Tag:

gene therapy

Latest Headlines

Latest Headlines

Celladon pulls the plug on R&D after a crushing gene therapy failure

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 provides a peek at progress with hemophilia gene therapy

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.

Kite and bluebird join forces with some cutting-edge cancer tech

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.

Avalanche tanks after its gene therapy posts mixed results in AMD

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 builds its case for gene therapy as first sickle cell patient responds

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 widens its CAR-T ambitions with a $130M Five Prime deal

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.

HIV biotech Calimmune bags $15M to move one-time gene therapy into new trials

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 offers a promising peek at a potential cure for sickle cell

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 beats a path to early approval for its pioneering gene therapy

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.

Northwestern team puts spherical RNA in common skin moisturizer to speed up wound healing

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.