Advanced Therapies Day Three


Advanced Therapies and Regenerative Medicine Day 3 - Friday 18th May 2018

CRISPR/Cell Line Development

New frontiers in repair and replacement of diseased tissues and organs

CRISPR/Cell Line Development

CRISPR goes in vivo to make preclinic more predictive

  • Past models in preclinic: what was good and what was bad
  • Application of genome editing for somatic cells engineering: advantages and perspectives
  • The future ahead: overoptimistic vs skeptical visions

Multipotent adult stem cells: characterization and regeneration 

  •     Unique adult stem cell with unlimited proliferation potential, ability to differentiate into phenotypes of all 3-dermal lineage, does not cause an immune-rejection response
  •     Used in several regeneration models in adult animals, where undifferentiated multipotent adult stem cells differentiate to the phenotypes at the site, thus regenerating the tissue
  •     Human multipotent adult stem cells survive in a rat and differentiate into phenotypes
Paul Lucas, Director of Orthopaedic Research, Department of Orthopaedic Surgery, New York Medical College
CRISPR/Cell Line Development

CRISPR-mediated genome engineering of CHO cell factories

  • Efficient and controlled genome engineering with CRISPR
  • Isogenic cell line generation and its applications
  • Lessons learned from working with CRISPR

How the development of tissue therapies can aid the development of functional organ replacements

  • Practical insights into Organovo’s tissue engineering technology that has capabilities working with a variety of cell types
  • Where are there opportunities to collaborate in order to advance developments in using tissue engineering for organ regeneration?
  • Next steps from supplemental tissue therapies towards larger replacement tissues and eventually full organ regeneration
CRISPR/Cell Line Development

Engineering cells and animals as models for therapies

  • Application of Synthetic Biology approaches  
  • Engineering transgene expression stability in cells and mice
  • Engineering mice as models for therapeutic vaccination
CRISPR/Cell Line Development

Cell therapy for neurorepair

  • Foetal stem cells are neuroprotective against neonatal hypoxia is he Mia
  • Transplantation reduces brain lesion
  • Mechanisms of action involve adenosine

Human PSC-derived cardiomyocytes/neurons on 3D micro-tissue devices for matured and functional cell products in drug discovery/cell therapy

  • Importance and needs of more matured/functional/reliable/low-cost cell products from human pluripotent stem cells (hESC/hiPSC) in drug discovery and cell therapy.
  • Robust & low-cost cytokine-free differentiation of cardiomyocytes from human ES/iPS cells by combination of small chemical compounds.
  • Construction of 3D micro-tissue devices with aligned nanofibers for more matured/functional/reliable cardiomyocyte/neuron products.

Networking refreshment break

Elizabeth Schwarzbach

Keynote: Harnessing patient data using stem cells to power precision medicine

Elizabeth Schwarzbach, Chief Business Officer, New York Stem Cell Foundation
Dr Yen Choo

Small molecule drug discovery to selectively stimulate regenerative processes

  • Collaborating with big pharma for successful target discovery
  • Using ATMP knowledge to target and stimulate specific biochemical signalling pathways
  • Use traditional pharmaceutical drug screening technologies to discover synthetic small molecule modulators of regenerative pathways
Dr Michael D West, Ph.D.

Keynote: What’s new in the field of aging and regeneration?

  • How can molecular and AI technologies help regenerate damaged tissue in humans?
  • What are the current challenges with induced tissue regeneration and how are we tackling them?
  • Combining pluripotent stem cell and iTR technology to form new company AgeX

Networking Lunch

Networking LunchClosing keynotes: What’s next for ATMPs?

Alain Vertes
Dr Frédéric Triebel

Panel: Combination immunotherapy strategies across pharma and biotech

  • High level pharma companies come together to discuss their strategies for tackling cancer
  • Each company will give a 5-minute presentation on their strategy, and a fire side discussion on oncology focused strategies will follow
  • Strategies will cover cytotherapy, checkpoint inhibitors, combination therapy, sequential therapy, approaches to solid tumours, treatments and diagnostics
Laszlo Radvanyi, Senior Vice President, Senior Scientific Advisor, EMD Serono

End of Day 3

last published: 14/May/18 16:05 GMT







Erica Baeta
+44 (0)207 092 1152


Jessica Robinson
t/ +44 (0)207 092 1150


Issa Mauthoor
+44 (0)207 092 1257