Pipeline
Beam is now in the strongest position of all companies advancing a genetic therapy for alpha-1 antitrypsin deficiency, according to analysts at William Blair.
Biomarker data for Sarepta Therapeutics’ RNA programs, licensed from Arrowhead Pharmaceuticals, are “competitive” and “strong,” according to analysts at Jefferies, which projected over $1 billion in peak sales.
Aardvark Therapeutics’ obesity asset is based on its lead molecule, which in February was linked to reversible heart safety signals in a healthy volunteer study.
Eli Lilly and Regeneron are leading the push to treat congenital deafness with gene therapies, seeking a piece of a potential billion-dollar market and banking on local delivery and the small amount of drug required to overcome key safety concerns.
The main beneficiary of Roche’s discontinuation of an investigational spinal muscular atrophy drug is Scholar Rock, which was hobbled by manufacturing concerns at a Novo Nordisk facility last year but is now nearing a potential resolution.
Gossamer Bio reported last month that its only late-stage candidate failed a Phase 3 study in pulmonary arterial hypertension, casting doubt on the asset’s future prospects.
Prime biotech buyout targets such as Revolution Medicines and Ascendis Pharma have recently found themselves in the middle of acquisition rumors—though no deals have panned out so far.
A “significant number of patients” could see a cure for multiple myeloma within the next two decades, one expert told BioSpace. Here are five therapies that could change the treatment paradigm.
Analysts are keeping a close eye on Vertex’s kidney disease portfolio, anchored by the IgAN drug povetacicept, as its nonopioid painkiller Journavx is poised for “incremental” growth in the first half 2026, according to BMO Capital Markets.
AstraZeneca will push the pill, elecoglipron, into a comprehensive late-stage program that will test the drug as a monotherapy, as part of a combination regimen and for several indications.
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