Celladon is circling the drain, suspending all research tied to its failed gene therapy and again halving its payroll as management searches for a sale and flirts with liquidation.
Baxalta won't formally debut as an independent Baxter drug spinoff for a few more days, but the company isn't waiting to discuss an early peek at efficacy results from a Phase I/II study of their long-term gene therapy for hemophilia.
Immuno-oncology innovator Kite Pharma is joining gene therapy luminary bluebird bio to craft new treatments for HPV-related cancers, combining two high-profile technologies in hopes of developing targeted therapies.
Gene therapy developer Avalanche Biotechnologies watched its value plummet after posting some discouraging Phase IIa results for its lead candidate, a treatment for the vision-destroying wet age-related macular degeneration.
Bluebird bio took another step forward today in its carefully planned march toward a possible accelerated approval for its lead program, noting that their first sickle cell disease patient has been responding remarkably well to their therapy while adding evidence of improved durability in responses for beta-thalassemia.
Bluebird bio, already making headlines with its promising gene therapy, is expanding its efforts in immuno-oncology, pairing up with Five Prime Therapeutics to develop cell therapies for cancer.
After getting started 9 years ago with backing from angel investors, Calimmune just landed a $15 million B round, positioning the biotech to move into the clinic with a gene therapy designed to durably stymie the lethal HIV with one treatment.
Bluebird bio, at work on a gene therapy for a rare blood disorder, unveiled some early but promising data on the one-time treatment's secondary indication of curing sickle cell disease, sending the biotech's value further skyward.
Bluebird bio, developing a potential cure for a rare blood disorder, is angling for an accelerated approval as it works through clinical trials, setting out a regulatory framework that could get the gene therapy on the market sooner than expected.
Combining spherical (as opposed to linear) RNA with a common commercial moisturizer, Northwestern University researchers developed the first topical gene regulation therapy to accelerate the healing of ulcers associated with diabetes, at least in animals.