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Interest is growing in genome-editing tools that can insert large chunks of DNA into the genome — and avoid the double-strand breaks associated with CRISPR–Cas9 genotoxicity.
Two upcoming regulatory decisions represent a tipping point for commercial gene therapy, with implications for work on existing viral vectors and the pursuit of new ones.
The Broad Institute’s enlightened licensing approach to CRISPR–Cas9 intellectual property stands out in the otherwise regrettable spat for patent rights over the foundational technology.
For the raft of new ventures developing epigenome editors, a compelling niche may be diseases of haploinsufficiency or genome imprinting that require exquisite control of gene expression.
Advances in technology and changes to healthcare during the pandemic may finally realize the vision of patient-centric blood testing espoused by disgraced Theranos CEO Elizabeth Holmes.
Outrage over the cost of insulin is driving drug-pricing reform. Industry must do more to support patients dealing with spiraling out-of-pocket costs for biotech medicines.
With supply-chain shortages and production problems exacerbating vaccine inequity, global manufacturing must expand to ensure the world’s poorest nations are better served.
Our new podcast series ‘Hope Lies in Dreams’ highlights the importance of visionaries in shepherding drugs through the setbacks of drug development to commercial success.
The Sequencing Quality Control 2 (SEQC2/MAQC-IV) project provides resources to aid sequencing reproducibility and highlights factors that can guide platform and software choice.
The International Society for Stem Cell Research has called for broad public dialogue on the ethics of human embryo research beyond 14 days post-fertilization. National jurisdictions should seize the moment.
An Alzheimer’s drug approval is being touted as a triumph for patients. But US healthcare is ill prepared for Aduhelm’s questionable efficacy, unfettered market access and high price.
New initiatives in N-of-1 drug development and clinical trial design offer the possibility of therapies for ultra-rare disease patients who have been long neglected by the drug industry.
To keep ahead of an increasing number of SARS-CoV-2 variants, pathogen surveillance and testing must become a joined-up global, rather than local, endeavor.
The US RADx program has spawned a phalanx of diagnostic products to market in just 12 months. Its long-term impact on point-of-care, at-home and population testing may be even more profound.
Since this journal launched in March 1996, biotech has become a US economic powerhouse. To reach its full potential over the next 25 years, touching all corners of the globe, it must become more inclusive.
With drug pricing a priority for the US Biden administration, the biotech industry must clean up its act: stop profiteering from old products and stamp out anticompetitive practices.
Rapid progress is being made in our understanding of RNA–protein interactions, their role in disease and their influence on the action of oligonucleotide drugs.
