Agenda
 

Virtual, 19 - 21 May 2021

Schedule

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May 1910:50
Conference pass

Welcome, introduction, housekeeping

Keynotes
Jessica Robinson, Project director, Terrapinn Ltd
May 1911:00
Conference pass

Clinical Progress & Global Competition in BCMA-targeted Immunotherapy

Keynotes
  • Multiple myeloma remain as an “incurable” cancer?
  • Clinical progress of CAR-T therapy in multiple myeloma
  • Competitive landscape of BCMA targeted therapy in early-stage commercial development
May 1911:30
Conference pass

Keynote panel discussion: State of the industry panel

Keynotes
  • How have events over the last 12 months affected the industry? Maintaining
  • Where is the ATMP field headed?
  • Who is making the best progress?
  • What is new on the market?
  • The Next Wave: moving beyond CAR-T and Gene Therapy, what’s next?
Vivienne Parry, Head Of Engagement, Genomics England
Dick Sundh, Vice President, Marketing & Head of Australia, Canada and Europe (ACE),, Kite Pharma, a Gilead Company
Pascal Touchon, President, Chief Executive Officer And Board Member, Atara Biotherapeutics
May 1912:00
Conference pass

Keynote panel discussion: How to ensure clinical success

Keynotes
  • Perfecting the art of regulatory submissions from pre-IND to final registration
  • How can we avoid idiosyncratic clinical responses to ATMPs?
  • Renewed guidance: are regulatory agencies changing how they view these approvals?
  • Safety and clinical considerations:Robust, novel pre-clinical models and standardised assaysWhat in vitro tests and animal models can you use to minimise clinical risk?What can you really extrapolate from preclinical investigation to humans?How much should you expand cells? Is a high or low cell dose important?Pharmacogenetics, biodistribution, toxicology.
  • Justification for routes of administration: what do the regulators think about delivery methods?
May 1912:30
Conference pass

Roundtable with the regulators: Ensuring clinical success

Keynotes
  • How do we move ahead?
  • Why is it important?
  • What’s the case to be made?
  • Why is it important that we get there?
  • Are there things we can do?
  • Smaller vs larger patient populations
May 1913:30
Conference pass

Panel discussion: Rise of allogeneic cells and allo-approaches to oncology & non-oncology

Cell Therapy
·The cell types·Impact of gene editing·Cellular Persistence·Cellular phenotypes·Expansion In vivo·Possibility of retreatment·Lymphodepletion·Administration of the cells/ Amount of the cells/Local and systemic administration
Stefanos Theoharis, Chief Business Officer, Bone Therapeutics
Pascal Touchon, President, Chief Executive Officer And Board Member, Atara Biotherapeutics
Patrick J Hanley, Director, Cellular Therapy Laboratory, Children's National Medical Center
May 1913:30
Conference pass

CAR T-cell combination immunotherapy for solid tumors

Gene Modified Cell Therapy
·Discuss preclinical strategies for combination immunotherapy with CAR T cells·Highlight ongoing clinical trials of combination immunotherapy·Outline next-gen combination therapy strategies
May 1913:30
Conference pass

Technologizing ATMP Processes – Efficient methods to enhance NK functionality

Manufacturing
·Are NK-Cells really so shear sensitive, that they can survive stirred/shaked conditions?·Can we treat NK to adapt to defined media to cut the dependency to the supply chain and to reduce costs?·Is a change in metabolism due to different growth conditions also effecting functionality?
May 1913:30
Conference pass

Providing a novel off-the-shelf implant to repair damaged organs

Innovation
(1st Indication: Oesophagus)
Derek Dashti, Founder And Chief Executive Officer, D and P Bioinnovations
May 1914:00
Conference pass

Panel: The next generation of cell therapies

Cell Therapy
  • Revisiting MSCs, what have we learned?
  • Repurposed tools and modified cells
  • Sourcing clinical grade iPSCs
Miguel Forte, Chief Executive Officer, Bone Therapeutics
Hardy Tadahisa S Kagimoto, President And Chief Executive Officer, Healios K. K.
Frank Barry, Scientific Director, Regenerative Medicine Institute
May 1914:00
Conference pass

Gamma delta T-Cells: Potential off the shelf treatment for solid tumours

Gene Modified Cell Therapy
·Circulating gamma delta T-cells expand more efficiently in the presence of TGF beta·These cells also exhibit superior fitness and resistance to cell death·TGFb supplemented gamma delta T-cells display enhanced anti-tumour activity, either when unmodified or genetically targeted using a CAR
May 1914:00
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Workshop: Automation, exploring new technologies

