Gene Therapy Ebook Free 14

Gene Therapy Ebook Free 14

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Dr. Bin Xiang, a biologist by training, has solid research experience in disease biology, intestine biology, genome editing, AAV and bacterial vector gene therapy. Dr. Xiang founded CommBio Therapeutics Co., Ltd. in 2019 and devote to develop novel medicine by focusing on intestine biology and bacterial delivery system. Currently, Dr. Xiang is the CEO of CommBio, and the College Advisor at ShanghaiTech University. Dr. Xiang obtained Ph.D in neurobiology from Fudan University Shanghai Medical School in 2001, followed by postdoctoral training in cancer biology at Cold Spring Harbor Laboratory and research fellow in cancer translational research at Vanderbilt University Medical Center. In 2008, Dr. Xiang switched career to drug discovery and joined AstraZeneca Innovation Center China working on oncology translational medicine. In 2011, Dr. Xiang joined China Novartis Institutes for Biomedical Research. At Novartis, Dr. Xiang was the project team leader having directed both small molecular drug and AAV gene therapy in oncology and metabolic disease. In addition, Dr. Xiang led an innovation group exploring novel disease mechanism from intestine biology and developing new class of drug approaches including genome editing and gene therapy.

Dr. Kuan co-founded ARCE Therapeutics in 2020 and is the President and CEO. He leads ARCE from discovery toward clinical trials in developing next generation cell therapies such as CAR-engineered cells, for cancer treatment, including liquid and solid tumors, and immunological disorder diseases. He has more than 30 years of experience in research and development of therapeutic antibodies, protein fusions, and cellular gene therapy for the treatment of cancer, autoimmune, neurodegenerative and infectious diseases, including Duke University Medical Center and Development Center for Biotechnology. His areas of expertise include antibody engineering, recombinant immunotoxins, bispecific antibodies, and CAR-T/NK cell therapies for cancer treatment. Dr. Kuan obtained his Ph.D. degree from Purdue University in the USA; and his B.S. degree from National Taiwan University in Taiwan.

Dr Domenica Martorana is an R&D scientist working on gene regulation and digital PCR assay product development at QIAGEN, Hilden, Germany. She studied Molecular and Cellular Biology at the Georg-August University in Göttingen (Germany), where she received her PhD in 2019. Her PhD research focused on the transcriptional regulation of the unfolded protein response in both fungi and higher eukaryotes. Along the way she has gathered extensive experience in gene editing and gene regulation in different international projects. Since joining QIAGEN in 2019, Dr Martorana has been involved in the development of CRISPR- and functional genomics-related products as well as in dPCR application products with focus on Cell and Gene therapy.

Professor Morishita is currently on the editorial board of over 30 scientific journals including Open Medicine Journal (Editor-in-Chief), Gene Therapy, ATVB, Circulation and Hypertension. He has published over 400 original articles and review papers, primarily on gene therapy and molecular biology in cardiovascular disease. In addition, Professor Morishita is a Board Chairman of Japanese Society of Gene & Cell Therapy. He is currently Strategic Advisor of Headquarter for HealthCare Strategy (Chief is Shizo Abe, Prime Minister of Japan). He is also Special Advisor of Osaka Prefecture and Osaka City.

Into its 7th edition, Cell and Gene Therapy World Asia has firmly established itself as Asia's THE cell and gene therapy gathering for new partnerships, ideas and knowledge. We are proud to continue the meeting platform where Asia's growing cell and gene therapy industry is at the forefront, receiving more and more global attention on its fast advancement in new therapies, R&D and biomanufacturing.

Since 2017, Cell & Gene Therapy World Asia haswitnessed huge success in bringing over 300industry pioneers from both cell & gene therapyindustry. With the mission to facilitate the research& development and manufacturing of high qualitycell & gene therapy treatments and regenerativemedicines in Asia, Cell & Gene Therapy World Asia isgoing to continue its legacy.

In addition, this year, speakers will be exploringinnovations in cell & gene therapy in Asia region,best practices on cell & gene therapymanufacturing and process development, scale outstrategies, cost optimization, next generation onCART, advances in CART manufacturing,preparation for commercialization, regulation casestudies and more.

Join the conference to interact with key andupcoming entities from Asia cell & gene therapycompanies including BeiGene, GracellBiotechnologies, Fosun Kite Biotechnology, TessaTherapeutics, CARSgen, Senlang Bio, KangstemBiotech, Medigen Biotechnology Corp, ShangaiUniCAR Therapy among others.

