7:30 am Morning Refreshments & Check In

7:55 am Chair’s Opening Remarks

8:00 am Panel Discussion & Live Audience Q&A: Is 2025 the Year of Curing Deafness? Reviewing Past Failures, Recent Successes & Future Opportunities for Inner Ear Disorders Therapeutics

  • Jonas Dyhrfjeld-Johnsen Chief Development Officer & Managing Director, Acousia Therapeutics
  • Terri Gaskell Chief Technology Officer, Rinri Therapeutics
  • Wan Du Physician Scientist, Massachusetts Eye and Ear/Harvard Medical School

Synopsis

  • Discussing the back-to-back positive results for gene therapies, large investments from key pharma into the inner ear disorders field, and uncovering the opportunities for 2024 and 2025
  • Leveraging lessons from past failures and understanding their downfalls
  • Understanding the current challenges with novel targets, preclinical models replicating the inner ear structures, less invasive delivery mechanisms and improved patient stratification with better clinical trial design 

EXPLORE NEW MECHANISMS OF ACTION TO TARGET GENETIC HEARING DISORDERS WITH RESTORATIVE & REGENERATIVE GENE THERAPY & STEM CELL THERAPY

9:00 am Developing Novel Restorative Gene Therapies for Hereditary Hearing Loss in Patients with OTOF & GJB2 Mutations

Synopsis

  • Developing restorative gene therapies for genetic hearing loss in patients with OTOF and GJB2 deficiency
  • Discussing genetically modified animal models to replicate disease-specific genetic mutations and administration modalities
  • Uncovering the standardized clinical endpoints and regulatory strategies to ensure expedited development of gene therapies

9:30 am Revolutionizing Inner Ear Disorders Therapeutics with Novel Gene Therapy for Replacement of Lost Hair Cells in Rare Genetic Hearing Loss

  • Kaiyu Gao Senior Manager, Refreshgene Therapeutics

Synopsis

  • Positive clinical results for transformative AAV-based gene therapy to restore and improve hearing loss for inherited hearing loss
  • Preclinical results showcasing safety profiles of novel gene therapy targeting OTOF deficiency
  • Discovering new protein recombinant-based otoferlin therapy to restore normal gene function in mutated mouse models

10:00 am Speed Networking

Synopsis

Chat one-to-one with leading genetic and acquired hearing loss experts developing gene therapies, cell therapies and small molecules. Use this informal networking opportunity that allows you to meet and speak with everyone in the room and note who you want to have deeper conversations with throughout the forum and after.

10:45 am Short Morning Break

11:30 am Developing a Regenerative Cell Therapy for Hearing Loss: From Early Research to Clinical Readiness

Synopsis

  • Generating a comprehensive preclinical safety and efficacy data package
  • Optimising manufacturing and establishing a GMP process
  • Developing novel surgical approaches and clinical endpoints for FiH trial
  • Gaining positive feedback from regulators supporting our translational approach

12:00 pm Exploring Novel Small Molecule & Gene Therapeutic Approaches for Tinnitus Indications for Improved Patient Care

Synopsis

  • Development of an operant behavioral mouse and gerbil model for Tinnitus drug discovery • GW-TT2 – Repurposing small molecule targeting Meniere’s disease-associated Tinnitus, delivered intra-nasally
  • Development of a novel two-component gene therapy approach (GW-TT5) for the treatment of severe debilitating Tinnitus
  • Incorporating biomarkers and improved diagnostics in preclinical and clinical designs for more predictable outcomes

12:30 pm Lunch & Networking Break

1:30 pm Panel Discussion & Live Audience Q&A: Gene Therapy Lessons for Rare Hearing Loss

Synopsis

2024 starts with a bang, thanks to back-to-back positive results in gene therapies for paediatric rare hearing disorders. Join this expert panel discussion to hear from gene therapy experts and leverage lessons from other diseases areas when developing and progressing gene therapy for rare hearing loss.

  • Discussing the expansion of the gene therapy field into a wider host of disease targets and increased patient populations
  • Uncovering safety, immunogenicity and dosing challenges for different patient populations outside hearing disorders
  • Dive deep into the exciting new advancements and technologies in gene therapy for other disease indications

DISCOVERING & VALIDATING BRAND-NEW DRUG TARGETS FOR NOISE-INDUCED, AGE-RELATED & CHEMICALLY-INDUCED DEAFNESS

2:30 pm Developing Preventative Therapeutics for Cancer Patients at Risk of Hearing Loss & Tinnitus Caused by Ototoxic Chemotherapy

Synopsis

  • Mitigating ototoxicity in chemotherapy with novel pharmacotherapeutic modalities as pivotal interventions for preserving auditory health
  • Discussing strategic administration of preventative agents prior to chemotherapy to optimize target engagement and efficacy
  • Uncovering clinical challenges for preventative drug formulations to ensure treatment adherence and effectiveness for a patient-centric approach
  • Localized and precise drug delivery to minimize systemic exposure, preserve chemotherapy efficacy and reduce risk of DDI

3:00 pm Repurposing Small Molecule Therapeutics to Prevent Cisplatin-Induced & Noise-Induced Hearing Loss

  • Tal Teitz Associate Professor, Ting Therapeutics

Synopsis

  • Preclinical studies with orally-delivered Dabrafenib for cisplatin-induced and noise-induced hearing loss
  • Uncovering genetic mouse models for Debrafenib activity and the MAPK pathway
  • Translation of repurposed FDA-approved small molecule drugs for prevention of hearing loss into clinical phase 1/2

3:30 pm Afternoon Break & Scientific Poster Session

Synopsis

Want to share your work but not ready for the big stage just yet? The Scientific Poster Session is your prime time to share your work with peers with discovery, preclinical, translational and clinical backgrounds, all working on developing inner ear therapeutics using novel drug modalities. Get their thoughts on how you can accelerate the progression of your drug pipelines and build connections for potential collaborations to expand your hearing disorder R&D pipelines.

4:30 pm Developing Novel Regenerative Therapeutics to Treat Damage to Inner Ear Hair Cells & Auditory Nerve Fibres Caused by Drug-Induced & AgeRelated Hearing Disorders

  • En Li Co-Founder and Chief Executive Officer, Salubritas Therapeutics

Synopsis

  • Uncovering the regeneration of inner ear hair cells and auditory nerve fibre to tackle drug-induced and age-related hearing loss
  • Discovering novel targets and combination therapeutics for cochlear hair cell regeneration
  • Outlining the potential clinical challenges when developing drugs for regeneration

5:00 pm Understanding How Targeting Receptor-Associated Protein Complexes Can Regulate & Restore Auditory Hair Cells in Patients with NoiseInduced Hearing Loss

  • David Bredt Founder, Director & Chief Scientific Officer, Rapport Therapeutics

Synopsis

  • Investigating the unique efferent system in the auditory and vestibular systems targeting inner ear disorders
  • Understanding the permeability of the blood-labyrinth barrier for revolutionized targeted drug delivery into the inner ear
  • Harnessing the complexity of receptor associated proteins to enable precision molecular solutions for inner ear disorders

5:30 pm Chair’s Closing Remarks

5:40 pm End of Conference Day One