Tag: gene therapy

July 24, 2024

The study results offer hope for patients who wish to avoid the burden of frequent Factor VIII injections. But some questions about the treatment remain. As with other gene therapies that hold out the promise of one-time solution for a […]

July 16, 2024

Affecting approximately one in every 40,000 people, FA is a neurodegenerative movement disorder and the most common form of inherited ataxia. The typical age of disease onset is between ten and 15 years, with initial symptoms including unsteady posture, frequent […]

July 15, 2024

Friedreich’s ataxia is known as a neurodegenerative disorder, as the progressive nerve damage it causes affects a person’s coordination and ability to walk or speak. But the most common reason people die from the condition is a type of cardiomyopathy […]

July 12, 2024

Affecting approximately 70,000 people in Europe and the US, Huntington’s is an inherited neurodegenerative disorder that causes nerve cells in the brain to decay over time. Patients can experience motor symptoms such as chorea, as well as behavioural abnormalities and […]

July 10, 2024

The Dutch biotech said a higher dose of the gene therapy slowed progression by 80% compared to a matched control group, measured using the Unified Huntington’s Disease Rating Scale (cUHDRS), 24 months after it was dosed, in a pair of […]

July 3, 2024

The Series B – led by Forbion and joined by other investors, including Syncona, Oxford Science Enterprises, and Oxford University Innovation – takes the total raised by Beacon since its formation last year to $290 million, making it a rising […]

July 1, 2024

The Lexington plant is a central production hub for UniQure, which describes it as “one of the world’s leading, most versatile, gene therapy manufacturing facilities.” The company has for years pitched it as a competitive advantage in the field, affording […]

June 28, 2024

The agency’s positive opinion, which specifically applies to adults with severe or moderately severe haemophilia B without a history of factor IX (FIX) inhibitors, reverses its previous decision to reject the therapy. Affecting more than 2,000 people in the UK, […]

June 20, 2024

The Food and Drug Administration has substantially loosened limits on the first gene therapy for Duchenne muscular dystrophy in a decision that could greatly expand its use even as questions remain about its effectiveness. The agency on Thursday made the therapy, called […]

June 13, 2024

The failure of a Pfizer medicine for Duchenne muscular dystrophy adds new uncertainty around the effectiveness of gene therapy for the muscle-wasting condition, days before the Food and Drug Administration is expected to decide on expanding use of a similar […]

June 5, 2024

Chlidren born deaf have had their hearing restored in both ears as a result of gene therapy, a new study reports. All five children showed hearing recovery in both ears, with dramatic improvements in speech perception and the ability to […]

May 23, 2024

Merck KGaA’s dual roles as a CDMO and traditional pharmaceutical research company make it increasingly rare in the industry. The company also has an electronics unit with expertise in semiconductors, displays and surface materials. The purchase of Mirus helps to shore up […]

May 8, 2024

Regeneron’s announcement follows news that Eli Lilly’s competing experimental gene therapy gave an 11-year-old boy who was born deaf the ability to hear. The treatment, which Lilly acquired in a buyout of biotechnology company Akouos, also targets otoferlin. The results […]

May 6, 2024

An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says. Researchers recruited 14 people with Leber Congenital Amaurosis (LCA), a rare genetic condition that causes babies to […]

March 26, 2024

The Dallas, Texas-based biotech said it plans to submit a dossier to the FDA later this year after its MCO-010 (sonpiretigene isteparvovec) gene therapy achieved almost all its primary and secondary efficacy objectives in the phase 2b RESTORE trial. Approximately […]

March 20, 2024

Gene therapy makers like Orchard are counting on insurers to recognize the value proposition of their products. Unlike traditional pharmaceuticals that might be taken regularly for the rest of a person’s life, gene therapies carry the promise of a long-lasting […]

March 6, 2024

Affecting an estimated one in 10,000 infants globally, SMA is a rare, genetic neuromuscular disease and a leading genetic cause of infant death. Caused by the lack of a functional SMN1 gene, the most severe forms of SMA result in […]

February 27, 2024

The FDA’s decision sets back many years of effort by Minerva to secure approval of its drug for schizophrenia, called roluperidone and designed to treat so-called negative symptoms of the condition. The company, which licensed rights to roluperidone from Mitsubishi […]

February 23, 2024

Roctavian’s approval was a scientific milestone, the culmination of years of research developing a genetic medicine for the chronic blood disease hemophilia A. Its launch, which began in Europe in 2022 and the U.S. last year, has been a focus […]

February 23, 2024

The regulator’s decision means that RRMM patients who have achieved a complete response or better for at least six months can receive a 1.5mg/kg dose of the BCMA-targeting bispecific antibody every two weeks. More than 35,000 new cases of multiple […]