A global team of scientists working with Johns Hopkins' Jordan Green has developed a new nonviral gene therapy for brain cancer, successfully testing it in the lab in cell lines as well as rat models for the disease.
The rapid maturation of gene therapy technologies has galvanized a wide range of projects throughout industry and academia, and now a team of researchers from Harvard and Boston Children's Hospital believe they may have come up with a one-time treatment for a genetic cause of deafness.
Parisian drug developer Gensight Biologics is swinging for a $100 million U.S. IPO to fund its work on potential one-time treatments for serious retinal diseases, angling to take advantage of a bullish market for biotechs.
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.