Artificial Intelligence in Medical Affairs: A New Paradigm with Novel Opportunities.

The advent of artificial intelligence (AI) revolutionizes the ways of working in many areas of business and life science. In Medical Affairs (MA) departments of the pharmaceutical industry AI holds great potential for positively influencing the medical mission of identifying and addressing unmet medical needs and care gaps, and fostering solutions that improve the egalitarian and unbiased access of patients to treatments worldwide. Given the essential position of MA in corporate interactions with various healthcare stakeholders, AI offers broad possibilities to support strategic decision-making and to pioneer novel approaches in medical stakeholder interactions.

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy.

Large genes including several CRISPR-Cas modules like gene activators (CRISPRa) require dual adeno-associated viral (AAV) vectors for an efficient in vivo delivery and expression. Current dual AAV vector approaches have important limitations, e.g.

A gene therapy for inherited blindness using dCas9-VPR-mediated transcriptional activation.

Catalytically inactive dCas9 fused to transcriptional activators (dCas9-VPR) enables activation of silent genes. Many disease genes have counterparts, which serve similar functions but are expressed in distinct cell types. One attractive option to compensate for the missing function of a defective gene could be to transcriptionally activate its functionally equivalent counterpart via dCas9-VPR.