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LATEST NEWS
Optogenetic Gene Therapy Gives Some Blind Patients Back a Sense of Light
In the NEJM-published PIONEER trial, an algae-derived light-sensing gene plus amber-light goggles improved light sensitivity in 6 of 10 people with late-stage retinitis pigmentosa, and 4 of 8 got better at finding objects. Here is how optogenetics works.
Oct 115 min read
Prime Medicine Doses First Patient With an In Vivo Prime Editor for Wilson Disease
Prime Medicine has dosed the first patient with PM577a, a one-time prime-editing infusion designed to correct the H1069Q mutation in liver cells and restore copper excretion in Wilson disease. It is the company's first prime editor delivered inside the body.
Oct 105 min read
The 2026 Nobel Prize for Optogenetics Is Also a Gene Therapy Story
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.
Oct 105 min read
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GENE TECH TIMES
The Future Has Arrived
FDA Approves BEQVEZ: A Breakthrough One-Time Gene Therapy for Adults with Hemophilia B
Gene therapy is transforming the landscape of treatment options, and the recent FDA approval of BEQVEZ marks a significant milestone for individuals living with hemophilia B. This innovative one-time gene therapy replaces the need for regular infusions of clotting factor, offering new hope for patients suffering from this genetic disorder. Understanding Hemophilia B Hemophilia B is a genetic condition caused by mutations in the human factor IX gene, leading to a deficiency i
May 1, 2024
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