Gene Editing

30 March -1 April 2020 | Business Design Centre London

 

 

2000+
Attendees
700+
1-2-1 Meetings
300+
Speakers
100+
Exhibitors
12
Streams

 

 

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

Prasad Adusumilli
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
Panel discussion
Bradley Campbell
10:00

Importance of patient focus in developing gene therapy for rare diseases

10:20

Morning Coffee Break

Martin Pule
11:20

Keynote presentation: War in the Blood - the latest in the front line of CAR-T clinical research

Mark Sawicki
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
round tables
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

Frank Hecht, Vice President Marketing And Sales, CellGenix GmbH

10. Exploring the crossover between human and veterinary applications for stem cells
Joanna Miller

Joanna Miller, Chief Scientific Officer, Cell Therapy Sciences

15. Industry-no profit alliances: how to optimize synergies
Elena Beltrami

Elena Beltrami, Business Development Manager, Fondazione Telethon

16. Industrialisation of ATMPs
Mark Lowdell

Mark Lowdell, Chief Scientific Officer, Inmunebio

17. How companies can interact with the regulator and various pitfalls to be aware of
John Johnston

John Johnston, Clinical Assessor, Medicines & Healthcare Products Regulatory Agency

19. Roundtable available
2. Physiological conditions and analytical and monitoring tools for 3D cell cultivations
Cornelia Kasper

Cornelia Kasper, Professor, Biopharmaceutical Production And Technology, BOKU

21. Neurological applications for ATMPs
John Isaac

John Isaac, Senior Director, External Scientific Innovation And Neuroscience, Johnson & Johnson

22. Development of regenerative medicines for cardiovascular indications
David Mazzo

David Mazzo, Chief Executive Officer, Caladrius Biosciences

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?
Diana Lober

Diana Lober, Product Manager, Schott AG

5. Considerations for manufacture scale-up from academia to commercial manufacturing
Johannes Van Der Loo

Johannes Van Der Loo, Clinical Vector Core Director, The Children's Hospital of Philadelphia

7. Reserved for Fujifilm Diosynth Biotechnology
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

Federico Mingozzi
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?
Steve Kanner
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
Frederic Chereau
10:00

In vivo gene editing paediatric patients with rare diseases

10:20

Networking Refreshments

GENE EDITING

Latest global developments

David Schaffer
GENE EDITING
11:20

Directed 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
Philip John Brooks
GENE EDITING
11:40

NIH Common Fund program on genome editing

Hans-Juergen Fuelle
GENE EDITING
12:00

The 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
Pinar Akcakaya
GENE EDITING
12:20

CRISPR off-targets: how to detect and avoid them

Pinar Akcakaya, Senior Research Scientist, AstraZeneca
Soekjoong Kim
GENE EDITING
12:40

Development of therapeutic genome editing strategies

13:00

Neworking Lunch

GENE EDITING

Dave C Morris
GENE EDITING
14:00

Targeted Elimination of viral DNA using ARCUS gene editing

Asael Herman
GENE EDITING
14:20

Target 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.
Stephen Hart
GENE EDITING
14:40

CRISPR/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
Marianna Romito
16:00

Networking refreshments

18:00

Close of conference

last published: 04/Jun/20 09:25