The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an
The trial, which is ongoing in the U.K. and New Zealand, has been on hold in the U.S. since late last year as the FDA sought more details from Verve. Verve Therapeutics plans to soon start testing a gene editing therapy for heart disease in the U.S. after federal regulators lifted an order that had
Researchers are testing an approach that involves replacing a mutated gene in the inner ears of children with severe hearing loss. Two companies have launched clinical trials to see if they can restore hearing to children with a rare type of genetic deafness. Akouos and Decibel Therapeutics, both based in Boston, are testing experimental therapies in
Glioblastoma (GBM), an aggressive brain cancer, is notoriously resistant to treatment, with recurrent GBM associated with survival of less than 10 months. Immunotherapies, which mobilize the body’s immune defenses against a cancer, have not been effective for GBM, in part because the tumor’s surrounding environment is largely impenetrable to assaults from the body’s immune system.
Can gene therapy trials for vision loss, hemophilia, and melanoma break through in 2023? fter another turbulent year in gene therapy development, all eyes are on five major trial readouts set for H1 2023. The clinical studies target rare blood disorders, inherited retinal diseases, and metastatic melanoma—each of which poses distinct trial design challenges. First, two small
Pfizer announced positive top-line results from the Phase 3 BENEGENE-2 study (NCT03861273) evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B. The BENEGENE-2 study met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post-fidanacogene elaparvovec infusion
H.T. Begay is a happy kid. He’s smiley, silly, and definitely trying to make you laugh. The four-year-old’s two neat braids of dark hair wave behind him as he runs among the dust, dogs, and sheep near his family’s ranch on their Navajo reservation in Arizona. On a table inside the family’s sweat lodge is
CAR T therapy can treat blood cancers by inserting new genes into a patient’s own immune cells using viruses. Early clinical trial results present an alternative that forgoes viral gene transfer: CRISPR technology. Such integration of CRISPR gene editing could improve the precision, speed and cost-effectiveness of CAR T cell production. In addition, researchers hope
A newly discovered link between protein misfolding and liver cancer could help improve gene therapy for hemophilia Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world’s deadliest diseases. The findings, published
Researchers have engineered a family of adeno-associated viral vectors that cross the blood-brain barrier in primate models. Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed. Researchers have engineered viruses called adeno-associated viruses (AAVs) to deliver
