Q&A
Three Major Challenges in Gene Therapy: Delivery, Delivery, and Delivery Again - 4D Molecular Therapeutics' Approach
Gene therapy is moving from rare diseases to broader markets, but delivery efficiency remains the core bottleneck. 4D Molecular Therapeutics uses directed evolution to optimize adeno-associated virus vectors, and collaborates with Arbor Biotechnologies and brings in UCSF professor Noriyuki Kasahara, aiming to elevate delivery capabilities to new heights. CEO David Kirn details the technical path and industry prospects.
Arbor Biotechnologies: Expanding the CRISPR Toolbox, Targeting More Disease Targets in the Liver and Central Nervous System
Arbor Biotechnologies is a preclinical-stage biotechnology company co-founded by CRISPR pioneer Feng Zhang and Illumina founder David Walt. The company aims to develop therapies for liver and central nervous system diseases by creating novel gene-editing tools that go beyond the limitations of existing Cas9 technology. CEO Devyn Smith stated that the company's lead program (targeting hyperoxaluria) is nearing the IND submission stage, with plans to file three applications over the next three years. The company is also collaborating with Vertex Pharmaceuticals to provide more precise gene-editing tools.
The Multiple 'Myths' of Clinical Trial Diversity and Genentech's Breakthrough Battle
Genentech's Chief Diversity Officer Quita Highsmith points out that there are multiple myths about clinical trial diversity and introduces how the company, through studies like Chimes, has proven that engaging diverse patients does not compromise scientific rigor.
Former CDC Director on Key R&D Areas, Public Trust, and Pandemic Lessons
Dr. Julie Gerberding, CEO of the Foundation for the National Institutes of Health and former CDC Director, shares insights in an exclusive interview on R&D hotspots such as cancer immunotherapy, women's health, ALS, preeclampsia, and mental health, as well as topics including rebuilding public health trust and PAHPA reauthorization.
Bristol Myers Squibb Business Leader Discusses 27 Years of 'Sea Changes' in the Industry and Future Directions
Adam Lenkowsky, who has spent over 26 years at Bristol Myers Squibb, rose from a sales representative to Chief Commercial Officer. He has witnessed multiple strategic shifts as the company transitioned from a diversified conglomerate to a pure biopharmaceutical firm, and has observed profound changes in industry innovation speed, market access policies, and digital marketing. In light of the portfolio expansion from the Mirati acquisition, he outlines the evolution of future commercialization models and his predictions regarding the impact of the Inflation Reduction Act in 2024.
Tibetan patients and advocacy offices that may face sudden raids - Gilead executives discuss the global HIV response
Janet Dorling, Senior Vice President at Gilead Sciences, visited nine countries within her first 90 days on the job, engaging with local healthcare providers and community members, and gaining a deep appreciation for the daily dangers HIV advocates face in some countries. She emphasizes advancing global health equity through purposeful partnerships and reveals that the company is exploring long-acting HIV treatments while still in the building phase in oncology.
AstraZeneca's Digital Health Business Leader on How the Pharma Giant Leverages Technology
Cristina Durán, President of AstraZeneca's digital health company Evinova, gave an exclusive interview exploring the value of data in drug development. She noted that technologies such as telemedicine and home monitoring help detect diseases earlier, and shared strategies for platform-based solutions across therapeutic areas, as well as a cautious approach to emerging technologies like AI.
Organon Chief Medical Officer: Reshaping Global Perceptions of Women's Health
Dr. Juan Camilo Arjona Ferreira, Chief Medical Officer at Organon, joined the company in September 2023, committed to advancing women's health with a fresh perspective. He proposes a three-dimensional matrix strategy that views the female life cycle as a continuum, emphasizing scientific leadership and inclusive research, while also focusing on 2024 pipeline progress and policy opportunities.
AstraZeneca Study Reveals: The Pandemic Is Far from Over for Immunocompromised Populations—What Comes Next?
AstraZeneca released a report indicating that among immunocompromised populations (such as cancer patients and organ transplant recipients), over 10% still fail to produce protective antibodies even after multiple vaccinations. Its two real-world studies, covering millions of health records in the UK and the US, found that immunocompromised individuals accounted for 22% of COVID-19 hospitalizations, 28% of ICU admissions, and 24% of deaths in 2022. Based on an interview with Professor Paul Moss from the University of Birmingham, UK, this article explores preventive antibody therapies, individualized risk assessment, and future policy directions.
