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3 Big Developments In Biotech This Week

|Includes: IBB, RHHBF, RHHBY, Sarepta Therapeutics, Inc. (SRPT), XBI

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This week in biotech was off to a bit of a quiet start. But by the end of the day Thursday, we had quite a few interesting events that are without a doubt noteworthy.

First Liquid Biopsy Approval

In the second big development for cancer this week (we wrote about the first big development here), yesterday we found out that Roche received FDA approval for it's liquid biopsy test for the EGFR mutation in non-small cell lung cancer. These mutations are present in 10-20% of non-small cell lung cancers, which are the most common type of lung cancer. Perhaps even bigger news than the approval itself, is the fact that this is the first liquid biopsy to ever be approved for use in the United States. According to Alberto Gutierrez, Ph.D., director of the Office of In Vitro Diagnostics and Radiological Health in the FDA's Center for Devices and Radiological Health: "Approvals of liquid biopsy tests make it possible to deliver highly individualized health care for patients," He continued, "Liquid biopsies also have the potential to allow physicians to identify patients whose tumors have specific mutations in the least invasive way possible." Different liquid biopsies have the potential to detect the existence and details surrounding many different types of tumors. But this first-ever approval is big news because it shows the FDA's recognition of the science behind liquid biopsies. Going forward, liquid biopsies should be able to provide patients a less invasive technique for detecting tumors, and providing information about them.

Sarepta Scare/Simplified Compassionate Use

Thursday morning, the FDA simplified the process for requesting "compassionate use" for investigational drugs. Of course, with the Sarepta Therapeutics' (NASDAQ:SRPT) Eteplirsen decision hanging in the balance, investors did what they do best - speculated that the FDA was preparing to reject Sarepta's NDA for Eteplirsen, and ask the company to provide the drug under the expanded use program. This would ultimately be a negative for Sarepta, because they'd have to provide the drug at cost, while incurring other operating expenses.'s Adam Feuerstein posted some additional arguments on Twitter as well. If this ends up being the case for Sarepta, they might as well re-incorporate as a 501c Non-profit Organization, and pray they can raise enough money each year to cover the true costs of operations & labor. Luckily for now, an FDA spokesperson said that the updated compassionate use process has nothing to do with any company in particular.

New CRISPR System For RNA Targeting

Today, we learned that multiple universities have identified and characterized a new CRISPR system that targets RNA instead of DNA, named C2C2. C2C2 is an RNA guided enzyme capable of targeting and degrading RNA. The implications for this new method are pretty big:

The new approach has the potential to open a powerful avenue in cellular manipulation. Whereas DNA editing makes permanent changes to the genome of a cell, the CRISPR-based RNA-targeting approach may allow researchers to make temporary changes that can be adjusted up or down, and with greater specificity and functionality than existing methods for RNA interference.

Some possibilities include adding molecules to RNA sequences to change their function, as well as the ability to fluorescently tag RNAs to study their trafficking and subcellular localization. You can read more about this new system here.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.