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

ZUG, Switzerland and BOSTON - CRISPR Therapeutics (NASDAQ:CRSP) reported a narrower-than-expected loss and revenue that surpassed analyst estimates for the fourth quarter of 2024, sending shares up 0.

Jones Trading initiated coverage on ProQR Therapeutics N.V. (NASDAQ:PRQR) with a Buy rating and set a price target of $11.00, representing significant upside from the current price of $2.19. The ...

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

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

BioSkryb Genomics, a Durham company focused on single-cell and ultra-low input multiomics, has introduced a new service called ResolveSEQ MRD, which allows more detailed classification and ...

Vertex has entered a reimbursement agreement with NHS England enabling eligible SCD patients to access Casgevy in the public ...

T-cell therapy represents a groundbreaking advancement in the field of immuno-oncology. This innovative treatment involves ...

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

Stuart Arbuckle, who’s been with Vertex since 2012, plans to retire from his role in July. His departure comes as Vertex ...

Antibody therapy Mim8 was found to be safe and effective for controlling bleeds in children with hemophilia A in a Phase 3 ...

Vertex sees 2025 revenue of $11.75 billion to $12.0 billion, the midpoint of which is above analysts' average expectation of ...