Rocket Pharmaceuticals voluntarily withdrew the FDA submission for its Fanconi anemia gene therapy. The move follows a corporate restructuring over the summer that refocused the company on its cardiovascular programs.
Rocket Pharmaceuticals adjusted the clinical trial design to include use of approved drug to avert an excessive immune response to its Danon disease gene therapy. The patient who died was one of two who received this drug under the revised protocol.
Across the nation, state-based health insurance exchanges are searching for ways to better serve their communities while making enrollment easier, more personal, and more accessible for everyone.
The gene therapy programs of Lexeo Therapeutics span rare forms of cardiomyopathy and certain types of Alzheimer’s disease. But Lexeo’s IPO comes as a rival gene therapy developer sues the biotech, claiming its work employs stolen trade secrets.
Rocket Pharmaceuticals is acquiring Renovacor, a gene therapies developer whose lead program addresses a particular genetic mutation that leads to a type of heart failure. The all-stock deal gives Rocket the opportunity to expand its reach in genetically driven cardiac diseases.
A Rocket Pharmaceuticals gene therapy for the rare immune disorder leukocyte adhesion deficiency-I has met the survival goals of a pivotal study, paving the way for regulatory submissions that the company expects to file in early 2023.
Data on four of nine patients in a Phase I/II study presented at a conference in Spain met or exceeded the threshold agreed to with regulators for the company's Phase II trial of the therapy, RP-L102.
Choosing the right brain injury rehabilitation program is a critical decision that can shape the future for you or your loved one.