At AHA 2026, CRISPR Therapeutics reported that one fixed dose of CTX310 lowered ANGPTL3 by 65% and triglycerides by 63% in 10 people with severe hypertriglyceridemia, with no treatment-related serious side effects.

A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions. New mutations could be added with lab data instead of new clinical trials, a path built for cases like KJ Muldoon's.

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.

A viral post claims scientists can now remove the extra chromosome behind Down syndrome. The 2025 Mie University study is real, but it was done in lab-grown cells, not babies, and it is far from a treatment. Here is the myth vs. fact.

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.

The FDA accepted Intellia's application for lonvo-z in hereditary angioedema with priority review, setting a March 10, 2027 decision date. It could be the first CRISPR therapy that edits genes inside the body.

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.

A June 2026 FDA draft guidance lets developers of genome-editing therapies reuse prior manufacturing, nonclinical and clinical knowledge.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.

Phase 1 data for VERVE-102, a one-time base-editing treatment that switches off PCSK9, showed LDL cholesterol cuts of up to 62% lasting up to 18 months. Here's what it means for heart disease.

Prime Medicine plans to seek FDA approval for a prime-editing treatment for chronic granulomatous disease based on two patients. Here's what prime editing is and why this filing is a test case.

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.

As CRISPR-based therapies edge closer to becoming mainstream, groundbreaking treatments for blood disorders and cancer are making their mark. One of the most exciting developments is Casgevy, a gene-editing therapy that has shown remarkable success in treating sickle-cell disease and β-thalassaemia. Approved in both the UK and US, Casgevy works by reactivating fetal hemoglobin, offering patients a chance at long-term relief with fewer blood transfusions and hospital visits. W

A team from the Broad Institute of MIT and Harvard, along with Harvard University and the Howard Hughes Medical Institute, has made exciting strides in using a gene-editing tool called prime editing to tackle the mutation responsible for cystic fibrosis in human lung cells. This innovative approach offers a more precise and safer alternative to traditional CRISPR methods, significantly lowering the chances of unintended changes to the genome. Cystic fibrosis is a genetic diso