Gene therapy has become a therapeutic alternative for patients with various pathologies such as diabetes, cancer, and orphan diseases. These innovations are classified into three large groups: gene therapy, cell therapy, or tissue therapy, also called tissue engineering. Cell therapy includes procedures that manipulate cells in a laboratory culture to increase their number or change their function. Stem cells that can generate the function that others have lost are generally used. For example, they can become cells of a needed tissue, such as heart muscle. After a heart attack where a piece of muscle has died, stem cells can generate normal heart tissue. Tissue therapy is about compensating for losing a tissue with another generated in a culture laboratory. The tissue loss can be due to genetic or traumatic causes and allows the initial function to be recovered. The best-known examples are covering large areas of burns with skin cultures grown in a laboratory or replacing a fragment of a bone with bone tissue grown in a laboratory culture.
Additionally, gene therapy is a strategy to correct mutations in the patient’s genetic information (DNA), thus modifying the evolution of the disease. For this, different techniques are used that add genetic material to the patient’s cells, restoring the function lost due to mutations in their own DNA. Although there are different definitions for ‘gene therapy’, some broader than others, broadly speaking, gene therapy would be any treatment that introduces genetic material into the patient’s cells.
Currently, countries such as the US and Canada have implemented health strategies to guarantee access to these therapies for patients who need them most. Mayflower BioVentures is an accelerator that was born from the collaboration of Mayo Clinic, Hibiscus BioVentures (Hibiscus), and Innoforce with the objective of unmet patient needs in the field and creating an environment for development between companies around the field. Mayo Clinic’s Center for Regenerative Biotherapeutics has been a major player in the development of regenerative technologies and clinical trials. The collaboration between the companies aims to create a network to accelerate gene therapy innovations by identifying patients’ needs that haven’t been addressed.
These therapies are a direct translation of basic scientific research into concrete therapeutic options for patients, opening the door to a new era of medicine.