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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
Base Editor Gene Therapy for CPS1 Deficiency Treats Baby’s Rare Disorder
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.
Jul 1, 2025
Gene Therapy Restores Enzyme Levels to Treat Hypophosphatasia - A More Permanent Treatment
Hypophosphatasia (HPP), or "soft bone disease" is a rare genetic disorder that impacts bone and tooth development, leading to weak bones...
Feb 4, 2025
6-Year Update on Gene Therapy for CALD: Positive Results with Continued Caution
A recent 6-year checkup on boys treated with gene therapy for cerebral adrenoleukodystrophy (CALD) reveals lasting benefits for most...
Oct 19, 2024
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...
May 1, 2024
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