The Gene‑Therapy Breakthrough Story You'll Be Mad You Ignored at These Prices

Core Viewpoint - Vertex Pharmaceuticals' stock has increased following its fourth-quarter results, but long-term investment potential lies with its partner CRISPR Therapeutics, which developed the gene-editing therapy Casgevy [1][2]. Group 1: Vertex Pharmaceuticals - Vertex reported higher sales projections for Casgevy, a gene-editing therapy for sickle cell disease and beta thalassemia, contributing to its profitability and revenue from cystic fibrosis treatments [2]. - The company's share price experienced a short-term spike but moderated afterward, losing most of its gains [2]. Group 2: CRISPR Therapeutics - CRISPR Therapeutics, based in Switzerland, is the developer of Casgevy, the first CRISPR-based gene therapy approved by the FDA, and stands to benefit from increased sales of this therapy [3]. - The company has a strong position in the market, as evidenced by its significant holding in Cathie Wood's Ark Innovation ETF, indicating investor confidence in its potential [4]. - CRISPR has five additional gene therapy candidates in clinical trials, which target larger patient populations than Casgevy, enhancing its long-term growth prospects [4]. - Notable candidates include CTX310, which has shown to reduce triglycerides and LDL cholesterol by over 80%, and CTX320 for atherosclerotic cardiovascular disease [5]. - The company is also developing SRSD107, a long-acting gene therapy for thrombosis, and CTX211, which aims to restore insulin production in type 1 diabetes patients [6]. - Despite not being profitable and experiencing a 64% decline in share price over the past five years, CRISPR has over $1.9 billion in cash, positioning it well for future development if Casgevy sales increase as anticipated [7].