Topic: Duchenne muscular dystrophy (DMD)
The initiative taps into the networks Invitae has built up to connect sponsors to patients and the clinicians responsible for their care.
Sarepta licenses five new gene therapies to its muscular dystrophy portfolio from Myonexus—and takes an option on buying the company.
Buoyed by safety data and evidence of exon-skipping activity, Daiichi is pushing ahead with further development of the antisense oligonucleotide.
Solid Bio fixed the manufacturing issue that led to a partial clinical hold on its DMD trial, but a full hold remains.
The reorg will cost about $1 million but will save the company about $3.3 million a year.
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.
The FDA has extended its partial hold on Solid Bio's phase 1/2 DMD trial to a full hold following a patient's hospitalization.
The latest analysis links the utrophin modulator to a statistically significant decline in muscle inflammation in boys with DMD.
Sarepta expects to resume dosing in a U.K. trial of its DMD drug golodirsen pending MHRA approval.
Targeting "clusters" of mutations with CRISPR could lead to a treatment that could work for up to 60% of patients with DMD.