Over the past two days, we looked at the state of the biotech industry (Pt.1) and talked about ways to invest in biotech using ETFs (Pt.2).

Today, we finish off the series by saying happy belated 10th birthday to CRISPR… sorry, who?

Nearly ten years ago, researchers made a breakthrough that changed the course of medicine…

Genetically modified superhumans

CRISPR is a gene-editing tool used to find specific DNA and alter or remove it. It was first discovered in 1987, but in 2012, scientists found that CRISPR can also be used to edit human cells.

Since the breakthrough, awards were won, CRISPR companies were founded and scientists were off to the races. Potential applications were immediately evident.

CRISPR is being tested to cure blindness with signs of progress, and other treatments for cancer, blood disorders and chronic infections are in clinical trials.

But messing with the human body doesn’t come without problems.

• Ethics: In 2018, Chinese scientists created the first gene-edited baby using CRISPR. With the human imagination, who knows how far we’ll use the power to edit genes…
• Safety: CRISPR has several side effects, including mutations and increased odds of triggering cancer under some treatment.

Coming soon to a lab near you….

The first FDA-approved CRISPR drug could come as early as next year. Vertex Pharmaceuticals (NASDAQ:VRTX) and CRISPR Therapeutics (NASDAQ:CRSP) are developing a CRISPR treatment together named exa-cel.

• The treatment targets two rare blood diseases, beta-thalassemia and sickle cell disease.
• In June, clinical trials for exa-cel showed strong results and are expected to be submitted for FDA approval by the end of 2022.

Verve Therapeutics (NASDAQ:VERV) is developing gene-editing medicines targeting heart disease. Yesterday, they dosed the first person as part of their clinical trials after successfully testing it on monkeys.

Investors: Challenges to adoption

Per Bloomberg biotech columnist Lisa Jarvis, the tech is still early and faces three significant challenges:

• Precision: In editing, genes can be easily turned off, but altering them is still challenging.
• Affordability: Treatments are expected to be costly, with the exa-cel treatment costing up to $2M per patient.
• Delivery: Editing works well in labs, but implementing CRISPR in human cells is difficult.

This could either be the start of Resident Evil or X-Men.

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