Annual Summit on Cell Therapy and Molecular Medicine September 27-28, 2017
(10 Plenary Forums - 1Event)
Chicago, Illinois, USA

Gene therapy basically involves the introduction or alteration of genetic material within a cell  or organism with an intention of curing the disease. Both cell therapy and gene therapy are overlapping fields of biomedical  research with the goals of repairing the direct cause of genetic diseases in DNA or cellular population respectively. The discovery of recombinant DNA technology in the 1970s provided tools to efficiently develop gene therapy. Scientists use these techniques to readily manipulate viral genomes, isolate genes and identify mutations involved in human disease, characterize and regulate gene expressions, and engineer various viral and non viral vectors. Various long term treatments for anemia, haemophilia, cystic fibrosis, muscular dystrophy, Gauscher’s disease, lysosomal storage diseases, cardiovascular diseases, diabetes and diseases of bones and joints are resolved through successful gene therapy and are elusive today. 

Molecular Medicine is a branch of medicine that develops ways to diagnose and treat diseases by understanding the ways genes, proteins and other cellular molecules work. It is a broad field where physical, chemical, biological, bioinformatics, and medical techniques are used to describe molecular structures and mechanisms, identify fundamental molecular and genetic errors of the disease, and to develop molecular interventions to correct them.  Molecular Medicine has now a days proved to be an exciting field of research as some of the recent advancements has led to improved clinical benefits for human health. These are LPS- induced inflammatory response is suppressed by Wnt inhibitors, Dickkopf-1 and LGK974, Selective inhibition of Ebola entry with selective estrogen receptor modulators by disrupting the endolysosomal calcium, ApoA-IV improves insulin sensitivity and glucose uptake in mouse adipocytes via PI3K-Akt Signalling and many more.  

Due to rapidly advancing field of cancer immunology in past few years, there has been production of several new methods of treating cancer called Immunotherapies. Immunotherapy is a type of treatment that increases the strength of immune response against tumors either by stimulating the activities of specific components of immune system or by counteracting signals produced by cancer cells that suppress immune responses. Some types of immunotherapy are also called as biologic therapy or biotherapy. Recent advancements in cancer immunotherapies have provided new therapeutic approaches. These include tumor-associated macrophages as treatment targets in oncology, in-situ activation of platelets with checkpoint inhibitors for post-surgical cancer immunotherapy, immune checkpoint blockade and associated endocrinopathies and many more. 

Genetic Medicine or Medical Genetics is the branch of medicine that differs from human genetics, and involves the diagnosis and management of hereditary disorders. Human genetics may or may not apply to medicine, but medical genetics refers to the application of genetics to medical care. Genetic Medicine basically involves different areas such as gene therapy, personalized medicine, predictive medicine and the rapidly emerging new medical specialty. Now a days, medical genetics has wide range of scopes in many conditions involving birth defects and dysmorphology, autism, mental retardation, skeletal dysplasia, mitochondrial disorders, cancer genetics, connective tissue disorders and some more.  

A clinical trial is a research study that tests how well new medical approaches work on people and determines if a treatment is safe and effective. The new cell and gene therapies (CGTs) that are advancing from the laboratory into early phase clinical trials has proven to be a complex task even for experienced investigators . As a result of wide variety of CGT products and their potential applications, a case by case assessment is warranted for the design of each clinical trial. Some of the latest and advanced clinical trials include safety and efficacy trial of AAV gene therapy in patients with CNGA3 Achromatopsia, A clinical trial for treatment of Aromatic L- Amino acid Decarboxylase (AADC) deficiency using AAV2-hAADC- An expansion and Glypican 3-specific Chimeric antigenic receptor expressed in T cells for patients with pediatric solid tumors. 

Cell Therapy Bioprocessing activity mainly focuses to accelerate the safe, cost- effective translations and clinical efficacious of cell therapies into commercial products. This activity covers the entire range of cell therapy activities as well as tissue engineering. In order to succeed, commercial success of at least a few late-stage products are required to develop which will be funded to develop next generation tools and technologies for this field. Recent achievements include, preclinical filing for Phase 1 clinical trials for cell therapy in acute spinal cord injury, clinical proof of concept studies in tissue- engineered trachea, clinical trials for tissue-engineered larynx and routine clinical practice in the regeneration of corneas. The future research priorities will focus on novel cell and bioprocess engineering techniques in order to improve the manufacturing efficacy and methods for health technology assessment to support rapid clinical adoption of new cell therapies.  

Cell  and Gene Therapy products manufacturing focuses on various strategies like the manufacturing process must protect the product, patient, should focus on product characterization, process control, high throughput and parallel processing to achieve scale. The process/analytical development throughout clinical trials involve ongoing, iterative development of manufacturing process and characterization of profile and FDA expecting increasing control and characterization as clinical development progresses. Steps involved in individualized manufacturing and running in parallel for high throughput involves cell selection, expansion, activation, centrifugation and cryopreservation. 

