Comment

The COP16 decision established a multilateral mechanism for digital sequence information (DSI) benefit-sharing. This Comment brings together insights from academia and commercial DSI researchers to assess what has been accomplished so far, identify remaining challenges and describe elements under discussion to support collective goals.

A vast landscape of ‘undruggable’ cancer targets remains beyond the reach of conventional therapeutic agents. Recent advances in artificial intelligence (AI), however, are challenging this paradigm. Synthesizing insights from a Cancer Moonshot workshop, we argue that systemically addressing the undruggable target space with AI requires a new conceptual framework. We highlight the failure of current target taxonomies and the need for benchmarking datasets, and re-evaluate clinical validation for novel AI-driven modalities.

Platform-based approaches for gene-editing therapies could markedly improve development efficiency, reduce costs and increase access for patients with rare diseases. Although gene editing has shown remarkable clinical success for a small number of Mendelian disease indications, broader adoption faces substantial hurdles. We propose strategies to overcome these challenges through modular platforms for nonclinical and chemistry, manufacturing and controls (CMC) data reuse, risk-based manufacturing quality, and streamlined umbrella clinical trials for regulatory efficiency and accelerated approval.

A new model for quantifying the impact of capital investments in life science interventions could help investors, policymakers and healthcare stakeholders to assess, monitor and optimize the societal impact of their investments.

23andMe’s bankruptcy serves as a moment of reflection for the direct-to-consumer (DTC) genomics industry. We analyzed 23andMe financial data and business practices to reveal the factors behind the fall of the company, once valued at US $6 billion and now being considered for acquisition by Regeneron for merely $250 million. Key challenges faced by 23andMe in monetizing its genomic data reveal that this information, at least in a typical DTC setting, is simply not worth that much.

Producing goods, such as foods and fuels, with minimal environmental impacts is urgently needed. Although advances in bioproduction are promising, there is often a noticeable gap in our understanding of whether and where new processes can compete with existing methods on an economic and environmental basis. Transparent lower bound calculations from basic principles highlight potential benefits of producing foods, but not fuels, from electro-microbial production of biomass.

Synergizing advances in artificial intelligence with mathematical modeling will improve our ability to computationally predict immunotherapy outcomes.

Spatial omics technologies offer insights into the organization of cellular and molecular components and their interactions within the tumor ecosystem. Overcoming the key challenges to integrating these advances into routine clinical practice will help unlock new treatment options for patients receiving cancer immunotherapy.

The identification of novel targets together with technological advances is transforming our ability to effectively modulate the tumor microenvironment.

Our ability to use engineered bacteria for cancer therapy is rapidly expanding. A survey of preclinical, clinical and commercial efforts provides an overview of the state of the field, revealing trends that could inform future directions.

To enhance the safety and security of the US bioeconomy, a new public–private partnership should be established to facilitate information sharing and threat analysis among industry, government and academia, and to develop and deploy safeguards.

After two decades of experience with biosimilars, physicochemical and in vitro biological comparison with their reference products appear sufficient to guarantee clinical safety and efficacy. Hence, the regulation of biosimilars has become redundant, and biopharmaceuticals should now be regulated through the generic pathway available for small molecules.

The Innovate Genomics Institute brought together regulators from 16 countries to discuss global capacity building for the regulation of genome-edited crops. The workshop provided insights into the suitable use of technical analyses to validate edits and raised future considerations regarding regulation reporting, offering suggestions to help countries meet their objectives in the ever-growing landscape of genome editing techniques.

The European Innovation Council aims to catalyze the formation and growth of science-based businesses in Europe through highly targeted initiatives, actively managed innovative portfolios and a stepped funding pathway of grants and equity investment.

The proper use of spike-in normalization in ChIP-seq improves sensitivity for detecting genome-wide changes between conditions, but improper use is common, calling some biological conclusions into question. A survey of public datasets generates guidelines for implementation of spike-in normalization for future ChIP-seq experiments.

Comparative studies that integrate genetically diverse mouse models and in vitro cell-based assays will accelerate drug discovery for precision medicine.

There is an ever-growing choice of single-cell and spatial ’omics platforms for industry and academia. The scTrends Consortium provides a brief historical overview of the established platforms and companies, revealing market trends and presenting possible angles for how technologies may differentiate themselves.

VitaDAO funds longevity research through a blockchain-based decentralized autonomous organization (DAO), showcasing the potential of collaborative, transparent and alternative systems while also highlighting the challenges of coordination, regulation, biases and skepticism in reshaping traditional research financing methods.

Biotechnology (biotech) clusters have evolved over time. Being a top biotech region in the early years 1978–1990 still positively correlates with the cluster’s biotech strength decades later. An entrepreneurial orientation of scientific actors as well as a variety of networks with partners outside the cluster both contribute to sustained biotech activity.

Pfizer created a ‘light-speed’ approach to meet the challenge of vaccinating the world against COVID-19. It involved developing new strategies for all aspects of vaccine development, from sourcing materials and scaling up manufacturing to transportation and dosing.