In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

Upfront fees and near-term development milestone payments totaling up to $11 millionSales milestones of $150 million or more ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

The Platform Technology Designation, which precedes the current FDA leadership, is designed to streamline the drug ...

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, ...

OCU400 is Ocugen’s novel modifier gene therapy for retinitis pigmentosa (RP). The OCU400 (AAV5-h NR2E3) gene therapy platform ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

An international group of gene editing leaders has put out a call for a 10-year ban on heritable human genome editing (HHGE), ...