Rhinovirus (RV) infection is among the most common respiratory infections causing exacerbations of chronic obstructive pulmonary disease and asthma. The high antigenic diversity of RV is a barrier to the development of effective cross-protective vaccines for RV infection.

Neuroblastoma is a pediatric solid tumor characterized by dysregulation of the CDK-RB-E2F axis, leading to elevated E2F activity, uncontrolled progression through the G1/S transition and an aggressive tumor phenotype. Researchers from Circle Pharma Inc. and collaborators presented preclinical data on CID-078, a first-in-class oral macrocycle with dual cyclin A and B RxL inhibitory activity, in models of neuroblastoma.

To address the various limitations of traditional CAR T therapy in autoimmune disease, Sail Biomedicines Inc. has developed an in vivo CAR T platform that enables in vivo transient programming of patient immune cells.

Frontera Therapeutics Inc. has developed FT-017, an adenovirus-associated vector(AAV)-based gene therapy that carries a human MYBPC3 optimized codon, for the treatment of hypertrophic cardiomyopathy (HCM).

Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.

Tissue factor (TF), a transmembrane protein overexpressed in tumors such as cervical, head and neck, NSCLC, and pancreatic cancers, initiates the extrinsic coagulation pathway under normal subendothelial expression. TF-targeted vedotin antibody-drug conjugates (ADCs) are in clinical evaluation for solid tumors, but toxicities, including peripheral neuropathy, ocular effects and epistaxis, limit their use.

Tumors with high levels of microsatellite instability (MSI-H), such as some subsets of colorectal, endometrial and gastric cancers, are associated with a hypermutated phenotype and a specific dependency on the WRN gene. Pharmacologic inhibition of WRN has been shown to selectively impair the viability of MSI-H cancer cells, effectively blocking tumor growth while sparing microsatellite-stable (MSS) cells.