Regenerative medicines in early-stage development have seen a change in drug targets compared to therapies in late-stage development.
According to analysts GlobalData, early-stage therapies are now focused on vascular endothelial growth factor (VEGF) as the top drug target, with three drugs currently in development.
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.
Collagen Alpha 1(VII) chain (COL7A1) and Cells Expressing B Lymphocyte Antigen CD19 lead the late-stage targets.
Two key regenerative medicines in late stages are beremagene geperpavec, a gene therapy by Krystal Biotech for the treatment of epidermolysis bullosa, a rare dermatological condition, and Yescarta, a gene-modified cell therapy by Gilead Sciences indicated for nodal marginal zone B-cell lymphoma.
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.
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.
Ikram Triki, Drugs Intelligence Analyst at GlobalData, commented: “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.”