Multimillion-dollar gene therapies being tested for sickle cell disease will be cost-effective in the United States if health disparities tied in part to decreased access to healthcare are taken into account, researchers said at a meeting of blood-disease specialists.
Vertex Pharmaceuticals agreed to a collaboration deal with the pre-clinical biotech Entrada Therapeutics, Thursday. The two companies announced a global partnership to develop intracellular Endosomal Escape Vehicle (EEV) therapeutics.
Odyssey Therapeutics raised $168 million in a Series B financing round, which will be used to advance its portfolio of immunomodulators and oncology therapies, according to founder and CEO Gary Glick.
Imagine Pharma’s founder Ngoc Thai’s ultimate vision for Imagine is to biopsy the pancreases of early Type 1 diabetics, grow out the islet cells, and reinfuse the now insulin-producing cells autologously as a way to treat and almost effectively cure T1D.
Vertex Pharmaceuticals announced Tuesday that the U.S. Food and Drug Administration has lifted its clinical hold on the company’s Phase I/II clinical trial of VX-880, a pancreatic islet cell replacement therapy for people with Type 1 diabetes (TID).
Precision BioSciences entered into an exclusive global in vivo gene editing research and development collaboration and license deal with Novartis Pharma. The two companies will work to develop a potential cure for hemoglobinopathies such as sickle cell disease and beta-thalassemia.
Vertex Pharmaceuticals announced on Wednesday that the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to the company’s candidate, inaxaplin (VX-147). Inaxaplin was granted the designation for the treatment of APOL1-mediated focal segment glomerulosclerosis (FSGS).
Weeks after announcing a significant partnership with Evotec to advance treatment for type 1 diabetes, Canada-based Sernova Corp. shared positive Phase I/II data from an ongoing clinical study of the company’s implantable Cell Pouch device that showed evidence of in vivo active insulin production following implantation of the device and islet transplantation.