GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

Experimental gene therapies like AMT‑130 lower toxic huntingtin protein and significantly slow Huntington’s disease, offering ...

DNA Nanobots, a pioneer in self-assembling nanoscale therapeutics, today announced it has secured $3.5 million in funding from a prominent, family office. This significant investment solidifies the ...

You're walking around right now carrying the genetic fingerprints of infections that happened millions of years ago. Deep inside your cells, woven into the very fabric of your DNA, sit sequences that ...

Bionova Scientific LLC, an Asahi Kasei company and a bespoke contract development and manufacturing organization (CDMO), announced a strategic manufacturing alliance with Syenex, a genetic medicines ...

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis. Primarily an inherited disease, its main symptoms concern degeneration of the ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

It was the priciest biotech startup sale in North Carolina history. Now five years under Bayer, AskBio advances more AAV gene ...