gene that has impacted the founder’s daughter, Rose, slowing her development and then slowly robbing her of the functional abilities she did develop. The company uses antisense oligos—short oligonucleotide strands designed to bind to complementary DNA or RNA sequences—to correct mutations that cause rare diseases. In the case of the H2 mutation, fibroblasts, which are the most commonly used cells, wasn’t a good model. Everlum needed neurons. bit.bio had just what they needed.
“The data blows me away,” Kotter told me. “Essentially, we’ve generated a manufacturing paradigm that is as good as manufacturing an iPhone - you cannot tell the difference between one that’s produced in January versus one that’s produced in March. If you think about biology being a fuzzy sort of science, we’ve proven that there is a reality in biology that is not fuzzy, that is, deterministic. That’s a watershed moment.
“We have a responsibility, a stewardship to bring this technology to its potential. Lots of ideas can die; we must allow this to flourish,” he says. “The ultimate achievement would be to allow cell therapy to become the mainstage treatment for various degenerative diseases and other conditions. It should be as simple and as cost-effective as biologics. And, if we can democratize access to human cells and enable others to generate drugs, there is the potential to impact a billion patients. I think it’s possible.”
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