Topic: Huntington disease
Ribometrix raised $30 million to fund its platform that fits small molecules into the 3D folds and pockets of RNA structures to modulate their activity.
Laquinimod was once hailed as a blockbuster heir to Copaxone but suffered a string of late-phase failures that dragged down Teva.
Active Biotech and Teva delivered more bad news from their clinical development of laquinimod, this time from a phase 2 trial in Huntington’s disease.
Vaccinex filed a $46 million Nasdaq IPO to support its development of antibodies and conjugates targeting neurodegenerative diseases and cancer.
Celgene launched a $60 million collaboration with Skyhawk Therapeutics to develop small molecules targeting RNA mis-splicing in neurological diseases.
EIP Pharma is enrolling a phase 2b trial of neflamapimod, which targets synaptic dysfunction.
Alnylam is gearing up to move RNAi therapies for central nervous system disorders into the clinic.
The small trial linked antisense drug RG6042 to a 40% drop in levels of the mutant protein that drives the neurodegenerative disease.
A Polish team is fine-tuning a Cas9 enzyme that can cut one strand of DNA instead of both strands—potentially providing a safe way to edit out the Huntington's gene.
Biotech discoveries over the past week included new way to harness the lethal power of Huntington’s disease and llama antibodies sans llamas.