The 20(+1) under 40: Inside the next generation of biotech leaders
Dr. Juliette Hordeaux, Senior Research Director at GTP is featured in Endpoints News’ annual “Under 40: Inside the next generation of biotech leaders.”
This program represents our ambitious commitment to developing cutting-edge vector technologies. Our diversified approach focuses on delivering advanced vectors for traditional gene therapy applications as well as vectors tailored specifically for genome editing. The design of these transformative next-generation AAV vectors is being guided by experimental results from studies on the basic biology of AAV that leverage our world-class resources in molecular, structural, cell, and computational biology.
GTP is innovating high-performance vectors to enhance the safety and efficiency of genetic therapeutics.
GTP provides preclinical pharmacologic and safety testing in appropriate in vitro and in vivo models, as well as IND-enabling studies designed to support the advancement of a clinical candidate to first-in-human clinical trials.
GTP provides strategic regulatory guidance and support for the Investigational New Drug (IND) application in order to advance a product candidate to clinical trials.
Phase I trials assess the safety and dose-dependent effects of a treatment in healthy volunteers.
Phase II trials assess the effectiveness of a treatment in a small group of patients, typically up to several hundred. For rare diseases, however, studies may only be able to enroll one to three patients per year for a given site.
Phase III studies involve randomized, blind testing on a large patient population. FDA approval for marketing of a therapy can be requested when Phase III studies are complete.
The cell and gene therapy industry represents a global market that is expected to exceed $20 billion by 2025. With recently approved products in both the US and EU, this industry is poised for dramatic growth.
In conjunction with the tenth anniversary of the Penn Orphan Disease Center (ODC), Penn Medicine will host a two-day symposium featuring ODC Director James Wilson, NIH Director Francis Collins, former Prime Minister David Cameron and others on February 24th & 25th