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.

One-time genetic treatments are moving from rare diseases to common ones like high cholesterol. In JAMA Internal Medicine, two University of Michigan cardiologists explain why permanence, rare side effects and cost call for a new framework.

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.

The FDA approved Ultragenyx's Fayuvi (UX111), an AAV9 gene therapy and the first treatment of any kind for Sanfilippo syndrome type A, often called childhood Alzheimer's. Here's how it works and what the data showed.

The FDA placed a clinical hold on Regenxbio's RGX-121 for Hunter syndrome after MRIs found spinal nodules in 5 of 48 patients, derailing plans to resubmit. Here's the science and the timeline.

Intellia's one-time CRISPR therapy lonvo-z cut hereditary angioedema attacks by 87% in the Phase 3 HAELO trial. In its August update, the company set a U.S. launch target of early 2027. Here's how it works.

The FDA expanded Vertex's CRISPR therapy Casgevy to children aged 2 and older with sickle cell disease or transfusion-dependent beta-thalassemia. Here's how it works and why the approval for toddlers is notable.

An arbitration tribunal confirmed Prime Medicine's rights to PM647, a prime-editing therapy for alpha-1 antitrypsin deficiency, ending a dispute with Beam Therapeutics. Here's the science and what comes next.

In June 2026 the FDA agreed that three-year data for uniQure's AMT-130 could support accelerated approval for Huntington's disease, reversing its late-2025 stance. Here's the science and the regulatory drama.

A June 2026 FDA draft guidance lets developers of genome-editing therapies reuse prior manufacturing, nonclinical and clinical knowledge. Here's why the platform approach could speed rare-disease cures.

At ASGCT 2026, Encoded Therapeutics shared one-year data for ETX101, an AAV9 gene regulation therapy for Dravet syndrome that cut seizures by about 76% at the top dose. A pivotal study is now underway.

Regeneron's Otarmeni became the first FDA-approved gene therapy for hearing loss and the first dual-AAV therapy, approved in a record 61 days. Here's how it restores hearing in OTOF deafness.

Grace Science's GS-100, an AAV9 gene therapy for NGLY1 deficiency, earned FDA RMAT designation after treated children showed gains in motor and cognitive skills. Here's how it works and why it matters.

The FDA has approved Kresladi, the first gene therapy for severe leukocyte adhesion deficiency type I (LAD-I). By adding a working ITGB2 gene to a child's own stem cells, it gives white blood cells back the grip they need to leave the bloodstream and fight infection, with no donor needed.

The FDA unveils a “plausible mechanism pathway” to speed approval of innovative therapies for ultra-rare diseases, paving the way for personalized and gene-based treatments.

Eli Lilly has announced its $1.3 billion acquisition of Verve Therapeutics, a leading biotech company specializing in gene editing for cardiovascular diseases. This strategic move aims to accelerate the development of one-time gene therapies using base editing technology, targeting high-risk heart disease patients. The deal marks a significant step forward in Eli Lilly’s expansion into precision genetic medicine.

In a historic first, doctors used base editor gene therapy to treat a baby with CPS1 deficiency, a rare genetic liver disorder. The personalized treatment—delivered using lipid nanoparticles—led to improved ammonia levels and reduced medication needs. Unlike traditional CRISPR, base editing made precise DNA changes without cutting, offering a safer, more targeted option. This breakthrough marks a major step forward in treating rare diseases with gene-editing technology.

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