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A lot has happened since our last update!

Today, an NPC gene therapy mouse study by the National Institutes of Health (NIH) was published in the journal Human Molecular Genetics.

Galyatech has a non-exclusive license with the NIH for the NPC gene therapy vectors used in this study, including the vector AAV9-EF1a-NPC1, which provides the proof-of-concept foundation for the ongoing development of GALYA-1.

Here are links to the Abstract and Full PDF (Free) and NIH's press release.

Here are videos from the published paper and a brief visual summary of efficacy data, along with other announcements.

Video G1: Three Mice – Treated, Untreated, Normal

Video G2: Untreated, Treated (Shown at Multiple Weeks)

Gene Therapy-Treated Mice Lived Longer with Improved Weight and Motor Activity

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Mean Survival ± SD: 69 days ± 3.1 vs. 166 days ± 89.2
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Week of Peak Weight: 6 weeks vs. 12 weeks
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Pct Weight Change (6-9 weeks): -17% vs. approx. +9%

Developing GALYA-1

The NIH mouse study publication is very exciting news! With this key proof of concept research now complete, our commitment remains to develop GALYA-1 as a safe and effective drug – including initiating a Phase I clinical trial – as quickly as possible. We are making progress toward this goal by preparing for efficacy studies in cats and GLP toxicology studies, as well as pushing forward with manufacturing of GALYA-1. Learn more below!

Testing in NPC1 Cats

We have a strong existing partnership with Dr. Charles Vite at the University of Pennsylvania –home of the only large animal model for NPC – and we are prepared to begin gene therapy efficacy studies in the NPC1 cat model soon. Demonstrating efficacy in cats will provide a stronger foundation for a Phase I clinical trial.

Pre-PreIND Scheduled

The FDA has agreed to our request for a pre-preIND interaction to give us advice about our remaining preclinical studies, especially toxicology. This communication with the FDA is a key step in the process of developing GALYA-1 as a safe and effective drug.

Manufacturing Moving Forward

The manufacturing of some components of the non-pathogenic virus is complete! We continue to move forward with manufacturing in parallel with remaining preclinical studies so that we can start toxicology studies for GALYA-1 as soon as possible.

NPUK Family Conference

Andrew Wilkinson and his wife Jennifer attended the NPUK Family Conference last month! It was great to meet many NPC families, as well as doctors and researchers, in person. :) It was a pleasure to be there.
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Where We're Going Next (China)

Andrew and Jennifer are going to China next week to attend the Chinese Niemann-Pick Disease Association (CNPDA) family conference! We will meet families, researchers and doctors in Beijing November 4-6 and will give a presentation on Saturday evening. For all the families in China and Asia, come join us in person! We really want to meet you face to face. :)