After gene therapy pivot, Axovant bolsters its executive ranks by Amirah Al Idrus Monday, November 26, 2018 After refocusing on gene therapy, Axovant is appointing five new executives to posts ranging from clinical development to commercial operations.
Sarepta sees muscle gains with DMD gene therapy by Phil Taylor Thursday, October 4, 2018 Preliminary signs of improved muscle function have been seen in Sarepta's Duchenne muscular dystrophy gene therapy trial.
Quality blip halts Sarepta DMD gene therapy trial by Phil Taylor Thursday, July 26, 2018 A trial of Sarepta’s gene therapy for muscular dystrophy was placed on clinical hold by the FDA but could be upgraded to a pivotal study as a result.
Sarepta taps Invitae to identify participants for DMD trials by Nick Paul Taylor Monday, May 7, 2018 The initiative taps into the networks Invitae has built up to connect sponsors to patients and the clinicians responsible for their care.
Sarepta adds to gene therapy pipeline via Myonexus pact by Phil Taylor Friday, May 4, 2018 Sarepta licenses five new gene therapies to its muscular dystrophy portfolio from Myonexus—and takes an option on buying the company.
Daiichi DMD trial misses primary efficacy endpoint, but vows to push on by Nick Paul Taylor Wednesday, April 25, 2018 Buoyed by safety data and evidence of exon-skipping activity, Daiichi is pushing ahead with further development of the antisense oligonucleotide.
Pfizer puts Bamboo’s muscular dystrophy therapy to the test by Phil Taylor Friday, April 13, 2018 Pfizer has dosed the first patient in a trial of a muscular dystrophy gene therapy, acquired as part of its $700 million takeover of Bamboo in 2016.
Summit’s DMD drug cuts muscle inflammation in POC trial by Nick Paul Taylor Monday, February 26, 2018 The latest analysis links the utrophin modulator to a statistically significant decline in muscle inflammation in boys with DMD.
Sarepta shines spotlight on next-gen DMD drug by Phil Taylor Monday, October 9, 2017 Sarepta is hoping to avoid the data-quality controversy that arose over its Duchenne muscular dystrophy drug Exondys51 last year with its next-generation drug golodirsen.
FDA panel pans PTC’s post hoc pitch, shredding DMD dream by Nick Paul Taylor Friday, September 29, 2017 A panel of experts convened by the FDA has overwhelmingly knocked back PTC Therapeutics’ filing for approval of ataluren.
