Pfizer announced in February 2025 that it was taking Beqvez (fidanacogene elaparvovec), its gene therapy for hemophilia B, ...

Pfizer and BioMarin have pulled their hemophilia gene therapies, citing low demand. To prepare for his presentation at the ...

The US Food and Drug Administration (FDA) has released draft guidance on how sponsors can utilize next-generation sequencing ...

A groundbreaking gene therapy tested through a UCLA-led clinical trial has received approval from the U.S. Food and Drug ...

The FDA’s recently unveiled plausible mechanism framework may have been forged with i | The FDA’s newly launched plausible ...

Shares of MeiraGTx Holdings (MGTX) added ~17% to reach a 52-week high on Tuesday after the company scheduled a presentation to disclose data from an early-stage trial for its gene therapy, AAV-hAQP1.

CRISPR Cas9 gene therapy explained with DNA scissors, hereditary diseases treatment, and designer babies ethical dilemmas ...

A compact Cas12f nuclease shows high editing efficiency in human cells, with structural insights enabling an engineered variant potentially suited for future AAV‑compatible delivery.

Cell and gene therapies are moving towards correcting root causes of diseases. Let's take a look at future cell and gene therapy trends.

I am deeply humbled by the decades of support from people around the world. It is an honor to share this painful yet ...

Peripheral nerve injuries, often caused by traumatic events such as car accidents, falls or battlefield injuries, can leave patients with long-term weakness, numbness or loss of function. Despite ...