RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO's investigational gene therapy, ...

As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...

Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...

Researchers have used gene therapy to partially reprogram the brain cells that store memories in aging mice, reversing signs of cellular decline and restoring learning ability. The technique, ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

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 ...

The MarketWatch News Department was not involved in the creation of this content. Issued on behalf of Avant Technologies Inc. VANCOUVER, BC, Jan. 2, 2026 /CNW/ -- Equity Insider News Commentary -- The ...

Gene editing refers to the precise alteration of an organism's DNA sequence at a specific locus. In cancer treatment, this technology is leveraged to modify the genetic makeup of cancer cells, either ...