First patient dosed in Opti-GAIN, a first-in-human Phase I/II clinical trial of CTx001 in Geographic Atrophy secondary to AMD CTx001 is an investigational AAV-based gene therapy designed to deliver ...

Multi-omics profiling of residual cancer burden after neoadjuvant hormonal therapy guides subtype-directed treatment in high-risk and locally advanced prostate cancer. Predictive associations between ...

The Food and Drug Administration (FDA) announced a new proposal Monday for flexible drug approval pathway treatments addressing ultrarare diseases. The FDA unveiled draft guidance on a proposed ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Adobe is putting on hold its plan to discontinue Adobe Animate following intense backlash from its customers after it announced plans to shut down the 2D animation software amid an increased focus on ...

Ocugen has reported preliminary phase 2 data on its eye disease gene therapy, providing more evidence that the candidate may outperform existing treatments from Apellis Pharmaceuticals and Astellas ...

This year’s 2026 Preview consists of all the entertainment — from movies to books to art shows — that Vulture writers and editors are excited to consume in the New Year. Below, our classical-music ...

Cedars-Sinai investigators have identified a genetic variant that increases people's risk of developing perianal Crohn's disease, the most debilitating manifestation of Crohn's disease. Science X ...

Performances in N.Y.C. Advertisement Supported by Listen to recordings by Maria Dueñas and Daniil Trifonov, works by Thomas Adès and Tania León, and more. London Philharmonic Orchestra; Thomas Adès, ...

The complement system is an innate immune response that leads to increased inflammation. The drug pegcetacoplan—a complement inhibitor—was approved for medical use in the United States in 2021 to ...

In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory pathway that could trigger a seismic shift in how bespoke gene editing therapies ...