After dropping development in December of its lead program, Spruce Biosciences Inc. has found new life by acquiring a BLA-ready enzyme replacement therapy for the rare genetic neurodegenerative disease Sanfilippo syndrome type B. If approved, the therapy, tralesinidase alfa, could bring Spruce a priority review voucher.
When an autoimmune disease disrupted the life of someone close to scientist Dario Gutierrez, an idea emerged for a new company called Merida Biosciences focused on removing misdirected antibodies and their negative effects using Fc biotherapeutics.
When an autoimmune disease disrupted the life of someone close to scientist Dario Gutierrez, an idea emerged for a new company called Merida Biosciences focused on removing misdirected antibodies and their negative effects using Fc biotherapeutics.
President Donald Trump’s executive order on global tariffs have pushed downward the stocks of biopharma and med-tech companies, even though the impacts of his 10% baseline tariff – which excludes pharmaceuticals – and his reciprocal tariffs affecting about 60 countries across the globe, are still unclear. “The story for the day is there’s still a lot of uncertainties in terms of pharmaceuticals,” said Wayne Winegarden, senior fellow and director of the Center for Medical Economics and Innovation at the Pacific Research Institute, a free-market think tank. “It’s going to pressure margins, pressure availability. This is just a complete negative for the industry. It’s self-inflicted. It’s not just unnecessary, it’s unwarranted.”
As it prepares to advance its lead RNA editing candidate, AIR-001, into a phase I/II trial for alpha-1 antitrypsin deficiency, Airna Corp. Inc. closed an oversubscribed $155 million series B financing less than a year after completing its series A round. The company, based in Cambridge, Mass., with research operations in Tübingen, Germany, focuses not only on repairing harmful genetic variants found in rare genetic disorders, but also on introducing beneficial variants that improve health in common conditions.
As it prepares to advance its lead RNA editing candidate, AIR-001, into a phase I/II trial for alpha-1 antitrypsin deficiency, Airna Corp. Inc. closed an oversubscribed $155 million series B financing less than a year after completing its series A round. The company, based in Cambridge, Mass., with research operations in Tübingen, Germany, focuses not only on repairing harmful genetic variants found in rare genetic disorders, but also on introducing beneficial variants that improve health in common conditions.
As companies continually search for next-generation obesity prospects, one of the leaders in the disease space, Novo Nordisk A/S, has obtained an exclusive license to a preclinical-stage, first-in-class, small-molecule inhibitor of Acyl-CoA synthetase 5 (ACSL5) developed by Lexicon Pharmaceuticals Inc.
As companies continually search for next-generation obesity prospects, one of the leaders in the disease space, Novo Nordisk A/S, has obtained an exclusive license to a preclinical-stage, first-in-class, small-molecule inhibitor of Acyl-CoA synthetase 5 (ACSL5) developed by Lexicon Pharmaceuticals Inc.
Becoming the first treatment for rare genetic disorder Prader-Willi syndrome, breakthrough therapy drug DCCR (diazoxide choline) gained U.S. FDA approval as Vykat XR the evening before its March 27 PDUFA date, sending shares of developer Soleno Therapeutics Inc. up 38%.
Losing nearly 60% of its value, Mural Oncology plc backed away from its lead interleukin-2 (IL-2) drug, nemvaleukin alfa, in platinum-resistant ovarian cancer based on interim results showing it failed to reach significance in the overall survival primary endpoint.
