Chief Technology Officer SmartCella and Head of Regenerative Therapies SmartCella, Sweden
Abstract: SmartCella is an innovative Swedish biotechnology company that combines the development and GMP manufacturing of regenerative medicines with methods for the targeted and precise delivery of different payloads (cells, genes, chemicals, and biochemicals) into tissues, organs, and tumour via our unique medical device - the Extroducer ®. Our pluripotent stem cell therapy pipeline aims to treat: - Advanced ischemic heart failure, cardiac progenitor cells (SMART01) - Parkinsons' disease, ventral midbrain dopaminergic neuron progenitor cells (SMART02) - Osteoarthritis, iMSCs transformed with modRNA (SMART03)
Results: SMART01: GLP toxicology studies and manufacturing development for Ph1/2a trial - engraftment and maturation into cardiomyocytes with improvement in function indicated by a reduction in infarction volume, and increase in ejection volume (regeneration of cardiac tissue) SMART02: pre-clinical results - increased % of Th1+ cells which mark dopamine-producing cells in vitro - Fewer contaminating cells that indicate poor engraft ability- low Col1A1 and FN1 - Manufacturing process with high efficiency and purity of drug product SMART03: pre-clinical results - Compared to the untreated group, treated rats experienced symptomatic pain relief from 1 week post-treatment (1wpt) until the end of the study (7wpt). The total cartilage degeneration volume showed a 15% improvement at 3wpt and peaked at 40% at 7wpt, indicative of cartilage regeneration in the damaged knee.®
Conclusions: SMART01- potential to improve cardiac function with reduced risks of current open chest surgical procedures - cardiac progenitor cells delivered as single cells endovascularly with the Extroducer SMART02- Faster improvement in rats vs current protocols/products SMART03- iMSC-mediated regenerative modRNA delivery system highlights the synergistic effect of our technology and represents significant therapeutic potential in the treatment of OA.
