Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

Alzheimer’s may be driven far more by genetics than previously thought, with one gene playing an outsized role. Researchers ...

But cell and gene therapies (CGTs) are catching up. During Cell and Gene Therapy International Europe 2025, held in Berlin, Germany, two prominent showcases in neurobiology have shown promise toward ...

Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

Future GLP-1 gene therapy could enable long-term hormone production, with Fractyl Health and others planning human trials in 2024.

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...

For decades, high cholesterol has meant a lifetime of daily pills, repeat blood tests and constant reminders that the biggest ...