Researchers at the Icahn School of Medicine at Mount Sinai Hospital used a virus to deliver genes directly to the heart to reverse heart failure, shrinking an enlarged heart and improving its function.
2012 Fierce 15 member Celladon has boosted its IPO goals, seeking as much as $92 million to help get its gene therapy for heart failure through a Phase IIb study and into Phase III.
Fidelity Biosciences is placing a big bet on gene therapy, funding the launch of startup Dimension Therapeutics with some licensed patents and a plan to develop novel treatments for hemophilia.
Combining gene therapy plus an infusion of stem cells helps heal wounds faster in mice, a discovery that could eventually lead to better treatments for older people with wounds, who fail to heal as well as younger patients.
Celladon and Xencor have cast lines into the still-blazing biotech IPO scene, seeking a combined $155.3 million and betting that the recent Wall Street excitement for drug developers will hold firm.
Scientists have used a gene therapy technique to reverse symptoms of Rett syndrome in mice, showing the potential for clinical application. Rett syndrome, an X-linked autism disorder that primarily occurs in girls, affects one in about 10,000 children a year.
Getting drugs into the brain to treat tumors and other ailments has for years proven a difficult hurdle to overcome, but neurosurgeons have begun using an MRI-based technique to guide the delivery of gene therapies to target brain cancers in real time.
The San Francisco-based gene therapy startup Audentes Therapeutics has rounded up an impressive $30 million A round. OrbiMed Advisors led the round with 5AM Ventures and Versant Ventures joining in.
More evidence points to the promise of wiping out HIV for good, but researchers believe that talk of a cure is premature. Two HIV patients who underwent bone marrow transplants to treat their blood cancers have been off their antiretroviral therapies for weeks without visible signs of the virus.
San Diego-based Celladon has made a big splash in the U.K. British scientists are playing a prominent role in a pair of clinical trials of Celladon's Mydicar, a gene therapy for heart failure which uses a benign virus to insert the Serca2a gene into heart cells.