Cell and Gene Therapy

Extracted Attributes

• Field

  Biotechnology and Regenerative Medicine

• Cell Sources

  Autologous (patient-derived) and Allogeneic (donor-derived)

• Key Applications

  Oncology, Rare pediatric disorders, Degenerative diseases, and Immunogenic pathologies

• Regulatory Focus

  Accelerated approval and shortened clinical trial timelines

• Primary Modalities

  Cell transplantation and Genetic modification

Timeline

• Origin of cell therapy through experiments involving the injection of animal material to treat illness. (Source: Wikipedia)

  1850-01-01

• Discovery that human cells could prevent organ rejection, leading to the development of bone marrow transplantation. (Source: Wikipedia)

  1950-01-01

• FDA approves the first cell-based gene therapy, a CAR-T therapy, for cancer treatment. (Source: Web Search (PhRMA))

  2017-08-30

• First regulatory approvals for CRISPR-Cas9 therapeutic gene editing for Sickle Cell Disease and transfusion-dependent beta-Thalassemia. (Source: Web Search (News-Medical))

  2024-01-01

• FDA Commissioner Marty Makary discusses accelerating cell and gene therapy approvals at the J.P. Morgan Healthcare Conference. (Source: Document 065d2e96-4d40-49bd-8511-d8d35f8b01f4)

  2025-01-13

Cell therapy

Cell therapy (also called cellular therapy, cell transplantation, or cytotherapy) is a therapy in which viable cells are injected, grafted or implanted into a patient in order to effectuate a medicinal effect, for example, by transplanting T-cells capable of fighting cancer cells via cell-mediated immunity in the course of immunotherapy, or grafting stem cells to regenerate diseased tissues. Cell therapy originated in the nineteenth century when scientists experimented by injecting animal material in an attempt to prevent and treat illness. Although such attempts produced no positive benefit, further research found in the mid twentieth century that human cells could be used to help prevent the human body rejecting transplanted organs, leading in time to successful bone marrow transplantation as has become common practice in treatment for patients that have compromised bone marrow after disease, infection, radiation or chemotherapy. In recent decades, however, stem cell and cell transplantation has gained significant interest by researchers as a potential new therapeutic strategy for a wide range of diseases, in particular for degenerative and immunogenic pathologies.

Web Search Results

• What is cell and gene therapy | Novartis

  ## The difference between cell therapy and gene therapy: Cell therapy aims to treat diseases by restoring or altering certain sets of cells or by using cells to carry a therapy through the body5. With cell therapy, cells are cultivated or modified outside the body before being injected into the patient. The cells may originate from the patient (autologous cells) or a donor (allogeneic cells)6. Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into cells— either inside the body (in vivo) or outside of the body (ex vivo)6. Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body. [...] 1. Home 2. About 3. Novartis Gene Therapies 4. What is cell and gene therapy # What is cell and gene therapy ## What are cells and genes? Cells are the basic building blocks of all living things, and genes can be found deep within cells. Genes are small sections of DNA that carry genetic information and instructions for making proteins, which help build and maintain the body1. Every person has around 20,000 genes and two copies of each of their genes—one from each parent. Small variations in genes result in differences in people’s appearance and, potentially, health1. ## What are genetic diseases? [...] ### Learn more about how we use cell and gene therapies and why they are important View the infographic References

• Cell & Gene Therapies - PhRMA

  Cell and gene therapies have the potential to cure disease in a one-time administration by targeting the root cause of disease at the cellular or genetic level. Since the first FDA approved cell-based gene therapy, a CAR T therapy in 2017, these medical technologies have transformed treatment for patients, like those with cancer and rare pediatric disorders. Rather than using traditional chemical compounds to treat disease, these therapies work by correcting or replacing damaged cells or genes. Today, the FDA has approved five CAR-T therapies, which use the body’s own immune system to fight disease, and two gene therapies. In cell therapy, cells are cultivated or modified outside the body before being injected into the patient to treat a condition in which the patient’s cells are damaged [...] ## The Complex Cell and Gene Therapy Production Process Most conventional drugs can be manufactured in bulk, stored and distributed as needed. Cell and gene therapies are different, each treatment is custom-made from an individual patient’s own cells, requiring rapid, time-sensitive production and immediate administration. This highly complex, personalized process demands advanced quality controls and a new level of manufacturing sophistication. Because these technologies are still evolving, many production methods continue to be refined and optimized. [...] treat a condition in which the patient’s cells are damaged or diseased. And in gene therapy, a patient’s genetic material (such as DNA) is used to treat, cure or even prevent a disease.

