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. Read More
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. Read More
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. Read More
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. Read More
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. Read More
Sanofi SA has synthesized new cyclopropane-aryl and vinyloxy-aryl compounds acting as transcriptional coactivator YAP1/transcriptional enhancer factor (TEAD) and/or WW domain-containing transcription regulator protein 1 (WWTR1; TAZ)/TEAD interaction inhibitors potentially useful for the treatment of cancer. Read More
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. Read More
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. Read More
CSPC Megalith Biopharmaceutical Co. Ltd. has discovered new antibody-drug conjugates (ADCs) consisting of antibodies targeting CUB domain-containing protein 1 (CDCP1) covalently linked to cytotoxic drugs potentially useful for the treatment of cancer. Read More
A recent Antelope Therapeutics SAS patent describes new antibody-drug conjugates (ADCs) comprising monoclonal antibodies targeting canAg (CA242) mucin glycoepitope covalently linked to exatecan designed for use in the treatment of cancer. Read More
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. Read More
Hangzhou Institute of Medicine Chinese Academy of Sciences has patented new nitrogen-containing heteroaryl compounds acting as CDK2/cyclin E1 inhibitors potentially useful for the treatment of cancer. Read More
Université de Montreal has identified new GTPase KRAS mutant inhibitors potentially useful for the treatment of cancer, inflammatory and immunological disorder. Read More
Werner syndrome helicase (WRN), belonging to the RecQ helicase family, represents a synthetic lethal vulnerability in MSI-H cancers, providing a strong rationale for therapeutic inhibition. Researchers from Simcere Zaiming Pharmaceutical Co. Ltd. reported the discovery and preclinical characterization of ZMS-4084, a novel WRN inhibitor. Read More
