Pre-Conference Workshop Day

Monday, December 5 | 8am-6pm

DISCOVERY & PRECLINICAL TRACK

WORKSHOP A | 8-11AM

Synopsis

Overcoming CNS Target & Discovery Challenges

  • Evaluating the most advanced in vitro models available for neurological disorders
  • Selecting the most appropriate targets to pursue for CNS specific vectors

 

WORKSHOP C | 11.30-2.30PM

Synopsis

Translating Dosage Data from Animals into Humans

  • Optimizing preclinical studies for dosage predictions
  • Translating species differences and considering administration routes
  • Reverse translation considerations using dosage data from the clinic

WORKSHOP E | 3.00-6.00PM

  • Bryn Martin Vice President, Research, Precision Delivery, & CSF Sciences, Alcyone Therapeutics, Inc

Synopsis

Use of Computational & In Vitro Modelling to Predict CSF Drug Biodistribution

  • Overcoming problems associated with preclinical study power, reliability and translation to humans
  • Highlighting the need for precision dosing of drugs to the brain
  • Understanding key elements of CSF anatomy and physiology for drug modelling

TRANSLATIONAL & CLINICAL TRACK

WORKSHOP B | 8-11AM

Synopsis

Translatable Biomarkers: Measuring Dose & Efficacy in the Clinic

  • Discussing the relevance and specificity of clinical biomarkers and endpoints are currently being used for neurological trials
  • Which gene therapy specific factors need to be accounted for?
  • Are there novel biomarkers being trialled?

WORKSHOP D | 11.30-2.30PM

Synopsis

Clinical Trial Design Challenges with Different CNS Administration Routes

  • What are the current clinical challenges being faced by neurological developers?
  • What are the specific clinical challenges for a gene therapy product?
  • Showcasing improvement to regulators

WORKSHOP E | 3.00-6.00PM

  • Serene Forte Senior Vice President, Head of Genetic Medicine, Relief Therapeutics
  • Kasia Szczepanska Executive Director, Global Launch Team, PTC Therapeutics
  • Gregory Fuest Vice President of Marketing & Commercial Strategy, Passage Bio
  • Steve Hill Senior Director Global Market Access & Pricing, PTC Therapeutics

Synopsis

Approaching Commercialization of Neurological Gene Therapies

  • What are the implementation challenges into different healthcare systems?
  • What are the payor and reimbursement challenges?
  • Achieving buy-in from relevant stakeholders: patients, doctors, systems, insurance companies