NRG Therapeutics Ltd., has nominated NRG-5051 as its first development candidate, and secured a $5 million grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support its preclinical development of as a disease-modifying treatment for Parkinson’s disease.
The FDA has granted orphan drug designation to Capsida Biotherapeutics Inc.’s CAP-002, an investigational gene therapy for the treatment of developmental and epileptic encephalopathy due to syntaxin-binding protein 1 (STXBP1) mutations.
The prospect of a gene therapy for Alzheimer’s disease has kept Wall Street steadily interested in Lexeo Therapeutics Inc., and another increment of intrigue was added when the New York-based firm offered positive interim results from the 52-week, 15-subject phase I/II study of LX-1001 for the treatment of the condition when APOE4-associated.
Two days after Monte Rosa Therapeutics Inc. signed a molecular glue degrader deal with Novartis AG, two other companies, Biogen Inc. and Neomorph Inc., are moving forward in the same space in a partnership worth up to $1.45 billion. Cambridge, Mass.-based Biogen and San Diego-based Neomorph will develop molecular glue degraders (MGDs) for priority targets in Alzheimer’s, rare neurological and immunological diseases, using Neomorph’s MGD platform to identify and validate novel small-molecule protein degraders.
Jiangsu Alicorn Pharmaceutical Co. Ltd. has described AP2-associated protein kinase 1 (AAK1) inhibitors reported to be useful for the treatment of Alzheimer’s disease, bipolar disorder, Parkinson’s disease, schizophrenia, diabetic peripheral neuropathy and postherpetic neuralgia.
Sandboxaq has received a $25 million research grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF) LRRK2 Investigative Therapeutics Exchange (LITE) program to advance drug candidates for Parkinson's disease (PD).
While phase II results of Coya Therapeutics Inc.’s low-dose IL-2 drug, COYA-301, showed promise in Alzheimer’s disease patients when dosed every four weeks, it was the more frequent dosing of every two weeks that led to exhausted regulatory T cells and no benefits, driving down the company’s stock by nearly 28%.
Regenerative medicine company Orthocell Ltd. raised AU$17 million (US$11.28 million) to launch its nerve repair product, Remplir, in the $1.6 billion U.S. market.
Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.
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