enGene's non-viral gene therapy detalimogene cleared detectable cancer in 56% of evaluated patients with BCG-unresponsive bladder cancer, and 86% kept their bladders. The company plans to start its FDA application by the end of 2026.

One-year data for CTX310, a single-infusion CRISPR therapy that switches off ANGPTL3 in the liver, showed LDL cholesterol down 53% and triglycerides down 48% at the top dose, with no treatment-related serious side effects.

Laru-zova met its primary goal in the pivotal VISTA trial: 31% of high-dose patients gained three or more lines of dim-light vision, versus none in the control group. It is the first XLRP gene therapy to win a late-stage trial.

Four-year data for AMT-130 show high-dose patients losing everyday function 61% more slowly than matched untreated patients, but a key composite score weakened. Here's what the results mean as the FDA reviews uniQure's application.

Hypophosphatasia (HPP), or "soft bone disease" is a rare genetic disorder that impacts bone and tooth development, leading to weak bones and other health complications. For years, enzyme replacement therapy has been the only effective treatment for HPP. This therapy involves injecting asfotase alfa, an enzyme that replaces the missing one in patients’ bodies. However, these injections must be given three to six times a week, which can be challenging for many patients. While t

Several clinical trials in 2025 are pushing the boundaries of gene and cell therapy, aiming to address a range of complex diseases. Below is an overview of five major trials that could provide significant breakthroughs for patients suffering from genetic disorders, cancer, and autoimmune conditions. Intellia Therapeutics’ Gene-Editing Therapy for Hereditary Angioedema Hereditary angioedema (HAE) is a rare genetic disorder that causes episodes of severe swelling, often in the

Vertex Pharmaceuticals has announced a pivotal milestone for its VX-880 therapy, a stem-cell-derived islet cell treatment to restore insulin production in people with type 1 diabetes (T1D). The treatment, which previously underwent a Phase 1/2 clinical trial, is now entering a Phase 1/2/3 trial, making it the first scalable T1D cure to reach this advanced stage. This combined trial streamlines the research process, integrating safety, efficacy, and confirmatory studies into a

Novartis's gene therapy, Zolgensma, is already approved in the U.S. for children under the age of two with spinal muscular atrophy (SMA), a severe genetic disorder that affects the muscles and is the leading genetic cause of infant mortality. Now, the treatment has the potential to expand its use to older children, following promising results from a late-stage trial. The trial focused on patients aged 2 to less than 18 who had never received prior treatment and were unable to

A recent international study has shown that lenadogene nolparvovec, a gene therapy based on adeno-associated virus (AAV), can provide long-lasting visual improvements for patients with Leber hereditary optic neuropathy (LHON). This genetic condition, caused by a mutation in the MT-ND4 gene, typically leads to severe vision loss. The study followed patients who received the therapy and found significant improvements in vision even five years after treatment. The therapy involv

A clinical trial for NGN-401, a gene therapy designed to treat Rett syndrome, recently faced a setback when a young participant experienced a severe immune reaction after receiving a high dose of the treatment. This reaction, called systemic hyperinflammatory syndrome, is a rare and dangerous immune response, causing her condition to become critical. As a result, high-dose treatments have been paused. Rett syndrome is a genetic disorder that primarily affects girls, leading t

A recent 6-year checkup on boys treated with gene therapy for cerebral adrenoleukodystrophy (CALD) reveals lasting benefits for most patients. CALD is a rare, severe brain disease that primarily affects young boys, often leading to a rapid decline in neurological function. In this follow-up, most patients showed stable brain function and minimal disabilities, showcasing the potential of gene therapy to bring hope to families facing this devastating diagnosis. A Closer Look at

On August 2nd, 2024, the FDA granted accelerated approval for afami-cel -- the first gene-engineered T-cell Therapy for advanced synovial sarcoma and the first T-cell therapy ever to get approved for solid tumors. Developed by Adaptimmune, afami-cel uses the patient's immune system to fight cancer, a mechanism similar to other existing T-cell therapies on the market. In this treatment, the patient's T cells are genetically modified to recognize and attack sarcoma cells that e

In a project funded by the National Institute of Mental Health (NIMH) and the National Institute of Neurological Disorders and Stroke (NINDS), a group of researchers have created a novel adeno-associated viral (AAV) vector to address brain disorders. This vector is more effective at reaching brain cells, opening doors for the development of treatment for brain disorders such as Parkinson's, Alzheimer's, and Huntington's Disease. Adeno-associated viral (AAV) vectors are common

On June 20th, 2024, the FDA expanded its approval of ELEVIDYS, an innovative AAV-based gene therapy developed by Sarepta Therapeutics, for the treatment of Duchenne Muscular Dystrophy (DMD) in patients aged 4 years and older. This landmark decision marks a significant advancement in the management of DMD, a serious neuromuscular condition primarily affecting young boys and severely impacting their quality of life and lifespan. Understanding Duchenne Muscular Dystrophy DMD is

The AK-OTOF-101 clinical trial by Akouos, Inc., a subsidiary of Lilly & Co., brings renewed hope to children suffering from congenital deafness through innovative AAV-based gene therapy. This pioneering research is taking place at The Children's Hospital of Philadelphia, which is leading the charge in developing effective treatments for this challenging condition. The clinical trial utilizes adeno-associated virus (AAV) vectors to deliver the OTOF gene, which encodes the otof

The FDA recently approved two groundbreaking CRISPR-based gene therapies for curing Sickle Cell Disease (SCD): Casgevy - developed by Vertex Pharmaceuticals and CRISPR Therapeutics Lyfgenia - developed by Bluebird Bio By targeting the BCL11A gene, these therapies successfully correct SCD mutations and increase fetal hemoglobin (HbF) levels in patients. This is exciting news for patients and families living with this painful disease, as it presents a potential cure rather than