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 The FDA unveiled draft guidance on a proposed ...

Quantum computers—devices that process information using quantum mechanical effects—have long been expected to outperform classical systems on certain tasks. Over the past few decades, researchers ...

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 ...

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 ...

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 ...

(RTTNews) - Biotechnology company Q32 Bio, Inc. (QTTB) announced Monday it has sold its Phase 2 complement inhibitor, ADX-097, to Akebia Therapeutics, Inc. (AKBA). ADX-097, the lead product candidate ...

– Single subcutaneous dose of ADX-038 achieved near-complete alternative pathway suppression for 6 months, supporting potent and selective CFB silencing – – Phase 2 studies are ongoing to assess the ...

SAN FRANCISCO -- The investigational complement-targeting agent claseprubart was safe and led to significant improvements in generalized myasthenia gravis (MG), the phase II MaGic trial showed. The ...