Rare diseases are life-threatening or chronically debilitating conditions, affecting no more than 5 in 10,000 persons in the European Community according to the Regulation (EC) N. 141/2000 of the European Parliament and of the Council. It is estimated that between 6000 to 8000 distinct rare diseases affect up to 6% of the total EU population. Therefore, these conditions can be considered rare if taken individually but they affect a significant proportion of the European population when considered as a single group. Several initiatives have been taken at international, European and national level to tackle public health as well as research issues related to diagnosis, prevention, treatment and surveillance of these diseases. An Orphan drug can be defined as the one that is used to treat an orphan disease. An orphan disease in USA is defined as the one that affects fewer than 200000 individuals, but in Japan the number is 50,000 and in Australia is 2000. In past 20 years efforts have been made to encourage companies to develop orphan drugs. The Orphan Drug Act in the USA (1983) was succeeded by similar legislation in Japan (1985), Australia (1997), and the European Community (2000). The encouragement takes three forms: tax credits and research aids, simplification of marketing authorization procedures, and extended market exclusively. 

Stem cells can self renew themselves and differentiate or develop into more specialised cells. They are the foundation for every organ and tissue in our body.  Due to this ability of the stem cells, they have tremendous promise to help us understand and treat a wide range of diseases, injuries and other health related problems. Bone marrow transplantation is the most widely used stem cell therapy , but some of the therapies are derived from umbilical cord blood are also in use today. Likewise, blood stem cells are used to treat diseases of blood, a therapy that has saved thousands of lives of children with leukemia. Some bone, skin and corneal (eye) injuries and diseases can be treated by grafting or implanting tissues and the healing process relies on stem cells with implanted tissue. Regenerative medicines aims to replace tissues or organs that have been damaged by disease, trauma, or congenital issues which is in contrast to the current clinical strategy that focuses primarily on treating the symptoms. These regenerative medicines have wide appropriateness in treating degenerative scatters including dermatology, cardio vascular, and neuro degenerative diseases. Cell treatment is the quickest developing fragment of regenerative drug and this undeveloped cell treatment is making up the biggest part of this business sector. 

Cancer is a process where the cells grow aberrantly and this growth of cancer cells results in damage of normal tissues, causing loss of function and often pain. The cancer therapeutic drugs are those drugs that block the growth and spread of cancer by interfering with specific molecules (molecular targets) that are involved in the growth, progression and spread of cancer. Moreover, gene therapy  approaches may be designed to directly kill tumor cells using tumor killing viruses, or through the introduction of genes termed as suicide genes into the tumor cells. The Food and Drug Administration (FDA) has approved many cancer therapies in order to treat specific types of cancers. To develop targeted therapies it requires the identification of good targets that is, those targets that play a key role in cancer cell growth and survival.  One way to identify potential targets is to compare the amounts of individual proteins in cancer cells with those present in normal cells. Gene silencing has also been designed to inhibit the expression of specific genes which are activated or over expressed in cancer cells and can drive tumor growth, blood vessel formation and allow resistance for chemotherapy. 

Nuclear medicine is a branch of medical imaging that involves the application of radioactive substances called radiotracers that are generally injected into the bloodstream, inhaled or swallowed. The radiotracer then travels through the area being examined and gives off energy in the form of gamma rays, which are detected by a special camera and a computer to create images of inside the body. It is used to diagnose or determine the severity of or treat different types of diseases like many types of cancers, heart disease, neurological disease, gastrointestinal disease, and other abnormalities inside the body. As nuclear medicine techniques are able to identify molecular activity within the body, they offer the capability to detect diseases in its very early stages as well as a patient’s immediate response to therapeutic interventions. There are two most common imaging methods in nuclear medicine, one is Single Photon Emission Computed Tomography or SPECT and the other is Positron Emission Tomography or PET scans.

In recent times, advancements in cell engineering, imaging and screening has reached a great height in the field of science & technology and also in the business world. It has attracted many scientists from academia and also established or emerging companies in the field to present their latest scientific achievements and exciting technological solutions through presentations in several sessions. This has helped in improving the scientific knowledge among the people, scientists, researchers and exhibitors from all over the world thus enhancing their scientific curiosity and providing robust solutions against technological issues. 

Synthetic Biology is one of the emerging field of research that can be broadly described as the design and construction of novel artificial biological pathways, organisms or devices or the redesigning of existing natural biological systems. Genome editing  with engineered nucleases is a type of genetic engineering in which DNA is either inserted, replaced or deleted in the genome of an organism using engineered nucleases or “molecular scissors”. These nucleases hence create site specific double stranded breaks (DSBs) at desired locations in the genome. The induced double stranded breaks are repaired through non homologous end- joining (NHEJ) or homologous recombination (HR), thus resulting in targeted mutations (edits”). Scientists now a days use various engineered nucleases in order to bring desired changes in the human genome.