Gene Therapy

30 March -1 April 2020 | Business Design Centre London

 

 

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

 

 

Gene Therapy

GENE THERAPY, 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

GENE THERAPY

New techniques and technologies

Bobby Gaspar
GENE THERAPY
14:20

Haematopoietic stem cell gene therapy for severe monogenic disorders

Bobby Gaspar, Professor Of Paediatrics And Immunology, UCL Great Ormond Street Institute of Child Health
Tanner Dockendorf
GENE THERAPY
14:40

Title TBC (GENE THERAPY)

Eric Michael David
GENE THERAPY
15:00

Novel approaches to building gene therapy programs: Lessons from BridgeBio

  • Asset identification
  • What to build and what to outsource
  • Putting patients first
Joery De Kock
GENE THERAPY
15:40

Reaching beyond the limits of nature to cure inherited metabolic disorders of the liver

Gerard Short
GENE THERAPY
16:00

AAVS3 gene therapy aimed at normalising Factor IX levels in patients with severe or moderately severe haemophilia B

16:20

Afternoon Break

Simon Waddington
GENE THERAPY
17:00

Developing gene therapy for genetic neurological disease in children

18:00

Close of Conference for the day - Please join us for an evening drinks reception

last published: 04/Jun/20 09:25

GENE THERAPY, 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 THERAPY

Clinical focus

Ted Tuddenham
GENE THERAPY
11:40

Latest data from haemophilia gene therapy trials

Graciana Diez-Roux
GENE THERAPY
12:00

Case study: TIGEM’s gene therapy projects

Angela Columbano
GENE THERAPY
12:20

Update on Genethon’s pipeline with fresh clinical developments

Erik Richardson
GENE THERAPY
12:40

A virtual model for gene therapy R&D to target the complement system

  • Introduction to Apellis: History, APL-2 and gene therapy pipeline expansion.
  • Components and operation of a virtual gene therapy network.
  • Ongoing gene therapy R&D at Apellis
13:00

Neworking Lunch

GENE THERAPY

Elena Piletska
GENE THERAPY
14:00

Solving problems with AAV neutralisation in gene therapy

Paul Gissen
GENE THERAPY
14:20

Developing Liver directed gene therapy for paediatric indications

  • Acute unmet need in paediatric liver based disorders
  • New approaches are needed to provide long-term efficacy
  • Challenges in translation of laboratory discoveries into clinical practice
Florence Allouche
GENE THERAPY
14:40

Saving Cones for saving vision; RdCVF a mutation-agnostic treatment in Retinitis Pigmentosa

Florence Allouche, Associate Professor, Faculty Of Pharmacy, University Of Paris; President And Chief Executive Officer, Sparingvision
Gabriel Brooks
GENE THERAPY
15:00

Leveraging industralized directed evolution for tissue targeted AAV capsids in Fabry disease

Janneke Meulenberg
GENE THERAPY
15:20

Preclinical AAV for inflammatory disease and arthritic applications

Janneke Meulenberg, Chief Operating Officer, Arthrogen Bv
Magali Taiel
GENE THERAPY
15:40

LUMEVOQ Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Subjects

  • Set the scene: LHON is a rare disease affecting young males
  • Main clinical results
  • Indirect comparison with Natural History data
  • Regulatory Pathways
16:00

Networking refreshments

David Venables
GENE THERAPY
16:30

Maximising value from the expression cassette in gene therapy product design

  • Expression cassette design attributes
  • Synthetic promoter design and validation
  • Regulatable gene expression
18:00

Close of conference

last published: 04/Jun/20 09:25