Brainstorm Cell Therapeutics Inc.’s Nurown got a thumbs down from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee Sept. 27, as the committee voted 1-17, with one abstention, that the data presented demonstrated substantial evidence of effectiveness for treatment of mild to moderate amyotrophic lateral sclerosis.
Long bones, vertebrae and skull bones have distinct types of stem cells, and new insights into those stem cells could lead to new ways to treat both rare developmental disorders of skull formation and the all-too-common phenomenon of bone metastases. Scientifically, the work, which was published in two papers by Matthew Greenblatt and colleagues in Nature, adds to the increasing understanding of bone’s complexities. “Bone may serve as an endocrine organ that is secreting factors throughout the body,” Greenblatt said.
Lipids are “very diverse, but also vastly understudied,” Anne Brunet told the audience at the recent meeting on Aging Research and Drug Discovery. Advances in the ability to predict protein structures have fueled a much better understanding of the human proteome and its estimated 20,000 members. The lipidome is much larger, numbering maybe 100,000 total. And what those lipids do remains much more fuzzy. “Very little is known about their function, and especially their function during aging,” Brunet said. Slowly, however, technological advances are enabling researchers to understand the roles of lipids as well.
With the U.S. FDA’s approval of Biolinerx Ltd.’s stem cell mobilization agent Aphexda (motixafortide) for treating multiple myeloma (MM) patients, the company is prepping for a launch in the next two or three weeks.
IPSirius SAS, an early stage French immuno-oncology firm, hopes to obtain a clinical trial authorization from the U.K.’s Medicines and Healthcare Regulatory Products Agency next year, to enable it to move its novel therapeutic cancer vaccine into a first-in-human trial in patients with non-small-cell lung cancer.
IPSirius SAS, an early stage French immuno-oncology firm, hopes to obtain a clinical trial authorization from the U.K.’s Medicines and Healthcare Regulatory Products Agency next year, to enable it to move its novel therapeutic cancer vaccine into a first-in-human trial in patients with non-small-cell lung cancer.
Gene therapy technology makes it possible to select diseased or mutated cells from a patient, modify them in the laboratory and reintroduce them to the body to treat different disorders. This is known as ex vivo autologous gene therapy. The difference with allogeneic cell techniques is whether the donor is oneself (autologous) or a compatible person (allogeneic), which would provide healthy cells that do not need genetic modification.
Restoring levels of the immune modulator mesencephalic astrocyte-derived neurotrophic factor (MANF) in the muscles of aged animals improved their regenerative capacity by promoting repair-associated injury responses, researchers from the University of Lisbon have discovered.
The science that led Garuda Therapeutics Inc. to a $62 million series B financing was a combination of hard work, luck and serendipity, according to co-founder and CEO Dhvanit Shah. At the Harvard Stem Cell Institute, Shah and his fellow researchers found that endothelial cells go through significant modifications before becoming hematopoietic stem cells. That simple discovery, as Shah told BioWorld, brought on research leading to the possibility that patients would not need a marrow donor before receiving a stem cell treatment.
Sensorium Therapeutics Inc. closed a $30 million series A round to fund the discovery and development of new psychiatric drugs, inspired by human ethnobotanical practices that date back hundreds or even thousands of years.
