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.
Close to 5 years after GlaxoSmithKline signed on to collaborate with the San Raffaele Telethon Institute for Gene Therapy in Italy, the partners have stepped up with a European application to start marketing a gene therapy for extraordinarily rare cases of immune deficiency triggered by ADA-SCID. And its delivery into regulatory hands comes as the gene therapy field has been recoiling from some notable setbacks that have begun to cloud what has been a bullish sector in biotech.
Kadmon, a biotech company founded by the once-jailed former CEO of ImClone, is spinning out its gene therapy division on the path to a long-promised IPO, touting a novel approach to the fast-moving field.
Gene therapies command avid attention due to their promise of providing a cure for some terrible conditions along with the prospect of a 7-figure price tag. But some prominent investigators at the University of Pennsylvania reported that their "cure" for blindness only worked for one to three years before patients' vision began to fade again. And their decline could rattle some of the hot biotechs in the group.
Celladon's lead program--as well as its stock price--crashed after the biotech announced Sunday evening that its heart therapy Mydicar failed the primary and secondary endpoints in a Phase IIb trial, losing out in a full lineup of efficacy tests.
A biotech with deep roots in the European gene therapy field has successfully tested a new approach to creating a personalized treatment for a rare disorder of the immune system.