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A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
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If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
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While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several companies including Wave, Dyne and Avidity are looking to answer the call with investigational therapies targeting greater efficacy and broader reach.
Less than a day into his second term, President Donald Trump ordered a freeze on communications at major public health agencies, among other moves that have sent waves through the biopharma industry.
The readout comes on the heels of CagriSema’s disappointing Phase III performance, where it missed Novo’s projection of 25% weight reduction.
The unsuccessful Phase III results are the latest to suggest that the blockbuster cancer drug is finally bumping up against its limits after racking up around 50 approvals since getting its first FDA nod in September 2014.
The San Diego–based company’s molecules avoid the well-trod GLP-1 pathway in favor of an alternate route in the gut.
The settlement is the largest deal to date with the people primarily who played an “instrumental role” in driving the opioid crisis, according to the office of Massachusetts Attorney General Andrea Joy Campbell.
Robert F. Kennedy, Jr.’s recent disclosures have revealed several potential conflicts of interest, including investments in two biopharma companies.
Vigil Neuroscience reported a strong safety profile and 50% sTREM2 reduction in an early-stage trial for VG-3927, potentially representing a new avenue for treating Alzheimer’s disease.
Protein degradation–focused Neomorph nabs its third Big Pharma deal of around $1.5 billion in less than a year.
In a good-news-bad-news week for Biogen, the company will cut an undisclosed number of employees, just as a higher dose of its Ionis-partnered therapy Spinraza for spinal muscular atrophy will be considered by the FDA and EMA.