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The newest gene editors are shifting away from tackling mutations one by one and toward universal therapies that will hopefully create cheaper and more broadly applicable medicines.
Startups are providing tantalizing glimpses of AI-assisted antibody discovery and claiming game-changing potential, even as new models remain hidden and industry awaits full disclosure and benchmarking.
Computer models are showing the potential to draw upon a person’s clinical records and current health data to forecast diseases and treatment outcomes. Could this presage a new era of preventive medicine?
First-in-human studies provide hope that islet replacement therapies derived from stem cells will prove safe and effective in people with type 1 diabetes, but hurdles remain.
Developers have married gene-modulating oligonucleotides with the targeted precision of antibodies, and the first filings using such conjugates in Duchenne muscular dystrophy are imminent.
In-body manufacturing promises a faster, cheaper and simpler alternative to CAR-T cell therapy — if the field can deliver clinical readouts that prove long-term safety and efficacy.
This year’s most popular stories are a testament to biotech ingenuity and a cause for celebration: the gene editing triumph that cured ‘baby KJ’ of a rare genetic disorder in record time; the bispecific molecules that trick HIV out of cells to destroy them; how Brazil is producing its own affordable CAR T cells; and the shift in regulators’ demands for animal testing for certain drug classes.
The first-in-class small molecule Jascayd targets a fibrotic signaling cascade, offering new hope in a stagnant therapeutic field, as several other programs near maturity.
After decades-long struggles with microRNA-based therapeutics, Regulus hits paydirt in the clinic and in the marketplace. Is it the beginning of a trend or a one-off?
Long considered undruggable, transcription factors have finally become fair game for a clutch of innovative biotechs who are combining biological insights with innovative chemistry to bring into play a vast number of new drug targets.
With a newly marketed product in Japan and 10 years of clinical data, regenerative cell therapies promise new options for patients facing a choice between invasive surgery or blindness.
Analogs of the pancreatic peptide hormone amylin are making strides in obesity as developers zoom in on oral drugs that can emulate or even improve on injectable GLP-1 agonists while avoiding muscle loss and gastrointestinal troubles.
