CRISPR’s one-shot moonshot to change medicine

Published on10 MAY 2023

Imagine instead of taking pills or undergoing regular treatments for a disease that you could simply have a one-time procedure and be cured of it forever.

That could very well be the reality for many in the coming years, according to Samarth Kulkarni, chief executive officer of CRISPR Therapeutics, one of the leading research companies attempting to use ground-breaking CRISPR genetic editing technology to cure diseases by rewriting faulty genes. His company, along with Vertex Pharmaceuticals, are awaiting FDA approval for a CRISPR treatment targeting sickle-cell disease and transfusion-dependent beta-thalassemia (TBT), which could become the first-ever commercially available treatment in the U.S. using the gene-editing technology.

“We’re going to think of medicine very differently 50 years from now because we’re not going to be popping pills,” Kulkarni said during a wide-ranging interview at Talks at Goldman Sachs. “We’re going to do one-time procedures to change your genome, and hopefully you’re preventing disease or completely curing yourself of disease.”

Kulkarni describes CRISPR as “molecular scissors,” capable of “deleting” genes that are susceptible to disease. “That’s why there’s so much excitement about CRISPR in biomolecular research or biomedical research,” he said, “because you can now think about tens of diseases, if not hundreds, that can all have a potentially curative therapy that acts on the basis or the fundamental aspect of the disease, and that’s the DNA.”

But CRISPR also has its skeptics, among them those who worry the technology will be used to do off-target editing or unintended genetic modifications. One worry is that such changes could increase a patient’s risk for cancer or other serious side effects. Kulkarni said that, while the risk of such outcomes is not zero, the U.S. Food and Drug Administration and industry experts are getting more comfortable with CRISPR the more they understand how precise it is at editing DNA. “The way I explain this is this is like a 20-letter passcode,” he said. “If you have a five-letter passcode, odds are it’s easier to hack or easier to figure out or easier to have off-target effects. But if you have a 20-letter sequence, there’s a trillion combinations. The odds that you’re going after a particular 20-letter sequence that happens somewhere else in the genome is very low.”

Still, because CRISPR leads to permanent changes in a person’s DNA, that creates a high bar for development and regulatory approval. Regulations around CRISPR Therapeutic’s treatment for sickle-cell disease requires the company to follow its trial patients for 15 years. But unlike drugs that can take time to work, CRISPR fixes the problem immediately. “There’s a black-and-white effect in these patients, and all of a sudden they’re normal,” Kulkarni said. While you still need to follow the patients for a very long time for safety, he says you don’t need large trials.

That opens the door to a potentially very promising future, Kulkarni said. At first, scientists assumed CRISPR would be perfect for rare diseases, which today number more than 600. But common diseases are the leading causes of mortality – such as heart disease, cancer and diabetes – and that could be the technology’s next frontier. “CRISPR is going to have a profound impact in common diseases as well,” said Kulkarni, “and that’ll extend to almost every disease that we know.”

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