CRISPR approval could rearrange the health market

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The Future. The first treatment using the gene-editing tech CRISPR has already been approved in the US and UK for patients suffering from sickle cell disease. There are still a lot of kinks to work out concerning costs and patient safety in the broader market, but the health industry may be close to having treatments that can tackle notoriously challenging diseases like Alzheimer’s and Parkinson’s.

CRISPR, which WSJ describes as “a molecular pair of scissors that can be used to cut and modify a DNA sequence,” is almost ready for prescription.

  • The UK has already approved CRISPR-based treatments from Vertex Pharmaceuticals and Crispr Therapeutics that tackle sickle cell disease and beta-thalassemia.
  • The US FDA is set to approve a treatment for sickle cell disease created by Bluebird Bio sometime early next month.
  • Both use a process called “ex vivo,” which involves collecting cells from the patient, shipping them to a manufacturing facility, genetically manipulating them in the lab with CRISPR, and shipping them back to the hospital. That process is a bit complicated.
  • So, some firms like Intellia (co-founded by Jennifer Doudna who won the Nobel Prize in Chemistry) are working on a therapy for ATTR that’s “in vivo” — meaning the therapy is delivered directly to the patient.

Those approvals could open the floodgates for a variety of new therapies, especially since CRISPR tech is apparently easy to use and administer. The best part is, by changing a patient’s genome, the disease should be cured in one cycle.

It’s a little early to call CRISPR therapies “miracle drugs,” but they’ll still likely create some new powerhouse pharmaceutical companies in the process. Even pharmaceutical giants are opening their wallets to acquire firms working in the space. No one wants to catch a case of being behind.

David Vendrell

Born and raised a stone’s-throw away from the Everglades, David left the Florida swamp for the California desert. Over-caffeinated, he stares at his computer too long either writing the TFP newsletter or screenplays. He is repped by Anonymous Content.


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