Pipeline

Alector is kicking off a resource realignment effort that will include a workforce reduction. The biotech expects its current cash position to last it through 2026.
Truqap’s positive clinical data comes after it failed a late-stage study in metastatic triple-negative breast cancer. It helps AstraZeneca position itself as a top player in the prostate cancer space, alongside its Big Pharma colleagues.
While taldefgrobep alfa failed to show improved motor function in spinal muscular atrophy, treated patients saw a marked reduction in body fat. Biohaven plans to launch a Phase II trial in obesity by the end of the year.
Adcendo joins a growing group of biotechs working to usher the next generation of antibody-drug conjugates to the market.
Despite the death, the FDA has allowed Neurogene to forge ahead with the Phase I/II Rett syndrome trial, but using only the lower 1E15 vg dose of its investigational gene therapy NGN-401.
Novartis’ up to $1.1 billion acquisition of gene therapy specialist Kate Therapeutics fits with the pharma’s plan to expand its new modality pipeline to ensure long-term business sustainability.
After previously failing studies in Parkinson’s and Alzheimer’s, dalzanemdor’s latest stumble in Huntington’s disease has pushed Sage Therapeutics to pull the plug on the NMDA receptor modulator.
While the full impact of the Supreme Court decision remains unknown, the new regulatory landscape could be a net positive for drug developers.
After failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells investors that it’s evaluating options for the program.
Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.
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