Fragile X

Latest Headlines

Latest Headlines

Genetic error highlights a new approach to Fragile X syndrome

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.

Roche abandons another Fragile X R&D program after PhII trials flunk out

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.

NIH awards Third Rock startup up to $10M for Fragile X work

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.

Roche rejects option on autism drug from struggling Seaside

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. 

UPDATED: Cash-strapped biotech fails PhIII test for pioneering Fragile X drug

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. 

Study: Asuragen's molecular Dx better at Fragile X detection

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.

Genentech plucks rights to Afraxis drug assets in $187M deal

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, Seaside join forces against autism and Fragile X

Roche has tapped assets from biotech startup Seaside Therapeutics to advance pioneering treatments for Fragile X syndrome and autism spectrum disorders.

Drug tackles Fragile X/autism symptoms in mice

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.

Roche headlines autism alliance

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.