Rare & Genetic Diseases

Mitochondria viewed under a microscope, depicted in a glowing style against a dark blue background.
| 4 min read
In a Phase 2b trial, Khondrion’s sonlicromanol led to significant health benefits for patients with mitochondrial disease linked to a common mutation. 
A photomicrograph of staining of the enteric nervous system, with neurons in red and glia in green.
| 3 min read
A gene therapy targeting organs beyond the brain could improve quality of life and possibly extend survival in children with the neurodegenerative disease.
White circles in the red sandstone of an Australian riverbed show evidence of the presence of ancient microbes there.
| 3 min read
An enzyme reconstructed from a three-billion-year-old bacterium widens gene editing capabilities to even more regions of the genome.
A ribosome shown in yellow translates an mRNA molecule into a protein.
| 5 min read
A damaged yellow axon extends from a pink neuron to a red muscle fiber.
| 4 min read
A woman holds a newborn baby at the hospital.
| 6 min read
Artwork of a piece of DNA with stiches along it.
| 3 min read
A visual representation of an adeno-associated virus used in gene therapies.
| 3 min read
A 3D rendering of motor neurons lit up with blue, purple, orange, and green coloring showing synapses against a black background.
An illustration shows epithelial cells in green within a lobule of normal breast tissue, with aneuploid cells highlighted in red.
A doctor holds a young boy’s hands in a doctor’s office.
| 3 min read
A conceptual illustration of a drug capsule filled with microchips, representing the integration of artificial intelligence in drug discovery and development
A 3D illustration of two DNA strands in a transparent bubble
A toddler holds an adult’s hands as she walks outside during an autumn afternoon.
| 9 min read
An illustration showing a DNA strand and scientists removing segments with tweezers, representing the CRISPR gene editing technology
A woman with ichthyosis sits at a table working on a laptop.
| 9 min read
A human ear against a backdrop of blonde hair
| 4 min read
A 3D illustration of protein molecules floating in a dark blue background
| 7 min read
Interconnected neurons send signals to each other.
| 3 min read
A digital rendering of a DNA base being removed from a DNA helix, representing genome editing
Page 1 of 19 - 374 Total Items
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A 3D rendering of motor neurons lit up with blue, purple, orange, and green coloring showing synapses against a black background.

Improving ALS research with pluripotent stem cell-derived models 

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A conceptual illustration of a drug capsule filled with microchips, representing the integration of artificial intelligence in drug discovery and development

A Technology Guide for AI-Enabled Drug Discovery

Learn practical strategies for using artificial intelligence to find the best drug candidate.
A 3D illustration of two DNA strands in a transparent bubble

Overcoming barriers in gene therapy 

Advanced gene editing, delivery, and analytical tools are driving better gene therapies.
Drug Discovery News March 2025 Issue
Latest IssueVolume 21 • Issue 1 • March 2025

March 2025

March 2025 Issue

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