Rare & Genetic Diseases

Learn practical strategies for using artificial intelligence to find the best drug candidate.

Advanced gene editing, delivery, and analytical tools are driving better gene therapies.

Rett syndrome currently has no cure. Now, innovations in gene therapy bring the possibility of treating the root cause of the disease closer than ever before.

Emerging CRISPR screening methods are shaping what’s possible in drug development and precision medicine.

Drug repurposing trials for ichthyosis reveal complexity in the long-untreated disease.

Previous genome editing techniques only work when delivered early in life. This approach could help preserve hearing that is lost later on. 

Achieving higher accuracy and depth in bottom-up proteomics involves navigating a multifaceted process with various steps and challenges.

Epilepsy onset and seizure severity are linked to variants in the SCN2A gene, potentially leading to improved diagnostics and treatments.

BioSpring and Pantherna agree on strategic cooperation in the field of genome editing

A new method called “REVeRT” expands the capabilities of gene delivery platforms by transporting larger genes than previously possible.  

Biologics like monoclonal antibodies and mRNA vaccines are complex drugs. Yongchao Su uses biophysical tools and innovative strategies to understand them better.

Researchers created an improved method for assays that detect viral vectors in a patient’s blood or urine. It’s used to ensure that bystanders will not be exposed.

People with achromatopsia have never seen color. Restorative gene therapies have had mixed success, leaving researchers wondering why.

The durability and capacity to boost protein expression of circular RNA makes it a promising candidate for next-generation vaccines and gene therapies.

Spatial transcriptomics technologies unveil a goldmine of biological information.

Cutting-edge DNA synthesis technology propels scientific advancements and drug discoveries forward at unprecedented speeds.

By watching brain organoids develop, researchers observed surprising abnormalities caused by mutations in a gene related to early-onset Alzheimer’s disease.

A gene therapy targeting excitatory neurons significantly reduced seizures in a mouse model of the most common cause of child epilepsy.

Biobanking gives scientists access to thousands of biological samples, moving precision medicine one step closer to reality.

Alnylam Pharmaceuticals develops therapeutics based on the RNA interference (RNAi) pathway discovered in 1998.