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What biotech innovation needs

Jun 05,2022 - Last updated at Jun 05,2022

By Phillip A. Sharp and Julien Patris

CAMBRIDGE  —  The COVID-19 pandemic has reshaped attitudes toward public health, fiscal policy, and the state’s role in the economy. Demands for greater supply-chain resilience and strategic autonomy in developing and producing medicines have given rise to the concept of “life-science sovereignty”.

French President Emmanuel Macron, for example, has announced an ambitious plan calling for France to produce at least 20 new biotherapies by 2030. With financing from the French public investment bank, his government’s La French Care initiative aims to support the domestic biotech ecosystem and turn France into a “pioneer mRNA nation”. Similarly, many other governments, from The Netherlands to the United Kingdom, are doubling down on their domestic biotech sectors.

This attention is welcome, but will it be enough? As the COVID experience has shown, securing approvals for a handful of vaccines and therapeutics requires hundreds of clinical trials for existing and new compounds, many of which fail. Medical innovation is expensive, and the costs and risks associated with it tend to be misunderstood by policymakers and ordinary citizens alike.

Consider the story behind ribonucleic acid interference (RNAi) therapeutics, a new category of medicines that target the genetic causes of disease and use small interfering RNA (siRNA) to “turn off” harmful proteins at their source. These treatments have virtually unlimited potential, but the journey from scientific possibility to real opportunity for patients has been very long.

The discovery of the structure and function of DNA in the 1950s launched a sustained research effort to understand the biological mechanisms underpinning the process of gene expression. Building on those breakthroughs, Andrew Fire and Craig Mello discovered RNAi, or “gene silencing”, in 1998, for which they won the Nobel Prize in 2006.

Fire and Mello’s discovery generated widespread excitement about siRNA’s possible use as a new type of therapy. Pharmaceutical companies invested heavily in this new area of research, but they soon encountered technical challenges in turning RNAi technology into therapeutics. The most salient problem was how to deliver siRNA to the right place in the human body for it to work effectively (that is, to the organ in which the disease gene was expressed). The difficulties of navigating this uncharted territory led many researchers and companies to lose hope.

By the early 2010s, most large pharmaceutical firms had terminated their investments in the technology entirely. Only a handful of companies, including our own, Alnylam, remained committed, eventually solving the delivery problem with lipid nanoparticles (LNP) as a carrier for RNAi. There are now four RNAi therapeutics approved by the US Food and Drug Administration and the European Medicines Agency, and LNPs are being used in the mRNA vaccines for COVID-19. But it is important to remember that it took Alnylam 20 years and nearly $7.5 billion to reach this point.

The story of RNAi holds important lessons about “life-science sovereignty”. First, success takes more than scientific excellence and public support. Today, Greater Boston is a world-class biotech hub, hosting more than 1,000 biotechnology-related companies. But building this ecosystem took 50 years, starting in the 1970s with the founding of Biogen.

The Boston biotech ecosystem owes its growth to several interacting forces. The presence of world-class biomedical expertise within Harvard University and MIT certainly helped, but the availability of interdisciplinary skills such as engineering, business, finance, computing, and data sciences was also essential. So, too, was the young industry’s proximity to some of the world’s largest research hospitals. Bringing together the scientific and medical communities was critical for clinical development. Finally, early venture capitalists and investors in Boston and New York provided the necessary funding.

A second lesson concerns “sovereignty”, a concept that can be problematic, because it implies a nationalist orientation. In fact, for any life-science ecosystem to succeed, it must be internationally oriented and open, so that it can tap scientific know-how, talent, and capital from around the world. There is a reason why many of the largest European and Japanese pharmaceutical companies, Sanofi, Novartis, Takeda, and Ipsen, invested in facilities in Boston.

To help domestic companies grow internationally, governments need to ensure that their policies are conducive to attracting both human and financial capital from abroad. The UK seems to understand this. Through its Biobank, a large-scale biomedical database and research resource, it is leveraging National Health Service (NHS) data to build partnerships with global companies and researchers, ultimately leading to the development of new medicines.

Third, medical innovation requires considerable funds from both the public and private sectors. Here, Europe continues to trail the United States. Much more funding will be needed for it to catch up and, perhaps more importantly, not be overtaken by China in the global biotech race.

Finally, to ensure financial sustainability and a continuous cycle of investment, market and policy incentives must be aligned to reward innovation. Here, too, Europe is far behind the US. Because the European market is so fragmented, it takes much longer for innovations to become accessible there, leading to lower returns on investment. Between the limited growth opportunities and the commercial risks associated with unfavorable market access, there are many disincentives for investment in research capacity and clinical trials.

A more unified European market, where new innovations are evaluated in a timely and reliable fashion, could fix these problems and create a virtuous circle of investment and growth. But it will require a change of mindset. Decision makers need to start looking at life-science innovation as a strategic investment instead of a health-care cost.

They also need to improve access to new innovations, as the NHS has done with its population health management initiative, using patient medical histories to provide early and broad access to new treatments. Many other innovative solutions exist, but shepherding them to adoption and use will require more dialogue and a new social pact between the biotech sector, policymakers and the public.


Phillip A. Sharp, institute professor of Biology at MIT, is director of Alnylam Pharmaceuticals Scientific Advisory Board and a recipient of the Nobel Prize in Physiology or Medicine. Julien Patris is head of policy for International Markets and country Manager for Belgium and Luxembourg at Alnylam Pharmaceuticals. Copyright: Project Syndicate, 2022.

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