Genomics & Bioinformatics
In October 2024, Demis Hassabis and John Jumper received the Nobel Prize in Chemistry for AlphaFold—the fastest Nobel win in science history. By December 2025, CRISPR therapies had expanded from sickle cell to at least a dozen active clinical programs. In May 2025, the FDA unveiled a new "plausible mechanism" pathway enabling bespoke gene therapies for conditions so rare they may affect only individuals. The field of genomics has crossed from theoretical breakthrough to clinical reality with unprecedented speed.
AlphaFold 3: Predicting All of Life's Molecules
AlphaFold 3, released May 8, 2024, represents a quantum leap beyond its predecessor. While AlphaFold 2 solved the 50-year-old "protein folding problem," AlphaFold 3 provides a universal "digital microscope" predicting interactions of nearly all life's molecules—proteins, DNA, RNA, and complex drug ligands.
The impact: over 3 million researchers in 190+ countries use AlphaFold. As of November 2025, the AlphaFold 3 paper has been cited more than 9,000 times. Isomorphic Labs—Google DeepMind's drug discovery spinoff—has secured multi-billion dollar partnerships with Eli Lilly and Novartis, focusing on "undruggable" targets associated with cancer and neurodegenerative diseases. By December 2025, early-stage drug discovery timelines have dropped by as much as 80%.
Rumors circulate about AlphaFold 4, expected to move beyond static structures to model the dynamics of entire cellular environments in real-time.
CRISPR Comes to Patients
Casgevy: The First Approval
Casgevy, approved December 2023, treats sickle cell disease and transfusion-dependent thalassemia by editing patients' blood stem cells to produce fetal hemoglobin. Cost: $2.2 million per one-time treatment. Of 44 patients treated, 29 of 31 monitored achieved relief from vaso-occlusive crises lasting at least 12 consecutive months. By spring 2025, Casgevy had been approved in the US, UK, EU, Switzerland, Canada, Bahrain, Saudi Arabia, and the UAE.
FDA's "Plausible Mechanism" Pathway
In May 2025, the FDA unveiled a revolutionary regulatory blueprint for bespoke gene therapies. Called the "plausible mechanism" pathway, it allows treatments for conditions so rare they may affect only individuals or handfuls of people—without requiring traditional clinical trial data. KJ became the world's first patient treated with a bespoke CRISPR-based therapy in May 2025, receiving a personalized treatment for a mutated carbamoyl-phosphate synthetase 1 (CPS1) gene via lipid nanoparticles.
Base Editing: Precise Single-Letter Changes
David Liu won the 2025 Breakthrough Prize for developing base editing and prime editing—technologies that correct genetic mutations without creating double-stranded DNA breaks, reducing certain safety risks.
Beam Therapeutics has dosed at least 17 adult patients with their base editing therapy for sickle cell disease, with FDA clearance now extended to adolescents. Additional trials in 2025:
- BEAM-302 for Alpha-1 Antitrypsin Deficiency - Positive initial data in March 2025 showing durable increases in functional AAT from a single dose
- Glycogen Storage Disease Type I - First patient dosed in May 2025 for the R83C mutation
- VERVE-102 for Heart Disease - Continuation after VERVE-101 was paused due to laboratory abnormalities
Prime Editing: First Human Results
In May 2025, Prime Medicine announced the first clinical data showing prime editing efficacy and safety in humans. A patient with chronic granulomatous disease (CGD) received PM359, an autologous cell therapy correcting the delGT mutation in the NCF1 gene. Results: complete restoration of NADPH oxidase activity in 66% of neutrophils by Day 30—significantly above the 20% threshold for clinical benefit—with no serious adverse events.
Prime Medicine is undergoing "strategic reorganization" and seeking external partners for continued development, but the proof-of-concept stands: prime editing works in humans.
Spatial Transcriptomics: Seeing Genes in Space
Traditional single-cell sequencing destroys tissue structure. Spatial transcriptomics preserves it—showing not just what genes cells express, but where those cells sit within tissue architecture.
10x Genomics 2025 Roadmap (AGBT February 2025)
- Xenium Prime Assay - 5,000-plex target panels for mouse and human
- Xenium RNA+Protein Multiomics - Simultaneous detection of RNA and proteins on the same tissue section
- Validated Protein Immunology Panel - Seven modular subpanels covering 28 protein targets for human FFPE tissues
- Visium HD XL - Expanded capture area for larger tissue sections at single-cell resolution
- Visium HD 3' - Whole transcriptome spatial profiling with de novo discovery capabilities
The Xenium platform delivers subcellular resolution with nanometer precision, mapping hundreds of genes in situ. Nature Methods published best practices in March 2025 benchmarking open-source computational tools for Xenium data analysis.
Other CRISPR Clinical Trials in 2025
- PBGENE-HBV for Hepatitis B - Precision Biosciences received FDA IND clearance in March 2025
- ZVS203e for Retinitis Pigmentosa - Fixes rhodopsin gene mutation via subretinal injection; 75% of participants showed improved vision
- EDIT-101 for Leber Congenital Amaurosis 10 - Initial safety established
- EBT-101 for HIV - First CRISPR therapy administered for infectious disease, using AAV9 to target three HIV genome locations
The Longevity Revolution
The global anti-aging market generated over $85 billion in 2025, projected to reach $120 billion by 2030. Key developments:
- Altos Labs - $3 billion funding, assembled top scientists including Jennifer Doudna (CRISPR Nobel), Shinya Yamanaka (reprogramming Nobel), and Steve Horvath (epigenetic clocks)
- NewLimit - $130M Series B for epigenetic reprogramming medicines targeting liver disease
- Senolytics - Drugs like dasatinib and quercetin selectively remove "zombie cells"; advanced trials for osteoarthritis, Alzheimer's, and cardiovascular disease
- Insilico Medicine - AI-designed senolytics reaching early-stage trials
In October 2025, a Cell paper from Belmonte and colleagues showed that turning off specific genes may prevent cellular drift and restore more youthful cellular states.
Where to Find Genomics Research
Preprint Servers
- bioRxiv - ~10 million monthly views, 38,700+ bioinformatics preprints
- medRxiv - Health sciences preprints
Data and Tools
- AlphaFold Protein Structure Database - 200+ million predicted structures
- GenBank - NIH genetic sequence database
- CRISPR Medicine News - Tracks ~250 gene editing trials
Why Genomics Shares
The Human Genome Project precedent proved decisive. When the most ambitious biology project in history committed to immediate data release, it established a norm that proved hard to reverse. COVID-19 cemented it—preprints proved essential infrastructure when speed saved lives. AlphaFold predictions would be useless locked in Google's servers. The result: Nobel Prize-winning discoveries become freely available tools within months.