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.
Menlo Park, CA's Avalanche Biotechnologies pulled off a $102 million Wall Street debut, planning to spend its new cash on a pipeline of gene therapies for ocular diseases.
Gene therapy 2.0 at bluebird bio is demonstrating some early signs of success with an upgrade to its experimental therapy for the genetic blood disorder beta-thalassemia demonstrating promising results in a pair of patients, weaning them off the regular blood transfusions needed to stay alive.
You can add another upstart to the swelling list of gene therapy biotechs hitting the industry radar around the world. Sofinnova stepped in to lead the $22 million A round for Paris-based Lysogene, which is already in the clinic with a new therapy for rare cases of Sanfilippo syndrome, with plans to branch out into other neurodegenerative conditions.
Parisian biotech AAVLife has come out of stealth with a Versant Ventures-led $12 million A round, looking to advance a gene-deleting therapy that promises to treat the rare and deadly Friedreich's ataxia.