Dewpoint Therapeutics Inc. has entered into a strategic research collaboration with Mitsubishi Tanabe Pharma Corp. to advance Dewpoint’s novel small-molecule condensate modulator targeting TDP-43 for amyotrophic lateral sclerosis (ALS).
Ractigen Therapeutics Co. Ltd.’s RAG-21, a novel siRNA therapy targeting the FUS gene, has been awarded orphan drug designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).
Eli Lilly & Co. has elected to pursue the development of therapeutics against two validated drug targets for amyotrophic lateral sclerosis (ALS) from its collaboration with Verge Analytics Inc. (dba Verge Genomics).
Sea Pharmaceuticals LLC has announced it is advancing two new orally administered neurotherapeutic molecules for neurological disorders. SPM-0404 and SPM-0606 are biologically active as dual AMPA receptor (AMPAR) and kainate receptor (KAINR) antagonists.
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.
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%.
At the ongoing European Society of Gene & Cell Therapy meeting in Rome, Aviadobio Ltd. presented preclinical data for a novel ATXN2-targeting miRNA-containing vector, AVB-205, developed based on previous research that has shown the potential of ATXN2 silencing as a promising therapeutic strategy for amyotrophic lateral sclerosis (ALS) and tau-negative frontotemporal dementia (FTD).
The first bispecific antibody to win regulatory approval is about to make a comeback 10 years after being taken off the market in Europe for commercial reasons. Catumaxomab, then called Removab, and now reborn with the brand name Korjuny, received a positive opinion for the treatment of malignant ascites from the EMA’s Committee for Medicinal Products for Human Use (CHMP,) at its monthly meeting Oct. 14 to 17.
