March 23, 2025
Cell and gene therapy is transforming modern medicine, offering targeted treatments for genetic disorders, cancers, and diseases once thought incurable. The global gene therapy market is expected to grow from $11.07 billion in 2025 to $55.43 billion by 2034, advancing at a rate of 19.60% per year. This rapid expansion places the industry at the forefront of medical innovation.
Breakthroughs in CRISPR gene editing, viral vector development, and AI-powered analytics are transforming how gene therapies are developed and delivered. Companies across the world are working to enhance precision medicine, from leading CAR-T cell therapies to next-generation RNA sequencing solutions. These transformations play a critical role in accelerating the next era of cell and gene therapy research.
This article explores the top companies working on gene therapy and their contributions to cell and gene therapy advancements.
Biostate AI is at the forefront of gene therapy, identifying key challenges faced by researchers in this field. This is why Biostate AI provides low-cost, fully integrated RNA sequencing solutions aimed at helping in the discovery of more sophisticated transcriptomic knowledge.
Biostate Ai’s Total RNA sequencing, powered by the proprietary Barcode-Integrated Reverse Transcription (BIRT) platform, provides researchers whole transcriptome coverage, including crucial non-coding RNAs that are instrumental in disease regulation.
By full-length transcript analysis, researchers can determine both polyadenylated and non-polyadenylated RNAs, which are of paramount importance for understanding complex disease processes and designing tailored therapeutics.
Biostate AI uses AI and high-throughput RNA sequencing to advance genomic research, biomarker discovery, and clinical decision-making. It integrates multi-omics data to support personalized medicine, drug development, and clinical trials. The key features and innovations include the following:
Biostate AI is revolutionizing multi-omics data acquisition by providing Total RNA-Seq on any sample type, from FFPE tissue to even 10uL of blood, at a mere $80 per sample. This level of flexibility and low cost allows researchers to achieve detailed transcriptomic data at a fraction of the cost compared to typical sequencing services.
For sensitive tissue samples, liquid biopsies, or small-size rare disease cohorts, Biostate AI ensures effective, high-quality sequencing data, leading to state-of-the-art advancements.
Breakthroughs in RNA sequencing, predictive modeling, and biomarker discovery are revolutionizing medicine and gene therapy. They make precision therapeutics possible, providing customized treatments with greater efficacy and reduced side effects.
They are leading the way in cancer therapies, regenerative medicine, and drug development, transforming the way we treat disease. They reveal new medical frontiers by discovering new drug targets and validating gene-editing methods.
A notable example involves the KRAS mutation in lung adenocarcinoma. RNA sequencing has helped identify novel mutations in genes like KRAS, PIK3CA, NOTCH1, and ERBB2, which are crucial for designing targeted therapies.
Bluebird Bio is a leading biotechnology company focused on developing gene therapies for rare genetic disorders. By leveraging genetic engineering, the company aims to provide curative treatments for conditions previously lacking effective solutions.
Its FDA-approved gene therapies target diseases such as beta-thalassemia, cerebral adrenoleukodystrophy (CALD), and sickle cell disease, addressing the root cause rather than just managing symptoms.
Key therapies include the following:
Bluebird Bio is revolutionizing the treatment of rare diseases using gene therapy. These therapies provide curative options for previously untreatable conditions. Bluebird Bio is an engineering genetics company developing methods for replacing dysfunctional genes with functional copies.
Bluebird Bio has made great strides in achieving long-term healing for patients suffering from diseases such as beta-thalassemia and sickle cell disease, shifting from experimental to available, curative treatments.
Novartis is a pioneer in CAR-T cell therapy, advancing the field of personalized medicine and immunotherapy. The company has played a crucial role in developing targeted treatments for hematological malignancies, leveraging gene and cell therapy innovations to offer curative solutions.
With a strong focus on reprogramming the immune system to fight cancer, Novartis continues to push the boundaries of next-generation therapies, shaping the future of oncology and genetic medicine.
Key therapies include the following:
Novartis has revolutionized oncology with CAR-T therapy, providing targeted therapy that reeducates a patient's immune cells to attack cancer. Gene therapy is revolutionizing medicine, making curative and personalized therapies across diseases possible and enhancing outcomes. These new therapies are transforming the treatment of complicated diseases, and Novartis is leading gene-based treatments.
