Pre-Conference Workshop Day
Tuesday August 20, 2024

9:30 am Check-In & Morning Refreshments

10:00am-12:00pm
Workshop A

Paving the Path to Effective Translation: Exploring Preclinical Models of Genetic & Acquired Inner Ear Hearing Loss & Measuring Toxicology Profiles for Efficient Translation into the Clinic

  • Seth Koehler Associate Director, Toxicology, Regeneron Pharmaceuticals
  • Meghan Drummond Director, Auditory Sciences, Regeneron Pharmaceuticals

Synopsis

As with other isolated systems, like the brain and the eye, accessibility to the inner ear poses a challenge in extracting samples to develop in vitro models. Furthermore, current animal models such as mice, guinea pigs and monkeys are only able to recapitulate the human inner ear to a certain extent.

This workshop will:

  • Bridge the gap and overcome challenges when developing animal models for genetic and acquired hearing loss
  • Uncover the lack in current in vivo models of the inner ear, unable to recapitulate the cochlear hair cells, supporting cells, spiral ganglion neurons, vestibular hair cells and stria vascularis cells
  • Understand the differences in size, anatomy and organization of the inner ear between in vivo models (such as mice, rates, guinea pigs, cats and monkeys) and humans, as well as differences in frequency, hearing range, lifespan and cellular composition
  • Explore novel in vitro models, such as organoids and iPSCs for better replication of the human inner ear and how these models could pave the way towards a patient-centric approach
  • Strategize how to optimize safety studies and validate non-conventional species are essential for overcoming regulatory barriers and fostering confidence in the development of inner ear therapeutics 

12:00 pm Lunch

1:00pm-3:00pm
Workshop B

Did it Work? Discussing Viable Clinical Endpoints & Objective Measures to Accurately Assess Drug Efficacy in the Inner Ear

Synopsis

With recent developments and positive clinical results with gene therapy for inherited deafness and great progress with cell therapies and small molecules showing promise as novel therapeutics for common hearing loss. a big question remains – how can you prove that your drug worked if you don’t have a checklist?

This workshop will:

  • Uncover objective measures that clinically correlate with clinical patient reported outcomes and improve structure and function
  • Examine quantitative outcomes that are measurable and clinically meaningful to develop better translational endpoints (working your way back)
  • Explore the gap in current criteria for predictiveness of various objective measures such as psycho-acoustics, psycho-physics, electrophysiological measures and protectiveness to allow selection of best targets and best candidates for clinical trials
  • Review previously used and acceptable endpoints that show clinical benefit in various patient populations for varying hearing disorders

3:00 pm End of Conference Workshop Day