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.

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.

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.

Eli Lilly’s once-daily oral weight-loss pill, orforglipron, helped patients lose 12.4% of their body weight in a Phase 3 trial, but fell short of rival Wegovy’s results. The announcement sent Lilly shares down 15%, the company’s steepest drop in 25 years, despite meeting all primary and secondary endpoints and showing improvements in heart health markers.

Eli Lilly has announced its $1.3 billion acquisition of Verve Therapeutics, a leading biotech company specializing in gene editing for cardiovascular diseases. This strategic move aims to accelerate the development of one-time gene therapies using base editing technology, targeting high-risk heart disease patients. The deal marks a significant step forward in Eli Lilly’s expansion into precision genetic medicine.

Capricor Therapeutics experienced a major setback after the FDA rejected its cell therapy, Deramiocel, for treating heart disease in Duchenne muscular dystrophy (DMD) patients. The decision led to a 30% drop in Capricor’s stock and highlighted the need for more efficacy data. Deramiocel, which uses cardiac-derived stem cells, aims to regenerate heart tissue and slow cardiomyopathy progression in DMD. Capricor plans to resubmit its application with additional clinical data lat

Ultragenyx has received an FDA rejection for its experimental gene therapy targeting Sanfilippo syndrome, a rare and fatal pediatric neurodegenerative disorder. The FDA cited concerns over efficacy data, delaying approval for the treatment. The decision is a major setback for families awaiting a one-time gene therapy option to address the root cause of this rare disease. Ultragenyx plans to work with regulators to resolve the issues and continue advancing its gene therapy pip

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

Sarepta Therapeutics has paused its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, after a second patient death due to acute liver failure. The pause affects ongoing trials and therapy shipments, raising safety concerns—especially for non-ambulatory patients. Approved under the FDA’s accelerated pathway, Elevidys aimed to slow DMD progression, but recent events have sparked debate over gene therapy risks, regulatory oversight, and treatment decisions for affected f

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.

Emerging research shows that the intestinal microbiome plays a vital role in the success of CAR T-cell therapy, influencing both efficacy and toxicity. Dr. Melody Smith’s studies reveal that certain antibiotics can disrupt the microbiome, reducing overall survival and increasing neurotoxicity risks. Optimizing gut health through diet or microbiome-targeted interventions may enhance CAR T-cell outcomes for cancer patients.

In an inspiring breakthrough for cancer treatment, a woman who underwent an experimental immunotherapy trial as a child has now been in...

A groundbreaking new approach in cancer treatment has emerged, utilizing light to disable the mitochondria, the energy-producing...

Hypophosphatasia (HPP), or "soft bone disease" is a rare genetic disorder that impacts bone and tooth development, leading to weak bones...

Several clinical trials in 2025 are pushing the boundaries of gene and cell therapy, aiming to address a range of complex diseases. Below...

Vertex Pharmaceuticals has announced a pivotal milestone for its VX-880 therapy, a stem-cell-derived islet cell treatment to restore...

Novartis's gene therapy, Zolgensma, is already approved in the U.S. for children under the age of two with spinal muscular atrophy (SMA),...

A recent international study has shown that lenadogene nolparvovec, a gene therapy based on adeno-associated virus (AAV), can provide...

As CRISPR-based therapies edge closer to becoming mainstream, groundbreaking treatments for blood disorders and cancer are making their...

Oxygen levels are a critical but often overlooked factor in scientific research. Conventionally, researchers grow cells at atmospheric...