The gene and cell therapy markets continue to grow in 2022, although companies are struggling to overcome new CMC, analytical, process development, and manufacturing constraints. The extended collaboration around gene and cell therapy science, technologies, manufacturing, and commercialisation is accelerated.
At the #VLGeneCell event on June 8-9, 2022, the cell and gene therapy experts will focus on the recent practical experiences and perspectives towards gene and cellular therapy-related advancements, while implementing and scaling up robust cost and quality efficient process development and manufacturing, and leveraging highly appropriate emerging technologies in the field.Read more
The gene and cell therapy markets continue to grow, and gene and cell therapies clinical trials are expanding, although companies are struggling to overcome new CMC, analytical, process development, and manufacturing constraints.
What are the major challenges, opportunities and milestones facing the cell and gene therapy industry in 2022? Which steps can be taken to advance gene and cell therapies progress from preclinical to manufacturing and commercialisation? How to assure that production processes meet technical, regulatory, and cost-efficiency excellence for manufacturing safe products, and commercially viable CGTPs are delivered?
The cell and gene therapy (CGTs) developers in 2022 will continue facing unique difficulties and perspectives:
At the 4th Gene and Cell Therapy: Quality Developments, Manufacturing & Commercialization Summit on 8-9 June 2022, while addressing manufacturing controls, cell and gene therapies experts will exchange valuable insights and expectations on:
At the #VLGeneCell in June 2022, the leading experts and advisers will focus on the recent practical experiences and perspectives towards implementing and scaling up robust cost and quality efficient process development and manufacturing, leveraging highly appropriate emerging technologies in the field.
Dr. Rajiv Gangurde is chief technology officer at SparingVision, leading technical operation activities including process and analytical development, manufacturing, and quality assurance.
Rajiv has over 14 years of industry experience in biopharmaceutical development, harnessed in the biotechnology hub of Massachusetts, USA. Before joining SparingVision, Rajiv served as senior director and head of chemistry, manufacturing, and controls (CMC) at Voyager Therapeutics, a gene therapy company based in Cambridge, MA. In this role, he was responsible for CMC strategy and management of the company’s entire gene therapy portfolio. Prior to Voyager, Rajiv was director of bioprocess development and manufacturing at Genocea Biosciences, where he led upstream and downstream process development and biochemistry groups for early- and late-stage recombinant-protein-based therapeutic vaccines. Prior to Genocea, Rajiv served as head of protein sciences at LakePharma (now part of Curia), leading therapeutic protein production efforts for pre-clinical studies.
Rajiv obtained his PhD in life sciences from Mumbai University in India and did postdoctoral research at the department of biochemistry at the New Jersey Medical School, and at the department of molecular and cellular biology at Harvard University.
Rajiv is based in Massachusetts, USA.
Roberto Nitsch graduated in medical biotechnology from the University of Naples (Italy), where he also obtained his PhD in molecular genetics. He later moved to Vienna, where he focussed on mouse genetics and cancer biology, and then recessive genetics. In 2014 he shifted his research topic to genome engineering with CRISPR/Cas9 and joined AstraZeneca, where he was responsible for the CRISPR mouse models for drug discovery and oncology. Since 2017, he has been associate director in the clinical pharmacology and safety sciences pioneering CRISPR safety assessments. Today, Roberto is the director of the gene therapy at AstraZeneca, and he is working towards the generation of safer CRISPR medicines.
Margit Jeschke recently transitioned from Novartis cell and gene technical development and manufacturing to become an independent consultant for ATMP CMC aspects. At Novartis, she was responsible for the analytical development strategy of the company’s CGT products including overall product control strategy, specification setting, comparability studies, as well as CMC correlational sciences. She was leading the team responsible for the analytical parts of CAR-T regulatory filings (BLA, MAA, post-approval changes, etc.). Margit joined CGT in 2012 after holding several positions of increasing responsibility within Novartis biopharmaceutical development, including leading analytical research and development of Novartis biologics in Basel, Switzerland. Margit holds a PhD in biochemistry from the University of Basel.
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).
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.
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.
Thea Ziegler studied biotechnology and graduated with her PhD in biochemistry in Berlin and Bayreuth. During her PhD project she used NGS to screen for new binding motifs of a bacterial transcription factor for further use in bioengineering contexts. After a brief postdoctoral period, she moved to the life sciences industry supporting Sanger sequencing, NGS, and RT-PCR applications. She now supports various HiFi applications as a sequencing application specialist at PacBio.
