Gene Therapy Market Size, Growth, Share & Trends Analysis

As of 2024, gene silencing therapy held the largest share of the gene therapy market and will continue leading the market through 2025. Gene silencing therapy is an approach in molecular biology and medicine that aims to regulate or suppress the expression of specific genes responsible for diseases. This therapy is based on shutting down the activity of genes, either temporarily or permanently, to treat a wide range of genetic and acquired disorders. Gene silencing can be achieved through several mechanisms, including RNA interference and oligonucleotides. Inherited genetic disorders caused by single gene mutations can be treated by silencing the defective gene.

In 2024, viral vectors dominated the gene therapy market. Viral vectors are genetically modified viruses used as a tool for gene delivery. They target cells to express the delivered gene for therapeutic purposes. Generally, retroviral vectors, adeno-associated virus vectors, and other viral vectors such as herpes simplex virus and adenovirus are used in gene therapy. In gene therapy, these viruses are engineered to carry the desired therapeutic genes instead of harmful viral genes. This makes them safe and effective tools for delivering genetic material to correct or treat genetic disorders and other diseases. The viral vectors segment is further divided into retroviral, adeno-associated virus (AAV), and other viral vectors.

The deficiency segment is expected to dominate the gene therapy market. Deficiency genes are introduced into the body to replace or supplement defective or missing genes, addressing genetic disorders caused by the absence or malfunction of specific genes. These therapies often use viral vectors to deliver functional gene copies directly to affected cells. The process involves a vector-commonly a viral vector like AAV or Lentivirus-engineered to carry the healthy gene into the target cells. Upon delivery, the cells produce the necessary protein, alleviating disease symptoms. This approach is often applied to genetic conditions without curative treatments, emphasizing its transformative potential.

In 2024, neurology segment dominated the gene therapy market. It encompasses a wide range of conditions such as neurodegenerative diseases (e.g., Alzheimer's, Parkinson's), neuromuscular disorders (e.g., ALS, muscular dystrophy), epilepsy, stroke, and genetic conditions like spinal muscular atrophy (SMA) and Huntington's disease. Gene therapy in neurology uses genetic material to treat or prevent neurological diseases. This involves techniques like gene replacement, where a missing or defective gene is replaced with a healthy one; gene editing using technologies like CRISPR-Cas9 to modify the faulty genes; and gene silencing to reduce harmful gene expression. Viral vectors, especially adeno-associated viruses (AAV), are frequently used to deliver therapeutic genes into the brain or spinal cord. Successful applications include treatments for SMA, such as ZOLGENSMA, which delivers a functional copy of the SMN1 gene to motor neurons, and ongoing research in conditions like Huntington's and Parkinson's disease.

The In Vivo segment is expected to dominate the gene therapy market by delivery method. In vivo gene therapy involves directly modifying a patient's genes inside their body to treat or prevent a genetic disease or other medical conditions. The in vivo delivery method administers the gene therapy vector directly to the target organ or via the vascular system into vessels feeding that organ. Therefore, depending upon the vector type and target organ, the in vivo gene therapy agent can be administered through the intravenous route (IV), intramuscular route (IM), infused/injected into an organ or bodily structure, or injected directly into a tumor. Currently, most marketed gene therapy products are delivered in vivo.

In 2024, Intravenous route of administration segment dominated the gene therapy market. The IV route of administration delivers medications and fluids directly into the patient's bloodstream. This route is characterized by the injection of drugs or fluids directly into a vein using a syringe, catheter, or IV bag. IV administration allows for the systemic distribution of gene therapies, reaching target cells and tissues throughout the body. Demand for intravenously administered therapies is growing because of their rapidity, effectiveness, and noninvasive nature, as they do not require a surgical procedure. Some viral vectors in gene therapy are also suitable for the IV route only.

Hospital segment is expected to dominate the gene therapy market by end user. Hospitals serve as the primary end users for gene therapies due to their specialized infrastructure and expertise in delivering these complex treatments. Gene therapies often involve sophisticated techniques such as genetic modification of cells, precise administration of therapy, and close monitoring of patient outcomes. Hospitals have the necessary facilities to ensure safe and effective delivery, including advanced laboratories, surgical units, and medical teams. The increasing number of hospitals in developed countries due to the increasing patient population and demand for advanced therapies is expected to drive market growth.