• Gene and Cell Therapy | North Carolina Biotechnology Center

  ## What is Gene and Cell Therapy? Cell and gene therapies use cells and genetic material to treat diseases. Cell therapies involve transplanting healthy human cells into a patient’s body to replace or repair diseased or damaged cells or tissue, whereas gene therapy is defined as the treatment of disease by the transfer of genetic material into cells. Gene and cell therapies provide a fundamental treatment option for disease conditions that cannot be treated with conventional drugs. ## The Difference Between Gene Therapy and Cell Therapy [...] Gene therapy restores the function of mutated genes by introducing a normal copy of the gene to ensure that correct proteins are produced. Cell therapy places new healthy cells into the body to replace diseased or damaged ones. Both cell and gene therapies provide new treatment options for diseases caused by genetic mutations including cardiovascular, blood clotting, neurodegenerative, and vision disorders. Source: Global Cell and Gene Therapy Market. Accessed Feb. 2022. About the Sector Workforce Development Business Climate Resources ### Gene therapy technology pioneered in North Carolina [...] Some genetic diseases arise from a single mutation, while others stem from a combination of mutations and other factors, including environmental exposure. It is currently possible to treat certain diseases with cell and gene therapy. However, research is ongoing in oncology, autoimmune diseases, musculoskeletal disorders, cardiovascular disorders, dermatological conditions and genetic disorders. ## What is Gene and Cell Therapy?

• Cell and Gene Therapy Insights

  {{#if interestId}} {{/if}} # CELL AND GENE THERAPY INSIGHTS Cell & Gene Therapy Insights is an online, open-access, peer-reviewed journal dedicated to the interdisciplinary exploration and advancement of cell and gene therapy. With a translational focus, we connect innovative research to practical clinical applications, providing valuable insights into one of biotechnology’s fastest-evolving fields. We address the key challenges and latest developments across advanced therapies, publishing original research articles, expert reviews, commentary, clinical trial reports, and more. Visit the Cell & Gene Therapy Insights Journal page for our complete collection. [...] Our popular webinar series provides expert-led discussions on important developments and methodologies in the field, designed to support ongoing professional growth. Explore our specialised channels, including detailed coverage of critical areas like the cell and gene supply chain, for targeted insights into manufacturing, logistics, and regulatory frameworks essential to therapy commercialisation. Learn more about our journal’s mission and publishing criteria by visiting our aims and scope. Sign-up for free to gain unlimited access to our extensive library of articles, webinars, podcasts, news, and interviews, and stay at the forefront of cell and gene therapy innovation.

• The Latest Advances in Cell and Gene Therapy

  13. Sabatini MT, Chalmers M. The Cost of Biotech Innovation: Exploring Research and Development Costs of Cell and Gene Therapies._Pharm Med._ 2023;37, 365–375. doi.org/10.1007/s40290-023-00480-0 14. Mireku A. Cell and gene therapy companies trip at scalability hurdle. 2024. Available at: [...] 5. Fermaglich LJ, Miller KL. A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act. _Orphanet J Rare Dis_. 2023;18(1):163. doi: 10.1186/s13023-023-02790-7. 6. Kakkaiyadi, K. Cell and gene therapy companies can shape the UK’s industrial strategy. 2024; Available at: 7. Cell and Gene Therapy Catapult. Annual Review 2024. Available at: 8. Parums DV. Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia. _Med Sci Monit_. 2024;30:e944204. doi: 10.12659/MSM.944204. [...] Systemic therapy for gallbladder cancer in the era of precision medicine： Coexistence of opportunities and challengesJournal of Clinical Hepatology, 2023