, and genetic diseases.
Focused on advancing gene therapies based on its NAV Technology platform
Expanding research into neurological and retinal diseases
Engaging with major pharmaceutical partners to bring therapies to market
Cutting-edge NAV Technology platform for gene therapy
Strong intellectual property portfolio
Collaboration with leading pharmaceutical companies such as AbbVie
Challenges in gene therapy scalability – Solved by investing in manufacturing innovations and building scalable processes.
High competition in gene therapy space – Solved by creating differentiated therapies and focusing on rare disease markets.
Regulatory hurdles – Overcome by closely working with FDA for accelerated approvals.
Partnered with AbbVie for ophthalmic gene therapy development
Research alliance with the University of Pennsylvania for neurological therapies
Collaboration with Novartis to develop gene therapies for rare genetic diseases
Headquarter: Amsterdam, Netherlands
Established: 1998
uniQure is a leader in gene therapy, focusing on developing treatments for hemophilia, Huntington's disease, and other rare genetic conditions.
Leading the development of gene therapies, particularly for hemophilia
Expanding its pipeline into neurological and liver diseases
Working to bring one-time gene therapies to patients with limited treatment options
Industry pioneer in gene therapy for hemophilia
Strong presence in the rare disease market
Strategic partnerships for expanding therapeutic reach
High manufacturing costs for gene therapies – Overcome by scaling up production and collaborating with contract manufacturers.
Regulatory challenges – Handled through proactive engagement with regulators to gain approvals.
Access to gene therapy – Solved by establishing patient support programs to increase access.
Partnered with Bristol Myers Squibb for gene therapy development
Collaboration with the University of California for gene therapies in neurological diseases
Research partnership with the Global Gene Therapy Initiative for global gene therapy access
The global cell and gene therapy (CGT) pharmaceuticals market size recorded US$ 16.75 billion in 2024, set to grow to US$ 19.91 billion in 2025 and projected to hit nearly US$ 91.56 billion by 2034, with a CAGR of 18.93% throughout the forecast timeline.
METiS Technologies is an international pioneer in AI-driven nanodelivery technology declared that it has successfully finished its 400 million RMB in a Series D funding round. The declaration was made at an important center for Beijing's life sciences industry is Zhongguancun (Daxing) Cell and Gene Therapy Industrial Park. Beijing Medical and Health Industry Investment Fund is co-leading the financing. Industrial Investment Fund Axing the Daxing Industrial Investment Fund and the Beijing Medical and Health Industry Investment Fund co-led the Series D round. The new funding will be utilized to expedite METiS's primary strategic goals, which include hiring top talent, developing an internal pipeline, expanding international alliances, and automating upgrades to its proprietary platform.
In August 2025, in order to create in vivo CAR-T treatments, Gilead's Kite acquired Interius BioTherapeutics for US$350 million. A GILEAD Company, Kite delivering therapeutic DNA to patients' cells with a single infusion. This purchase attempts to streamline and reduce expenses for Interiüs treatment with CAR-T. Separately, Cabaletta Bio and Cellares have teamed up to automate the production of their respective CAR-T cell therapies, with the goal of facilitating greater accessibility to these cutting-edge treatments.
Steps:
Key Organizations/Companies:
Novartis, Gilead/Kite Pharma, CRISPR Therapeutics, Sangamon Therapeutics – Gene therapy development, Cellectis,
Academic & research institutions: Harvard Medical School, Stanford University, University of Pennsylvania
Steps:
Key Organizations/Companies:
Novartis, Bluebird Bio, Sarepta Therapeutics, etc., FDA (USA) – Regulatory oversight, EMA (Europe) – Approval and regulation, PMDA (Japan) – Regulatory review, Contract research organizations (CROs): IQVIA, Parexel, Charles River Labs
Steps:
Key Organizations/Companies:
Lonza, WuXi AppTec, Samsung Biologics, Catalent – Contract manufacturing
Steps:
Key Organizations/Companies: Novartis Patient Support Programs, Gilead’s KiteCare, Bluebird Bio Patient Services, Hospitals & specialized infusion centers (e.g., Mayo Clinic, MD Anderson Cancer Center)
In August 2025, the U.S. Food and Drug Administration announced the clinical hold on the pivotal phase 2 clinical trial conducted by Rocket Pharmaceuticals. In order to treat Danon Disease, RP-A501, an experimental adeno-associated virus serotype 9 (AAV9) vector-based gene therapy, is being evaluated (NCT06092034).
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