Estuary Biotherapeutics launched with $103 million to develop one-time, kidney-directed AAV gene therapies for ADPKD, the most common inherited kidney disease. Here is how restoring polycystin proteins could stop, or even reverse, kidney cysts.

The 2026 Nobel Prize in Physiology or Medicine went to Karl Deisseroth, Peter Hegemann and Georg Nagel for light-gated ion channels and optogenetics. Here's how an algae gene became a tool for neuroscience, and why the first optogenetic gene therapy for blindness is now under FDA review.

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.

A groundbreaking new approach in cancer treatment has emerged, utilizing light to disable the mitochondria, the energy-producing organelles inside cancer cells. Researchers at Ohio State University have developed a targeted gene therapy using viral vectors, specifically engineered adeno-associated viruses (AAVs), that deliver genes designed to disrupt mitochondrial function directly to cancer cells. This therapy uses light to activate these genes, leading to the collapse of t

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

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

Heart failure, which affects over 64 million people worldwide, is currently considered irreversible without a heart transplant. Researchers at the University of Utah have developed a gene therapy that could change this. Using minipigs as a model, scientists successfully reversed heart failure by delivering a therapeutic gene called cBIN1 directly into heart cells. The therapy, which targets a key protein often deficient in heart failure patients, resulted in significant recov

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

In exciting news for Parkinson’s disease research, MeiraGTx has announced positive results from a Phase 2 clinical bridging study of AAV-GAD, an innovative gene therapy. The study, which involved 26 weeks of evaluation included three participant groups: high dose, low dose, and a sham control group. The clinical trial aimed to assess both safety and effectiveness, showing promising results in both areas. Key Findings: AAV-GAD demonstrated a strong safety profile, with no seri

The fight against diabetes is being revolutionized with an innovative approach from Genprex, with its gene therapy candidate, GPX-002. Genprex, a gene therapy company focused on developing therapies for cancer and diabetic patients, has announced its plan to launch a new subsidiary, NewCo, which would solely focus on developing and commercializing GPX-002. GPX-002 was designed to treat both Type 1 and Type 2 Diabetes. This gene therapy employs an engineered AAV vector to deli

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