Manufacturing
·Poll·Panel: How are people using these products? How can they get maximum efficiency? Is there some way of using these machines more optimally?·Roundtable
Robin Ng, Strategy Lead, Cellular Biomedicine Group
Farid Ighemat, Senior Director Cell and Gene Therapy, GlaxoSmithKline
May 1914:00
Conference pass

Multimodal imaging for quantitative, longitudinal cell tracking in the clinic

Innovation
Mangala Srinivas, CSO, Cenya Imaging; Chair Group Leader,, Wageningen University and Research Centre
May 1914:30
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Roundtable: neurological & ophthalmic applications for ATMPs

Cell Therapy
May 1914:30
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New real-world data: better expansion, PERSISTANCE and disease control of CAR-T

Gene Modified Cell Therapy
May 1914:30
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When Rapid Results Matter: The value of QC testing in ATMP

Manufacturing
·Improving the time to result of QC testing with rapid and automated solutions to reduce patient to patient time·Advance safety testing of ATMP from raw materials to product release·Bringing flow cytometry analysis to point-of-need
May 1914:30
Conference pass

Using Myeloid Cells to tackle solid tumours

Innovation
May 1915:00
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Caladrius Biosciences; Progress, challenges and learnings during the COVID-19 pandemic

Cell Therapy
·CD34+ cell therapy platform yielding a multi-product development pipeline with 2 clinical programs having regenerative medicine “breakthrough” designation·Multiple potential value creating events in the next 12-24 months based on milestones across the pipeline·Strong balance sheet; ~$116 million in cash & investments (2/25/2021) with no debt and cash runway projected to fund operations for several years
May 1915:00
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CAR-Macrophages - a novel approach to treating solid tumors

Gene Modified Cell Therapy
Michael Klichinsky, Co-Founder, Vice President Of Discovery And Research, Carisma Therapeutics
May 1915:00
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Establishing a process intensification and adaptive manufacturing strategy for CAR-T production

Manufacturing
·Establishing a process control strategy facilitates process intensification, increasing yield and efficiency·Automated strategies for process and product development enable increased consistency·Improved control allows for the potential of patient-specific adaptive manufacturing
May 1915:00
Conference pass

Streamlining digital transformation and reducing data and process silos in advanced therapeutics

Innovation
·Reducing time to market·Reducing the cost of developing·Ease of scaling and distribution post-FDA approval
Vasu Rangadass, President CEO, L7 Informatics
May 1915:30
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Panel: Tackling solid tumours

Gene Modified Cell Therapy
·Hurdles to solid tumour cell therapy and novel strategies to overcome·Autologous and allogeneic cell therapy for solid tumours·Potential role for combination cellular therapies
Michael Klichinsky, Co-Founder, Vice President Of Discovery And Research, Carisma Therapeutics
May 1915:30
Conference pass

Journey to the Center of the Cell Therapy – Manufacturing and Analytics

Manufacturing
  • Manufacturability and sustainability
  • Beyond Development: today and tomorrow trends
  • Real live case studies
Stefano Baila, director of operations, Anemocyte
May 1915:30
Conference pass

CAR-T model for gamma-delta T cell therapy for GBM

Innovation
Lei Ding, Lead Scientist, in8bio
May 1916:00
Conference pass

Update on Healios K.K.

Cell Therapy
Hardy Tadahisa S Kagimoto, President And Chief Executive Officer, Healios K. K.
May 1916:00
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Workshop: Upscaling cell therapy manufacturing

Manufacturing
·Deciding on which bioreactor is best for large volumes, how to freeze large batches of cells at a time, etc.
May 1916:00
Conference pass

The latest advances on a monitoring system integrated bioreactor for 3D cell culture

Innovation
May 1916:30
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Cord blood and cord tissue derived cellular therapies for pediatric brain diseases

Cord Blood
·Discuss differences between cord blood and cord tissue cells and their roles in potential treatment of pediatric brain injuries.·Update results of ongoing studies of cord blood therapies in children with cerebral palsy and autism·Update results of ongoing studies with cord tissue MSCs in children with autism, babies with HIE
Joanne Kurtzberg, Professor Of Pediatrics And Pathology; Director, Marcus Center For Cellular Cures,, Duke University Medical Center
May 1916:30
Conference pass
May 1916:30
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Workshop: product and process characterization and validation