Catch the latest cell & gene therapy development inAsia. From current best R&D practices to advancingtowards manufacturing and commercializationfrom most-exclusive case studies to industry's keyneeds. All this and more under 1 roof.

Polyplus is a leading upstream solutions provider for advanced biologic and cell and gene therapy production from research to commercial scale. An innovator in nucleic acid delivery, the legacy portfolio features process-centric transfection reagents, kits, and support services. Custom plasmid vector design was integrated into the offer in 2022 as a first measure to expanding the products and services portfolio to help customers (or the industry) optimize process economics while meeting strict scientific and regulatory standards. Headquartered in Europe, the Polyplus team continues to grow globally with operations in the United States and Asia. Learn more at www.polyplus-transfection.com.

Asia Pacific CGT Excellence Awards 2022 seeks to give recognition to exceptional Cell & Gene Therapy Pioneers, Researchers, Innovators & Manufacturers that facilitate fast advancement in new therapies, R&D and manufacturing. Featuring top Cell & Gene Therapy leaders in the industry, along with the new cell & gene therapy research development, latest advances and technologies and best practices in Cell & Gene manufacturing, the Asia Pacific CGT Excellence Awards 2022 applauds extraordinary leaders & trend-setters of today and inspires innovators of tomorrow.

Cell & Gene Therapy growing fast in the Asia region with a lot of support seen from the regulatory and its advancement in research. Many of the Academic & Research Institutes have been contributing to the cell & gene space with great research, discovery & advances. There also have been spin-offs come out of the institutes with promote cell and gene therapy in the region. The Academia plays a crucial role supported by the government and the industry in the growth of the cell and gene space.

Abstract:Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that encodes a cAMP-regulated anion channel. Although CF is a multi-organ system disease, most people with CF die of progressive lung disease that begins early in childhood and is characterized by chronic bacterial infection and inflammation. Nearly 90% of people with CF have at least one copy of the ΔF508 mutation, but there are hundreds of CFTR mutations that result in a range of disease severities. A CFTR gene replacement approach would be efficacious regardless of the disease-causing mutation. After the discovery of the CFTR gene in 1989, the in vitro proof-of-concept for gene therapy for CF was quickly established in 1990. In 1993, the first of many gene therapy clinical trials attempted to rescue the CF defect in airway epithelia. Despite the initial enthusiasm, there is still no FDA-approved gene therapy for CF. Here we discuss the history of CF gene therapy, from the discovery of the CFTR gene to current state-of-the-art gene delivery vector designs. While implementation of CF gene therapy has proven more challenging than initially envisioned; thanks to continued innovation, it may yet become a reality.Keywords: history; viral vectors; animal models; clinical trials; adeno-associated virus; adenovirus; lentivirus; retrovirus; non-viral vectors

Cell therapies repair or replace damaged cells or tissue and involve the injection of intact, living cells into a patient. These are either derived from the patient (autologous cell therapy) or a donor (allogeneic cell therapy).

Cell and gene therapies are combined in some techniques, such as ex vivo chimeric antigen receptor (CAR) T-cell therapy (figure on the right side). But they can also be utilized entirely independently, for example, in mesenchymal stem cell (MSC) therapy (cell therapy only) and in vivo viral gene transfection (gene therapy only).

Allogeneic cell therapy entails the isolation of cells from a single human leukocyte antigen (HLA)-matched donor, which is then expanded to treat many different patients. MSCs have been the pioneer cells for such applications, as they are easily accessible, their isolation is straightforward, and they can be biopreserved and stored with minimal loss of potency for point-of-care delivery. Moreover, MSCs have thus far shown no adverse reactions when used in allogeneic therapies to treat cardiovascular, neurological and immunological diseases.

Ex vivo gene therapy (cell-based therapy) involves removing target cells from the body, modifying their genome in vitro and subsequently delivering these cells back to the patient. Ex vivo gene therapy applications have focused on immune cells such as T cells or NK cells to treat vari-ous diseases, particularly cancer.2

Owing to its targeted and personalized nature, ex vivo gene therapy benefits from an improved safety profile. However, cell retrieval and in vitro manipulation can be a costly and intricate process. In vivo gene therapy is potentially more straightforward, but faces challenges such as toxici-ty or the induction of immune responses. 2b1af7f3a8