Meet the 4 Biotechs That are Disrupting Healthcare

Published: Nov 14, 2017 By

Scientist

It’s not always easy to see when a company is a “disruptor,” which is to say, that it causes a paradigm shift in how an industry operates. A classic example is Amazon, which definitely disrupted bookselling, then retail in general, publishing, and just about every industry it touches. Keith Speights, writing for The Motley Fool, takes a look at four biotech companies that he believes are in the process of disrupting healthcare.

The first three companies are all using CRISPR/Cas9 gene editing technology to try to develop therapeutics. The tech essentially allows researchers to identify a specific stretch of DNA, cut it out and replace it with something else. It’s a very exciting technology, although there are two looming concerns. The first is the concern over the technique creating unexpected mutations throughout the genome. The jury appears to still be out on the extent to which this occurs.

The second concern regards intellectual property. CRISPR-Cas9 as a technology was discovered by a University of California Berkeley professor Jennifer Goudna and Emmanuelle Charpentier of the Helmoltz Centre for Infection Research in Braunschweig, Germany.

There’s a big patent battle ongoing with Feng Zhang, a researcher at the MIT-Harvard Broad Institute, who filed a broad U.S. patent claim on the technology. The patent battle revolves quite a bit around changes in U.S. patent law in the last few years. For a long time, it was “first to invent,” meaning that whoever was the first person to invent or discover a technology was the patent owner. They had to prove it, which creates its own bars to hurdle. Then, in late 2011, the U.S. switched to “first-to-file,” and it went into effect on March 16, 2013.

Although some of these have been at least somewhat settled, others continue.

Speights is optimistic, and writes, “The possibilities for CRISPR gene editing are tremendous. Some envision a day when many genetic diseases can be cured, or even prevented altogether. Others see the potential for even more effective cancer treatments using genetically altered cells. Regardless of whether these dreams come to pass, the work that CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics are doing today could greatly change how diseases are treated in the future.”

1. CRISPR Therapeutics

With U.S. headquarters in Cambridge, Mass., CRISPR is working on developing therapeutics using its CRISPR/Cas9 gene-editing platform. Just yesterday the company announced it had signed a collaboration agreement with Casebia Therapeutics, a joint-venture established by CRISPR Therapeutics and Bayer AG with Tubingen, Germany’s CureVac AG.  Under the deal, CureVac will develop novel Cas9 mRNA constructs that improve on already existing gene editing applications.

2. Editas Medicine

Also based in Cambridge, Editas is also using CRISPR/Cas9 to develop therapeutics. On Oct. 19, the company released results from a pre-clinical study of its editing platform in transgenic mice. Its pre-clinical product candidate is for the treatment of Leber Congenital Amaurosis type 10 (LCA10), an inherited retinal degenerative disease caused by mutations in the CEP290 gene that can lead to blindness.

3. Intellia Therapeutics

Again, headquartered in Cambridge, Intellia is working on developing therapeutics using CRISPR/Cas9. On Nov. 1, it announced the pricing of its initial public offering of 6,250,000 shares of its common stack at $24 per share. Shares are currently trading for $19.80.

4. Illumina

Based in San Diego, Illumina is the world leader in gene-sequencing technology. And in January 2016, Illumina also launched Grail Bio, whose goal is to develop a blood test to identify all types of cancers.

Speights writes, “Not only has Illumina pioneered next-generation gene sequencing, a high-throughput method of mapping genes, it has also helped greatly reduce the costs, which in turn has paved the way for more research to be performed. Consider that in 2009, the average cost of sequencing a human genome, the complete set of DNA, was $200,000. Just five years later, the cost plunged to $1,000—thanks in large part to Illumina’s technology.”

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