Manufacturing
·Choosing a relevant assay for a certain product, discussing what surrogate markers to go for, pros and cons with those, measurability of output data and sensitivity of the assay·How to handle a change of production system, what does it take from a regulatory point of view to justify that it is still the same product?·Set up of stability studies and stress tests of drug product
May 1916:30
Conference pass

Cell therapy in children’s renal disease

Innovation
May 1916:45
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Cord Blood Derived Immune Effector Cells

Cord Blood
John Wagner, Executive Medical Director Of B.M.T. Program, University of Minnesota
May 1917:00
Conference pass

Roundtable: The future of cord blood use. Business models, new uses for CB, ethics of pioneering new trials, updating the delivery room

Cord Blood
·A poll will be taken to learn more about the people who are interested in CB, to learn about their activities in the CB field, and we will show the statistical results in the event and even discuss them
Marcie Finney, Associate Director, Cleveland Cord Blood Center
John Wagner, Executive Medical Director Of B.M.T. Program, University of Minnesota
Joanne Kurtzberg, Professor Of Pediatrics And Pathology; Director, Marcus Center For Cellular Cures,, Duke University Medical Center
May 1917:00
Conference pass

Development of SARS-CoV-2-Specific T cells for the treatment and prevention of infection in immune compromised patients

Gene Modified Cell Therapy
Susan Conway, Critical Care Specialist, Children's National Medical Center
May 1917:00
Conference pass

Roundtable: Suspension, perfusion and continuous processing

Manufacturing
May 1917:00
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Generating a patient specific thymus

Innovation
·Developing a thymus ex vivo for transplant
May 1917:30
Conference pass

Immunotherapy keynote: Precision medicine tools have played a role in establishing PD-1 antibodies as foundational monotherapy for- and have informed combination therapies for cancer

Keynotes
  • PD-1 Antibodies have become foundational as monotherapy in a number of cancers and new data continue to accumulate
  • Precision Medicine Tools enable patient selection and provide information regarding resistance biology
  • This information informs on combination therapy selections and an expanding number of these are further transforming cancer treatment

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May 2012:00
Conference pass

Keynote panel discussion: Where are we with COVID now?

Keynotes
·There are 70+ CT clinical trials registered for COVID and ARDS related to COVID·Round up of COVID & ARDs related CT clinical trials·What are the short term and long-term effects of COVID on the industry going forward?·Head to head comparison of data from key players·Regulatory standpoint on cell therapies for COVID ARDs
Susan Conway, Critical Care Specialist, Children's National Medical Center
Hardy Tadahisa S Kagimoto, President And Chief Executive Officer, Healios K. K.
May 2012:30
Conference pass

Advanced Therapies: development and regulation, from an EU point of view

Keynotes
Klaus Cichutek, President, Paul Ehrlich Institute
May 2014:00
Conference pass

Panel discussion: Immunogenicity for gene therapy

Gene Therapy
Key unresolved clinical issues in gene therapy: immunosuppression, re-dosing, and in-utero dosing
May 2014:00
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Beyond “one disease at a time” : Increasing efficiency in rare genetic disease therapeutics development

Rare disease
May 2014:00
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Workshop: In house vs outsourcing manufacturing

Manufacturing
·AAV materials made by different platforms and what difference will this make in the clinic?·Why is it better?
May 2014:00
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Episomal gene transfer for gene therapy

Innovation
·The Mitotic Stabilityof the episomes within the host cell·The Deliveryissue of non-viral episomes into cells
Aristides D Tagalakis, Senior Lecturer, Edge Hill University
May 2014:30
Conference pass

Latest in rare/orphan drug research

Rare disease
Graciana Diez-Roux, Chief Scientific Officer, Telethon Institute of Genetics and Medicine
May 2014:30
Conference pass

Panel: Analytics: having the right toolbox to go forward to commercial scale

Manufacturing
·Capsids: full vs empty vs partially filled
May 2014:30
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Update on Phase 1 study of AGT103-T: Cell and gene therapy for HIV disease

Innovation
·Implementing a GMP manufacturing process·Product characteristics including new product identity profiles·Real world results on cell yields and potency
May 2015:00
Conference pass
May 2015:00
Conference pass

