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Sekar Kathiresan's older brother died prematurely from heart disease, which drove him into the study of heart disease genetics and led him to found Verve Therapeutics, aiming to use gene-editing technology to develop a one-time drug to prevent heart disease. This article reviews his upbringing, scientific breakthroughs, and entrepreneurial journey.
By constructing a dense patent network, Amgen extended the market exclusivity period for its arthritis drug Enbrel from the standard 20 years to 37 years, generating cumulative revenue of over $74 billion. This strategy has raised concerns among policymakers about drug pricing and faces legislative challenges.
After setbacks in its alpha-1 antitrypsin deficiency (AAT) drugs, Vertex Pharmaceuticals CEO Reshma Kewalramani and Chief Scientific Officer David Altshuler publicly defended the company's diversified R&D strategy. Despite the stock falling nearly 30% over the past year and mounting analyst criticism, the two emphasized that 'this is not the time to retreat but to double down,' citing the company's success in cystic fibrosis as evidence.
In the field of drug development, deals are as important as scientific breakthroughs. In recent years, large pharmaceutical companies have frequently acquired innovative biotechnology companies to supplement their product pipelines. This database compiles biotechnology M&A deals valued at least $50 million since 2018, covering information such as acquirer, target, deal date, total consideration, price per share, and premium, and is regularly updated.
The experimental CRISPR gene editing drug co-developed by Intellia Therapeutics and Regeneron Pharmaceuticals showed positive results in an early trial for treating the rare genetic disease transthyretin amyloidosis (ATTR). After treatment in six patients, levels of the disease-causing protein dropped significantly, with only mild side effects. The results, published in the New England Journal of Medicine, represent the first clinical evidence of CRISPR gene editing successfully treating a disease in the human body, and were hailed by experts as a 'milestone event in modern medicine.'