Web Content Display

2017 was a productive year at Galyatech! And we're hoping to make much more progress in 2018! Read more below!

A Look Back on 2017

In 2017, we:


  • performed 3 in vitro preclinical tests in cells

  • designed 3 in vivo preclinical animal model studies (plus 2 additional supplementary studies)

  • initiated 1 in vivo preclinical study in mice (and another study coming soon)

Animal Model Colonies

  • established 1 Npc1 mouse colony (with an existing mouse model)

  • worked closely with 1 existing NPC1 cat colony


  • manufactured more than 10 preparations of non-pathogenic AAV9 virus

  • experimented with more than 3 different variations of the virus components


  • entered more than 10 new legal agreements, including:

    • 1 new research collaboration agreement with a major scientific research institution and

    • 6 agreements with organizations and consultants in areas of manufacturing, animal models, and process development

Project Management

  • participated in 100's of phone and/or teleconference calls

  • sent and received around 4,000 emails

  • received great advice from our 3 scientific advisory board (SAB) members: Brittney Gurda, Cristin Davidson and Steven Gray


  • were in-person at 5 conferences/symposiums focused on gene therapy and/or rare disease:

    • WORLD Symposium (Feb 2017)

    • NYAS: Gene Therapy for Rare Diseases Conference (Apr 2017)

    • Hanson Wade: Gene Therapy for Rare Disorders (Apr 2017)

    • Michael, Marcia, and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research (June 2017)

    • Global Genes 2017 RARE Patient Advocacy Summit (Sep 2017)

Next Update

We hope to share more details with you soon in the next update!