Roundtable: Translating the technique of gene editing into new treatments

Rare disease
May 2015:00
Conference pass

Using Plasticell combinatorial screening technology for optimisation of gene therapy for rare diseases

Innovation
May 2015:30
Conference pass

Roundtable: Moving from ultra-orphan to more common diseases

Gene Therapy
May 2015:30
Conference pass

Development of a Manufacturing Platform Process for Multiple AAV Gene Therapy Vectors

Manufacturing
·Leveraging platform based approaches to accelerate the development of cGMP compliant processes for manufacturing viral gene therapy products from pre-clinical stages to the clinic·Establishment of a comprehensive, end-to-end service offering, including HEK293 suspension master cell bank, cGMP plasmid process development and manufacturing capabilities, and drug substance / drug product manufacture·Flexible approaches to producing other viral vectors to support cell & gene therapy and vaccine products
May 2015:30
Conference pass

Avoiding the endosomal trap through membrane fusion: Efficient delivery of CRISPR protein systems in vitro and in vivo

Innovation
May 2016:00
Conference pass

CRISPR’s Progress for Patients: Clinical Development of Therapies

Gene Editing
May 2016:00
Conference pass

New technologies related to engineering AAV production systems and engineering enhanced promoters for transgene expression

Manufacturing
May 2016:15
Conference pass

Development of ARCUS-based Allogeneic CAR-T Cell Therapies

Gene Editing
·Discussing patient conditioning without biologic immunodepletion·Outlining dosing considerations·Evaluating cell expansion, persistence, and durability of response
Christopher R Heery, Chief Medical Officer, Precision BioSciences
May 2016:30
Conference pass

Q&A panel: what’s next in ex-vivo gene therapy and gene editing?

Gene Editing
Sven Kili, CEO, Antion Biosciences, Principal, Sven Kili Consulting
Pascal Touchon, President, Chief Executive Officer And Board Member, Atara Biotherapeutics
May 2016:30
Conference pass
May 2016:30
Conference pass

Innovations in immune monitoring technologies to monitor the immune system for next generation ATMPs

Innovation
May 2017:00
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Bioethics discussion: Silencing the extra chromosome in trisomy 21: Bioethics of eliminating Down’s syndrome at a cellular level

Gene Editing
·Current state of experimental procedures·Potential Impacts on affected individuals, family and society·Ethical frameworks to consider

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May 2111:30
Conference pass

Keynote panel discussion: Tackling large-scale, commercial regulatory

Keynotes
·Regulatory harmonisation issues·Covering multiple regions·Aligning regulatory approvals·State of affairs: a lot of modernisation
May 2112:00
Conference pass

Keynote panel discussion: Silver linings from COVID-19, reshaping a better future from the pandemic

Keynotes
Consequences of COVID19 on the advanced therapies industry·Short term, reactive:oclinical trials and supply chainoImpact on businesses: need to resolve impact on finances and stock·Learnings, long term positive effects:oTechnology advancesoRegulatoryoEventsoGM organisations & legislation regulators lifted·What can we implement as a positive consequence?·Culture/evolutions of companies, how is working from home effecting how we work?
May 2112:30
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Strengthening the chain of custody data stream in the cell and gene therapy supply chain

Keynotes
·Services and systems integration through consolidation and strategic partnership·Implementation of process and data integration·Optimization of supply chain workflow
May 2113:00
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Keynote panel discussion: Finding the right framework for reimbursement and market access for cell and gene therapies

Keynotes
·Europe vs USA vs ROW?·Reimbursement strategies·What’s driving the decision makers at country and national level?
Luc Boileau, Chairman And Chief Executive Officer, INESSS
Sheela Upadhyaya, Accelerated Access Collaborative Relationship & Delivery Lead & HST Specialist, National Institute for Health and Care Excellence
May 2113:30
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Panel: Patient Delivery in the COVID environment

Patient delivery including logistics, transportation & distribution
Kris Elverum, Senior Vice President Of Business Development And Strategy, Rubius Therapeutics
Jim Kovach, Director, Innovation
May 2113:30
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Regulatory session for academic and early-stage companies: Regulatory submissions: gathering the evidence

Innovation
Margareth Jorvid, Head of Regulatory Affairs and QA, Immunicum
Nneka Onwudiwe, Founder And Chief Executive Officer, Pharmaceutical Economics Consultants of America (PECA) LLC
May 2114:00
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Roundtable: Hospital exemption vs in-house preparation

