Gene therapy startup Spark Therapeutics has picked up the FDA's coveted breakthrough-therapy designation for its lead candidate, a one-time treatment that promises a permanent solution some rare eye diseases.
Japan's Astellas Pharma is the latest big drugmaker allured by the promise of gene therapy, mounting an R&D effort that could lead to a new treatment for an inherited eye disease.
Using a new type of cell transplantation, researchers at Cincinnati Children's Hospital Medical Center corrected a lung disease in mice that mimics a similar rare disease in humans.
As researchers and investors toast renewed optimism for a new generation of gene therapies, biotech bellwether Biogen Idec is deepening its commitment to the space, another sign of life for the resurgent field.
New technologies have inspired a wave of biotech startups developing gene therapies. Now, one of the field's leading lights is worried about sticker shock for the treatments working their way down the pipeline.
Over the last few years, new technologies have inspired a wave of biotech startups developing gene therapies. Now, one of the field's leading lights is worried about sticker shock for the treatments working their way down the pipeline. No wonder: They're likely to cost $1 million-plus. So, how about a new payment model for these therapies?
ReGenX Biosciences is one of the most intriguing gene therapy biotechs you never heard of. And now the executive team in charge intends to start changing that.
Using a gene-editing method, investigators were able to delete certain genes in the human papillomavirus, prompting cancer-causing cells to self-destruct. The antiviral technique could be replicated to target other DNA-based viruses like hepatitis B and herpes simplex, researchers say.
After making international headlines with the West's first approval for a gene therapy, pioneering biotech uniQure is working to flesh out its pipeline of one-time treatments, buying into an early-stage shot at heart failure that could put its technology on the biggest stage yet.
Celladon has announced the beginning of a trial it hopes will determine the feasibility of using MYDICAR in heart failure patients using left ventricular assist devices.