Biotech's Crispr gene editing: Intellia, Beam, Verve, inside the lab

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Investors are betting billions that cutting-edge cures can upend how we treat heart disease and transform pharma

— said technologies such as gene editing are fundamentally different from the drug industry's usual approach."Biotech is just beginning," Afeyan said."In the past, biotech was a little bit of bio and a little bit of tech. I think the amount of it that is bio now in terms of actually understanding has gone up and the amount of technology is incredibly higher. The result is going to be more drugs, more predictably, hopefully with less cost.

Even the most advanced gene-editing programs have provided only preliminary results. But that early data has been enough to drive further enthusiasm, reinforcing the belief that the technology can usher in a new era of drug research."We are part of this revolution in medicine to go toward one-time cures," said John Evans, CEO of the gene-editing specialist Beam Therapeutics, which commands a $6.2 billion valuation.

The scientists who discovered Crispr were awarded the Nobel Prize last year, and the public got a major introduction to Crispr's story through the biographerStill, gene-editing therapies are years away from reality, and therapies that rely on altering our genes have run into trouble before. In 1999, the field of gene therapy suffered a massive setback after the. It wasn't until 2017 that the first gene therapy was approved in the US.

Gene editing's supporters argue it is more precise and powerful than gene therapy. Some leading gene-therapy programs have faced recent setbacks over toxicity and durability concerns. While gene therapy typically uses a virus to replace a faulty gene with a working one, gene editing corrects errors in the genetic code, or DNA itself, similar to fixing a typo in a line of computer programming.

At the turn of the 21st century, the Human Genome Project completed the first rough draft of the entire genetic sequence of the human body. Since then, knowledge of human genetics has exploded, and the cost of sequencing a genome

 

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