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Category: Cell and Gene Therapy


Cell and Gene Therapy, Industry

uniQure’s investigational gene therapy shown to slow Huntington’s disease progression

July 12, 2024

Via: PMLiVE

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 […]


Cell and Gene Therapy, Industry

uniQure rockets on Huntington’s gene therapy result

July 10, 2024

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry, Mergers and Acquisitions

Genezen to Acquire Massachusetts Gene Therapy Manufacturing Operations from uniQure

July 8, 2024

Via: Biopharm International

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, […]


Cell and Gene Therapy, Industry

Beacon lights up with $170m for retinal disease gene therapy

July 3, 2024

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

With revised IPO pitch, Artiva touts ‘natural killer’ cell therapy for autoimmune disease

July 1, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

NICE recommends CSL Behring’s haemophilia B gene therapy Hemgenix

June 28, 2024

Via: PMLiVE

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, […]


Cell and Gene Therapy, Industry

Vertex islet cell therapy gets type 1 diabetics off insulin

June 24, 2024

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, FDA, Industry, Regulations

Sarepta Duchenne gene therapy wins broader use from FDA

June 20, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Pfizer setback brings questions for Duchenne gene therapy ahead of Sarepta decision

June 13, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Gene Therapy in Both Ears Restores Hearing to Babies Born Deaf

June 5, 2024

Via: Drugs.com

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 […]


Cell and Gene Therapy, Industry

Merck KGaA to buy gene therapy tools maker for $600M

May 23, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry, Manufacturing, Research and Development

Addressing Performance, Scalability, and Regulatory Challenges to Accelerate Cell Therapy Manufacturing

May 16, 2024

Via: Biopharm International

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 […]


Cell and Gene Therapy, Industry

Regeneron gene therapy improves hearing in two children

May 8, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Gene Therapy Improves Vision in People With Inherited Blindness

May 6, 2024

Via: Drugs.com

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 […]


Cell and Gene Therapy, Industry

Astellas adds to ‘off-the-shelf’ cell therapy capabilities with Poseida deal

May 1, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, FDA, Industry, Regulations

FDA rejects Abeona cell therapy, asks for more manufacturing data

April 23, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Bristol Myers taps startup to boost cell therapy production

April 22, 2024

Via: Biopharma Dive

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. […]


Biotech, Cell and Gene Therapy, Industry

Century buys a startup, raises funding in cell therapy expansion

April 11, 2024

Via: Biopharma Dive

Drugmakers are moving quickly to study whether cell therapy, already a proven treatment for certain blood cancers, can tamp down autoimmune conditions, too. Lupus, a chronic disease that can cause inflammation and pain across the body, is one of the […]


Cell and Gene Therapy, Industry

Ginkgo, in search of more cell therapy tools, buys another startup

April 2, 2024

Via: Biopharma Dive

Ginkgo Bioworks is expanding its ability to help other companies make cell and gene therapies, this time by acquiring a startup with tools to improve cell-based treatments. Ginkgo on Tuesday announced it has bought Modulus Therapeutics, a Seattle-based biotechnology startup. […]


Cell and Gene Therapy, Industry

Nanoscope preps filing for retinitis pigmentosa gene therapy

March 26, 2024

Via: Pharmaphorum

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 […]