The gene and cell therapies clinical impact, production, and processes efficiency depend on the unique challenges related to CMC, analytical, process development, automation, manufacturing, facility design, aseptic processing, supply chain and others that developers of gene and cell therapies must resolve.
Key therapies experts, advisers, and practitioners will focus primarily on:
The #VLGeneCell brochure 2023 contains additional key insights as well as information about the concurrent #VLasepticRead more
Gene and cell therapies breakthroughs are advancing rapidly.
Autologous and allogenic cell-based therapies, including CAR-T, TCR, NK-cells, TIL, viral and non-viral vector mediated therapies, and other ATMPs demonstrate tremendous progress.
The full extent of their clinical impact, production, processes performance, and efficiency hardly depend on the unique challenges related to CMC, analytical, process development, automation, manufacturing, facility design, aseptic processing, supply chain and others that developers of gene and cell therapies must resolve.
At the #VLGeneCell Summit, key experts, advisers, and practitioners will focus primarily on:
The leading gene and cell therapies practitioners and consultants will share insights, technical and regulatory requirements, and expectations on successfully achieving and scaling-up robust, cost- and quality-efficient products, process developments, and manufacturing, as well as explore recent developments, emerging technologies, methods, and tools, including the updates on the aseptic processing at the concurrent event.
Join the #VLGeneCell to explore the industry challenges, standards, and advancements on June 20-21, 2023, and take an advantage of the concurrent #VLaseptic.
Chief Executives, Vice Presidents, Directors, Heads, Leaders, and Managers specialising in:
The demand of aseptic manufacturing is rising, and, therefore, the challenges of aseptic processing will continue to grow in complexity.
Ensuring product quality at faster speed and with better flexibility is becoming the key priority.
The Annex 1 of the EU GMP Guide "Manufacture of Sterile Medicinal Products" is considered to be the most important European regulatory standard for the manufacture of sterile pharmaceutical products. The changes to Annex 1 are bringing new obstacles to pharma company operations that should be overcome from both technical and organisational perspectives.
The 6th Aseptic Processing Summit on June 21-22, 2023, will bring together pharmaceutical developers, manufacturers, CDMOs, suppliers, engineering companies, and experts from the field to highlight:
Join the #VLaseptic to explore the industry challenges, standards, and advancements, as well as develop new partnerships and showcase innovative technologies at the premium conference and exhibition on June 21-22, 2023.
Chief Executives, Directors, Vice Presidents, Heads, Leaders, Senior Managers, Scientists, Chemists, Engineers, and Fellows specialising in:
Aidan Luttrell is scientist within the cell process MSAT group at Orchard Therapeutics. In 2019 he joined Orchard, where he manages pre-commercial and commercial cell and gene therapy drug product manufacture. His role includes supporting regulatory filing via authoring content and delivering technical content for variations and amendments to existing filings. Aidan has a BSc from Dublin City University in biotechnology, in collaboration with the National Institute of Cellular Biotechnology (NICB). He initially worked in clinical research with CROs over a range of therapeutic areas before he joined Pfizer, moving into a technical operations role. There he built knowledge in technologies transfer, process development, and GMP manufacturing working on late stage and commercial products (Nimenrix and ReFacto) before joining Orchard Therapeutics.
Dr. Behnam Ahmadian Baghbaderani is the global head of process development and cell and gene technologies at Lonza. He has over 18 years of experience in development of cell and gene therapies for clinical and commercial applications.
Behnam holds a PhD degree in biomedical engineering from the University of Calgary (Canada), where he developed bioreactor protocols for large-scale expansion of human neural stem cells for clinical applications. He completed nearly three years of a postdoctoral program, including a two-year postdoctoral fellowship at the NIH focussing on generation of human-induced pluripotent stem cells (iPSCs), bioprocessing of human embryonic stem cells and human iPSCs, and controlled differentiation into neuronal lineage.
After joining Lonza in 2011, he started to lead a group of scientists in process development, establishing pluripotent stem cell platform technologies and a cGMP compliant manufacturing process for iPSCs. Behnam then led the cell therapy development department, focussing on the development of cGMP compliant processes and analytical methods for different autologous and allogeneic cell therapy applications. In his current role, Behnam has been leading a global organisation offering development services for a wide range of early- and late-stage viral vector gene therapies as well as autologous and allogeneic cell therapy applications at Lonza.
