New types of therapies, such as gene therapy and oligonucleotides, are paving the way toward treating rare diseases that have so far been thought of as “undruggable”. Gene therapy focuses on adding a corrected copy of a gene or directly altering a mutated gene. Oligonucleotide therapies focus on blocking the formation of a protein or restoring the function of a protein. In this session, leaders from patient advocacy and industry will discuss the promise of these new approaches.
Speaker: Bartholomew Tortella, MBA, MD, Global Medical Affairs Lead of Rare Disease Hematology and Transplantation, Pfizer
Speaker: Alex Mori
Day 2, Track 4