Regenerative Medicines
Our science
At SmartCella we specialize in SMART cell-based therapies. With our proprietary and highly efficient human pluripotent stem cell (hPSC) differentiation protocols, we are developing first-in-class allogeneic regenerative medicines with clinical indications including advanced heart failure and Parkinson’s disease.Leveraging our extensive expertise in hPSC differentiation, SmartCella has expanded our pipeline to include novel modified cell therapies to tackle an even broader range of disease indications through prolonged and sustained protein expression via our proprietary induced mesenchymal stem cell platform with targeted mRNA delivery.
Having the commercial product in mind, we leverage our bioprocessing and analytical platform capabilities and to develop robust and scalable manufacturing processes, which are then implemented in our GMP-certified facility for the production of our SMART cell-based therapies for clinical use.
Pipeline
SMART01
SMART02
SMART03
Cell Therapies
Our current allogeneic stem cell therapy platform is being developed by our ProCella business unit. The ProCella pipeline consists of stem/progenitor cells obtained from the differentiation of hPSCs as regenerative medicines to address conditions including heart failure and Parkinson’s disease.
SMART01
Our lead program, SMART01, utilizes allogeneic cryopreserved Human Ventricular Progenitor cells (HVPs) derived from hPSCs to regenerate cardiac tissue in patients with advanced heart failure with reduced ejection fraction (HFrEF). SMART01 is entering a Phase 1/2a trial for advanced heart failure in the UK.
- PSCs are differentiated in vitro to HVPs with a proprietary differentiation protocol before cryopreservation.
- HVPs are delivered as single cells directly to the heart using the Extroducer®.
- Following delivery, the HVPs engraft in the host tissue and further differentiate to form new functional heart tissue.
- Following delivery, the HVPs engraft in the host tissue and further differentiate to form new functional heart tissue.
- In pre-clinical studies, SMART01 HVPs re-muscularized the myocardium three months after injection in post-myocardial infarction (MI) minipigs.
- The SMART01 HVPs reduced the infarct size and attenuated decline in cardiac function three months after injection in post-MI minipigs.
- We have initiated the GMP manufacturing campaign to support Ph1/2a clinical trial of SMART01.
- We have initiated the GMP manufacturing campaign to support Ph1/2a clinical trial of SMART01.
SMART02
SMART02 consists of cryopreserved allogeneic human ventral midbrain progenitor dopamine producing cells (mDAs) derived from PSCs for the treatment of Parkinson’s disease (PD). Grafted cell preparations have shown exceptional therapeutic performance in preclinical transplantation studies in parkinsonian rat models, suggesting potential to become a best-in-class cell product. Professor Johan Ericson (Karolinska Institutet) and his team have developed a novel method to differentiate PSCs into mDA neurons progenitor cells with improved efficiency and yield in therapeutic mDA neurons after transplantation. We have built upon Ericson’s work to develop our SMART02 program.
- The novel xeno-free protocol for the in vitro differentiation of PSCs substantially increases the yield of therapeutic mDA neurons after transplantation and concomitantly reduces undesired cell impurities, resulting in small transplants that are highly enriched for mDA neurons exhibiting identity of endogenous neurons
- SMART02 will be delivered as single cells injected directly into substantia nigra area of the brain
- Functional recovery is observed four months after injection in a Parkinsonian rat model (6-OHDA rats), which is notably faster than the time reported in other preclinical studies
- We are adapting Professor Ericson’s protocol to establish an efficient, GMP-compliant manufacturing process to support pivotal safety and toxicology studies, as well as the production of SMART02 for first-in-human clinical trials.
Modified Cell Therapies
Our cell-based mRNA delivery technology is being developed by our Solutions business unit. This platform leverages a first in class approach that combines the innovation of hPSC differentiated mesenchymal stem cells with an mRNA therapeutic cargo for prolonged protein expression to create a truly novel regenerative therapy.
SMART03
We are currently developing SMART03, our proprietary induced mesenchymal stem cell (iMSC) delivery platform, which builds on the paracrine anti-inflammatory properties of mesenchymal stem cells, paired with the sustained expression of regenerative factors for cartilage regeneration in osteoarthritis.
- mRNA is considered an efficient and cost-effective way of delivering and inducing protein expression.
- Protein expression with conventional mRNA therapies peaks within one day, and lipid nanoparticle (LNP)-based delivery can trigger serious immune responses.
- SmartCella’s iMSC delivery platform extends protein expression to up to 1 week in vivo, enabling new treatment options for various diseases.
- The iMSCs themselves exhibit well documented anti-inflammatory effects through paracrine mechanisms, thus further enhancing the regenerative potential of our therapy.
- The anti-inflammatory profile of iMSCs provides an ideal carrier for the mRNA, while the iMSC’s stealth properties support immune system evasion, and thus prevent mRNA degradation and avoids the immunogenic side effects of lipid nanoparticles.
- Promising in vivo data for pain relief demonstrates potential for disease-modifying applications in the future
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