A recent paper has identified the enhancer ETS2, located in a so-called gene desert, as a contributor to five separate immune disorders. It also showed that one of ETS2’s target genes mediating this inflammation was the eminently druggable MEK, a kinase that is the target of the FDA-approved inhibitors Mekinist (trametinib, GSK plc), Mektovi (binimetinib, Array Biopharma Inc.), Cotellic (cobimetinib, Roche Holding AG) and Koselugo (selumetinib, Astrazeneca plc/Merck & Co. Inc.). Read More
EG 427 SAS has received IND clearance from the FDA for EG-110A, a gene therapy for the treatment of neurogenic detrusor overactivity in patients with spinal cord injury. A phase Ib/IIa study is being initiated. Read More
Astrazeneca plc has added a novel target for systemic lupus erythematosus (SLE) to its discovery portfolio through its collaboration with Benevolentai Ltd. The novel target was discovered using Benevolentai’s artificial intelligence (AI) drug discovery platform and experimentally validated by Astrazeneca. Read More
Immutep Ltd. has signed a license agreement with Cardiff University granting the company exclusive rights to develop and commercialize anti-LAG-3 small molecules. Read More
Work at Neumora Therapeutics Inc. has led to the identification of new casein kinase I isoform δ (CKI-δ) inhibitors reported to be useful for the treatment of neurological disorders. Read More
Epilepsygtx Ltd. has raised a total of $10 million in seed funding to support its development of gene therapies to treat focal refractory epilepsy. Read More
4D Molecular Therapeutics Inc. has obtained IND clearance by the FDA for 4D-175, an R100 vector-based intravitreal genetic medicine, for the treatment of patients with geographic atrophy. Read More
Ranok Therapeutics (Hangzhou) Co. Ltd. has prepared 4-(3,8-diazabicyclo[3.2.1]octan-3-yl)-pyrido[4,3-d]pyrimidine derivatives acting as son of sevenless homolog 1 (SOS1)/GTPase KRAS (G12D mutant) interaction inhibitors and reported to be useful for the treatment of cancer. Read More
Checkpoint kinase 1 (Chk1) inhibitors have been described in a Boundless Bio Inc. patent and reported to be useful for the treatment of cancer. Read More
Adicet Bio Inc. has obtained FDA clearance of its IND application to evaluate ADI-270, an armored allogeneic λδ CAR T-cell therapy candidate targeting CD70-positive cancers, for the treatment of relapsed or refractory renal cell carcinoma (RCC). Read More
Cerevel Therapeutics Inc. and Pfizer Inc. have jointly developed azaindole compounds acting as phosphodiesterase 4 (PDE4) inhibitors potentially useful for the treatment of psoriasis, atopic dermatitis, idiopathic pulmonary fibrosis, diabetes, sepsis, autoimmune disease, cystic fibrosis and inflammatory bowel disease, among others. Read More
Sumitomo Pharma America Inc. has patented 2-phenylmorpholine and 2-phenyl(thio)morpholine compounds acting as 5-HT1A and trace amine-associated receptor 1 (TAAR1; TAR1) agonists. Read More
Flamingo Therapeutics BV and Ionis Pharmaceuticals Inc.’s FTX-001 (also known as FLM-7523) is a potential first-in-class antisense oligonucleotide (ASO) targeting the human metastasis-associated lung adenocarcinoma transcript 1 (MALAT1) long noncoding RNA (lncRNA); it is being developed for the treatment of cancer. Read More
Additional early-stage research and drug discovery news in brief, from: Arvinas, Clene, Dyne Therapeutics, Kura Oncology, Mira Pharmaceuticals, Scholar Rock, Turn Therapeutics, Viking Therapeutics. Read More
