Symposium Goal

To gather scientists, clinicians, patients, advocates, and members of industry together to determine how to overcome the last hurdles to bring genetic therapies for Usher Syndrome to clinic.

Symposium Aims

1) To summarize current clinical and research findings of genetic diagnosis for Usher Syndrome while highlighting progress made in the Children’s Rare Disease Cohort Initiative (CRDC) at Boston Children’s Hospital.
2) To critically evaluate the data available from trials of gene therapy for Usher Syndrome in animal models.
3) To discuss the best methods for clinical evaluation of Usher syndrome patients undergoing clinical trials and determine the first patients for initial clinical trials.

Symposium Program

The symposium will include:
1) A scientific program targeted for clinicians and scientists in academia and industry.
2) A public program targeted for patients, families and advocates in the Usher Syndrome community.

Symposium Schedule

Scientific Program 1-5pm (clinicians and scientists only)

Boston Children’s and Invited Faculty

• 1:00-1:05 — Introduction / Eliot Shearer MD PhD, Boston Children’s Hospital

• 1:05-1:30 — Accurate and Early Diagnosis for Usher Syndrome / Eliot Shearer MD PhD, Boston Children’s Hospital

• 1:30-1:55 — Results from the Children’s Rare Disease Cohort(CRDC)/Bilateral and Unilateral Genetic Hearing Loss (BAGHL)Study: How does USH compare? / Margaret Kenna MD MPH, Boston Children’s Hospital

• 1:55-2:20 — Development of therapeutic approaches for Usher Syndrome / Gwen Géléoc PhD, Boston Children’s Hospital

• 2:20-2:45 — Usher Syndrome: paradigms for mutation-specific therapy / Timothy Yu MD PhD, Boston Children’s Hospital

• 2:45-3:00 — Break & Refreshments

• 3:00-3:30 — A Primate Model for Usher 1B / John Brigande PhD, Oregon Health & Sciences University

Industry Representatives

• 3:30-3:50 — Optimization of a Dual AAV Vector Mediated Approach for the Treatment of Usher Syndrome type 1B / Eva Andres-Mateos PhD, Atsena Therapeutics

• 3:50-4:10 — Development of a CRISPR-based gene editing therapy for USH2A-associated retinitis pigmentosa / Benjamin Diner PhD, Editas Therapeutics

• 4:10-4:30 — Development of an AAV-Based Gene Therapy for Children With Congenital Hearing Loss Due to Otoferlin Deficiency (DB-OTO)/ Jonathon Whitton AuD PhD, Decibel Therapeutics

Public Program — This is USH Conversations 5-8pm (open to all registrants), Organized by the Usher Syndrome Society

• 5pm-6pm: Refreshments and hors d’oeuvres

• 6pm-8pm: Special guests Rebecca Alexander with Steven McCoy

For more information about This is USH Conversations click here.

Symposium Location

The Joseph B Martin Conference Room

New Research Building
Harvard Medical School
77 Avenue Louis Pasteur
Boston, MA 02115

Directions

Campus Map

Google Map (with hotels nearby)

Parking

Organized in Coordination With: