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Another Win for CRISPR By Jeff Brown, Editor, The Bleeding Edge 
Caused by an altered gene that controls the C1 inhibitor protein, hereditary angioedema (HAE) is a rare genetic disease. It can cause swelling in the stomach, gastrointestinal tract, or mouth/throat, which can potentially be fatal. It’s one of more than 5,000 diseases caused by genetic mutations that have no known cure – despite our understanding of the root cause. And it is a great example of a disease that may be cured for life through the use of CRISPR genetic editing technology. Not Real Solutions More than 150,000 people worldwide have the genetic mutation leading to HAE, and the average age of diagnosis is 20 years old. This in itself is a problem, as symptom onset typically occurs by 12 years of age. This is a fundamental problem with the global healthcare system today. And it is something that could easily be solved with childhood genetic screening. A simple genetic sequencing of a child would reveal any known genetic mutations that are associated with a disease. Catching the mutated gene responsible for HAE is actually easy to do… as long as a patient’s whole genome is sequenced to find it. Doing this would have been impossible 20 years ago, as the cost to sequence a single whole human genome was more than $10 million. 
Source: National Institutes of Health But today, the price of sequencing is well below $1,000 – currently around $200-300 at scale. And with Illumina’s latest generation NovaSeq X sequencing machines, the cost will rapidly drop to just $100. That price will be accessible to most of the global population. And at current levels, it makes sense for insurance companies to pay for the diagnostic test as well. The reality is that understanding the root cause of a disease like HAE can save millions for these insurance companies – by not wasting money on therapies for symptom management that won’t in any way help cure the disease. But what can patients with HAE do about it? Patients with HAE suffer dangerous swelling attacks that can be triggered by a range of conditions. The attacks can result in hospitalization, chronic treatment to manage the disease, and death. A disease like HAE requires incredible vigilance, living cautiously, and always being close to emergency care. Today, there are only two options for HAE patients that provide some support for managing the swelling attacks. BioCryst has Orladeyo, which is taken once a day. And Takeda has Takhzyro, which is administered every two to four weeks. Ionis Pharmaceuticals is also in a Phase 3 trial that looks to reduce swelling linked to HAE. But none of these therapies cure the disease. And all of them need to be administered regularly for life. These aren’t really solutions. And the annual cost of the leading prophylactic treatment for HAE is around $500,000 every year. These are just the only options available today to provide some level of reduction in swelling attacks. But since last week, there is hope. And it comes from Intellia Therapeutics (NTLA) – one of the original pioneers in CRISPR genetic editing. | Recommended Link | | | | Chaos is Coming as Soon as November 7th Legendary "Market Wizard" says there are no safe havens for this one. He predicted the 2020 crash and the 2022 crash, and he had one of the best years of his life during the 2008 crash. Now he has a new prediction that's incredibly dire and urgent for the near future… It's tied directly to the election – and you have less than a week to prepare. In an exclusive interview Tonight, October 30, at 8 p.m. ET, he'll explain everything and reveal a free ticker for the coming chaos. | | | -- | Stunning Trial Results Intellia announced the results of its Phase 2 study of NTLA-2002, which is an in vivo genetic editing therapy designed to provide a lifelong cure for HAE. In vivo refers to a therapy that can be directly administered to a patient. In this case, shown below, NTLA-2002 is packaged in lipid nanoparticles and administered through an IV, which sends the therapy to the liver. 
Source: Intellia Therapeutics The CRISPR guide RNA finds the correct gene that it needs to edit and disables that gene. More specifically, CRISPR knocks out the KLKB1 gene, which reduces the production of kallikrein, an enzyme. This is relevant because too much kallikrein results in the overproduction of bradykinin – a peptide that can cause smooth muscle contractions and blood vessel dilation – which is what causes the swelling attacks. By knocking out the trouble-causing gene, in theory, the swelling attacks will greatly subside. But here is the best part. With an in vivo genetic editing therapy, only one shot is required. And a patient can be cured for life. Once the genetic edits have taken place, there is no longer any need for further therapy. This is why Intellia’s Phase 2 results were so exciting: The Phase 2 trials were randomized, with 27 patients receiving either a 25mg or 50mg dose or a placebo. Intellia decreased monthly swelling attacks by 75% and 77%, respectively, for the low and high doses. The reduction rates rose to 80% and 81% when looking at attacks from weeks 5 to 16 of the trial. This is not surprising, as it takes some time for the genetic edits to proliferate in the patient’s cells. Better yet, 8 of the 11 patients who received the 50mg dose were completely attack-free after just the single infusion of NTLA-2002. This suggests a functional cure for HAE. And there were no serious adverse events. Stunning results – and life-changing news for the HAE community. And yet, Intellia’s stock has dropped by 25% since it announced the incredible results. Absolutely nuts. In a healthy biotech market (not even a bull market), these results would have sent Intellia’s stock screaming. Institutional Malaise A functional cure to a genetic disease… With a global market size around $6 billion… And the stock drops. This is nothing but a reflection of the current state of the small-capitalization segment of the stock markets. Persistent inflation and high interest rates continue to keep institutional capital out of the biotech sector, especially biotech companies that are still in research and development mode (i.e., still burning cash). The general institutional malaise for biotech can easily be seen in the chart below of the equally weighted SPDR S&P Biotech ETF (XBI). 
While XBI is up about 50% from its 2022 and 2023 lows, it is nowhere near the ETF’s highs in 2021. Biotech remains like a coiled spring. Major breakthroughs are happening every month, largely catalyzed by the ever-increasing power of artificial intelligence. Genetic sequencing costs have plummeted, making AI-assisted data collection and analysis cheap. And CRISPR genetic editing technology continues to prove its utility, allowing scientists to correct unwanted mutations – almost like finding and fixing bugs in a software program. The future is ridiculously bright. Interest rates will fall materially in the next couple of years as economic and fiscal policy is returned to a state that stimulates growth and curtails rampant money printing and devaluation of the dollar. This will not only be good for the biotech industry – it will be great for the stock markets. And it will be even better for our health, as we’ll develop a new generation of therapies to cure thousands of diseases that currently have no known cure. Regards, Jeff | 
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