Dr. Shengjian Shawn Liu is an experienced biotechnologist, virologist, biochemist, and serial entrepreneur. Prior to Avirmax, Shengjiang held executive positions including chief scientist and global head of pathogen safety of Bayer Pharmaceuticals for 15 years. He was a co-founder and the president of Abmaxis, Inc., which was acquired by Merck in 2006. He was also the group leader of Genentech BSL-3 Research Virology Lab (1996-2000). In his scientific career, he has developed bioprocess technologies to prevent commercial scale bioreactors from virus contamination and secured virus safety for biotherapeutics. In 1984 he discovered and characterised a new virus, rabbit hemorrhagic disease virus (RHDV), the rabbit calicivirus. During the past 25 years of his biotechnological career, he has played important roles in innovation, development, tech transfer and manufacturing support, and regulatory submission for approvals at various development stages of many biologics including GT therapy AAV vectors, out of which 10 are widely used biologics in the market. Now he is focussing primarily on AAV and GOI engineering, innovation, development, and production for ocular disorders.
Shengjian received his PhD in biochemistry from Kansas State University (1990-93) and his post-doctoral training was with Dr. Arthur Kornberg, Stanford University (1994-96). He is a member of ASGCT and ARVO as well as an advisor of the bioengineering department at Santa Clara University (Santa Clara, CA).
Dr. Kuldip Sra has over 25 years of work experience in biotech and pharmaceutical industries. Currently, Kuldip is executive director in tech ops at Crisper
Therapeutics. Previously, he was executive director in tech ops at Crisper, and he worked at Kite Pharma/Gilead, where he was director of QC. Kuldip is involved in developing and validating analytical assays
for lot release and characterisation of autologous and allogenic cell therapy drug products. In the past, he worked at MedImmune/AstraZeneca for 15 years.
Kuldip led the vaccine analytical development and characterisation group at MedImmune at its Mountain View site.
Christiane Niederlaender has spent nine years at the UK medicines regulator MHRA, most recently as the unit manager of the biologicals unit. At the same time, Christiane was a biologicals senior quality assessor and the UK delegate at the EMA committee for advanced therapies (CAT). As UK CAT rapporteur, she was responsible for managing the portfolio of all UK ATMP products (cell and gene therapies). She was also the rapporteur for the EMA CAT gene therapy guideline, and a drafting group member in all other quality related ATMP drafting groups, including GMP for ATMPs. Prior to joining MHRA, Christiane worked with the UK regulator for the European Tissues and Cells Directive as a quality manager and inspector.
Christiane joined Parexel in January 2021 as vice president technical for CMC and now works with developers to get cell and gene therapies into the clinic and to market. Since joining Parexel, Christiane has done a significant amount of work within the US regulatory system and can advise on EU, UK, and US CMC considerations.
Matthias Bozza, PhD, is the asociate director for gene regulation at Vector BioPharma. A molecular biologist by training, he joined Vector Biopharma from the German Cancer Research Centre (DKFZ) in Heidelberg, Germany, where he served as a senior staff member in the department of Dr Richard Harbottle. Matthias got his master's degree in Italy and his PhD from the University of Heidelberg. He received multiple international awards (ASGT and AACR) for his research and was elected one of the most talented Italian scientists under 35 in 2021. Matthias developed new highly engineered gene and cell therapy vectors by implementing cutting-edge synthetic biology systems and was responsible for their implementation into clinical pipelines gaining extensive experience in vector design and GMP manufacturing.
Chaminda Salgado is a highly experienced CMC Leader, with experience in CMC biological assets and cell and gene therapies, ensuring the smooth flow of programs from lead candidate through to commercialisation. Before joining T-Cypher Bio, Chaminda was leading the analytical innovation capability projects for CGT at GSK to address testing issues to releasing autologous therapies for indications with high patient numbers.
Dr Houria Bachtarzi is a director of regulatory, CMC, and gene and cell therapies at Precision for Medicine. She has 16-plus years of experience in the scientific, CMC and regulatory/development strategic aspects of advanced biological therapies, including virally vectored gene therapy, in vivo and ex vivo gene editing, cell-based immunotherapy, genetically modified cells for tissue regeneration, genetically engineered immune cells targeting cancer cells, and stem cell-based therapies.
Houria started her career by first training and registering as a pharmacist in the UK before embarking into a unique journey in gene therapy and regenerative medicine. She completed her PhD in cancer gene therapy at the University of Oxford, department of oncology (previously department of clinical pharmacology). She subsequently followed up with additional post-doctoral work in AAV-based gene therapy and shRNA therapeutics for degenerative neuro-muscular disorders at the Centre for Biomedical Sciences, Royal Holloway – University of London, in collaboration with Benitec Biopharma, Inc. and the Institute of Myology at the Pitié-Salpétrière Hospital in Paris, France. In 2013, Houria was appointed as a lecturer in biopharmaceutics and biotechnology at the school of pharmacy and biomolecular sciences, University of Brighton.
