
18 Aug What are ATMPs (Cell and Gene Therapies)?
Learn more about cell & gene therapies
Advanced Therapy Medicinal Products (ATMPs) represent a cutting-edge category of treatments in the pharmaceutical industry, designed to address unmet medical needs, particularly for complex and chronic conditions. These therapies fall under four broad categories: gene therapies, somatic-cell therapies, tissue-engineered products, and combined ATMPs. This article will provide an overview of ATMPs, focusing on their significance, types, development, and regulatory considerations, as well as current challenges and future prospects.
Understanding ATMPs: A Brief Overview
ATMPs are therapies that employ genes, cells, or tissues to treat or cure diseases by modifying the patient’s genetic code or restoring normal function to damaged tissues. Unlike traditional medicines, ATMPs have the potential to provide long-lasting or even curative solutions to diseases. ATMPs can be divided into four main types:
- Gene Therapy Medicinal Products (GTMPs): These aim to introduce genetic material into cells to compensate for abnormal genes or to produce a beneficial protein. An example is treating inherited genetic disorders by replacing faulty genes.
- Somatic-Cell Therapy Medicinal Products: These involve cells that have been manipulated to treat or prevent disease. Unlike gene therapy, these cells are not modified at the genetic level but rather expanded and sometimes altered to enhance their therapeutic efficacy.
- Tissue-Engineered Products (TEPs): These products contain or consist of engineered cells or tissues used to regenerate, repair, or replace human tissue. They aim to restore normal function by replacing damaged tissues with lab-grown alternatives.
- Combined ATMPs: These therapies incorporate medical devices as part of the treatment, combining cellular or gene therapy components with a physical structure to repair or replace damaged tissues or organs.
The Regulatory Landscape for ATMPs
ATMPs are regulated by both European and US agencies, although differences in terminology and regulatory approaches exist. In Europe, the European Medicines Agency (EMA) oversees ATMPs, defining them under the “Advanced Therapy Medicinal Products Regulation (EC) No. 1394/2007.”
In Europe, the EMA classifies ATMPs into four categories:
- Gene therapy medicinal products (GTMP): These contain recombinant nucleic acids and are used to regulate, repair, replace, or add genes.
- Somatic-cell therapy medicinal products (sCTMP): These contain cells or tissues that have been manipulated to treat, prevent, or diagnose diseases.
- Tissue-engineered products (TEP): These are composed of engineered cells or tissues to repair or regenerate human tissue.
- Combined ATMPs: These contain one or more medical devices as an integral part of the therapy.
In the US, the FDA regulates ATMPs under the broader categories of cell and gene therapies and regenerative medicine. The FDA defines gene therapy as a treatment intended to modify or manipulate a patient’s genes to treat disease. In the US, the term “regenerative medicine” is also frequently used, which overlaps with some cell therapies.
The EMA’s regulation focuses on ensuring the safety, efficacy, and quality of ATMPs, providing a pathway for conditional approvals, especially for innovative therapies targeting life-threatening diseases. These regulations encourage early access to promising treatments while maintaining stringent oversight throughout their lifecycle.
The Promise of Gene Therapy
Gene therapy represents one of the most exciting developments within the ATMP space. It seeks to modify or manipulate the expression of a gene or alter the biological properties of living cells for therapeutic use. This includes introducing healthy genes to replace faulty ones, deactivating problematic genes, or repairing defective DNA in vivo.
One example of an approved gene therapy is Luxturna, which treats patients with inherited retinal dystrophy. By delivering a healthy copy of the RPE65 gene, Luxturna helps restore visual function in individuals who would otherwise face blindness.
Gene therapy has the potential to treat a wide range of conditions, from monogenic disorders like cystic fibrosis to complex diseases such as cancer. However, challenges remain, particularly in ensuring the long-term stability of these treatments and their safe integration into patients’ genomes.
CAR T-Cell Therapy: The Frontier of Cell Therapy
CAR T cell therapy, also known as chimeric antigen receptor T-cell therapy, is a form of cell-based gene therapy. This treatment combines elements of both gene therapy and cell therapy. In cell therapy, specific cells are introduced into the body to perform a therapeutic function. In cell-based gene therapy, these cells are genetically modified to enhance their function.
