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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.
23 hours ago5 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.
2 days ago5 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.
2 days ago5 min read
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GENE TECH TIMES
The Future Has Arrived
Biogen’s Salanersen SMA Treatment Shows Promise After Gene Therapy
Biogen’s investigational drug, Salanersen, shows strong potential as a once-yearly treatment for spinal muscular atrophy (SMA), even after gene therapies like Zolgensma. Early trial results reveal slowed neurodegeneration and improved motor milestones in children. With reduced neurofilament levels and notable functional gains, Salanersen is heading directly into Phase 3 trials, signaling a possible breakthrough in SMA care and a promising alternative to existing therapies lik
Jul 1, 2025
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