Friday, 14 June 2024

Science Fiction or Reality? The Role of Gene Therapy in Regenerative Medicine

 The increasing awareness regarding advanced medical therapies supports the global regenerative medicine market demand. In addition, a strong investigational pipeline of promising candidates and expected approvals in the coming years are projected to favor market expansion. Furthermore, advancements in nanotechnology and the increasing prevalence of degenerative disorders have emphasized research to introduce novel regenerative therapies.

According to a study by Grand View Research, in 2023, the global market size of regenerative medicine was valued at USD 30.4 billion and is anticipated to expand at a CAGR of 16.8% from 2024 to 2030. Regenerative medicines have the exceptional ability to modify the elemental mechanisms of diseases. Regenerative therapies in trials deliver favorable resolutions for certain chronic indications. These solutions involve the use of multiple approaches and products, such as gene therapy, tissue-engineered products, small molecules, biologics, allogeneic & autologous stem cells, and combinational products. Supportive initiatives from governments, biotechnology & pharmaceutical companies, and healthcare providers are projected to boost the industry growth.

Where Technology meets Medicine

Novel technologies such as tissue engineering, gene therapy, and cell-based therapies are employed in regenerative medicine to offer augmented patient care with regeneration or replacement of genes, cells, organs, & tissues in patients. The increasing applications of regenerative medicine have encouraged researchers and professionals to explore the potential of novel technologies in regenerative medicine therapeutic models. For instance, developments in the fabrication of tissue mimics & advanced grafts, technologies that can integrate grafts, and the host vasculature have been gaining traction in recent years. In addition, using immune modulation and cell injection strategies to enhance the host’s intrinsic regenerative capacity is also set to gain traction.

On the other hand, cell sheet technology is a relatively newer concept dependent on retrieving confluent cell sheets from a substrate that responds to temperature. This enables the deposition of signaling molecules, Extracellular Matrix (ECM) molecules, and cell adhesion molecules by the cells, further assisting them to remain intact. The adoption of successive layers of these sheets to develop constructs of higher thickness is presently being witnessed and is explored in corneal reconstruction.

Emerging Applications of Gene Therapy in Regenerative Medicine

Gene therapy has high potential in treating diabetes and cancer with the help of restoring gene function. In the present scenario, gene-based therapies are used in treating patients suffering from cancer, monogenic diseases, cardiovascular disorders, infectious diseases, genetic disorders, ophthalmological indications, and central nervous system diseases.

The use of gene therapy to treat blood cancer has shown positive results in a study that included around 35 individuals. According to the American Cancer Society, gene-based drugs help in treating over 70% to 80% of blood cancer patients. The treatment is CAR-T therapy and has been tried on patients with lymphoma and leukemia. Gene-based therapy is one of the most efficient therapies as it provides treatment at genetic levels. Restoring the genetic alterations can help regain the lost functionality in cells, which provides opportunities to develop regenerative therapies.

Several healthcare entities are now investing in the R&D of gene-based therapies to meet the increasing demand for regenerative medicines. For instance, in March 2023, Pharmaron Beijing Co., Limited invested USD 190.26 million into the UK gene therapy CDMO with support from the British government. This investment was aimed at boosting the enhancement of gene therapy development across the UK. The advancements in gene therapy are higher in oncology due to the high disease burden and genetically driven cancer pathophysiology.

The steady commercial success of gene therapies is projected to accelerate the regenerative medicine market growth in the coming years. Furthermore, researchers are required to address certain technical challenges to make gene therapy practical. Researchers have begun using RNA Interference (RNAi) for developing gene therapy against chronic infectious diseases, such as Human Immunodeficiency Viruses (HIV). This is expected to support the future development of gene therapies.

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