Core Insights - The company has shifted its business model from partnering with pharmaceutical companies to producing and commercializing its own drugs in-house, aiming to retain more value and control over its products [3][4][16] - The focus is on developing anti-sense drugs for severe rare diseases, with recent approvals for treatments targeting high triglyceride levels and related conditions [5][6][7][8] Company Strategy - The CEO emphasizes the importance of controlling the drug development process to ensure urgency in addressing patient needs and maximizing reinvestment into research and development [4][16] - The company has a history of successful drug development, with a strong pipeline that includes four FDA-approved medicines in the last two years and three more expected next year [16] Drug Development Focus - The company has recently received approval for a drug called olarsen, which significantly reduces triglyceride levels by 72% and lowers the risk of pancreatitis by 85% in patients with severe hypertriglyceridemia [7][8] - The company is also working on treatments for rare neurological diseases, having previously developed drugs like Spinraza for spinal muscular atrophy and a treatment for ALS in partnership with Biogen [12][13] Market Potential - The company believes that the prevalence of rare diseases will become better defined as new treatments become available, leading to increased diagnosis rates and market opportunities [14][15] - Investors are showing strong interest in the company's new business model and the potential for high rewards despite the inherent risks associated with rare disease treatments [4][16]