Towards Healthcare

Cell and Gene Therapy (CGT) Pharmaceuticals Companies and Company Background

Date : 15 October 2025

Leaders in the Cell and Gene Therapy (CGT) Pharmaceuticals Market

Cell and Gene Therapy (CGT) Pharmaceuticals Market Companies

  • Novartis
  • Gilead Sciences / Kite Pharma
  • Bristol Myers Squibb (BMS) / Juno Therapeutics
  • Roche / Spark Therapeutics
  • bluebird bio
  • Sarepta Therapeutics
  • REGENXBIO
  • uniQure
  • Orchard Therapeutics
  • Sangamo Therapeutics
  • CRISPR Therapeutics
  • Intellia Therapeutics
  • Editas Medicine
  • Beam Therapeutics
  • Allogene Therapeutics
  • Fate Therapeutics
  • Catalent (Cell & Gene Services)
  • Lonza (Cell & Gene Solutions) 
  • Thermo Fisher Scientific (including Patheon services)
  • WuXi AppTec / WuXi Advanced Therapies

Company Background and Historical Data

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

Novartis

Overview

  • 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.

Top Role and Responsibilities

  • 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

Strengths

  • 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

Challenges Faced and Solutions

  • 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.

Recent Collaboration and Impact

  • 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.

Gilead Sciences / Kite Pharma

Overview

  • 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.

Top Role and Responsibilities

  • Development of antiviral drugs and cell therapies

  • Advanced research in oncology, particularly immuno-oncology

  • Improving patient access to therapies through collaborations

Strengths

  • Industry leader in HIV and HCV therapies

  • Leading position in cell therapy with Kite Pharma

  • Innovative approaches in CAR-T therapies for cancer

Challenges Faced and Solutions

  • 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.

Recent Collaboration and Impact

  • 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

Bristol Myers Squibb (BMS) / Juno Therapeutics

Overview

  • 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.

Top Role and Responsibilities

  • 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

Strengths

  • Strong oncology pipeline, particularly in immuno-oncology

  • Advanced expertise in CAR-T cell therapies

  • Strategic acquisitions and collaborations, including the purchase of Celgene

Challenges Faced and Solutions

  • 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.

Recent Collaboration and Impact

  • 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

Roche / Spark Therapeutics

Overview

  • 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.

Top Role and Responsibilities

  • 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

Strengths

  • 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

Challenges Faced and Solutions

  • 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.

Recent Collaboration and Impact

  • 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

bluebird bio

Overview

  • Headquarter: Cambridge, Massachusetts, USA

  • Established: 1992

  • Bluebird bio is a biotechnology company specializing in developing gene therapies for rare genetic diseases and cancer.

Top Role and Responsibilities

  • 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

Strengths

  • Leader in gene therapy innovation

  • Strong pipeline targeting hematological disorders

  • Deep commitment to rare disease research and patient outcomes

Challenges Faced and Solutions

  • 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.

Recent Collaboration and Impact

  • 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

REGENXBIO

Overview

  • Headquarter: Rockville, Maryland, USA

  • Established: 2009

  • REGENXBIO is a biotechnology company focused on developing gene therapies for neurological, ophthalmic, and genetic diseases.

Top Role and Responsibilities

  • 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

Strengths

  • Cutting-edge NAV Technology platform for gene therapy

  • Strong intellectual property portfolio

  • Collaboration with leading pharmaceutical companies such as AbbVie

Challenges Faced and Solutions

  • 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.

Recent Collaboration and Impact

  • 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

uniQure

Overview

  • 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.

Top Role and Responsibilities

  • 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

Strengths

  • Industry pioneer in gene therapy for hemophilia

  • Strong presence in the rare disease market

  • Strategic partnerships for expanding therapeutic reach

Challenges Faced and Solutions

  • 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.

Recent Collaboration and Impact

  • 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

Market Growth

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.

Investment & Funding

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.

Adoption of Inorganic Growth Strategies

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.

Value Chain Analysis for Cell and Gene Therapy (CGT) Pharmaceuticals Market

Research & Development (R&D)

Steps:

  • Target identification and validation
  • Preclinical studies (in vitro and in vivo)
  • Development of vectors, gene-editing tools, or cell therapy products
  • Process optimization for manufacturing

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

Clinical Trials & Regulatory Approvals

Steps:

  • Phase I–III clinical trials (safety, efficacy, dose optimization)
  • Regulatory submissions to FDA, EMA, PMDA, or other national authorities
  • Fast-track or breakthrough designations for accelerated approval
  • Post-approval monitoring

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

Manufacturing & Distribution (optional step for completeness)

Steps:

  • In-house or outsourced production (CDMOs)
  • Quality control and batch release
  • Cold-chain distribution to hospitals and infusion centers

Key Organizations/Companies:

Lonza, WuXi AppTec, Samsung Biologics, Catalent – Contract manufacturing

Patient Support & Services

Steps:

  • Patient education and counseling
  • Therapy administration support
  • Monitoring and follow-up care
  • Insurance, reimbursement, and financial assistance programs

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)

Latest Announcement by Industry Leaders

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).

Recent Developments in the Cell and Gene Therapy (CGT) Pharmaceuticals Market

  • In March 2025, Bharat Biotech International Limited (BBIL), a company that produces reasonably priced domestic vaccines, declared its entry into the viral vector and cell and gene therapy (CGT) fields. Genome Valley production, according to the company's statement. Extending beyond vaccine development, the business seeks to promote regenerative and customized treatments, providing millions with fresh hope.
  • In April 2025, the company Neurotech Pharmaceuticals, Inc. chose Cencora to support the launch of Neurotech's cell therapy in the US by offering integrated distribution and commercialization services. Neurotech Pharmaceuticals' revakinagene taroretcel-Iwey (ENCELTO) was approved by the US Food and Drug Administration (FDA) on March 6, 2025, for the treatment of Macular Telangiectasia type 2 (MacTel). When ENCELTO is approved, it will be the first and sole MacTel treatment authorized by the FDA.
  • In August 2025, the U.S. Food and Drug Administration (FDA) approved PAPZIMEOS (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). Precigen, Inc. is a biopharmaceutical company that specializes in the development of innovative precision medicines to improve the lives of patients. The first and only FDA-approved treatment for RRP in adults is PAPZIMEOS.

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