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Wave has met its goal in a Duchenne muscular dystrophy study, positioning it to talk to regulators about accelerated approval. [ABSTRACT FROM AUTHOR]

Having scooped up the U.S. rights, Japan's Nippon Shinyaku is fronting $35 million in cash and a stock purchase to secure the same deal in Europe. [ABSTRACT FROM AUTHOR]

After escaping a clinical hold several years back, Dyne Therapeutics has revealed new phase 1/2 data for it [ABSTRACT FROM AUTHOR]

Avidity impressed investors, but closer examinations revealed details that present a more nuanced picture than what the headline result implies. [ABSTRACT FROM AUTHOR]

Pfizer disclosed the discontinuation of a DMD gene therapy—and the financial fallout—alongside the axing of a RSV combination vaccine. [ABSTRACT FROM AUTHOR]

Pfizer's phase 3 gene therapy trial has failed to demonstrate significant improvement in motor function for boys with DMD. [ABSTRACT FROM AUTHOR]

The FDA has maintained the nearly yearlong clinical hold on Entrada's Duchenne muscular dystrophy candidate despite the company submitting more info. [ABSTRACT FROM AUTHOR]

Vertex may be looking forward to a "milestone-rich period coming up," but one candidate will no longer be joining the biotech on its journey. [ABSTRACT FROM AUTHOR]

PTC Therapeutics' layoffs will go deeper than initially thought as European regulators consider halting sales of its Duchenne muscular dystrophy drug. [ABSTRACT FROM AUTHOR]

Pamrevlumab flunked a second phase 3 Duchenne muscular dystrophy trial in quick succession to send FibroGen deeper into the mire. [ABSTRACT FROM AUTHOR]

Eli Lilly's Prevail unit has changed up a licensing pact with Precision, dropping the value and seeking a bigger share of pre-clinical duties. [ABSTRACT FROM AUTHOR]

Once heralded as a potential blockbuster, FibroGen's pamrevlumab did not live up to expectations in a phase 3 study for a chronic lung disease. [ABSTRACT FROM AUTHOR]

Santhera is bidding farewell to critical commercialization rights for vamorolone, selling North American access to Catalyst for up to $231 million. [ABSTRACT FROM AUTHOR]

After Evrenzo struggled last month, now the biotech's Duchenne muscular dystrophy candidate has flunked a late-stage study. [ABSTRACT FROM AUTHOR]

The FDA needs more time to consider Sarepta's gene therapy for Duchenne muscular dystrophy—which calls back to the approval of Exondys 51 in 2016. [ABSTRACT FROM AUTHOR]

Researchers tied the death of the DMD patient to their immune reaction to the viral vector, raising concerns about dosing older, more advanced people. [ABSTRACT FROM AUTHOR]

The FDA has "eased" a partial clinical hold on Avidity's lead program for an inherited muscle wasting disorder seven months after it was placed. [ABSTRACT FROM AUTHOR]

With days to go, Sarepta set out what is expected to be asked at the meeting—and made the case that it can allay worries about its confirmatory trial. [ABSTRACT FROM AUTHOR]

Securing fast-track status positions Regenxbio to benefit from more frequent interactions with the FDA as it works to bring its DMD therapy to market. [ABSTRACT FROM AUTHOR]

The European Medicines Agency gave a letter of support to the Critical Path Institute's model-based clinical trial simulation platform that supports a consortium looking for treatments for Duchenne muscular dystrophy. [ABSTRACT FROM AUTHOR]

Entrada Therapeutics has been dragged back to Earth thanks to a clinical hold placed by the FDA on its Duchenne muscular dystrophy program. [ABSTRACT FROM AUTHOR]

Sarepta has raised $1.2 billion to prepare for a launch of the DMD gene therapy SRP-9001, which was filed for accelerated approval during the quarter. [ABSTRACT FROM AUTHOR]

The biotech has decided its lonodelestat dreams may have to die so vamorolone can fly. [ABSTRACT FROM AUTHOR]

Sarepta Therapeutics has broken free from an FDA hold—put in place this June—on its phase 2 Duchenne muscular dystrophy (DMD) clinical trial. [ABSTRACT FROM AUTHOR]

Sarepta's shares saw a nice boost Friday as the biotech declared its intent to seek an accelerated approval for a Roche-partnered DMD gene therapy. [ABSTRACT FROM AUTHOR]

Dyne Therapeutics is back on track to move its first asset into the clinic in the coming weeks after the FDA lifted a clinical hold on the drug. [ABSTRACT FROM AUTHOR]

Sarepta Therapeutics has successfully defused concerns about a FDA clinical hold on one of its Duchenne muscular dystrophy (DMD) clinical trials. [ABSTRACT FROM AUTHOR]

Solid Biosciences is cutting over a third of its workforce—for the second time in two years. [ABSTRACT FROM AUTHOR]

Roche has pushed back the regulatory timeline for its Duchenne muscular dystrophy treatment by a year. [ABSTRACT FROM AUTHOR]

Capricor Therapeutics will get enrollment underway in a phase 3 trial for its Duchenne muscular dystrophy (DMD) cell therapy next quarter, while development of a COVID-19 vaccine is coming to a hal [ABSTRACT FROM AUTHOR]

