ETEncoded Therapeutics is a biotechnology company focused on developing transformative gene therapies for CNS disorders.
STSarepta Therapeutics is a leading biotechnology firm dedicated to advancing precision genetic medicine for rare diseases.
ShapeTX is dedicated to revolutionizing programmable RNA medicines with its innovative AI-driven platform, aimed at repairing genetic disorders and providing effective solutions for many diseases.
PTC Therapeutics, Inc. is a global biopharmaceutical company focused on developing innovative therapies for patients with rare diseases.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy, utilizing its proprietary NAV® Technology Platform to develop a pipeline of AAV therapeutics for retinal, metabolic, and neurodegenerative diseases.
Dyne Therapeutics is dedicated to developing transformative therapies for neuromuscular diseases using its FORCE™ platform.