Data demonstrating superior selectivity, safety and pharmacokinetics of RGT-0474060 were presented at the 21st Annual Huntington’s Disease ...

The modulation of RNA splicing by small molecules has emerged as a promising strategy for treating pathogenic infections, human genetic diseases, and cancer; however, the principles by which splicing ...

Akari Therapeutics, Plc (Nasdaq: AKTX), an oncology biotechnology company developing antibody drug conjugates (ADCs) with ...

Akari Therapeutics is advancing its research on a novel spliceosome modulator, PH1, which has shown potential in killing cancer cells and activating the immune system. The company is focusing on the ...

Neuroendocrine tumors, including small cell lung cancer and neuroendocrine prostate cancer, are very aggressive with high chances of spreading. However, many individuals develop resistance to few ...

With big pharma signalling interest in novel RNA-targeted approaches, the term “druggable” is being redefined as technology advances.

RNA splicing is a cellular process that is critical for gene expression. After genes are copied from DNA into messenger RNA, portions of the RNA that don't code for proteins, called introns, are cut ...

Ipsen has swooped on a $1.8 billion R&D opportunity, joining with Skyhawk Therapeutics for the chance to option two candidates that could benefit from its “neuroscience expertise in movement disorders ...

Skyhawk Therapeutics Inc. signed on to work with Ipsen SA in a neurological disease-focused deal worth potentially $1.8 billion to discover and develop novel small molecules that modulate RNA. The ...

RNA splicing is a cellular process that is critical for gene expression. After genes are copied from DNA into messenger RNA, portions of the RNA that don't code for proteins, called introns, are cut ...

RNA splicing is a cellular process that is critical for gene expression. After genes are copied from DNA into messenger RNA, portions of the RNA that don't code for proteins, called introns, are cut ...