Every year, scientific journals and major universities publish “Top Discoveries of the Year” lists, and they’re genuinely worth reading. But they also have built-in biases: journals tend to feature work they’ve published in their own focus areas, and university press offices highlight breakthroughs from their own labs. So they’re not always ideal if you want to see the full landscape of biomedical research in a given year.

This list was put together differently. It was curated by the Rosetta Institute using a series of AI prompts that pulled in discoveries from many journals, institutions, and fields, with an emphasis on both basic molecular/cellular biology and translational/clinical advances. The goal is to give students a broader, more integrated snapshot of biomedical research in 2025—from CRISPR cures and aging mechanisms to RNA droplets and AI-designed proteins. As you read, ask yourself: what do you want to work on?  Where can you see the future?

2025 in Review

  1. A one-of-a-kind CRISPR cure for a single baby

Doctors used CRISPR base editing to design a personalized in-vivo gene therapy for one infant with CPS1 deficiency, targeting his exact mutation in liver cells and correcting a deadly metabolic defect.
Source: New England Journal of Medicine (NEJM), 2025.

  1. RNA “storage droplets” that help decide cell fate

Tiny droplets called P-bodies were shown to store and silence RNAs from earlier developmental stages; releasing them can push cells back toward more stem-like states and make hard-to-generate lineages easier to produce.
Source: Nature Biotechnology, 2025.

  1. Translation slowdowns that help drive brain aging

In aging killifish brains, ribosomes stall on certain mRNAs (often for mitochondrial and RNA-binding proteins), so the mRNAs stay but less protein is made, promoting misfolded proteins and dysfunction before other aging signs appear.
Source: Science, 2025.

  1. A tRNA-editing glitch that breaks the heart

The protein AIMP3 helps methionyl-tRNA synthetase edit out toxic homocysteine; when AIMP3 is lost in heart cells, homocysteine builds up, mitochondria fail, proteins misfold, and mice develop lethal cardiomyopathy.
Source: Nature Cardiovascular Research, 2025.

  1. Base-edited “universal” CAR7 T cells for tough T-cell leukemia

Researchers built base-edited, off-the-shelf CAR7 T cells that don’t attack themselves or get rapidly rejected, and used them to treat relapsed/refractory T-cell acute lymphoblastic leukemia, where options are usually exhausted.
Source: New England Journal of Medicine (NEJM), 2025.

  1. Fixing short telomeres with gene therapy

A first-in-human trial of a telomere-repair gene therapy in telomere biology disorders lengthened telomeres in blood stem cells and improved blood counts, with early safety looking acceptable.
Source: NEJM Evidence, 2025.

  1. Nanoflowers that turn cells into mitochondrial donors

Molybdenum disulfide “nanoflowers” taken up by donor cells trigger mitochondrial biogenesis; those cells then donate extra mitochondria to damaged neighbors, rescuing ATP production and respiration in cell models.
Source: Proceedings of the National Academy of Sciences (PNAS), 2025.

  1. PDGrapher: AI that suggests how to flip diseased cells back to healthy

The AI tool PDGrapher looks at omics data from healthy vs. diseased cells and ranks gene targets and drug combinations predicted to push cells back toward a healthy state, outperforming earlier models across many cancers.
Source: Nature Biomedical Engineering, 2025.

  1. A mostly synthetic human chromosome that works in human cells

The Synthetic Human Genome Project built a largely synthetic human chromosome, matured it in mouse stem cells, then moved it into human cells where it functioned stably, opening a path to systematically testing chromosome design and behavior.
Source: Science, 2025.

  1. Protective microglia that behave like regulatory T cells

A Nature study identified a microglial state with a regulatory T-cell-like gene program that calms inflammation and protects neurons in Alzheimer’s models, pointing to specific transcription factors and metabolic cues that could be targeted by drugs.
Source: Nature, 2025.

  1. AI-designed proteins that neutralize snake venom

Using de novo protein design, scientists created small synthetic proteins that bind and neutralize deadly “three-finger toxins” in snake venom and protect animals in vivo, hinting at safer, animal-free antivenoms.
Source: Nature, 2025.

  1. A fluorescent-protein “biological qubit” inside living cells

University of Chicago researchers turned a fluorescent protein into a functioning quantum bit (qubit) whose spin state can be controlled inside living cells, suggesting future quantum sensors that read out ultra-tiny changes within cells.
Source: Nature, 2025.

  1. Lenacapavir: a twice-yearly shot that nearly eliminates HIV infection in trials

A first-in-class capsid inhibitor, lenacapavir, given as a twice-yearly injection for HIV pre-exposure prophylaxis (PrEP), showed ~96–100% efficacy in large PURPOSE 1 and 2 trials, with no HIV infections in lenacapavir groups in key analyses and strong real-world momentum after FDA approval in 2025.
Source: New England Journal of Medicine (PURPOSE-1 & PURPOSE-2), 2024–2025.