Delivering functional copies of genes to correct genetic disorders using viral vectors like adeno-associated virus (AAV) or lentivirus. Promises one-time curative treatments for monogenic diseases.
Ultragenyx submits its BLA for DTX401, an AAV8-delivered gene therapy expressing the human G6PC gene for glycogen storage disease type Ia. The submission marks another rare disease gene therapy approaching the FDA finish line in a crowded 2026 regulatory calendar.
Apr 3, 2026 · 5 min read
Wraps AlphaFold2 with MMseqs2-based MSA generation, making AF2 runs 40-60x faster. Accessible via Google Colab or local install.
Diffusion-based generative model that treats docking as a generative problem over ligand poses. No pre-specified binding pocket needed.
Genetic algorithm-based docking supporting full ligand flexibility and partial protein side-chain flexibility. Four scoring functions available.
Commercial platform predicting 175+ ADMET properties with integrated PBPK (GastroPlus) and generative drug design (AIDD module).
Diffusion-based generative model for de novo protein backbone design. Generates novel protein structures conditioned on binding targets, symmetry, or functional sites.
Inverse folding model: generates amino acid sequences predicted to fold into a target 3D backbone structure. Standard component of all modern protein design pipelines.