All of the companies associated with the gene-editing technique are having a good day thanks to the data. Although the treatment for the fatal disease -- transthyretin (ATTR) amyloidosis -- is great news, the implications go far beyond any one disease.
The results are the first to show the gene-editing approach known as CRISPR-Cas9 can be both safe and effective when performed in the human body. Previous applications used cells that had been removed, edited, and reinserted into the patient.
The excitement should increase attention on Beam's alternative approach, called base editing. While CRISPR-Cas9 has been likened to a pair of genetic scissors, Beam's approach is like a pencil and eraser. Instead of cutting both strands of DNA, Beam is able to chemically change one base letter at a time. Many believe this is an easier approach that leads to fewer accidental insertions or deletions -- known as off-target effects.
Intellia's results are impressive but only an early step toward commercializing a treatment. Beam is a year or two behind that. The company doesn't plan to submit its sickle cell disease drug for investigational new drug approval to the FDA until later this year. That's only the first step of testing the drug in humans.
Even if the it gets the green light to enter phase 1 trials, it could be two years before results are available. Although developments in genetic medicine are exciting and advancing rapidly, investors will have to take a long-term view while waiting for any sales and profits.