Ashley Winslow is president and chief scientific officer of Odylia Therapeutics, a non-profit biotech focussed on developing gene therapies for rare diseases. Odylia develops therapeutics in collaboration with patient groups and a strategic network of CROs, academic partners, and industry, with the aim of bringing life-changing therapeutics to patients.
Ashley received her PhD in medical genetics from the University of Cambridge and completed her postdoctoral work at Massachusetts General Hospital and Harvard Medical School. Before joining Odylia, Ashley worked in the precision medicine and human genetics and computational biomedicine group at Pfizer, and the Orphan Disease Center at the University of Pennsylvania. She has experience overseeing drug development from target discovery to early-phase clinical programs and brings to Odylia more than 15 years of drug development experience in academic, industry, and non-profit sectors. Ashley serves on the International Angelman Syndrome Research Council (INSYNC-AS) and the CHAMP1 scientific advisory board.
Matthew Li is a biomedical scientist and engineer with over 13 years of experience across an array of areas in the biotech field including CGT R&D and manufacturing, translational medical device development, and clinical research.
He is currently a director at Tome Biosciences working on CMC strategies. He has held roles at Vor Biopharma and Takeda, engaging across the CGT value chain interfacing with research, development, manufacturing, quality, regulatory, clinical, business development, and commercial taking an integrative approach to address challenges across the life cycle of a therapy. Matthew holds a PhD in medical engineering and medical physics from the joint health sciences and technology program of Massachusetts Institute of Technology and Harvard Medical School.
Stefano Baila received his PhD in 2007 based upon translational research and development of gene therapies for hemophilia at the Children’s Hospital of Philadelphia. Since that time he has been actively involved in the process development and manufacturing of advanced therapeutic medicinal products through business development and strategic marketing roles at Areta International, a CDMO, and by leading field implementation and commercialization activities for the cell processing unit of Terumo BCT. Stefano also worked as Industrialization Manager at Celyad where he led process development and automation efforts for CAR-T. Now he serves as Director of Operation and BD for Anemocyte
Dr. Sullivan is program manager and international liaison officer for the Global Alliance for iPSC Therapies (GAiT).
GAiT works to establish an HLA-homozygous haplobank network for clinical-grade-induced pluripotent stem cell (iPSC) lines [Barry et al. 2015, Sullivan et al. 2018]. Such cells would represent an appropriate starting material for allogeneic therapeutic development worldwide.
GAiT activity is funded by an international consortium of organisations: the French Institute of Health and Medical Research, INSERM (Paris, France), the University of Hong Kong (Hong Kong, Hong Kong), the Cell and Gene Therapy Catapult (London, UK), the Centre for Commercialisation of Regenerative Medicine (Toronto, Canada), the Korea HLA-Typed iPSC Banking Initiative (Seoul, Korea), and the New York Stem Cell Foundation (New York, USA). More information on GAiT and its sponsors is available at www.gait.global.
Stephen initially earned his PhD at the Roslin Institute (Edinburgh) under Professor Ian Wilmut and Dr. Jim McWhir, becoming one of the first researchers in Europe to culture human embryonic stem cells. His work focussed on pluripotency induction in human somatic cells.
Thereafter, Stephen worked as a research fellow at the University of Cambridge, Harvard University, and UCSD, deepening his expertise of other human stem-cell types (including induced pluripotent stem cells) and their medical applications.
While at Harvard, he read Dr. Paula Stefan's "How Economics Shapes Science", which stimulated his interest in the financial, as well as the scientific and clinical, hurdles that must be surmounted to bring stem-cell therapies to the clinic. Thereafter, he also worked for Novartis, dealing with clinical trial management and compliance, to better understand manufacturing, logistic, and regulatory challenges to bringing new therapeutics to market and earned an MBA at Trinity College Dublin focussing on risk mitigation during AMTP development.
Prior to his current position, Stephen was the chief scientific officer of the Irish Stem Cell Foundation, where, working with others, he facilitated the introduction of stem-cell legislation into the Irish Program for Government. He was also a project support officer for the Scottish National Blood Transfusion Service (2017-21). In March 2021, he founded Lindville Bio, an ATMP consultancy service specialising in scalable manufacture of pluripotent stem cells (PSC) and therapeutic development therefrom.
