Category: Cell and 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 8, 2024

Genezen, a gene therapy contract development and manufacturing organization (CDMO), and uniQure, a gene therapy company, announced on July 1, 2024 that they have entered into an agreement under which Genezen will acquire uniQure’s commercial gene therapy operations in Lexington, […]

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

Like many of its cell therapy peers, Artiva reshuffled its strategy hoping to ride a recent wave of investor interest in autoimmune disease research. The company is one of a number of biotechs to emerge in recent years to develop […]

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 24, 2024

The new data from Vertex’s phase 1/2 trial of VX-880, presented at the American Diabetes Association (ADA) annual congress in Florida, suggest that the therapy can restore the physiological activity of the insulin-producing islet cells of the pancreas and restore glucose […]

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 16, 2024

To date, the FDA has approved 36 cell and gene therapy products. In the past decade, new cell therapy modalities such as chimeric antigen receptor T-cell (CAR-T) immunotherapies have emerged as promising treatments especially for many types of cancers. There […]

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 […]

May 1, 2024

Astellas Pharma is betting again on cell therapy, announcing Wednesday a deal with Poseida Therapeutics to develop donor-derived treatments for cancer. Through the partnership, the companies aim to create two allogeneic, or “off-the-shelf,” cell therapies for solid tumors. Astellas will […]

April 23, 2024

The Food and Drug Administration has rejected an experimental cell therapy for a rare skin condition, asking its developer, Abeona Therapeutics, to provide more information about how the treatment is made. Abeona on Monday said the FDA issued a so-called […]

April 22, 2024

CAR-T therapies are personalized treatments made from patients’ own immune cells. Their production is precisely choreographed, as frozen cells are brought to manufacturing hubs where they are engineered and expanded into a cancer-seeking drug product. It’s laborious, time-consuming and costly. […]