The American Society of Hematology (ASH) Annual Meeting & Exposition began December 5 with numerous presentations, abstracts and posters.
Switzerland-based Noema Pharma raised $59 million in a Series A financing round that will support the development of four clinical-stage assets the company licensed from Roche for the treatment of orphan neurological diseases with severe unmet needs.
Eli Lilly and Company and Precision BioSciences Inc. announced a research collaboration and exclusive license agreement to use Precision’s ARCUS genome editing platform to research and develop potential in vivo therapies for a series of genetic disorders.
The U.S. Food and Drug Administration is wrapping up the month of September with a few PDUFA dates, including an approval review of Aquestive’s Libervant for the management of seizure clusters in epilepsy.
Passage Bio’s first attempt to develop a GM1 gangliosidosis gene therapy treatment ran into a roadblock in the form of a clinical hold.
The Committee for Medicinal Products for Human Use of the European Medicines Agency recommended the expanded approval of Kyowa Kirin Co. Ltd.’s Crysvita (burosumab) to include older adolescents and adults living with the rare disease X-linked hypophosphatemia.
U.S. Food and Drug Administration PDUFA dates on the calendar for July include an NDA review for Jazz Pharmaceuticals’ experimental medicine for cataplexy.
Sana Biotechnology, which launched during 2019, raised $700 million in initial financing that will be used to advance the company’s discovery and development programs that create and deliver engineered cells as a treatment for different disease types.
San Diego-based Neurocrine Biosciences is partnering with Japan’s Takeda Pharmaceutical to develop and commercialize molecules in Takeda’s early-to-mid-stage psychiatry pipeline.
AMO Pharma, a British biopharmaceutical company, is targeting a pathway most often associated with cancer to treat people with genetic encephalopathy (GE).