Regeneron Leads $60 Million Consortium to Accelerate UK Biobank Genome Sequencing for Alzheimer's and Parkinson's Drug Discovery
核心洞察
Regeneron has formed a consortium with AbbVie (搜索), Alnylam, AstraZeneca, Biogen, and Pfizer, with each company committing $10 million to accelerate sequencing of 500,000 UK Biobank (搜索) genomes by end of 2019.
The project aims to identify new biological targets for drug development, particularly for neurological diseases like Alzheimer's and Parkinson's, where current failure rates exceed 90%.
Regeneron's automated sequencing capabilities through its Genetics Center have dramatically reduced processing time, potentially revolutionizing drug discovery by linking genetic data with health records from 500,000 volunteers.
Regeneron Pharmaceuticals has assembled a major pharmaceutical consortium to dramatically accelerate the sequencing of 500,000 genomes from the UK Biobank (搜索), targeting breakthrough discoveries for Alzheimer's disease (搜索), Parkinson's disease (搜索), and other challenging conditions. The consortium includes AbbVie (搜索), Alnylam, AstraZeneca, Biogen, and Pfizer, with each company contributing $10 million to complete the ambitious sequencing project by the end of 2019.
The UK Biobank (搜索) project, originally launched over a decade ago, reached its initial milestone of collecting 500,000 genetic samples from volunteer NHS patients in 2010. However, the sequencing of these samples has proceeded slowly, with Regeneron and GSK previously funding the first 50,000 sequences completed last year. The new consortium funding represents a significant acceleration of the timeline, moving the completion date forward by several years.
Automated Sequencing Revolution
Regeneron's unique position in the pharmaceutical industry stems from its dedicated genetics division, The Regeneron Genetics Center (搜索) (RGC), which has developed highly automated sequencing capabilities. According to Aris Baras, head of the Regeneron Genetics Center, the company has automated nearly every step of the sequencing process, with robots now handling tasks such as filling flasks with reagents.
"This is going to revolutionise drug discovery and development," Baras stated in an interview. The automation, combined with increased computing power, has vastly accelerated a process that originally took a decade when first attempted at the end of the last century.
The RGC maintains one of the world's most comprehensive genetics databases, pairing sequenced exomes with de-identified electronic health records from more than 180,000 people. This integration of genetic and clinical data provides unprecedented insights into disease mechanisms and potential therapeutic targets.
Targeting Neurological Diseases
The consortium's focus on neurological conditions addresses one of medicine's most challenging frontiers. Unlike cancer (搜索), where biological mechanisms are increasingly well understood, the underlying causes of Alzheimer's and Parkinson's diseases remain largely mysterious, contributing to exceptionally high failure rates in drug development.
The pharmaceutical industry has witnessed a string of failures in Alzheimer's research, with only a single new Parkinson's drug approved in the last decade. Current drug development success rates hover around 10%, with the remaining 90% of candidates failing to reach patients, often due to incomplete understanding of the relationship between biological targets and disease mechanisms.
Baras expressed particular optimism about the potential for neurological disease research, noting that "for many reasons a lot of those diseases have not had the opportunity to have large scale gene sequencing. There is a lot of hope there – the Parkinson's community has done a lot of the fundamental work, there are large cohorts and some of the first round of analysis."
Expanding the Target Universe
The project aims to dramatically expand the universe of potential drug targets beyond the approximately 1,000 molecular targets currently exploited by the pharmaceutical industry. With around 20,000 active genes in the human genome, researchers believe they are "merely scratching the surface" of therapeutic possibilities.
Particularly valuable are patients with "knock out" mutations – individuals who lack certain genes entirely. These natural experiments provide crucial insights into gene function and suggest how specific genes could be targeted therapeutically, either by inhibiting or amplifying their activity.
The sequencing focuses on exomes, the protein-coding sections of DNA that represent about 1% of the total genome but contain roughly 85% of disease-causing mutations. This targeted approach maximizes the clinical relevance of the data while maintaining cost efficiency.
Data Sharing and Future Applications
The consortium operates under a "pre-competitive" model, with members sharing costs and initial access to data before making findings publicly available. After a limited exclusivity period, all sequencing data will be accessible to the broader scientific community through the UK Biobank (搜索), and consortium members have committed to publishing significant research findings in peer-reviewed journals.
Beyond neurological diseases, the project could yield insights into other challenging conditions such as non-alcoholic steatohepatitis (搜索) (NASH (搜索)), the fatty liver disease currently targeted by numerous pharmaceutical companies. The comprehensive genetic and health data linkage enables researchers to identify previously unknown disease mechanisms and validate potential therapeutic approaches.
GSK's president of R&D, Patrick Vallance, emphasized the collaborative nature of the effort: "Having been actively involved in UK Biobank (搜索) as a board member since 2013, I'm delighted that, through our collaboration with Regeneron, we can enrich this resource for the wider scientific community and also provide potential new opportunities for companies such as ours to develop new medicines for patients."
The UK Biobank (搜索) continues to track participants' health for 25 years or more, creating an unprecedented longitudinal dataset linking genetic information with real-world health outcomes. This long-term follow-up capability distinguishes the project from other genetic databases and enhances its value for understanding disease progression and treatment responses.
Additional pharmaceutical companies are reportedly considering joining the consortium, potentially expanding both the funding base and the collaborative expertise applied to the sequencing effort. The project represents one of the largest coordinated efforts to translate population-scale genetic data into therapeutic insights, with implications extending far beyond the participating companies to benefit global drug discovery efforts.