Conference Day One

8:15 am Registration & ‘Coffee Room’ Networking

8:50 am Chair’s Opening Remarks

9:00 am Panel Discussion: Advancing Inner Ear Therapeutics Towards Industrial Success


• What criteria do you look for in terms of selecting candidate diseases to search for therapeutic opportunities?
• What are the major challenges lying ahead in translating novel therapeutics from bench to bedside?
• What are the most promising therapeutic candidates in the pipeline, and why?
• How do you see the inner ear therapeutics field panning out in the next 5 years? What challenges do you anticipate post-approval?

9:50 am Virtual Speed Networking


Grab a bite to eat from the comfort of your own kitchen or office and virtually connect with old and new colleagues from the Inner Ear Disorders space. Exchange digital business cards, catch-up with colleagues and strike long lasting connections using our unique speed networking feature.

10:20 am Morning Break

Understand Translation Challenges to Propel Therapeutic Candidates Towards the Clinic

10:50 am AC102 vs Standard of Care in Acute Hearing Loss


• AC102 as a neuro-regenerative therapeutic
• Wheal and woe of steroids
• Acute hearing loss and other acute conditions

11:15 am Translational Survival Challenges of Inner Ear Drug Development


• The “Three Pillars of Survival” principles of Pfizer has been proposed as framework of limiting attrition through the pharmaceutical clinical development lifecycle
• Due to sensory organ anatomy and the immaturity of the therapeutic field, inner ear disorders pose specific translational challenges for assessing adequate local exposure, target engagement and PK/PD relationships
• This talk will focus on the current understanding and limitations in vertical integration of these key aspects for the development of small molecule, inner ear therapeutic candidates

11:40 am Discussion/Q&A

12:00 pm Lunch & Networking Break

Leveraging Next Generation Gene Therapy Approaches to Treat Hearing Loss

1:00 pm Development of an AAV-Based Gene Therapy for Children With Congenital Hearing Loss Due to Otoferlin Deficiency (DB-OTO)


• Genetically driven otoferlin deficiency causes congenital severeprofound hearing loss
• Tightly controlled expression of otoferlin in the sensory hair cells with cell-selective promoter provided robust and durable hearing rescue in preclinical studies
• We are advancing DB-OTO to the clinic for patients with congenital hearing loss due to biallelic OTOF mutations

1:25 pm Gene Discovery & Therapy for Hearing Loss


• Discovery of genes associated with hearing loss by next-generation sequencing, HEar-Seq panel and whole exome sequencing.
• Regulatory elements in the auditory system
• Gene therapy of Syne4 gene in Syne4 knock-out deaf mice

1:50 pm Precision Genetic Medicines for Inner Ear Disorders

  • Manny Simons President, Chief Executive Officer & Co-Founder, Akouos


• Session details to be confirmed

2:15 pm Advances in Hearing Loss Diagnostic Testing: A Gateway to Therapeutic Development

  • Barbara Vona Junior Group Leader , University of Tübingen


• Molecular genetic diagnostic testing for patients with hereditary hearing loss has rapidly changed in the past decade
• Genetic testing allows for a much more comprehensive understanding of the genes and genetic variants involved in hereditary hearing loss
• Genomis has the potential to shape precision medicine by uncovering the molecular landscape of hearing loss, allowing for prioritization and selection of therapeutic strategies

2:40 pm Discussion/Q&A

3:05 pm Virtual Networking

3:35 pm Afternoon Break

Leveraging Next Generation Gene Therapy Approaches to Treat Hearing Loss

4:05 pm Strategy to Trigger Hair Cell Regeneration and Ultimately Restore Hearing Function

  • Michael Franti Director & Head of Regenerative Medicine , Boehringer Ingelheim


• Hearing loss associated with age is predominantly due to the loss of cochlear hair cells. Unlike fish, birds or amphibians, the hair cells of mammals do not spontaneously regenerate; consequently opportunities which lifted the block on regeneration of hair cells from supporting cells, or which allowed proliferation of hair cells in the cochlea would be valuable therapies to address age-related hearing loss
• Recent progress made around the molecular pathways and soluble factors involved in the regeneration of functional hair cells will be presented, and modes of administration and different modalities will be discussed
• Providing a clear overview on the recent advancements in the regeneration of auditory hair cells

4:30 pm Development of First-in-Class Cell Therapy for Hearing Loss


• Founded in 2018, Rinri Therapeutics is a therapeutic discovery and development company working in the field of hearing loss supported by Boehringer Ingelheim Venture Fund and UCB Ventures, BioCity and The University of Sheffield
• Rinri has capitalized on its unique deep understanding of auditory biology to build developmentally informed protocols to produce pluripotent cell-derived auditory neurons that match natural cochlear phenotypes and functional physiological characteristics

4:55 pm Discussion/Q&A

5:15 pm Close of Conference Day One