After showing its cell-based therapy slowed disease progression in Duchenne muscular dystrophy last September, Capricor Therapeutics is now ready to take the drug into a phase 3 trial with $30 million upfront from Nippon Shinyaku to help out. [ABSTRACT FROM AUTHOR]

Sarepta Therapeutics will ask the FDA to approve its gene therapy for Duchenne muscular dystrophy next year if the data from a pivotal trial is consistent with existing evidence, the biotech said at JPM 2022 on Monday. The biotech, with three approved DMD meds, said its gene therapy helped kids improve their motor skills. [ABSTRACT FROM AUTHOR]

Pfizer reported the death of a young male patient who was participating in an an early-stage trial for the Big Pharma's Duchenne muscular dystrophy gene therapy. The company paused screening and dosing in the study while it works with the trial site investigator to figure out what happened. [ABSTRACT FROM AUTHOR]

There are a number of potential blockbuster drugs on the list, but it's not all about revenue potential. Many of the compounds are high-risk, high-reward candidates trying to push the boundaries of drug treatment. [ABSTRACT FROM AUTHOR]

Pfizer's vision for a banner 2022 in gene therapies is over. Having previously expected to post interim data on a trio of phase 3 programs next year, Pfizer has now delayed all the readouts, setting back its plans in hemophilia and Duchenne muscular dystrophy. [ABSTRACT FROM AUTHOR]

Investors are enthusiastic about the next generation of gene-focused treatments, most notably in vivo gene editing, which recently got a major boost from Intellia's positive first-in-human data. But all of these companies are advancing their gene therapies at a time when safety concerns are dogging developers of first-generation products. [ABSTRACT FROM AUTHOR]

Pfizer is tightening up the criteria for Duchenne muscular dystrophy patients to participate in a phase 3 clinical trial for an experimental gene therapy after three serious adverse events were seen in ongoing studies. [ABSTRACT FROM AUTHOR]

Capricor Therapeutics' Duchenne muscular dystrophy therapy appears to have slowed disease progression in older boys and young men who are in the later stages of their disease and unable to walk, setting the biotech up for a phase 3 trial. [ABSTRACT FROM AUTHOR]

Solid Bio will present new biopsy results today for three patients who show "durable expression and function of microdystrophin protein" at 1.5 years after dosing at the World Muscle Society 2021 Virtual Congress. [ABSTRACT FROM AUTHOR]

Catabasis Pharmaceuticals has culled staffers, axed pipeline meds and endured some major trial flops. Now, it's looking to hit the reset button with a new name and focus. [ABSTRACT FROM AUTHOR]

PepGen has raised $112.5 million to take a treatment for Duchenne muscular dystrophy into the clinic early next year. The crossover round positions PepGen to start showing if its technique for getting more oligonucleotides into cells can improve on the efficacy of Sarepta's Exondys 51. [ABSTRACT FROM AUTHOR]

Two weeks after Sarepta reported a new safety concern in a Duchenne muscular dystrophy clinical trial, the company says another gene therapy candidate has shown some early success and will be brought to the FDA for permission to kick off further testing. [ABSTRACT FROM AUTHOR]

Pfizer has paused enrollment in a pivotal trial of its anti-BCMA bispecific antibody after seeing three cases of peripheral neuropathy in an earlier-stage study. The Big Pharma shared the news alongside details of a delay to the start of the U.S. part of its Duchenne muscular dystrophy (DMD) phase 3. [ABSTRACT FROM AUTHOR]

Sarepta Therapeutics says its RNA therapy for Duchenne muscular dystrophy, SRP-5051, could be more effective than the company's existing therapies, but a dose-escalation study also dug up some new—and serious—adverse events in patients. [ABSTRACT FROM AUTHOR]

Entrada Therapeutics has raised $116 million to develop intracellular biologics. The series B round will enable Entrada to start clinical development of a pipeline featuring treatments for diseases such as Duchenne muscular dystrophy (DMD). [ABSTRACT FROM AUTHOR]

Solid Biosciences has given its Duchenne muscular dystrophy gene therapy without causing the sort of adverse events that forced it to shutter the study in 2019. The experience provides early evidence that the manufacturing changes and clinical mitigation strategy could rescue the program. [ABSTRACT FROM AUTHOR]

While big companies can shrug off a failure, the wasted investment and missed commercial opportunities can still be damaging. For smaller companies, flunking a trial can jeopardize the future of the business. [ABSTRACT FROM AUTHOR]

Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy has failed to beat placebo in a phase 2 clinical trial. Functional motor ability scores in the SRP-9001 arm were statistically no better than in the placebo group, almost halving Sarepta's stock price and raising doubts about a key program. [ABSTRACT FROM AUTHOR]

Once wary of inking deals around gene therapy, Eli Lilly is jumping headfirst into developing treatments that edit genes within the body. It's teaming up with cell and gene therapy biotech Precision Biosciences to develop in vivo gene therapies for three gene targets, starting with Duchenne muscular dystrophy. [ABSTRACT FROM AUTHOR]

Nearly one month ago, Santhera admitted defeat for its Duchenne muscular dystrophy drug idebenone, culling the program. [ABSTRACT FROM AUTHOR]