SMRTS, a smart mRNA system, enables cell‑selective gene expression, expanding the mRNA toolbox for precision cancer therapies.

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College ...

A 16-year-old with a rare blood disease was cured with innovative gene therapy by doctors at the Children’s Hospital at ...

Major is one of the first pediatric patients in Florida to receive gene therapy for sickle cell disease since the FDA ...

Charli qualifies for a gene therapy trial in California through the Charlotte and Gwenyth Gray Foundation but the family needs to raise $850,000 between now and the end of the year to continue with ...

In a new study, scientists at the Max Planck Institute for Evolutionary Anthropology in Leipzig analyzed the impact of more ...

Researchers at the Icahn School of Medicine at Mount Sinai have developed a first-of-its-kind mRNA system that switches on therapeutic genes preferentially inside targeted cells—an advance ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College ...

Milestone Pharmaceuticals won FDA approval for a new nasal-spray medicine to treat episodes of paroxysmal supraventricular ...

If the FDA ceases to be a predictable source of regulatory guidance, then China will gain a significant advantage in the race ...

Discover top biotech companies for 2026, featuring emerging stars and leaders set for major clinical and regulatory ...