Clinical trial complexity, logistical challenges associated with treatments, and resource constraints at trial sites cited as top factors that ...

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

No doubt 2025 will usher in many more advances in CRISPR therapies, thanks to the FDA’s new Platform Technology Designation ...

Advanced Therapies Week 2025—a conference focused on the latest innovations in cell and gene therapies, regenerative medicine ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

Researchers have been "making great inroads" in precision medicine, and a 15 percent cap on indirect costs could quash the momentum, experts worry.

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

Scientists in the polo-obsessed nation have produced world's first genetically edited horses, designed to outrun champion ...

Ongoing launch of CASGEVY ® continues to gain momentum; new cell patient collection initiations expected to significantly grow in 2025- -More than 50 authorized treatment centers (ATCs) activated ...

New gene therapy modalities, such as CRISPR guide RNA (single guide ribonucleic acid [sgRNA]) and messenger RNA (mRNA), ...

When William was days old, he was diagnosed with adrenoleukodystrophy — or ALD — a rare genetic disease affecting one in 17,000 people.