Prior to joining Precision for Medicine, Houria was an associate director of regulatory sciences for gene and cell therapies at ProPharma Group (formerly Diamond Pharma Services). She also held senior consultant positions at other product development and regulatory consultancies, including ERA Consulting (UK) Ltd. (now part of PharmaLex), NDA Group, and Dark Horse Consulting.
Houria holds a first-class master of pharmacy degree (with honours) from the University of Bath, UK, and is a registered pharmacist with the General Pharmaceutical Council in the UK. She is also a member of the British Society for Gene and Cell Therapy (BSGCT), the American Society of Gene and Cell Therapy (ASGCT) and the International Society for Cell and Gene Therapy (ISCT).
Eugenio Montini started his scientific career in 1995 in the field of human molecular and medical genetics at the Telethon Institute of Genetics and Medicine (TIGEM, Milan, Italy). Later, in 1999, he worked in the field of liver gene therapy and liver regeneration and characterised the therapeutic potential and the genomic integration events of Sleeping Beauty transposons and showed for the first time that adeno associated vectors (AAVs) integrate in the genome of hepatocytes in vivo.
Since 2009, he has been leading the safety of gene therapy and insertional mutagenesis research unit at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget, Milan, Italy), a research group aimed at unravelling the basic mechanisms governing the genotoxicity in gene therapy applications and understanding the impact of cellular senescence upon oncogene activation and identification of markers and targets for their elimination. In 2012, he established and has since directed the vector integration core in SR-Tiget, a team whose mission is to monitor clonal composition of genetically modified cells in gene therapy patients and to devise and validate novel molecular readouts for safety assessment.
His main scientific contributions include the demonstration that self-inactivating lentiviral vectors (SIN-LVs) have a superior safety profile over gamma-retroviral vectors, which has resulted in the adoption of SIN-LVs as the preferred platform for therapeutic gene delivery internationally. He has also successfully used integration site analysis to perform clonal tracking and cell lineage specification and differentiation in preclinical models and clinical trials. He has also utilised novel ad-hoc designed LV vectors to uncover novel cellular oncogenes and novel pathways involved in drug resistance in human cancers, developed a liquid biopsy integration site sequencing (LiBIS-seq), a polymerase chain reaction technique optimised to quantitatively retrieve vector (including AAV) integration sites from cell-free DNA released into the bloodstream by dying cells residing in diverse tissues, and discovered that oncogene-induced senescence in hematopoietic progenitors features myeloid restricted hematopoiesis, chronic inflammation, and histiocytosis in a humanised model.
Since his time in the Gene Therapy Research Group at Imperial College, Richard has worked on non-viral gene therapy. His PhD was focused on getting DNA into cells and even then he realised that the genetic component of these vectors was arguably more important than the methods of delivery.
Richard Harbottle’s research has subsequently focused on generating novel, next-generation DNA vectors for cell and gene cell therapy.
The DNA Vector Lab has refined and developed a novel DNA vector platform that can be used for the persistent genetic modification of dividing cells without causing molecular or genetic damage. These non-integrating non-viral vectors can be used comparably in gene and cell therapy applications to integrating vector systems such as Lentiviral and Sleeping Beauty vectors and gene editing systems such as CRISPR/CAS9; in some instances, these DNA vectors can be used where viruses and editing systems cannot. We are continuously refining these DNA Vectors by reducing the impact they have on target cells and improving their capabilities.
This improved DNA Vector design has clear ramifications for its clinical application and our recent work using it for the engineering of T Cells for immunotherapy is now one of our primary avenues of research.
Francesco Cicirello is currently director of quality assurance at Evelo Biosciences in Cambridge, MA (USA), and he enjoys helping the company bring to the market an entirely new therapeutic modality.
Prior to Evelo, he was site quality head at Adaptimmune, an ATMP company headquartered both in the UK and USA, with a gene therapy drug products with PRIME and RMAT designation.
Before joining Adaptimmune, Francesco worked in a regulatory agency, where he was an expert inspector at the Therapeutic Good Administration (TGA) in Australia. He also was elected to several roles in PIC/S (Pharmaceutical Inspection and Cooperation Scheme) including chairperson of the working group that revised the PIC/S Annex 2 and deputy chair of the subcommittee on harmonisation. He also represented PIC/S at the EMA IWG and was a member of the PIC/S coordinating committee on human blood, tissues, cells, and ATMPs. Francesco is the author of the recently published PIC/S Annex 2A and 2B, and he contributed to the revision of the latest published draft of the EU Annex 1 and the PIC/S transposition of Annex 16.
