We recently published a list of 8 Best Gene-Editing Stocks to Buy. In this article, we are going to take a look at where Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) stands against other best gene-editing stocks to buy.
Gene editing is an advanced medical technique within gene therapy that involves precisely modifying an individual’s DNA to treat or prevent diseases. This approach directly alters genetic material to correct mutations, enhance cellular functions, or eliminate disease-causing genes. Industry experts believe that gene editing has the potential to revolutionize the treatment of genetic disorders, cancers, and various other conditions.
According to IMARC, the U.S. healthcare infrastructure has adapted to support gene-editing therapies. IQVIA reported that 114 gene therapy trials were initiated in 2023, with approximately 77% sponsored by the healthcare industry. Kella Kapnisi, Head of Cell and Gene Therapy at Team Consulting, noted that the FDA has approved 38 cell and gene therapies, many of which have reached commercialization through predominantly manual laboratory manufacturing processes.
The U.S. gene editing market has experienced significant growth, valued at $3.19 billion in 2024, while genome editing stood at $3.55 billion in 2023. Looking ahead, projections indicate a substantial surge, with gene editing expected to reach $13.99 billion and genome editing forecasted at $16.49 billion by 2034. These growth trends reflect compound annual growth rates (CAGRs) of 15.93% and 16.6%, respectively, underscoring the increasing adoption and investment in gene-editing technologies.
Several breakthrough therapies are paving the way for advancements in gene editing. Precision BioSciences’ PBGENE-HBV represents the first FDA-cleared in vivo gene-editing trial for chronic hepatitis B. Early data from the trial indicate a 70% reduction in the hepatitis B surface antigen (HBsAg) in two out of three patients at the lowest dose (0.2 mg/kg), targeting covalently closed circular DNA (cccDNA) to address the root cause of HBV persistence. YolTech Therapeutics has also made strides with its hyperoxaluria treatment, demonstrating a 70% reduction in harmful oxalate levels in patients with primary hyperoxaluria type 1 through lipid nanoparticle-delivered gene editing. Additionally, AccurEdit’s cholesterol therapy has shown promising results, achieving a 70% reduction in LDL cholesterol with a single-dose treatment by silencing PCSK9.
The number of patients receiving gene therapies is expected to fluctuate over the next decade. In 2020, 16,244 patients were treated, with this figure projected to rise to 94,696 by 2025 before gradually declining to 65,612 by 2034 as existing patient stocks deplete. Over the next decade, an estimated 1.09 million patients could benefit from gene therapies, with cancer patients making up approximately 48% of recipients.
From a financial and economic standpoint, annual spending on gene editing therapies is anticipated to peak at $25.3 billion in 2026 before stabilizing at $21.0 billion by 2034. These therapies are projected to yield an additional 5.12 quality-adjusted life years (QALYs) per patient, with each QALY valued at $43,110. Insurance coverage is expected to play a crucial role in facilitating access, with Medicare, Medicaid, and private insurers projected to cover $8.1 billion, $5.44 billion, and $12.2 billion annually, respectively.
Despite these advancements, the gene-editing industry faces key challenges. Delivery risks remain a concern, as adverse events associated with viral vectors and conditioning regimens pose safety hurdles. Regulatory scrutiny is also increasing, with the FDA’s pending decision on Abeona Therapeutics’ recessive dystrophic epidermolysis bullosa (RDEB) therapy, expected by April 29, 2025, highlighting the evolving standards for gene-editing approvals.
For this list, we scanned Insider Monkey’s database of over 1,000 hedge funds as of Q4 2024 and selected companies involved in the development and commercialization of gene-editing technologies. From there, we picked eight companies with the highest number of hedge fund investors and ranked them in ascending order.
Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter’s strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (see more details here).
Is Vertex Pharmaceuticals Incorporated (VRTX) the Best Gene-Editing Stock to Buy?
A pharmacist delivering a specific medication to a patient in a specialty pharmacy.
Number of Hedge Fund Holders: 68
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a biotechnology company that develops transformative medicines for serious diseases, with a strong focus on gene editing, particularly in collaboration with CRISPR Therapeutics. The company specializes in treatments for genetic diseases, especially cystic fibrosis, and generates revenue by selling medications to patients with rare genetic conditions. Recently, the company has expanded its focus to include sickle cell disease and beta-thalassemia.
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has partnered with CRISPR Therapeutics to develop CASGEVY (exagamglogene autotemcel), a gene-edited cell therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). This treatment involves editing the BCL11A gene in a patient’s stem cells to increase the production of fetal hemoglobin, which helps manage these inherited blood disorders.
The company’s Q4 2024 financial results highlight continued growth, with a 16% increase in revenue from the same period in 2023, reaching $2.91 billion. Full-year 2024 revenue reached $11 billion, exceeding expectations. The growth was driven by strong sales of CF treatments and the launch of CASGEVY. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is also expanding its reach with new therapies like ALYFTREK for CF and JOURNAVX for acute pain, solidifying its place among the best gene editing stocks.
With a broad total addressable market (TAM) covering diseases like CF, SCD, TDT, acute pain, type 1 diabetes, and renal diseases, the company aims to treat millions of patients worldwide. The business is focused on growing its market presence and advancing its innovative therapies.
Overall, VRTX ranks 2nd on our list of best gene-editing stocks to buy. While we acknowledge the potential of VRTX, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns, and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than VRTX but that trades at less than 5 times its earnings, check out our report about thecheapest AI stock.