He is also a member of the steering committee for the International Stem Cell Banking Initiative (ISCBI), the Autologous iPSC Therapy Consortium, the Human Pluripotent Stem Cell Advisory Committee at University College Cork, Ireland, and is chief editor of "Human Embryonic Stem Cells – The Practical Handbook" published by Wiley & Sons.
Nick is an experienced cell and gene therapy executive, innovator, and people leader. He has over 15 years' experience developing an assortment cell and gene therapies and associated technologies. Currently chief development officer at Artisan Bio, he previously held leadership roles at BlueRock Therapeutics, the Center for Commercialization of Regenerative Medicine (CCRM), and the Center for Advanced Therapeutic Cell Technologies. Nick's global experience includes time living and working in Canada, Switzerland, Australia, and New Zealand, engaging with a diverse assortment of partners, colleagues, and clients from industry and academia.
Abhishek Srivastava is vice president of cell therapy development at Athenex, overseeing early development, manufacturing, and technical operations of the autologous and allogeneic cell therapy programs. He has extensive clinical and translational research experience in the field of adoptive cell therapies serving both academia and industry. Most recently, he was an assistant professor (research) at the division of surgical oncology at the University of Pittsburgh, where he led the process development and manufacturing of tumor infiltrating lymphocytes (TIL) to support the multiple adoptive cell therapy Phase II clinical trials. He received his postdoctoral training from surgery branch at the National Cancer Institute and earned his PhD in immunology. He has authored numerous papers including research articles, abstracts, book chapter, and expert opinions.
Dr. Lara Ionescu Silverman has a PhD in bioengineering from the University of Pennsylvania and an undergraduate degree in chemical engineering from Princeton University. She spent 10 years as the head scientist at an allogeneic cell therapy company developing a product to treat low back pain. She has experience managing R&D, manufacturing, clinical trials, process and method development, and intellectual property portfolios. She is the author of numerous peer-reviewed papers and the inventor of many patents. Currently, she owns her own consulting business where she provides advisory services to early-stage cell therapy organisations, helping them navigate the pathway to IND.
Isabelle Gennart is a biologist by training. She earned her PhD in 2018 at the University of Namur (Belgium), where she focussed on cancer development mechanisms using viral-induced lymphoma models in avian species. Since then, she has had experience with various CAR-T development and clinical manufacturing projects working as a QC specialist at Catalent. She joined Celyad Oncology in 2020 within the R&D team to setup new analytical tools to support innovative CAR-T cells product development and characterisation.
Hemant Dhamne obtained his PhD from the ACTREC, Mumbai (2014) in applied virology. His PhD thesis was on the lentiviral vector mediate long-term expression of therapeutic proteins. He completed his doctoral work in the labs of Dr. Robin Mukhopadhyaya and Dr. Rajiv Kalraiya. His doctoral studies mainly focussed on developing the novel LV platform using the inhouse HIV-1 derived vector system and its application for cell engineering and tumour targeting in pre-clinical models. From 2014-15, he was a postdoctoral fellow in the laboratory of Dr. Amit Dutt in the same institute, working on functional genomics aspects spanning novel miRNA discovery and novel mutations in cancer-causing genes and associated pathways. Thereafter, he joined the gene therapy division at Intas Pharmaceuticals, Ahmedabad. He worked on early-stage product development of AAV-mediated gene therapy for diseases such as hemophilia, neuronal, and eye disorders. He successfully developed the pilot-scale manufacturing platforms using suspension cell lines in upstream and chromatography in downstream processes. He also contributed to assay development. He served as research scientist from 2015-17 and as a senior research scientist from 2017-19. He also developed early-stage designs and assays for CAR-T research.
From 2018-19, he completed a diploma in international business management from Ahmedabad Management Association in affiliation with California State University. In 2019, he joined Immuneel Therapeutics as head of vector and CAR-T manufacturing. His work encompasses the process development and GMP-grade manufacturing of autologous CAR-T cells for clinical trials. The disease areas mainly include leukemias and lymphomas.
He has seven peer-reviewed publications in the field of gene therapy and functional genomics.
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The conference was extremely well-organized! Thank you the Vonlanthen group for the efforts they take to bring on board the leading experts, who explore from practical perspectives the hottest topics in gene and cell therapies.
It has been a great event and it was lovely to be back in person.
I appreciated the conference a lot and especially liked the mixture of scientific talks with updates on the current technical development.
I appreciated the conference a lot and especially liked the mixture of scientific talks with updates on the current technical development.
Jun 6, 2019
Gene and Cell Therapy: Quality Developments to Commercialization Summit