2. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)
Number of Hedge Fund Holders: 57
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a biotechnology company focused on developing and commercializing therapies for cystic fibrosis. It is one of the best gene editing stocks to buy. On January 16, Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced that it recently received FDA approval for its groundbreaking gene editing therapy, Casgevy, developed in collaboration with CRISPR Therapeutics. The approval is for individuals aged 12 years and older with transfusion-dependent beta-thalassemia (TDT). This follows the earlier FDA clearance for Casgevy in sickle cell disease, marking it as the first gene editing therapy to be approved in the U.S. Vertex also announced that nine clinical centers in the U.S. are now authorized to provide Casgevy for TDT patients, with additional sites scheduled to be launched in the coming weeks.
According to Insider Monkey’s third quarter database, 57 hedge funds were bullish on Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX), compared to 54 funds in the last quarter. Ian Simm’s Impax Asset Management is the largest stakeholder of the company, with 558,138 shares worth $193.7 million.
Global Equity Strategy stated the following regarding Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) in its fourth quarter 2023 investor letter:
“Health Care outperformed due to a strong showing by US-based Vertex Pharmaceuticals, even as other holdings in the sector posted weak returns. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) reported positive phase 2 trial data for a drug to address diabetic neuropathic pain.
The potential for technological innovation to create shareholder value goes far beyond the effects of Al, of course. For example, Vertex Pharmaceuticals is building a significant competitive advantage through its unique approach to drug discovery.
Unlike most drug developers, which focus on mitigating symptoms of an illness, Vertex seeks to understand and address the causal biology of a disease more fully. This approach has helped the company establish a wide lead in treating cystic fibrosis (CF), a rare disease that causes mucus buildup in organs such as the lungs. Vertex’s research and treatments have targeted the root cause of CF, which is that a particular protein becomes misfolded and unable to move chloride out of a cell, resulting in an imbalance between salt and water. Its scientists have been able to do this by working with the lung cells of real CF patients, an advantage over using engineered animal cells to simulate the disease-causing mechanism to determine which drug candidate is most promising for humans. Vertex’s latest therapy, approved in 2019, is Trikafta, a combination of three medicines that targets a mutation found in 90% of CF patients. Vertex is now using the same approach to address other illnesses. In 2023, Casgevy, its treatment for sickle cell disease, became the first CRISPR-based gene-editing therapy to receive regulatory approval. (CRISPR, a technology honored by the Nobel Prize in 2020, allows scientists to make precise changes to parts of the genome, the set of DNA instructions found in a cell.) By correcting the faulty gene that causes sickle cell disease, Casgevy could offer a one-time therapy to cure approximately 16,000 patients who have the red blood cell disorder-a significant improvement over the usual course of treatment, which is merely to manage symptoms through blood transfusions and pain medicines…” (Click here to read the full text)
