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.

Interleukin-4 receptor α (IL-4Rα), a shared receptor subunit for both IL-4 and IL-13, along with thymic stromal lymphopoietin (TSLP), are key drivers of the type 2 inflammatory cascade involved in asthma and other inflammatory disorders.

The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.

Pulmonary fibrosis is a lung disease with limited therapeutic options and the development of new therapeutics is a clinical unmet need. Little is known about the role of endoplasmic reticulum stress and unfolded protein response seen in macrophages during pulmonary fibrosis.

Peptide-drug conjugates (PDCs) are emerging as a promising alternative to antibody-drug conjugates (ADCs), offering enhanced tumor penetration and reduced immunogenicity. Carbonic anhydrase IX (CAIX) and epidermal growth factor receptor (EGFR) are both well-validated targets in oncology due to their role in cancer cell survival, invasion and migration.

Chronic obstructive pulmonary disease (COPD) is among the most leading causes of death around the world and there are insufficient treatment options that prevent exacerbations or alter the progression of the disease. COPD is a complex disease with multiple factors driving inflammation, emphysema or small airway remodeling, among others, where interleukin-1 receptor-associated kinase 4 (IRAK-4) plays a crucial role in the pathogenesis of the disease.