Investigators say that a single mutation in the gene linked to Fragile X--which is the largest single-gene cause of autism--can be tied to specific symptoms of the disease, suggesting that a new approach to treating the disease could work where others have failed.
A long, rough patch in Fragile X syndrome drug R&D just got longer and rougher. Roche has notified patient groups that both of its mid-stage studies for RG7090--an mGluR5 therapy--failed to hit the primary and secondary goals, prompting the pharma giant to shut down the program.
The money is earmarked for discovery work through Phase I. And interim CEO Kevin Starr--a partner at Third Rock who launched the company with a $35 million A round in 2011--hopes that Sage can break new ground in a tough R&D field.
Seaside Therapeutics has suffered a string of setbacks recently. Swiss drug giant Roche has passed on taking an option on Cambridge, MA-based Seaside's lead drug candidate arbaclofen, which the biotech startup revealed last month flunked a Phase II autism study and Phase III trial in Fragile X patients.
Seaside Therapeutics has revealed its second failed study for its lead drug STX209 this month. In one of two Phase III studies for the compound for the genetic mental disorder Fragile X syndrome, STX209 treatment missed its main goal of reducing social withdrawal.
Asuragen is celebrating encouraging clinical data, suggesting one of its molecular diagnostic tests is better than existing standards of care in determining how likely a woman will have a child with Fragile X syndrome--a genetic cause of autism spectrum disorders and other intellectual disabilities.
Primarily known for its work on Fragile X, a well-known autism target, Afraxis says that the giant Roche subsidiary will pay up to $187.5 million in milestones and an undisclosed upfront in order to develop the compounds for a "novel target."
Roche has tapped assets from biotech startup Seaside Therapeutics to advance pioneering treatments for Fragile X syndrome and autism spectrum disorders.
Scientists at F. Hoffman-La Roche in Switzerland and the Picower Institute for Learning at the Massachusetts Institute of Technology believe they've found a new compound that reverses most Fragile X-related symptoms in adult mice.
A European research effort led by Roche ($RHHBY) and others to tackle autism spectrum disorders nailed down $38.7 million in financing for what will be a massive, 5-year project seeking to come up with new diagnostic and treatment options.