National Organization for Rare Disorders’ Post

#GeneTherapy and #GenomeEditing may feel futuristic and out-of-reach, but it's here. Now, how do we make it accessible to the average person? Some ideas from Peter Marks, Director of #FDA CBER; David Barrett, CEO of American Society of Gene & Cell Therapy; and Nicole Guadelli of GV (Google Ventures): - Explore non-bespoke therapies - Get #RareDisease patients access to technologies and drugs in late stage development - Further explore the potential of #mRNA and #GuideRNA for #GeneEditing - Improve education about the complexities of these therapies, both within the #pharma industry and outside of it - Collaborate and share knowledge about what works and how #NORDSummit #Biotech #Biotechology #CRISPR #DrugDevelopment

  • NORD Summit Panel, "How Can We Make Gene Therapy More Accessible?" featuring, from left to right, moderator PJ Brooks of NCATS, Peter Marks of FDA CBER, Nicole Gaudelli of Google Ventures, and David Barrett of ASGCT.

To view or add a comment, sign in

Explore topics