Prominent Scientists will discuss Evolution of Cell and Gene Therapy

GTCbio | July 11th, 2017

Cell and gene therapy are overlapping fields of biomedical research that address the repair of the direct cause of genetic diseases in the cellular population or DNA, respectively.

Cell therapy can be defined as a therapy in which cells or cellular material are injected into a patient. The origins of cell therapy can perhaps be traced to the nineteenth century, when Charles-Édouard Brown-Séquard (1817–1894) injected animal testicle extracts in an unsuccessful attempt to stop the effects of aging. In 1931 Paul Niehans (1882–1971), who has been called the inventor of cell therapy, attempted to cure a patient by injecting material from calf embryos. In 1953, researchers found that pre-inoculating cells from donor animals could reduce rejection of transplanted tissue. In 1968, in Minnesota, the first successful human bone marrow transplantation took place. Dr. Richard Gatti, our keynote speaker, was a member of that team. In recent times, cell therapy using human material has emerged as central process in the treatment of human disease.

Gene therapy is the process of introducing foreign genomic materials into the DNA of host cells to elicit a therapeutic benefit. The therapy was conceptualized in 1972 and the first attempt of modifying human DNA was performed in 1980 by Martin Cline. However, nuclear gene transfer in humans was not approved until the late 1980s . The first approved gene therapy clinical research in the US took place on 14 September 1990, at the National Institutes of Health (NIH), under the direction of William French Anderson. Further, in 1993 the first somatic treatment that produced a permanent genetic change, was performed. The first commercial gene therapy Gendicine was approved in China in 2003 for the treatment of certain cancers. Clinical successes since 2006 regained researchers’ attention, although it remains an experimental technique. In recent years US companies have invested over $600 million in this field. In 2016, the Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis and it was approved by the European Commission in June of this year.

Recent clinical trials of cell and gene therapy have shown remarkable therapeutic benefits and an excellent safety record. The exciting possibility of treating many genetic and infectious disorders is now close to a reality through the success of recombinant adeno-associated virus (rAAV) as genome editing platform, novel vector engineering, and the recent disruptive discoveries of micro RNA (miRNA), and CRISPR/Cas9. We are on the brink of having therapies approved for clinical use in the United States, while the European Medicines Agency has two approved therapies. AAV encoded miRNAs will soon be tested in clinical trials, whereas technologies, such as CRISPR/Cas9, are still in proof-of-concept stages but hold great  clinical promise. Some of the breakthrough stories of year 2017 include use of stem cells in growing new human retinas; helping stroke victims to walk; and aiding paralyzed patients in the use of their arms. Also, the use of bioreactors in cell therapy applications is on the rise, as clinical trials and commercialization of cell therapy–based products are moving to the forefront of treatment opportunities.

Although numerous cellular and gene therapy products are currently in different phases of clinical trials, only 14 have so far been approved for use by FDA. The 14 FDA-approved products include cellular immunotherapies and other types of both autologous and allogeneic cells for certain therapeutic indications. FDA has not approved any stem cell-based products, except ones that utilize cord blood-derived haematopoietic progenitor cells.

Cell and gene therapy are now in a maturation phase with all indications suggesting breakthrough therapies in the near future. Research based on stem cells and genes for treating various diseases is being undertaken in many countries. Recent work suggests that chimeric antigen receptor-T (CAR-T) cells might prove to be a breakthrough therapy for myeloma. The expected focus of cell and gene therapy appears to be retinal diseases, ageing, type 1 diabetes, and especially immuno-oncology. In the field of gene therapy, the advent of new next-generation vectors, preservation technologies for gene therapy, and gene-carrying nanoparticles may  dominate in near future.

CGT keynote speakers

The keynote speakers at GTCbio’s 4th Cell & Gene Therapy (CGT) Conference will be discussing the evolution of cell and gene therapy. The CGT Conference will be held on September 11-12, 2017 in Boston, MA. The following are the plenary keynote speakers at this summit.

  • Richard Gatti, Distinguished Professor in the Departments of Pathology & Laboratory Medicine and Human Genetics, David Geffen UCLA School of Medicine.

Dr. Gatti is a molecular biologist and geneticist who joined the UCLA School of Medicine faculty in 1974 as full professor. He served as Director of Pediatric Hematology, Oncology and Immunology at Cedars-Sinai Medical Center (1974-1980). He joined the Department of Pathology & Laboratory Medicine at UCLA in 1981. He has co-authored over 330 scientific articles. He pioneered the field of hematopoietic stem cell transplantation and other treatments for primary immunodeficiencies. His current research focuses on the molecular genetics, diagnostics, radiobiology, and treatment of children with ataxia-telangiectasia, a progressive neurological disease of children.

  • James Wilson, Professor, Internal Medicine and Pathology & Laboratory Medicine; Director, Gene Therapy Program, University of Pennsylvania.

Dr. Wilson’s laboratory focuses on the development of gene transfer vectors and their application in the treatment of a variety of acquired and inherited diseases. He has recently isolated new families of simian-based adenoviruses and adeno-associated viruses.


We invite you to join us at the 4th Annual Cell & Gene Therapy Summit, which includes the following sessions:

  1. Opening Keynote Session – Historical Perspective & Current State of the Industry
  2. Preclinical Development of Novel Technologies
  3. Translational Studies & Lessons Learned
  4. Round Table Discussions
  5. Breakfast with Mentors from Academia and Industry
  6. Industry Partnering with Academia & CROs to Advance Research
  7. Funding & Regulatory Challenges
  8. Award Ceremony & Closing Keynote Session–Future of CGT