Repligen’s spinal muscular atrophy program, including development of the experimental drug RG3039, now has the support of a major pharmaceutical company

posted on January 4, 2013 - 8:48am
Update (Feb. 2, 2015): Pfizer announced in January 2015 that it will not continue its collaboration with Religen for development of RG3039. The reasons are not clear, but industry observers suspect that the change of plan is related to an announcement in summer 2014 that the compound failed to increase production of the SMN protein.

In a phase 1 trial in healthy volunteers, Repligen's experimental therapy for spinal muscular atrophy was safe and well-tolerated

posted on April 25, 2012 - 4:00am
Update (June 21, 2012): This story was updated to include the fact that MDA funding helped support the phase 1 trial, as well as the preclinical development of RG3039.

A phase 1 clinical trial has begun in Turin, Italy, to test the safety of RG2833 in adults with Friedreich's ataxia

posted on March 15, 2012 - 11:00am
The biopharmaceutical company Repligen Corp., headquartered in Waltham, Mass., has launched a phase 1 clinical trial to test its experimental compound, RG2833, in adults with Friedreich's ataxia (FA). The trial is the first of a therapy specifically developed to treat the underlying molecular cause of FA. The phase 1 trial is being conducted at San Luigi Gonzaga University Hospital in Turin,...

Reports on research in amyotrophic lateral sclerosis, Duchenne muscular dystrophy, myotubular myopathy and spinal muscular atrophy

posted on August 12, 2010 - 11:33am
Duchenne muscular dystrophy Acceleron Pharma announced Aug. 4 that it has received fast track designation from the U.S. Food and Drug Administration (FDA) for its experimental compound ACE031 for the treatment of Duchenne muscular dystrophy (DMD). ACE031 is designed to interfere with the actions of myostatin, a protein that inhibits muscle growth.

Repligen Corp. is proceeding with development of an experimental compound to treat Friedreich's ataxia, with help from a new MDA grant.

posted on December 17, 2009 - 1:47pm
Development of a promising experimental medication to treat Friedreich's ataxia (FA) is proceeding, with help from a $731,534 grant MDA awarded to Repligen Corp. of Waltham, Mass., this month (December 2009). This is the second research grant that MDA has awarded to the small biopharmaceutical company, through the Association's translational (laboratory-to-clinic) research program.

A compound being developed to treat Friedreich's ataxia specifically targets an unwanted molecular brake

posted on September 29, 2009 - 11:32am
Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA) and determined that this brake is specifically targeted by an experim
posted on November 1, 2008 - 1:57pm
QUEST Vol. 15, No. 6
New MDA clinical research network to target ALS, DMD