Since our last update we've made a lot of progress toward developing GALYA-1. Read more below!
Dr. Steven Gray Joins SAB
We are very happy to announce that Dr. Steven Gray, PhD (UNC Gene Therapy Center) joined Galyatech's Scientific Advisory Board (SAB) in November 2016. Steven's gene therapy expertise and preclinical development experience are an invaluable contribution to our goal of initiating a Phase I clinical trial as quickly as possible. Dr. Gray successfully directed the development and testing of an AAV9 gene therapy for the rare neurological disorder, Giant Axonal Neuropathy (GAN), which began a Phase I clinical trial in 2015.
Pre-PreIND with FDA
Toward the end of 2016, Galyatech had discussions with the FDA and received valuable advice about GALYA-1 development which we've since put into action, described below.
Preclinical Study Designs
Building on the FDA's advice, we came up with robust preclinical study designs to evaluate GALYA-1 in NPC1 mouse and cat models. These studies will generate the necessary preclinical data to support an FDA investigational new drug (IND) filing for GALYA-1.
The FDA also gave us advice about manufacturing the gene therapy vector – a critical step, and a common bottleneck, in the process of preparing for a Phase I clinical trial. It takes time to evaluate different manufacturing facilities and to lay the groundwork for vector production for a clinical trial. We are committed to doing the process right and work is underway to produce GALYA-1.
When we launched Galyatech in June 2016, we aimed for starting a Phase I clinical trial in the second half of 2017. Though Galyatech continues to work diligently on moving gene therapy toward a clinical trial, the manufacturing and preclinical study processes indicate this timeline needs to be modified.We now anticipate filing an IND with the FDA by the end of 2018. Galyatech is blazing a new trail into the unknown with an optimistic timeline, though unexpected delays may occur. We remain dedicated to developing GALYA-1 as a safe and effective drug – including initiation of a Phase I clinical trial – as quickly as possible.
CNPDA Family Conference
We wanted to share with you one more piece of exciting news. Last November, Andrew Wilkinson and his wife Jennifer attended the CNPDA family conference in Beijing! It was a pleasure to meet many NPC families, doctors and researchers in person. :) While there, Andrew presented results from the NPC gene therapy mouse study published by NIH (see October 2016 update).