What is cell and gene therapy?
We’re living at a pivotal moment in which human biology can be understood and remastered at a microscopic level, creating new personalized therapeutics for once-untreatable diseases. These therapies belong to a new category of medicine that is often summed up under a variety of different names — cell and gene therapy (CGT), advanced therapies, regenerative medicine, or ATMPs (advanced therapy medicinal products).
What do these different category names — and the various therapies contained within them — all have in common? They all represent therapeutics for human use that are based on the building blocks of life — genes, cells, DNA information, and/or tissues. Vineti’s mission is to create the digital technology required to provide these transformative treatments to all patients in need. Here’s an overview of the therapies we support.
What are cells and genes?
Cells are the basic structural and functional units of all living things. Genes are small segments of genetic information found within cells, and are made up of a hereditary material called DNA. Genes are passed from parent to offspring, and often contain instructions that help cells function.
In some cases, serious health conditions may arise due to disorders rooted in these basic biological units. Cancer, for example, arises when changes in a cell’s genetic information cause that cell to divide rapidly and out of control. Inherited disorders such as sickle cell anemia arise when an irregular gene issues an abnormal instruction to cells, impairing the body’s ability to function.
What are cell- and gene-based therapies?
Therapeutics focused on cells and genes aim to provide new medical options by addressing the root causes of many serious disorders. This innovative therapeutic category is constantly evolving and expanding thanks to ongoing scientific research.
Gene-based therapies seek to either introduce genes into a patient’s body, or modify genes that are not functioning normally.
Gene therapies set out to replace a disease-causing gene with a more functional version, or introduce a new copy of a gene to fight disease. One gene therapy approved by medical regulators, for example, treats an inherited form of childhood blindness. Gene therapies are typically delivered in vivo, meaning that the therapeutic genes are transferred into cells inside the patient’s body.
Gene-modified cell therapies also work to alter patient cells at the genetic level, enabling the cells to perform new or enhanced functions. One category of approved gene-modified cell therapies is CAR-T cell therapy, which retrains immune cells to effectively recognize and destroy cancer cells. Gene-modified cell therapies are typically produced ex vivo, meaning that important cells are extracted from the patient’s body, genetically modified, and then reintroduced back into the body.
Cell therapy is the introduction of living cells into a patient’s body to replace or repair damaged tissue or treat a variety of diseases. These therapeutic cells are selected for their beneficial qualities and then may also boosted in some way, such as genetic modification for greater ability to detect and fight disease (see gene-modified cell therapy, above).
Cell therapies may come from a variety of sources. Some cell therapies are autologous, meaning that the cells come from the same patient receiving the final treatment. Some cell therapies are allogeneic, meaning that the cells come from a donor.
In just one example of cell therapy, bone-forming cells taken from healthy donors are developed into a clinical-phase therapy intended to treat broken bones that are failing to heal.
These new types of vaccines, also sometimes called neoantigen cancer vaccines or inactive cell vaccines (such as dendritic cell vaccines), are intended to treat disease rather than prevent it. These treatments are based on a detailed analysis of unique proteins, or neoantigens, found on the surface of each cancer patient’s tumor cells.
These vaccines representa a type of immunotherapy that rallies the immune system to identify and kill cancer cells.
By understanding this aspect of a patient’s tumor at a molecular level, biopharmaceutical innovators are working to develop personalized treatments that target each patient’s cancer. Many of these treatments, which are still in the research phase, include a combination of patient-specific materials, such as tumor samples, patient cells, and patient-specific genetic information. One such clinical-phase product, for example, is being studied for the treatment of common cancers such as breast or colorectal cancer.
Genome editing focuses on making changes to disease-related sections of DNA, often by using molecular technologies to “cut” or remove a particular section of DNA and replace it with a segment of DNA intended to function normally. Genome editing is still in the research phase, and is being studied as a way to treat cancer and a wide variety of genetic disorders. CRISPR, a technology that won the Nobel Prize for Chemistry in 2020, is one example of genome editing.
In tissue engineering, treatments are often formed from a combination of living cells and a biological foundation, or “scaffold,” upon which the cells can grow ex vivo to form new tissues. These treatments must be produced in such a way that they are a biological match for the intended patient. Approved tissue-engineered products, for example, treat cartilage disorders of the knee or severe skin damage and loss after deep burns.
Why do these therapies require new digital technology?
While these new therapeutics may rely on a wide variety of approaches, they are all highly patient-specific. Some must be closely matched to the intended patient, while others are manufactured from cells taken from the very patient needing the treatment. This patient-specific paradigm requires a whole new kind of tracking, tracing, and control to make sure that the right patient receives the right therapy, safely and efficiently. Legacy technologies used in biopharmaceutical development were created to serve mass-produced drug products, and cannot effectively or safely create and deliver personalized, patient-specific treatments. Vineti was founded to address this essential requirement for a personalized medicine ecosystem and make these new therapeutics available to all patients in need.