30 March -1 April 2020 | Business Design Centre London



1-2-1 Meetings




INNOVATION, Tuesday 31 March 2020

CONFERENCE DAY 1 - TUESDAY 31st March 2020

Opening Keynotes Day 1 – CAR-T & Immunotherapy focus

Prasad Adusumilli

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

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

Morning Coffee Break

Mark Sawicki

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

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

11. Cord blood as a source of new cells
12. Tissue engineering for organ regeneration
Vaidehi Joshi

Vaidehi Joshi, Scientist II, Therapeutics, Organovo

13. TBC
14. Making allogeneic cell therapy a reality
Devyn Smith

Devyn Smith, Chief Operating Officer And Head Of Strategy, Sigilon

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

Elena Beltrami, Business Development Manager, Fondazione Telethon

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

18. Ensuring ATMP access to patients
Delphine Roulland

Delphine Roulland, Director Government Affairs And Public Policy, European Confederation of Pharmaceutical Entrepreneurs (Belgium)

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

Cornelia Kasper, Professor, Biopharmaceutical Production And Technology, B.O.K.U.

21. Neurological applications for ATMPs
22. Development of regenerative medicines for cardiovascular indications
David Mazzo

David Mazzo, Chief Executive Officer, Caladrius Biosciences

23. Renal applications for ATMPs
24. Tackling diabetes with ATMPs
3. TBC (Reserved for Schott)
Sebastian Kress

Sebastian Kress, Post Doc, BOKU

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

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

Joanna Miller, Chief Scientific Officer, Cell Therapy Sciences



Yochi Slonim

Translation Control Therapeutics: discovery of small molecule drugs that control mRNA translation into proteins

  • The novel target space of mRNA translation regulation
  • Discovery of small molecule drugs that selectively control mRNA translation
  • Anima Biotech:bi-directional and tissue selective translation controlling drugs

Afternoon Break

Julian Gough

Using big data applications to transform any cell type

  • Network-based algorithms to find transcription factors that lead to a change is cell state
  • Generating cell conversion protocols
  • Characterise and understand the cell type using microscopic and genetic markers
Elena Costariol

Process Development and Manufacture of CAR-T cells in an automated, stirred-tank bioreactor

Evelina Vågesjo

Taking a novel next-generation biologic from idea to clinical proof of concept

  • On-site delivery of human proteins to body surfaces by lactic acid bacteria
  • Clinical trials skin wounds-ILP100
  • Preclinical MOA IBD-ILP100

Close of Conference for the day - Please join us for an evening drinks reception at the iconic Emirates Stadium

last published: 28/Jan/20 14:05

INNOVATION, Wednesday 1 April 2020


Opening Remarks

Federico Mingozzi

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

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
Adrian Woolfson

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

In vivo gene editing paediatric patients with rare diseases


Networking Refreshments

Dr Bo Kara

Engineered Exosome Therapeutics – Development and Manufacturing

Randolph Corteling

An Exosome-based Drug Delivery Platform Derived from a Human Neural Stem Cell (hNSC) Line

  • To ensure the scale required for clinical research and commercialisation producer cell immortalisation and clonal isolation is a practical strategy to produce consistent, functionally bioactive exosomes for use as therapeutic agents.
  • The cell line can be rapidly modified to alter exosome cargo or reprogrammed to change its phenotype while maintaining its conditional immortalisation.
  • hNSC derived exosomes demonstrate favourable distribution across the blood brain barrier.
Chirag Desai

Exosome derived from peripheral blood plasma of chronic pancreatitis patients and healthy volunteers and explore therapeutic potential in auto-islet transplant

  • Produce and characterize serum derived exosome products from CP and healthy subjects and to establish the feasibility of cGMP-compliant production.
  • Evaluate if CP patient derived exosome has impaired anti-inflammatory response by comparing the exosome molecular profile and potency with healthy individuals.
  • Evaluate in mouse model therapeutic potential

Neworking Lunch

Antonio Rodriguez

Developing the First External Quality Assurance (EQA) Program for ATMPs

  • Design and development of the first External Quality Assurance (EQA) programme for ATMPs. Adapting the acquired experience from other fields of knowledge.
  • Promoting the harmonization of relevant Quality Control methods to guide manufacturers to develop safe and effective ATMPs.
  • State of development. Joining the programme and benefits to participants.
Vittorio Colizzi

Plant Micro-vesicles containing smallRNAs in delivering and adjuvancy for sustainably cancer therapy

Sergey Piletsky

Development of novel assays and sensors to support manufacturing and therapy


Networking refreshments


Stem cells secretome and their efficiency in the treatment of soft tissue injury and skin rejuvenation


Cell manufacturing for cell therapy and regenerative medicine and cell expansion for iPSCs

last published: 28/Jan/20 14:05