Francesco has a degree in chemistry and pharmaceutical technology from the University of Florence, an MSc in oncology pharmacology from the University of Milan, and an MSc in cosmetic production from the University of Siena (he may still remember how to make sunscreens!). He is a member of the PDA ATMP board, and of the ISPE SIG Biotech and CoP in ATMP.
Dr. Ana Sofia Coroadinha has a degree in biochemistry (University of Lisbon) and a PhD in gene therapy performed at Généthon, Helmholtz Centre for Infection Research (HZI) and iBET, granted in 2005 by ITQB-UNL. Since July 2009, she has been the head of cell line development and molecular virology lab at iBET and ITQB-NOVA.
She has over 20 years of experience in viral vectors and animal cell culture, areas where she has published over 65 peer-reviewed papers.
Ana’s group research ties virology and technology in order to find treatment solutions through the use of recombinant virus in the areas of gene therapy and vaccine development.
Dr. Roland Pach holds a PhD in molecular parasitology at the University Fribourg analyzing the intracellular trafficking of transgenic RNA in human pathogens.
Prior Roche, he was leading the Analytical Development department at Berna Biotech (former Swiss Vaccine and Serum Institute) and the QC department of Bio-Process Development at Merck Serono.
Roland is the global CMC Analytical Technical Lead in the cancer vaccines and cell- & gene therapy (CGT) area of Roche more than 10 years. In his assigned area, he represents Roche in external development projects, industrial consortiums like CGT BioPhorum and numerous due diligences of in-licensing candidates or companies in the CGT fields.
In his second role at Roche as global technical development leader, he had led successfully new formats like immunotoxins from pre-clinics into entry to human (EiH).
Amnon Eylath is a seasoned quality leader who's experienced in the complete life cycle of biological and small molecule drug development, from discovery through nonclinical and tox studies, clinical trials, process and method development, GMP manufacturing, validation, regulatory submissions, and US/EU/MHRA commercial product approvals. Amnon has direct experience with the manufacturing of biomolecules and peptides, cell and gene therapy, and with developing and troubleshooting aseptic processes, including design and fabrication of isolators and the implementation and validation of highly effective decontamination and sterilisation technology. Amnon has worked at various capacities in multinational companies, such as Amgen, Ely Lilly, and Genzyme-Sanofi, as well as at cell therapy companies such as Cellcor Therapies, Histogenics, and Minovia Therapeutics. Currently, Amnon is vice president of quality assurance at Rentschler Biopharma Inc. in Milford, Massachusetts, managing the GMP quality system, overseeing the quality and compliance of clinical and commercial biomolecule therapeutics and intermediate manufacturing, health authority inspections, client audits, and implementation of continuous improvement methods to the quality management system and related processes.
Dr. Gregory Fiore is a co-founder and is the President and CEO of Exacis Biotherapeutics. He is a pharmaceutical/biotechnology executive and entrepreneur and is a co-founder and former CEO of Sollis Therapeutics, a clinical-stage pharmaceutical company. As CEO of Sollis, he led fundraising efforts and raised up to $50M to perform a pivotal trial for the company’s lead asset.
Before this, Gregory provided senior medical support as consultant and acting CMO for several early-stage biotechnology companies through two successful consultancies he founded, SSI Strategy and Fiore Healthcare Advisors. He was the first CMO of The Medicines Company and held leadership roles at Merck and Abbott Laboratories, and previously he had been a management consultant at McKinsey and Company.
Gregory completed an internal medicine residency and received clinical pulmonary/critical care training at Harvard Medical School following graduation as class valedictorian with an MD degree from New York Medical College. He serves on several medical and business advisory committees/boards. He is on the executive board of the Institute of Neuromodulation, the scientific advisory board for Advera Health Analytics, the business advisory board for the Advanced Group of Companies, and the editorial advisory board of the industry publication Clinical Leader. He serves as an advisor to several biotechnology companies.
Shi-Jiang (John) Lu, PhD, MPH, is currently the president and CEO of HebeCell Corporation, focussing on the development and clinical translation of regenerative medicine and cell therapy technologies, especially iPS-CAR-NK cells for the treatment of cancer, autoimmune and viral infectious diseases. Before establishing HebeCell, he was the senior director of research at Advanced Cell Technology/Ocata Therapeutics, which was acquired by Astellas Pharma in 2016. John is an expert in stem cell biology and regenerative medicine with 20 years of experience.
