Gene editing is revolutionizing medicine, but not all companies are benefiting equally. While one company's stock soared, another's barely budged, even with promising advancements. Let's dive into why, and what it means for the future of healthcare.
At the American Heart Association conference, Crispr Therapeutics (CRSP) revealed exciting results. Their gene-editing drug showed impressive dose-dependent reductions in ANGPTL3, a protein crucial for processing lipids like LDL cholesterol and triglycerides. This could potentially offer a 'one-and-done' treatment for patients with genetic high cholesterol and triglyceride levels.
The data revealed that patients treated with Crispr's CTX310 experienced significant ANGPTL3 reductions after 30 days. The low-dose group saw about a 10% reduction, the middle dose achieved 33% to 80%, and the highest dose resulted in an impressive 89% reduction. Furthermore, the highest dose led to a 55% decrease in triglycerides and a 49% reduction in LDL cholesterol. This is a significant positive development for Crispr.
But here's where it gets controversial: despite these promising results, Crispr's stock traded sideways. Why?
Meanwhile, Intellia Therapeutics (NTLA) presented compelling data on their gene-editing treatment for hereditary angioedema (HAE), a severe swelling disorder. In a study, 10 out of 11 patients were attack-free at two years after treatment. The remaining patient saw a 59% reduction in swelling attacks. This is considered a 'functional cure' for HAE.
Intellia's stock surged on this news. However, this positive momentum was tempered by a setback. A patient died after receiving a gene-editing treatment for transthyretin amyloidosis, leading investors to question the safety of Intellia's approach.
Crispr's approach to treating high cholesterol and triglycerides could potentially compete with existing lipid-lowering treatments. They are testing their gene-editing approach in patients whose cholesterol and/or triglycerides remained high despite treatments like statins, Merck's Zetia, or PCSK9 inhibitors from Regeneron Pharmaceuticals, Sanofi, and Amgen.
Crispr Therapeutics also reported its third-quarter results. Sales of their approved gene-editing treatment, Casgevy, are gaining traction, potentially exceeding $100 million this year. The company will also have test results in children at the upcoming American Society of Hematology meeting in Orlando. However, they have deprioritized CTX131, a potential treatment for solid tumor cancers.
Intellia's gene-editing platform, which edits genes inside the patient's body (in vivo editing), has raised safety concerns. While 24 out of 27 patients remained free of HAE attacks at six months, some analysts point out that three patients are still experiencing attacks, which weakens the 'one-time cure' proposition. Additionally, Ionis Pharmaceuticals' Dawnzera has shown a median of 2.7 years of attack-free periods, with 71% attack-free after a year.
So, what does this mean for the future? Both companies are making strides, but challenges remain. Crispr is facing market competition, and Intellia is dealing with safety concerns.
What are your thoughts? Do you think the potential benefits of gene editing outweigh the risks? Share your opinion in the comments below!