CAR-T therapy involves genetically modifying a patient’s own T-cells to recognise and attack cancer cells. CAR T-cells are engineered by introducing a receptor specific to cancer antigens into the patient’s T-cells, which are then expanded in a lab and reintroduced into the body to fight cancer.
CAR T-cell therapies, such as Kymriah and Yescarta, have shown remarkable success in treating certain types of blood cancers like leukaemia and lymphoma. However, their application to solid tumours remains limited due to the complex nature of these cancers, which makes them more resistant to immune system targeting.
Tissue-Engineered Products: Regenerating the Future
Tissue engineering aims to create functional tissues to repair or replace damaged tissues and organs. This branch of ATMPs has promising applications in fields such as orthopaedics, cardiovascular diseases, and wound healing.
For example, Carticel, one of the earliest approved TEPs, uses a patient’s own cartilage cells to repair cartilage damage in the knee. While this field holds significant promise, scaling up production and ensuring reproducible results remain key challenges.
Challenges when Developing ATMPs
Despite the transformative potential of ATMPs, their development poses numerous challenges.
- Manufacturing complexities: Producing ATMPs requires advanced biotechnological processes that must be tailored for each patient in some cases, such as in autologous cell therapies. This creates logistical and cost challenges.
- Delivery systems: Ensuring that gene therapies, in particular, reach their target cells effectively is one of the main hurdles. Viral vectors are often used to deliver genetic material, but these can lead to immune responses or other safety concerns.
- Cost and accessibility: ATMPs, especially gene and cell therapies, are often extremely expensive. This limits their accessibility and raises concerns about affordability and health equity.
- Long-term safety: Gene therapies involve permanently altering a patient’s genetic makeup, raising concerns about long-term risks such as unintended genetic changes, immune reactions, or even cancer.
- Regulatory landscape: Agencies are supportive of innovation, however ATMPs require robust evidence of safety and efficacy before granting full approval. Conditional marketing approvals or expedited pathways are available, but these come with rigorous post-market surveillance requirements to ensure patient safety over time.
Future Directions and Opportunities
As ATMPs continue to evolve, the future holds exciting possibilities for expanding their impact across a wider range of diseases. Innovations in CRISPR gene-editing technology are paving the way for even more precise genetic modifications, with the potential to correct genetic diseases at their root. Researchers are also exploring ways to make CAR T-cell therapies more accessible by developing “off-the-shelf” solutions that do not require customising the treatment for each patient.
Advances in the field of exosome-based therapies, which utilise small vesicles to deliver genetic material or proteins, are also gaining traction as a less invasive alternative to viral vectors in gene therapy. This could open up new treatment options for diseases previously considered untreatable.
Conclusion
ATMPs represent a revolution in the treatment of diseases, offering the possibility of long-term remission or even cures for conditions that were once deemed incurable. While significant challenges remain, including cost, manufacturing complexity, and regulatory hurdles, the progress made so far suggests that ATMPs will play a pivotal role in the future of medicine.
As researchers continue to refine and expand these technologies, the hope is that ATMPs will become more widely accessible, transforming the landscape of personalised medicine and offering new hope to patients around the world.
Written by Educo Life Sciences Expert, Melody Janssen
Dr. Melody Janssen is an expert in bioanalysis and immunogenicity testing of biopharmaceuticals with in-depth experience as a project manager, trainer and senior expert in CROs and pharma/biotech companies. She worked predominantly on developing and validating ligand-binding and cell-based assays for PK/Tox and immunogenicity studies for biologics, biosimilars and vaccines and her work supported numerous regulatory filings. Dr. Janssen is experienced in the regulatory framework related to bioanalytical method establishment and validation including FDA, EMA, ICH guidelines and USP and Ph.Eur. monographs.
This article was written from an interview (which you can read above) and from materials taken from the course, An Introduction to Cell and Gene Therapies: A Beginner’s Guide.
Watch the interview here:
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