Regenerative medicine in the early stages of development (preclinical, drug discovery, investigational drugs) [IND]/ clinical trial application [CTA] Changes in drug discovery targets compared to late-stage (Phase II to pre-registration) therapeutics. According to GlobalData’s Drugs database, vascular endothelial growth factor (VEGF) has emerged as the most important drug target for early-stage therapy, with three drugs currently in development.
Regenerative medicine is an approach to replace or regenerate human cells, tissues, or organs in order to restore or establish normal function. Early-stage targets in regenerative medicine are currently being led by VEGF due to its potential as a druggable target for multiple indications in both the central nervous system (CNS) and cardiovascular therapeutic areas. Surprisingly, the majority of early-stage drug targets are not among the top 10 late-stage drug targets. This highlights the changing targets of regenerative medicine.
As shown in Figure 1, cells expressing collagen α1(VII) chain (COL7A1) and B-lymphocyte antigen CD19 are leading late-stage targets for each of the three drugs in development. Two major late-stage regenerative medicines are beremagene geperpavec, a gene therapy by Krystal Biotech for the treatment of epidermolysis bullosa, a rare skin disease, and Yescarta, a genetically engineered cell therapy by Gilead Sciences It is indicated for perinodal zone B-cell lymphoma. , B-cell non-Hodgkin lymphoma, and extranodal marginal zone B-cell lymphoma, both of which are currently pre-enrolled.
VEGF is an angiogenic protein with neurotrophic and neuroprotective properties and a target to help central nervous system indications such as Alzheimer’s disease (AD) and Parkinson’s disease, two common neurodegenerative diseases. There is increasing interest in VEGF as a target for regenerative medicine, due to its potential use as a Significant unmet needs that currently affect more than 55 million and 10 million people, respectively, in the world. One of his early-stage regenerative medicines in development targeting VEGF, the University of Basque Country’s genetically engineered cell therapy ‘encapsulated VEGF-secreting cells’, hopes to elicit these angiogenic properties and ameliorate cognitive deficits. purpose. In the past 30 years, only nine innovative drugs have been approved for the treatment of AD. Gene cell therapy was only 11%. Surprisingly, of the 61 drugs approved for Parkinson’s disease in the last 20 years, none are for gene therapy.
There is a clear shift in drug targets between early- and late-stage therapy, with drugs shifting from COL7A1 and CD19 to VEGF targets. However, gene therapy remains an important molecular type for these targets. Therefore, the positive results of these gene therapies, including those in early-stage development targeting VEGF, will trigger a shift in the market landscape, leading to diversification within the pharmaceutical market across therapeutic areas such as oncology and the CNS. It can bring you a target.