Profiles
Months after winning approval for the first CRISPR gene-editing therapy, Vertex Pharmaceuticals is approaching a critical FDA review milestone for its non-opioid analgesic suzetrigine. The company's Chief Scientific Officer, David Altshuler, points out that 'target conviction' in targets with human causal biological evidence, along with a high-intensity strategy allocating over 70% of operating expenses to R&D, forms the cornerstone of its 'serial innovation.' From betting on cystic fibrosis in 2012 to now addressing pain, kidney disease, and diabetes, Vertex is attempting to prove its model is replicable.
Debra Weiss was persuaded to transition to GMP during her tenure at Shire, where she learned the essence of leadership. Now as COO of the Gates Medical Research Institute (Gates MRI), she applies this philosophy to global health projects such as tuberculosis vaccines, emphasizing the critical role of operations teams in scientific execution.
Marcus Conant, the doctor who identified early signs of AIDS in San Francisco in 1981, is now 87 years old and serves as Chief Medical Officer at American Gene Technologies. More than four decades later, he is still pursuing a cure for HIV and points out the gap between the public's perception that HIV is 'solved' and the reality of continuously rising infection numbers.
Gianluca Pirozzi is a senior vice president at Alexion Pharmaceuticals (AstraZeneca's rare disease unit), and his 10-year-old daughter Valentina has Smith-Magenis syndrome. This personal experience has given him a deep understanding of the struggles faced by families with rare diseases and has shaped his patient-centered approach to drug development.
The biopharmaceutical industry generally has business development leads, but the Chief Investment Officer (CIO) title is uncommon. Dr. Mayukh Sukhatme, President and Chief Investment Officer of Roivant, said in an interview that this atypical title precisely matches the company's atypical 'hub-and-spoke' operating model. This asset-centric, therapy-area-agnostic model has led to ten positive Phase 3 studies and multiple multi-billion-dollar deals. Sukhatme, combining a Wall Street investment background with medical training, explains how to track potential partners over the long term, strike at the right time, and emphasizes that 'the only way to guarantee being in the right place at the right time is to always be present.'
DBV Technologies' Chief Medical Officer Pharis Mohideen stated that after the FDA issued a Complete Response Letter in 2020, the company redesigned the patch and focused on younger patient groups, now seeing a clear approval pathway. Phase III trial results for patients aged 1-3 were published in the New England Journal of Medicine, and a new trial for patients aged 4-7 is expected to release top-line data by mid-2025.
After experiencing layoffs and terminated partnerships, SQZ Biotech founder and former CEO Armon Sharei shares his decade-long entrepreneurial insights, emphasizing the practical difficulties and future hopes in the biotech field.
Murray McKinnon, who helped bring blockbuster drugs such as Stelara, Tremfya, and Remicade to market, has chosen to join Empress Therapeutics, a startup with fewer than 40 employees, after over 35 years at major pharmaceutical companies. The company, incubated by Flagship Pioneering, aims to use genetics to find small-molecule drugs within the human body. Its platform has already produced 15 molecules, with plans to submit multiple IND applications over the next 24 months.
Dr. Vivian Cheung is a rare disease researcher who herself has a rare disease, and she has filed a discrimination lawsuit against the Howard Hughes Medical Institute after her contract was not renewed in 2018. The case will go to trial on December 4. This article recounts her personal experiences, her laboratory's discoveries in RNA research, and the systemic neglect of disabled scientists in academia.
On the first anniversary of the overturning of Roe v. Wade, Afaxys, a privately held pharmaceutical company named for 'affordability and accessibility,' is supplying oral contraceptives and other products to 8,400 health centers within the U.S. public health system through its unique low-margin, high-coverage model. CEO Ronda Dean, drawing from her own adolescent healthcare experiences, emphasizes the urgency of preserving the FDA approval system and the accessibility of reproductive health services.