The findings show precise correction of the SCD mutation in the β-globin gene in three SCD patients as well as restored expression of adult hemoglobin (HbA).

Many of the blood proteins that could flag neurodegeneration are so scarce that standard assays barely register them. A new UK Biobank study will test whether an ultrasensitive platform can pick them ...

In this GEN eBook readers will discover how epigenomic insights are improving every stage of the drug discovery pipeline, ...

Findings may offer insights into disorders associated with dysregulated forgetting, including dementia and PTSD.

The FDA has set a May 1 target action date for uniQure’s Huntington’s disease gene therapy candidate ifezuntirgene inilparvovec, after accepting the company’s Biologics License Application (BLA) under ...

The FDA has delayed a decision on Agios Pharmaceuticals’ supplemental New Drug Application seeking additional approval for mitapivat in sickle cell disease until next year.

Genomics launches Sentira, an Anthropic Claude-powered AI platform designed to accelerate single-cell data analysis.

Study findings reframe pulmonary hypertension as a multi-organ inflammatory disorder and point to potential diagnostic, prognostic targets.

Single-cell atlas gives researchers a baseline for exploring how injury, aging, drugs, or other changes affect cells across ...

Parse Biosciences and Nomic Bio are partnering to create Cascade, a 100-million-cell multimodal dataset combining single-cell ...

Patient safety, expanding access to gene therapy, and progress towards an HIV vaccine were prominent themes among the early ...

Shares of gene editing therapy companies mostly surged this past week on two growth catalysts: Data readouts at the European Society of Gene and Cell Therapy’s 33rd ESGCT Annual Congress, and the ...