Post Conference Day

Gene Therapy for Inner Ear Disorders

10:00 am Chair’s Opening Address

10:15 am From Lab to Clinic – Clinicians Perspective Working on the Frontline

  • Kaukab Rajput Consultant Audiovestibular Physician, Great Ormond Street Hospital NHS Trust

Synopsis

• The common causes of genetic hearing loss may require the attention of the gene therapy industry. Get the number of cases right – rare diseases are rare, consider going for common gene, may be hard but worth it in the long run
• The proof of gene therapy is in the outcomes. This has to be better than what is being offered at the moment such as hearing aids for moderate hearing losses and cochlear implants for severe to profound hearing losses
• The long-term sustained ability is important, the inner ear therapeutic development was needed to look at the world of cancer and long-term survival. The same rule needs to apply inner ear therapies such as hearing preservation on medium to long-term

10:45 am Single-Cell Genomics for Inner Ear Gene Therapy Design

  • Adam Palermo Vice President, Molecular Platform, Decibel Therapeutics

Synopsis

• Long-term success in gene therapy and its application to regenerative medicine relies on the ability to restrict transgene expression to specific cells
• We are applying proprietary analyses of gene expression in the cochlea and vestibule and have generated a comprehensive database of every known inner ear cell type to enable target identification and evaluation, as well as identification and selection of promoters for gene therapies

11:15 am Population-Scale Analysis of Common & Rare Genetic Variation Associated with Hearing Loss In Adults

Synopsis

• Talk details TBC

11:45 am Break

12:45 pm Gene Therapies for Treating Monogenic Hearing Disorders

  • Nora Yang Chief Scientific Officer, Sensorion

Synopsis

• Hundreds of millions of people suffer from genetic based hearing loss without effective treatments
• More than a hundred genetic loci have been mapped to cause hearing loss in humans, ranging from congenital to presbycusis
• AAV delivered gene replacement therapies hold great hope of improving and restoring hearing in patients suffering from monogenic hearing loss disorders

1:15 pm Gene Replacement Therapy for GJB2-Related Hearing Loss

Synopsis

• Review of GJB2 gene mutations, the most common cause of congenital hearing loss
• Development of OTO-825 for GJB2 gene therapy
• Preclinical proof-of-concept: OTO-825 rescues cochlear morphology and hearing function

1:45 pm DB-OTO, Gene Therapy Product Designed to Provide Hearing to Individuals with Otoferlin Mutations

Synopsis

• DB-OTO is an AAV-based dual-vector investigational product candidate designed to restore hearing to individuals with profound, congenital hearing loss due to otoferlin deficiency
• DB-OTO utilizes a proprietary, cell-selective promoter to express otoferlin in hair cells
• Pre-clinical studies have demonstrated restoration of hearing in a mouse model after administration of DB-OTO

2:15 pm Chair’s Closing Address