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

In 2017, we:

Studies

• 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

Manufacturing

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


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

Legal

• 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

Conferences

• 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)

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