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.
Baxter is buying out collaborator Chatham Therapeutics for $70 million, planning to absorb the gene therapy specialist into its growing hemophilia R&D operation as it prepares to separate its drug and device businesses next year.
Baxter International is the latest life sciences player to see better living through mitosis, unveiling a plan to separate its drug development arm from its sprawling med tech business in hopes of helping each grow faster.
Oregon Health & Science University is opening a new Center for Embryonic Cell and Gene Therapy to study experimental treatments for Parkinson's disease, multiple sclerosis and a range of other conditions caused by diseased or injured cells in the human body.
A busy Servier wants to explore Celladon's technology to see if there are some small molecule applications to diabetes and other metabolic conditions that warrant clinical development.
An experimental gene therapy developed by investigators at the Icahn School of Medicine at Mount Sinai successfully regenerated heart muscle in pigs following a heart attack.
Third Rock Ventures has cut the ribbon on its latest startup, pledging $45 million to launch Cambridge, MA's Voyager Therapeutics, a gene therapy biotech with a promising platform for central nervous system treatments.
An experimental gene therapy has restored eyesight to 6 patients in a small Phase I clinical trial in the U.K., exceeding expectations of investigators, according to a new study in The Lancet.