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Looking to the Future of Cell and Gene Therapies

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At the front line of advances in personalized medicine are cell and gene therapies. These are two overlapping fields of medical research whereby the overall aim is to treat the underlying causes of both genetic and acquired diseases.

What is cell therapy?

In cell therapy, intact live cells are delivered to a patient via either graft, injection or implantation to eradicate or relieve the burden of a disease. This is achieved by either using cells to carry a therapy through the body or restoring a set of cells impacted by disease. At present, many cell therapies are based on autologous (meaning the cells used are the patient's own) transplants, however the landscape is moving towards the development of allogeneic (meaning the cells used are not the patient's own) transplants.

What is gene therapy?

The basic premise of gene therapy is to modify, inactivate or introduce new components to the genetic makeup of cells. By altering the DNA that is present in a cell, the protein or group of proteins that are encoded by that DNA are also altered. This means that the gene therapy can reduce the levels of dysfunctional proteins that may be causing disease, introduce the DNA code required to produce a functional protein, or increase the level of production of proteins required for disease-response.

The promise of personalized medicine

A select number of cell and gene therapies have already been approved by the Food and Drug Administration for commercialization and are available in the clinic to treat specific diseases. You may be familiar with immunotherapy for cancer treatment, for example. This is a type of cell therapy. Some therapies are also considered to be both cell and gene therapies, as they achieve their mode of action by altering genes in specific cells that are then inserted into the body.

Is the promise of personalized medicine on the edge of being delivered? Decades of research and advances in genomics, cell biology, cancer and analytical technologies have permitted exciting progress in the cell and gene therapy space recently.

Technology Networks
recently spoke with three of the leaders in this space, Allogene Therapeutics, bluebird bio Inc. and Mogrify® to gain their insights on the next developments in cell and gene therapy.

What key trends can we expect to see in the cell and gene therapy space in 2020?

"Cell therapy methods are likely to move from ex vivo approaches (where cells are isolated from the patient, reprogrammed, and delivered back into the patient), to in vivo approaches, where cell therapies are delivered directly to the recipient, for example, through the use of small molecules present in a “reprogramming cocktail” or direct gene editing."

– Joe Foster, COO, Mogrify.  

"We are on the cusp of new breakthroughs in this field and that evolution of engineered cell therapy has the potential to expand beyond cancer. Engineering, synthetic biology and gene editing has opened the door beyond allogeneic cell therapy. There is a bright future with transformative technologies that may hold the key to addressing solid tumors."

 – David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder, Allogene Therapeutics.

"Although gene therapies are still a nascent technology, they present a new paradigm for healthcare, offering a one-time treatment that can address the underlying genetic cause of certain severe genetic diseases and cancer. We anticipate that the three techniques being studied for gene therapy – gene editing, gene addition and gene-based immunotherapy – will become more distinct and that the entire treatment journey will become more efficient."

– Martin Butzal, Head of Medical Europe, bluebird bio.