CRISPR is (Gene) Editing The Future For Those Who May Not Have a Long One

Weight loss drugs aren’t the only miracle drugs making their way into the American diet. This one is completely rewriting the future for those who have all but lost hope. Over 2K gene therapies are in the pharma pipeline to treat difficult-to-cure and life-threatening diseases — but one kind of therapy stands out: gene-editing technology CRISPR, which slices and dices DNA and replaces it with new strands.

Into the microscope lens: Its significance was highlighted when the discovery won the 2020 Nobel Prize in chemistry. Now, CRISPR is leaving the lab. Over 200 people have been treated with experimental CRISPR therapies, and studies could clear the way for more treatments soon:

• At the end of 2022, there were 71 ongoing clinical trials related to CRISPR — with 45 directed towards human products.
• CRISPR therapies will come in two flavors: in-vivo treatments (gene editing inside the body) and ex-vivo treatments (infusing edited genes back into the body).

US drug regulators weigh first CRISPR therapy

The initial CRISPR treatments have targeted simple-to-edit diseases like sickle cell disease or beta-thalassemia, known for causing severe pain. A mother of one patient told NYT, “Gene therapy is our last hope.” The US Food & Drug Administration now deems CRISPR therapy “safe enough” for sickle cell disease patients and a final approval decision is expected in December — opening the doors for this groundbreaking therapy to be approved and possibly many more.

• CRISPR Therapeutics (NASDAQ:CRSP), which developed the sickle cell therapy in collaboration with Vertex Pharmaceuticals (NASDAQ:VRTX), is also working on therapies for cancer, diabetes and ALS.
• Intellia Therapeutics (NASDAQ:NTLA) and Editas Medicine (NASDAQ:EDIT) are both building similar products.

Life saving? Yes. Outrageously expensive? Also yes. The first CRISPR therapy costs between $1.35M-2M — while other treatments are estimated to be as much as $3M. According to Bloomberg, industry execs have justified the high cost by saying the lifetime treatment costs could run into the millions. But Jennifer Doudna, one of the researchers who discovered CRISPR, is “confident we can reach a point where genome editing reaches all who would benefit most” (IGI).