30 March -1 April 2020 | Business Design Centre London
2000+
Attendees
700+
1-2-1 Meetings
300+
Speakers
100+
Exhibitors
12
Streams
2020 KEY SPEAKERS
Gene Editing
GENE EDITING, Tuesday 31 March 2020
CONFERENCE DAY 1 - TUESDAY 31st March 2020
Opening Keynotes Day 1 – CAR-T & Immunotherapy focus
08:45
Welcome – Jessica Robinson, Project Director, Advanced Therapies 2020, Terrapinn
08:48
Chairman’s introduction
09:00
Keynote presentaton: Autologous Mesothelin-targeting CAR-T to tackle solid tumours in the clinic
- The presentation will highlight results to date of mesothelin-targeted CAR T-cell clinical trial results
- Advances in preclinical research of CAR T-cell therapy for solid tumors will be presented
- Novel strategies for overcoming hurdles to solid tumor CAR T-cell therapy will be discussed
09:20
Realizing the promise of ATMPs for patients around the world
- Working with regulators to navigate scale up challenges in manufacturing
- Strategies in supply chain and patient delivery
- Tackling post manufacturing commercialization challenges such as pricing and finding highly skilled experts
- Durability
10:00
Importance of patient focus in developing gene therapy for rare diseases
10:20
Morning Coffee Break
11:20
Keynote presentation: War in the Blood - the latest in the front line of CAR-T clinical research
11:40
Effective Integration Strategies Through a Compliance Unified Ecosystem (CUE)
- Systems Integration continues to be a major issue in the production and distribution of Advanced Therapies
- Well-defined strategies for the integration of independent informatics platforms will be critical for future standardization and workflow optimization
- Cryoport’s Compliance Unified Ecosystem (CUE) is driving standard practices for systems integration in the industry
12:00
Technology and Strategy Roundtables will run from 12:00-12:45 with 15 minutes for a quick summary from each table at the front of the room. These will take place in the conference rooms: look for you
Technology and Strategy Roundtables will run from 12:00-12:45 with 15 minutes for a quick summary from each table at the front of the room. These will take place in the conference rooms: look for your table number to participate.
1. Raw material supply for the commercial manufacturing of cell and gene therapies
Frank Hecht, Vice President Marketing And Sales, CellGenix GmbH
10. Exploring the crossover between human and veterinary applications for stem cells
Joanna Miller, Chief Scientific Officer, Cell Therapy Sciences
12. Cord blood as a source of new cells
John Wagner, Executive Medical Director Of B.M.T. Program, University of Minnesota
13. Longevity/new approaches to diseases of Ageing
Peter Hamley, Chief Scientific Officer, Samsara Therapeutics
14. Making allogeneic cell therapy a reality
Devyn Smith, Chief Operating Officer And Head Of Strategy, Sigilon
15. Industry-no profit alliances: how to optimize synergies
Elena Beltrami, Business Development Manager, Fondazione Telethon
16. Industrialisation of ATMPs
Mark Lowdell, Chief Scientific Officer, Inmunebio
17. How companies can interact with the regulator and various pitfalls to be aware of
John Johnston, Clinical Assessor, Medicines & Healthcare Products Regulatory Agency
18. Ensuring ATMP access to patients
Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
19. Roundtable available
2. Physiological conditions and analytical and monitoring tools for 3D cell cultivations
Cornelia Kasper, Professor, Biopharmaceutical Production And Technology, BOKU
20. Tackling solid tumours in the clinic
21. Neurological applications for ATMPs
John Isaac, Senior Director, External Scientific Innovation And Neuroscience, Johnson & Johnson
22. Development of regenerative medicines for cardiovascular indications
3. Ensuring safe delivery to patients and fast time-to-market of advanced therapies - How to address challenges of drug administration, fill & finish, packaging & stability?