He has been conducting translational research and discovery of novel therapeutic strategies utilising human pluripotent stem cells (PSC) and their derivatives. The goal of his research is to generate human PSC-derived products for the treatment of human diseases. He also has extensive experience in process development and large-scale production of human PSC derivatives under defined conditions for clinical trials. John is the inventor of more than 20 patents in the stem cell field: in an analysis of global stem cell patent landscape by Nature Biotechnology in 2014, John’s patent application and citation ranked No. 7 and No. 5, respectively. In addition to stem cell research, John also has more than 10 years of experiences in cancer research.
John received his BS degree in biochemistry from Wuhan University, MSc degree in oncology/pathophysiology from Peking Union Medical College, MPH degree in molecular toxicology/environmental sciences from Columbia University and PhD degree in molecular cancer biology from University of Toronto.
Dr. Li Ou is currently the vice president at Genemagic Bio, a startup focussed on AAV gene therapy for neurological and ocular diseases. Previously, Li was associate director of gene therapy at Capsida Biotherapeutics and assistant professor at the University of Minnesota, USA. Li obtained his PhD in genetics (minor in biostatistics) at the University of Minnesota. He has over 10 years of experience in leading IND-enabling studies of gene therapy (AAV, lentivirus, ZFN, CRISPR, gene regulation), resulting in two IND approvals, six pending patents, 23 manuscripts, 62 abstracts, and 23 oral presentations.
Christopher P. Locher, PhD, is CEO and co-founder of Versatope Therapeutics, a preclinical-stage Massachusetts-based company currently focussed on the development of technology platform using nano-vesicles. He previously led drug discovery projects for infectious diseases at Vertex Pharmaceuticals in Boston, inflammatory diseases at Opsona Therapeutics in Ireland, and malaria and alphaviruses vaccine development programs at Maxygen in California. He completed postdoctoral training at the University of California, San Francisco, was a Fulbright Hayes Research Fellow at the Institute of Tropical Medicine in Antwerp, Belgium, and received his PhD in tropical medicine and bachelor's in biological sciences from the University of Hawai`i.
Matthew Hewitt, PhD, currently serves as vice president, technical officer of cell and gene therapy and biologics at Charles River Laboratories. In this role, Matt leads one of Charles River’s CGT sites in helping multiple partners move assets to clinic and commercial markets. Matt is also responsible for driving many conversations on cell and gene therapy strategic approach.
Before joining the company, Matt was head of clinical development for Lonza’s personalised medicine business unit, where he was responsible for the Cocoon platform, a closed, automated, scalable cell therapy manufacturing solution that relieves many cell therapy manufacturing difficulties. He played a key role in accelerating clinical development timelines all while overseeing numerous academic and industry collaborations. These collaborations included a lead program in Israel that culminated in dosing the first patient with a cell therapy manufactured in the Cocoon. He led the R&D innovation group, which included launching the personalised medicine scientific advisory board. This board is responsible for directing current and future personalised medicine activities. In addition, Matt managed a personalised medicine analytics initiative to better understand cell therapy products, enhance clinical efficacy, and manufacturing process robustness.
Prior to Lonza, Matt led the tumour immunology and microenvironment program at Bellicum Pharmaceuticals, which focussed on improving cell therapy efficacy in solid tumours. He also led the immunology group at the University of Pennsylvania’s gene therapy program under Dr. James Wilson, leading numerous AAV gene therapy programs.
Matt received his PhD in biophysics and physiology from the University of Alabama at Birmingham and completed his post-doctoral fellowship at Johns Hopkins University.
Ales Štrancar has been the executive managing director of BIA Separations since it was founded in 1998.
Aleš is one of the main inventors of the CIM Convective Interaction Media® monolithic columns technology and co-inventor of many analytical methods including pDNA, mRNA, AAV, Adeno, and other viruses. He co-developed a number of DSP processes including pDNA for Boehringer Ingelheim and AAV for AveXis/Novartis.
Aleš is author or co-author of more than 100 scientific papers dealing with separation and purification technologies. He is a co-author of several granted patents.
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It was a well-prepared, practical review of the major issues affecting the cell and gene therapy area, with recommendations from the experts in the field of CGTPs.
This conference brought many opportunities to exchange the contacts and the recent cases in the field. Must attend for those looking to overcome the numerous challenges in the process of gene and cell therapies development and manufacturing.
The success of this summit & exhibition is in the excellent speakers board, emerging and critical topics, and networking opportunities. I will be glad to attend it again.
Thank you for your interest!