Aavantgarde Bio Ush1B study at Moorfields

The purpose of this study is to test a new treatment for loss of vision due to a genetic, inherited disease called Usher syndrome type 1B. In this disease, due to a defective gene, a protein needed for proper health of the light-sensing cells in the back of the eye called photoreceptors is defective. The treatment will involve inserting a gene inside these cells to produce a properly functioning protein. This treatment could lead to slowing of the disease progress over time.
The study involves a one-time surgery to inject the gene therapy underneath the retina.

(is this a new para or should it line up with the last one) There are no costs involved for patients to participate.  The sponsor will cover all travel and accommodation costs associated with making each study visit. The sponsor will also organise and cover the cost for a BSL interpreter to be present at each study visit if required.

The study is looking for patients with the following general criteria:
1. Male or Female between 18-60 years of age.
2. Confirmed diagnosis of Usher 1B due to MYO7A mutations confirmed by an approved lab.
3. No previous treatment with an investigational gene therapy

All study visits including the screening, surgery, and follow-up visits will be completed at Moorfields Eye Hospital London by the clinical trials and medical team led by Professor Michel Michaelides.

Further details on the study are on clinicaltrials.gov Study Details | Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa | ClinicalTrials.gov

Patients who are interested in the study can contact Sarah Hill at Moorfields Eye Hospital London:  [email protected]