For instance, Novartis' CAR-T treatment has been quite successful in treating leukemia by destroying and targeting cancer cells. It has the potential for a cure while having a few side effects compared to conventional treatments.
Gilead Sciences is a global biopharmaceutical leader recognized for its groundbreaking work in antiviral treatments, oncology, and cell therapy. The company has played a pivotal role in developing life-saving therapies for infectious diseases, including HIV, hepatitis, and COVID-19, and has expanded into oncology and immunotherapy through cutting-edge research.
With the acquisition of Kite Pharma, Gilead has strengthened its position in cell therapy, driving advancements in personalized medicine and immune-based cancer treatments.
Key therapies include the following:
These therapies are revolutionizing the landscape of gene therapy by introducing cell-based immunotherapies that reprogram the immune system to attack and then kill cancer cells.
They are a significant advancement in precision medicine and cancer therapy, significantly enhancing survival rates for patients with hard-to-treat cancers such as large B-cell lymphoma and mantle cell lymphoma (MCL).
CRISPR Therapeutics, in collaboration with Vertex Pharmaceuticals, has developed Casgevy (formerly known as Exa-cel, or exagamglogene autotemcel), a groundbreaking CRISPR/Cas9-based gene therapy designed to treat sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). This therapy involves editing a patient's hematopoietic stem cells to produce fetal hemoglobin, thereby alleviating disease symptoms.
Casgevy’s approval is a historic milestone as the first CRISPR-based gene therapy to receive regulatory authorization, setting the stage for future precision gene-editing treatments.
Impact on Cell and Gene Therapy
CRISPR Therapeutics is working on gene editing to correct mutations causing sickle cell disease and beta-thalassemia, aiming for potential cures. CRISPR technology allows for accurate DNA editing, transforming precision medicine.
However, while CRISPR-based therapies focus on somatic cell editing, ethical concerns remain around the potential risks of germline editing, which could introduce heritable genetic modifications. Additionally, the possibility of unintended genetic alterations (off-target effects) is an area of ongoing scientific debate and regulatory scrutiny.
Gene therapy is advancing rapidly, and companies working on gene therapy, such as Biostate AI, Bluebird Bio, Novartis, Gilead Sciences, Oxford Biomedica and CRISPR Therapeutics, are driving this revolution. Next-generation RNA sequencing and AI analytics can speed up research, improve treatment protocols, and optimize patient outcomes.
At Biostate AI, we are dedicated to providing cost-effective, high-accuracy RNA sequencing and AI-driven analytics to enable the advancement of your gene therapy research and clinical trials effectively.
As gene therapy advances every day, the convergence of multi-omic information and predictive modeling will be at the heart of tailoring treatments and enhancing patient success rates.
If you need to remain at the forefront of companies working on gene therapy, Biostate AI can assist you. Discover our RNA sequencing solutions and be among the leaders of the future of personalized medicine and revolutionary gene therapies.
The information provided in this article is for informational purposes only and should not be considered medical advice. Any applications in clinical settings should be explored in collaboration with appropriate healthcare professionals.
Frequently Asked Questions
1. What is the cell and gene therapy model?
The cell and gene therapy model is an advanced medical approach that involves modifying or replacing genetic material or cells to treat or cure diseases. Gene therapy works by correcting or replacing faulty genes, while cell therapy involves using live cells to repair or regenerate damaged tissues. These therapies are particularly effective in treating genetic disorders, certain cancers, and degenerative diseases.
2. What is the future of cell and gene therapy?
The future of cell and gene therapy is promising, with advancements in personalized medicine, gene editing, AI-driven diagnostics, and scalable manufacturing. Emerging technologies are improving therapy precision, reducing costs, and expanding applications to a broader range of diseases, including neurological disorders and autoimmune conditions. As regulatory frameworks evolve and clinical success rates improve, these therapies will become more accessible to patients worldwide.
3. What kinds of diseases does gene and cell therapy treat?
Gene and cell therapy are used to treat a wide range of diseases, including:
