Increasing evidence supports metabotropic glutamate mGlu7 receptor as a promising target in psychiatric disorders. Neurosterix Pharma Sarl has presented data on NTX-819, a potential first-in-class, potent and selective negative allosteric modulator (NAM) of mGlu7. NTX-819 was tested in vivo in rats using behavioral tests that are relevant to disorders such as obsessive-compulsive disorder (OCD), agitation and mania/psychosis.
HCW Biologics Inc.’s HCW11-040 has been shown in IND-enabling studies to prevent bronchopulmonary dysplasia (BPD), a rare pediatric disease affecting underweight premature infants.
Ensem Therapeutics Inc. has presented data for ETX-929, a small-molecule, oral pan-KRAS inhibitor with potent ON and OFF dual-state inhibitory activity for both wild-type and mutant KRAS.
The American Society of Gene & Cell Therapy (ASGCT) and Orphan Therapeutics Accelerator (OTXL) have announced the public launch of CGTxchange, an AI-enhanced clearinghouse and marketplace built to help reactivate cell and gene therapy programs that have been shelved despite strong scientific and clinical evidence.
Create Medicines Inc. has closed a $122 million series B funding round to support progression of its pipeline of in vivo CAR therapies across autoimmune disease and oncology. The company’s proprietary mRNA-LNP platform directly engineers T cells, NK cells and myeloid cells inside the body to enable scalable, repeat-dose, off-the-shelf immunotherapies.
Camp4 Therapeutics Corp. has highlighted new preclinical data for CMP-002, the company’s lead investigational antisense oligonucleotide (ASO) therapeutic candidate for SYNGAP1-related disorder. CMP-002 administration resulted in a statistically significant improvement in seizure phenotypes and parameters in a SYNGAP1 haploinsufficient mouse model.
Korean researchers reported the discovery and preclinical characterization of KSI-028, a novel tetrahydroquinoline-based STING inhibitor with activity across multiple models of STING activation.
Directed evolution has become a central pillar in gene therapy. This engineering strategy enables the generation of more efficient variants of genetic editors and delivery vectors. Molecular diversification methods are increasingly sophisticated and are now accelerated by machine learning and AI tools, as showcased at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Boston this week.
Pfizer Inc. has synthesized substituted fused heteroaryl lactam compounds acting as dual calcitonin (CALCR; CT-R)/amylin receptor agonists potentially useful for the treatment of obesity and type 2 diabetes.
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