Living therapeutics on several occasions is referred to as the “new third pillar” of medicine. It is followed by small molecule drugs (simple compounds that can be manufactured chemically) and biologics (proteins and other molecules synthesized in the cells or microorganisms).
Researchers across UCSF (University of California, San Francisco) are investing their energy towards building the next generation of cellular therapies that would be able to treat diseases such as diabetes, solid tumors, neurodegeneration, autoimmunity, and other infectious diseases. The recent innovations would be revolutionary for the Microbiome Therapeutics Market as the therapies would be safer, smarter, and more effective than CAR-T therapies (therapies which one of the first to be developed under living therapeutics). This is possible mainly because of recent breakthroughs in the field of cell engineering and gene editing.
The primary concept of living therapeutics is to take advantage of normal cellular processes and making them much more efficient. However, there is usually a huge gap between what occurs inside the lab and what is received by the patient. Under LTI (Living Therapeutics Initiative), started by UCSF, researchers will try to bring new and innovative therapies directly to the kids and adults who require them and can get them anywhere else. The team is optimistic that they have come to a juncture where their therapies can help revolutionize the ways diseases are treated.
There are some disorders that the living therapies would be able to target easily. One of them is heritable disorders caused by single-gene mutations. Under this, clinical trials have already begun for CRISPR therapies for sickle cell disease. The research team is also looking towards CRISPR to help cure a combined disease of immunodeficiency syndrome and repair a genetic mutation in T cells that leads to immune deficiency.
Similarly, such cell therapies could also be used in utero to help cure a disease that occurs before birth. Against this, a clinical trial has already begun which would use blood stem cell transplanted before birth. Under the clinical trials, cells donated by the mother and transfused into her fetus would be used to treat alpha thalassemia major, which is known to be a fatal blood disorder.
The researchers believe that these are transformational times for the medical sector. Brilliant minds at UCSF would bring together their expertise of fundamental science of stem cell biology and world-class clinical capabilities to develop revolutionary Living Therapies.
Experts state that down the line, it might be possible for cell therapies to restore and regenerate tissues damaged due to aging or disease. Such applications may involve engineering immune cells to treat HIV and other infectious diseases, restoring cardiac function, and regenerating neurons.
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