Pricing & Market Access
May 2114:00
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Partnering with big pharma: potential strategic and manufacturing alliance

Commercialisation
May 2114:00
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Roundtable: How and when you should talk to the regulators

Innovation
·Rules vs expectations·Examples·‘Interact scheme’ FDA – est. 2018
May 2114:30
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May 2114:30
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Roundtable: Business development strategies

Commercialisation
May 2115:00
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Developing an Ideal CAR-T Patient Experience

Patient delivery including logistics, transportation & distribution
  • Significant opportunities exist to improve the patient experience centered around patient information sharing
  • Leveraging Human Centered Design can dramatically and positively impact the patient experience
  • Through this process not only was the patient experience improved, but staff experience was also improved as was efficiency and teamwork
Allison Matthews, Service Designer, Mayo Clinic Center for Innovation
May 2115:00
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Cell and Gene therapy standardisation

Innovation
May 2115:30
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Roundtable: All the things you were too afraid to ask about funding a new technology or financing a new company

Investment
May 2115:30
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Looking at value for advanced therapies in Quebec

Commercialisation
Michele de Guise, Scientific Vice-President, INESSS
May 2115:30
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The need to Create a collaborative network to provide a framework for hiPSC generation of hiPSC homozygous for frequent HLA haplotypes

Innovation
May 2116:00
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Perspectives on the ATMP Regulatory landscape: approaches to harmonisation and identifying early translational issues

Pricing & Market Access
  • Introduction to the Accelerating Research & Development for Advanced Therapies (ARDAT) project
  • Regulatory issues encountered during early stage ATMP development in small biotechs and SMEs
  • Future scope and tools for regulatory harmonisation and improving ATMP development
Christopher Mann, Scientific And Regulatory Affairs Director, Asphalion, S.L.
May 2116:00
Conference pass

Roundtable: Tackling the challenges of getting to commercial level

Commercialisation
·How do you differentiate yourself from the competition in a clinical and commercial setting?
May 2116:30
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Panel: VC financing during COVID

Investment
·Money is flowing freely·What happens once these funds are paid out?·What drove them to high valuations? (without having met in person, over zoom)
May 2116:30
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Workshop: Financing, innovation & company creation

Commercialisation
·How to scale up your business to a commercial level
Kris Elverum, Senior Vice President Of Business Development And Strategy, Rubius Therapeutics
Pierre-Albert Colcomb, Genethon
May 2116:30
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Workshop: New commercial products: moving from clinical to commercial

Patient delivery including logistics, transportation & distribution
·Interacting with pharma companies (i.e. how to administer X)·Moving from bags to vialsoPreparing the dose on-site, how to calculate the correct dosage all the way to patient delivery·POC/commercialisation·Moving from proprietary -> streamlining·Allogeneic:oDosage/amountoDispensing as a pharmacyoVerification process
May 2116:30
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Attracting, Retaining & Training Talent

Innovation
Where are the skills shortages? What can be done to train the next generation of talent to minimise the shortage of experts in this area and advance the collective area of advanced therapies for patient benefit?
May 2117:00
Conference pass

Quell Therapeutics Case Study - Building and funding the journey to develop an engineered Treg cell therapy platform

Investment
May 2117:00
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Tech Transfer - Driving Translation from Academia to Industry

Commercialisation
·The effectiveness of the Gates Grubstake Program to advance regenerative medicine therapies to the clinic & industry
May 2117:00
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Workshop: Bedside delivery of cell therapy nursing perspective

Patient delivery including logistics, transportation & distribution
·Screening patients·Infusion related issues and how to avoid them·Avoiding neurotoxicity·Monitoring after cell infusion·How to spot signs of complications·Finding the right medication·Working with other departments
Matthew J Frigault, Clinical Director, Cellular Immunotherapy Program, Massachusetts General Hospital
Sara Geary Alexander, Clinical Nurse Specialist, Massachusetts General Hospital
Kelly Foster, Moffitt Cancer Center
Alix Beaupierre, Transplant and Cellular Therapy Nurse Coordinator, Moffitt Cancer Center
May 2117:00
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Gates Biomanufacturing Facility: The Experience of Building a Multi-product Cell Therapy/Biologics Phase I cGMP Facility

Innovation
Matthew Seefeldt, Executive Director, The Charles Gates Biomanufacturing Facility
last published: 15/Apr/21 11:05 GMT