Research and Innovation 2023 – Accelerating future drug discovery
Wednesday, 29 March 2023 to Thursday, 30 March 2023

Presentations by Session

Programme : Presentations by Session

Ultra-rare disease drug discovery and personalized therapies

Session Introduction
Wed29  Mar11:00am(10 mins)
 Dr Joanne Hackett Dr Joanne Hackett Dr David Fischer Dr David Fischer 
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
Novel therapies in developmental and epileptic encephalopathies
Wed29  Mar11:10am(30 mins)
 Dr Amy McTague Dr Amy McTague  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
Using patient data to enable drug discovery in rare diseases
Wed29  Mar11:40am(30 mins)
 Dr Guido Hartmann Dr Guido Hartmann  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
Realising the benefit to healthcare through population-wide application of polygenic risk scores
Wed29  Mar02:00pm(30 mins)
 Prof Gilean McVean Prof Gilean McVean  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
Advanced spectral cell sorting solutions for immune monitoring and cell purification
Wed29  Mar02:30pm(5 mins)
 Ms Katrina Kidd  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
3-D patient-derived models of rare cancers
Wed29  Mar02:35pm(5 mins)
 Dr Rebecca Charlton  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
Development of antisense oligonucleotide therapeutics for the treatment of TUBB4A-related leukodystrophies
Wed29  Mar02:40pm(30 mins)
 Dr Laure Humbert Dr Laure Humbert  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
SLC transporters and drug discovery: unlocking the "gatekeepers" as therapeutic targets for rare diseases
Wed29  Mar03:10pm(10 mins)
 Mrs Lucia Azzollini Mrs Lucia Azzollini  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
AI augmented Target Discovery
Wed29  Mar03:50pm(30 mins)
 Dr Anne Phelan  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
Cell and Gene Therapy: the ddPCR Solution
Wed29  Mar04:20pm(15 mins)
 Mr Jose Martins Mr Jose Martins  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room
Nonclinical Development for Rare/Ultra-rare diseases; a Balance of Safety Assessment and High Need
Wed29  Mar04:35pm(30 mins)
 Dr Michael Templin Dr Michael Templin  
Session : Ultra-rare disease drug discovery and personalized therapies
Room: The Rosalind Franklin Room

Programme

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