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.

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.

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.

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.

Maipl Therapeutics Inc. has outlined preclinical progress with MA-4604, its lead investigational therapy for endometriosis. MA-4604 is an oral nonhormonal prostaglandin F2α receptor (FP) antagonist that offers a differentiated mechanism intended to address key pathological drivers of endometriosis without altering hormone levels.

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.