![]() We have a combination of advanced diagnostic technologies coupled with computer processing power, artificial intelligence, and machine learning. We’re at the very beginning of a multiyear bull market for the biotech space. Van: For investors, that sounds like they should pay close attention to the biotech sector next year. We are still just at the beginning.Īnd once the applications of CRISPR are well understood, we’re going to continue to see an explosion of investment in this area. The novel therapies for these genetically caused diseases will absolutely continue in the new year. But I believe at least five CRISPR therapies will demonstrate in either preclinical or clinical trials that they have cured the diseases they were meant to cure. To be clear, I don’t expect that five therapies will be approved by the FDA in 2020. Jeff: So beta thalassemia and sickle cell disease were the first two, but I predict that CRISPR will cure at least five more diseases caused by genetic mutations in 2020. I’m expecting identical results compared to the first two patients.īut going off of Leiden’s comments, it appears CRISPR technology has cured these diseases caused by genetic mutations. The next step for Vertex is to expand the clinical trials to another 45 patients. Of course, it is not Food and Drug Administration (FDA) approved yet. We have cracked the biology of sickle cell and beta. This is amazing because patients with this disease typically need blood transfusions every 2–4 weeks.Īnd the sickle cell patient had not suffered a crisis in four months.Īnd Leiden stated directly, “We have essentially cured these diseases. The beta thalassemia patient had not needed a blood transfusion in nine months. Leiden talked about how two patients have been treated with CTX001 so far – one with sickle cell disease and the other with beta thalassemia. Bad mutations in the hemoglobin gene cause these blood disorders. ![]() Vertex has been working with CRISPR Therapeutics (CRSP) on a therapy for sickle cell disease and beta thalassemia. One of the most incredible presentations at the 2019 STAT Summit came from Vertex Pharmaceuticals (VRTX) CEO Jeffrey Leiden. It was the first year the event was held, and it was fantastic. Jeff: In November, I attended a biotechnology conference, the STAT Summit. Van: And when you look back at 2019, what CRISPR developments stand out to you? And thanks to CRISPR, we will cure diseases in patients years before symptoms ever present themselves. As a reminder, CRISPR can edit our genetic code as if it were software. Then, thanks to CRISPR genetic editing technology, we’ll be able to fix these mutations – these “typos” – in our DNA. We will have our genome – our genetic “blueprint” – sequenced to identify mutations in our genetic code that lead to disease. In the very near future, medicine will be proactive and personalized. This approach to medicine is reactionary. Very often, each patient is treated with a “one size fits all” therapy. Then, we visit a hospital, where our symptoms are treated with varying degrees of success. Jeff Brown: These days, we typically wait until we feel sick before we seek medical treatment. But for readers just joining us, could you get us up to speed on what precision medicine is? Van Bryan: Jeff, today we’re talking about precision medicine and your predictions in the space for 2020. We hope you enjoy today’s edition of The Bleeding Edge. Today, we continue Jeff Brown’s 2020 prediction series by taking a close look at an emerging technology trend: precision medicine.Īs you’ll see today, we’re on the cusp of a complete reordering of health care… and at the very beginning of a major bull run in biotech. Over the past couple days, we have seen what Jeff forecasts for 5G and AI. We hope you are spending the day with friends and family. Jeff, the team, and I would like to wish all readers happy holidays and a merry Christmas. Van Bryan here, Jeff Brown’s longtime managing editor.
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