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No, CRISPR Hasn't Cured Down Syndrome. Here's What the Viral Study Really Showed
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.
6 hours ago4 min read
One CRISPR Infusion Kept Cholesterol and Triglycerides Down for a Full Year
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.
6 hours ago4 min read
Beacon's Gene Therapy for X-Linked Retinitis Pigmentosa Succeeds in a Pivotal Trial
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.
1 day ago4 min read
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No, CRISPR Hasn't Cured Down Syndrome. Here's What the Viral Study Really Showed
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.
6 hours ago
One CRISPR Infusion Kept Cholesterol and Triglycerides Down for a Full Year
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.
6 hours ago
Beacon's Gene Therapy for X-Linked Retinitis Pigmentosa Succeeds in a Pivotal Trial
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.
1 day ago
Four Years In, uniQure's Huntington's Gene Therapy Still Slows Decline as It Heads to the FDA
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.
2 days ago
FDA Approves Fayuvi, the First Treatment for Sanfilippo Syndrome Type A
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.
Sep 17
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