The instructions for making a human are long and complicated. They involve over 3 billion sets of microscopic letters, so tightly coiled that, if unwound, they would be taller than the average person.
The daunting task of reading these instructions involves a type of protein called transcription factors. But, while integral to life, these proteins can also cause disease when they mutate or malfunction.
For drug developers, transcription factors are an attractive target. “Every biotech company that I’ve been a part of has been interested in at least one or two,” said Abbie Celniker, a well-known industry veteran and a partner at the healthcare investment firm Third Rock Ventures.