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LATEST NEWS
FDA Launches New Pathway to Fast-Track Treatments for Ultra-Rare Diseases
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.
Feb 25
Stem Cell Breakthrough Lets Paralyzed Patient Stand Again
Japanese researchers restored movement in a paralyzed man using reprogrammed stem cells injected into his spinal-cord injury. The treatment, tested in four patients, helped one man stand independently and another regain limb movement, offering early evidence that iPS cell therapies may support neural repair and future paralysis treatments.
Dec 3, 2025
Scientists Cure Type 1 Diabetes in Mice Using a Gentle Immune System “Reset”
Stanford Medicine researchers cured Type 1 diabetes in mice using a gentle immune-reset approach that combines donor blood stem cells and pancreatic islet transplants. The method prevented autoimmunity, required no immunosuppressive drugs, and may pave the way for future treatments for diabetes and other autoimmune diseases.
Dec 2, 2025
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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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