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As a follow up to our earlier post back in January, here's more info. We are working diligently to move gene therapy toward a clinical trial, yet we also have some significant obstacles to overcome which we want to share with you.

Delays: Two Sources

Manufacturing

As we said in our February 2017 Update, manufacturing of gene therapy vector is a common bottleneck. Unfortunately, that has been our experience, with repeated manufacturing difficulties resulting in delays, pushing back timelines for preclinical studies and further manufacturing activities. We've been actively working to resolve these issues – we have multiple strategies in place, plus some hopeful results to bolster our efforts – and we continue to push for a Phase I clinical trial as soon as possible.

Route of Administration

We expected our gene therapy to be administered via a lower back (intrathecal) injection into the central nervous system. While that might still prove to be the case, the route of administration is not set in stone and we will follow where the preclinical data leads us. We're evaluating two alternative routes of administration: intrathecal and intravenous.

IND Timeline: Three Alternatives

As a result of these delays and because we learned some processes take longer than previously estimated, we need to push back our timeline, again. We face some pretty significant unknowns, and therefore we can't give a more hard-and-fast date (sorry), but we still want the NPC community to know the facts.

Depending on how things go in 2018 with our manufacturing and evaluating the route of administration, here are three estimates for timeline to file an IND.

Best-case scenario. File IND in early 2020.

Moderate-case scenario. File IND in early 2021.

Worst case scenario. It's possible that Galyatech won't be able to resolve the manufacturing issues we've encountered. (We hope this is not the case, but it is possible.)

(For reference: In February 2017, we anticipated filing an IND in 2018.)

NPC Community: One Commitment

While these delays are disappointing and the unknowns are daunting, we remain committed to developing a safe and effective gene therapy for NPC1 disease – including initiation of a Phase I clinical trial – as quickly as possible.

We hope to have more encouraging news to share with the NPC community this year. However, even if results are not good, we will do our best to keep you informed.