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Development of Novel Technologies for Cell and Gene Therapy

GTCbio | July 26th, 2017

Gene therapy is the use of genetic material (usually DNA) to manipulate a patient’s cells as a means to treat diseases, whereas cell therapy is the infusion or transplantation of whole cells into a patient for the treatment of an inherited or acquired disease, both are overlapping fields of biomedical research.

In 1990, the first approved gene therapy clinical research took place in US, at the National Institutes of Health (NIH). Recently, development of recombinant adeno-associated virus(rAAV), miRNA, CRISP/Cas9, nanocarrier systems for drug and gene delivery and novel vector engineering have been intensively studied in both preclinical and clinical trials, and their utility has been demonstrated. Stem cell based therapies, however, are still mainly under preclinical development. Gene therapy for retinal blinding disorders is in clinical trials for inherited retinal degenerations. RNA-based mechanism of LentiFlash vector production platforms are already validated in clinical settings, making it a promising tool for therapeutic applications. Recent data show that CAR T cells might prove to be a breakthrough therapy for myeloma. To date, FDA has approved the clinical investigation of more than 1,100 different gene therapy trials and has approved gene therapy products for clinical phase investigations in humans as part of an Investigational New Drug (IND) application. Ongoing preclinical developments in cell and gene therapy have the potential to treat patients with neurological disorders, cancer, HIV/AIDS and many other genetic and acquired disorders.

The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and with an increasing understanding of the immune response to AAV administration. In particular, liver-directed therapy has made remarkable strides, with a number of clinical trials currently planned and ongoing in hemophilia A and B, as well as other liver disorders. In order for cell therapies to be commercially successful and efficient, cost effective manufacturing must be developed. BioSpherix Medical platform is a flexible platform that facilitates automation, reduces production cost and removes contamination risk.

Dendritic Cells (DCs) play an important role in emerging cell-based cancer immunotherapies ranging from antigen-pulsed autologous dendritic cell therapies to dendritic cell-stimulated autologous T cell therapies. DCs are usualy obtained manually by the conversion of blood-derived monocytes using well-established protocols but this process can have high variability in yield and has significant operator dependence. Recently, a new automated closed system, MicroDEN has been identified that overcomes these challenges. Further, next generation cell therapies such as the use of afibrotic polymers that do not trigger a fibrotic response, and encapsulated cell therapeutics that do not require immunosuppression, are expected to revolutionize the current healthcare industry.

Some of the above mentioned topics will be discussed at GTCbio’s 4th Cell and Gene Therapy Conference to be held on September 11-12, 2017 in Boston, MA. The following speakers will discuss about development of novel technologies for cell and gene therapy.

CGT speakers

•    Sam Wadsworth, Chief Scientific Officer, Dimension Therapeutics

Dr. Wadsworth holds a Ph.D. from the University of Chicago and a B.A from Southern Illinios University. He brings extensive drug discovery and development experience in the gene therapy and rare disease area. In his more than 20 years of industry experience at Genzyme and later Sanofi, he oversaw the discovery and translational research on multiple rare disease and gene therapy programs, including Genzyme’s only clinical stage gene therapy program. Most recently before joining Dimension, he served as the head of gene therapy research and early development at Sanofi Genzyme. He will talk about “Development of AAV Products for Rare Diseases”.

•    Devyn Smith, Chief Operating Officer, Sigilon

Dr. Smith received his Ph.D. in Genetics from Harvard Medical School where his research culminated in 12 publications in leading journals such as Cell, Nature, and Development. He also holds an M.S. in Biology from Idaho State University and a B.S. in Zoology from Brigham Young University. In his consulting experience, he led a wide range of projects, across multiple therapeutic areas and a host of technology platforms, including basic R&D tools, regenerative medicine, gene therapy, and macromolecules/biologic products. Regenerative Medicine has been an area of strong focus with over 30 engagements, several published papers, and numerous invited conference presentations. He will talk about “Next Generation Cell Therapies – Will These Revolutionize Healthcare“.

•    Shashi Murthy, Professor of Chemical Engineering , Northeastern University

Dr. Murthy earned his Ph.D. in Materials Science & Engineering at the Massachusetts Institute of Technology (MIT) in 2003 and his B.S. in Chemical Engineering at Johns Hopkins University in 1999 Dr. Murthy has co-authored over 70 publications and is an inventor on 7 issued or pending patents. He is also founder of Quad Technologies, which has commercialized hydrogels as releasable magnetic beads for cell separation and reagents for cell activation. He is an expert in the areas of cell separation and automated cell culture and current projects in his lab focus on patient-specific dendritic cell generation and dendritic cell-mediated T cell expansion for therapeutic use. He will talk about “Automated Closed System Production of Dendritic Cells”.

•    Kevin Murray, Vice President, Global Sales, BioSpherix Medical
Mr. Murray did BSc BioChemistry from Virginia Polytechnic Institute and State University and MBA Finance & Marketing from California State University. He has 20+ Years of extensive experience in the Pharmaceutical/Biotech/Medical Research Industry. He will talk about “Rapid, Efficient, Scale up/Scale out for any Cell Therapy Production Process”.


We invite you to join us at the 4th Annual Cell & Gene Therapy Conference to be held on September 11-12, 2017 in Boston, MA.