4. Automation and continuous manufacture
Lior Raviv, Vice President Of Development, Pluristem Therapeutics
5. Considerations for manufacture scale-up from academia to commercial manufacturing
Johannes Van Der Loo, Clinical Vector Core Director, The Children's Hospital of Philadelphia
6. Defining standards for gene therapy
Jim Richardson, Senior Science And Standards Liaison, U.S. Pharmacopeia
7. Reserved for Fujifilm Diosynth Biotechnology
8. Mechanisms of MSC action and potency assays
Mary Murphy, Principle Investigator, Remedi National University of Ireland Galway
13:00
Lunch
16:20
Afternoon Break
18:00
Close of Conference for the day - Please join us for an evening drinks reception
last published: 04/Jun/20 09:25
GENE EDITING, Wednesday 1 April 2020
CONFERENCE DAY 1 - WEDNESDAY 1st April 2020
Opening Keynotes Day 2 — The latest science in gene therapy and gene editing
08:55
Opening Remarks
09:00
Gene therapy 2.0: New clinical data, therapeutic applications and next generation technologies
- Latest updates on LUXTURNA studies to treat blindness, haemophilia and rare disease
- Tackling the challenges of viral gene therapies i.e. immunogenicity
- Developing the next generation of gene therapies, what does the future hold?
09:20
Next-generation gene editing technology for allogeneic immune cell therapeutics
- Next-generation CRISPR-Cas9 technology
- Significantly enhanced editing specificity
- Editing in immune cells for generation of functionally tuned therapeutics
Amy Pooler, Director, Neuroscience, Sangamo Theraputics
09:40
Repairing the human genome with designed zinc finger editing reagents
- Overview of Sangamo’s zinc finger protein gene editing technology, including new innovations that facilitate specificity, precision, and gene editing efficiency
- Will address specific examples of defined therapeutic applications including the generation of reagents suitable for use in tauopathies, Huntington’s disease, and lysosomal storage diseases
10:00
In vivo gene editing paediatric patients with rare diseases
10:20
Networking Refreshments
GENE EDITING
Latest global developments
GENE EDITING
11:20Directed evolution of novel aden-associated viral vectors for clinical gene therapy
- Adeno-associated viral vectors are increasingly succeeding in the clinic, leading to several regulatory approvals
- Vectors based on natural versions of AAV suffer from numerous shortcomings, requiring high dosages, invasive routes of administration, and/or limited efficacy for many targets
- Our directed evolution approach involves high throughput selection of highly optimized AAVs from a library of a billion variants
GENE EDITING
11:40NIH Common Fund program on genome editing
GENE EDITING
12:00The promise of advanced therapies for 21st Century Medicines
- Novartis is re-imagining how we treat and care for patients: Innovative platforms for Advanced Therapies
- Genome editing technologies with a focus on CRISPR/Cas9: Opportunities and challenges of CRISPR as therapeutic modality
- Genome editing clinical trials: Current status
Hans-Juergen Fuelle, Vice President, Head Global Regulatory Strategy, AveXis
GENE EDITING
12:20CRISPR off-targets: how to detect and avoid them
Pinar Akcakaya, Senior Research Scientist, AstraZeneca
GENE EDITING
12:40Development of therapeutic genome editing strategies
13:00
Neworking Lunch
GENE EDITING
GENE EDITING
14:00Targeted Elimination of viral DNA using ARCUS gene editing
GENE EDITING
14:20Target optimized variants of CRISPR associated nuclease enables allele-specific gene-editing therapy
- High precision genome editing is key when developing a genetic cure for congenital diseases.
- To achieve high precision, optimization of the nuclease per target sequence is a must. Our approach to achieving high precision editing with no off-targets is to employ advanced protein engineering using directed evolution.
- The approach we present here is novel and broad; it opens up unlimited opportunities for gene editing therapy in cases of dominant negative mutations, compound heterozygous mutations and haploinsufficiency.
GENE EDITING
14:40CRISPR/Cas9 Gene Editing Therapies for Cystic Fibrosis
- Cystic fibrosis is caused by more than 200 different mutations in the CF gene, many of which are likely to remain untreatable by small molecule drugs.
- Gene editing by CRISPR/Cas9 offers many different approaches to the correction of CF gene mutations which could lead to new therapies
- The big challenge for therapeutic gene editing in CF is delivery of the therapy to the lung, the main affected organ.
- Nanoparticle delivery and cell therapy methods offer alternative delivery strategies
GENE EDITING
15:00Panel discussion: Exploring advances in gene editing technology, research and ethical questions
Marianna Romito, Field Applications Scientist, MaxCyte
Hans-Juergen Fuelle, Vice President, Head Global Regulatory Strategy, AveXis
16:00
Networking refreshments
18:00
Close of conference
last published: 04/Jun/20 09:25
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jessica.robinson@terrapinn.com
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