Regenerative medicine displays new focus on VEGF target: GlobalData

Regenerative medicines in early-stage development have seen a change in drug targets compared to therapies in late-stage development. Early-stage therapies are now focused on vascular endothelial growth factor (VEGF) as the top drug target, with three drugs currently in development, according to GlobalData.

Regenerative medicine is an approach to replacing or regenerating human cells, tissues, or organs to restore or establish normal function. Early-stage targets for regenerative medicine are currently led by VEGF. Surprisingly, the majority of the targets for drugs in early-stage development are not included in the top 10 late-stage drug targets, highlighting a shift in the targets for regenerative medicines.

Ikram Triki, Drugs Intelligence Analyst at GlobalData, comments, “Collagen Alpha 1(VII) chain (COL7A1) and Cells Expressing B Lymphocyte Antigen CD19 lead the late-stage targets with three drugs in development each. Two key regenerative medicines in late stages are beremagene geperpavec, a gene therapy by Krystal Biotech Inc for the treatment of epidermolysis bullosa, a rare dermatological condition, and Yescarta, a gene-modified cell therapy by Gilead Sciences Inc indicated for nodal marginal zone B-cell lymphoma, with both therapies currently in pre-registration.”

VEGF is an angiogenic protein with neurotrophic and neuroprotective actions. Increased focus on VEGF as a regenerative medicine target is attributed to its potential use as a target to help CNS indications such as Alzheimer’s disease (AD) and Parkinson’s disease, two common neurodegenerative diseases with severe unmet need.

Triki continues, “One of the regenerative medicines in early-stage development focusing on VEGF as a target, is ‘Encapsulated VEGF Secreting Cells’ by the University of the Basque Country, a gene-modified cell therapy that aims to reveal these angiogenic properties and improve cognitive impairment.”

Triki concludes, “There is a distinct shift in drug targets between early- and late-stage therapies, with drugs moving away from COL7A1 and CD19 to the VEGF target. However, gene therapies remain key molecule types for these targets. Thus, positive outcomes for these gene therapies including those in early-stage development targeting VEGF may lead to changes in the marketed landscape, resulting in diversified targets within the drug market across therapy areas such as oncology and CNS.”