Hypertrophic cardiomyopathy is characterised by pathologic cardiac growth as a result of genetic mutations and/or chronic cardiac stress. It is the primary cause of sudden cardiac death in young adults, and is common among men aged 35 and above, diabetics, and in obese and hypertensive adults There are currently no approved therapies for hypertrophic cardiomyopathy and thus unmet need is very high .
IND & CTA filing.
Loss of cardiomyocytes (heart cells) is the primary hallmark of heart failure. However, unlike other cell types, there is a limited capacity for cardiomyocytes proliferation in the adult heart and in the failing human heart. Therefore, regenerative approaches to replenish the lost cardiomyocytes are necessary to recover the contractile capacity of the heart and prevent death. Targeting cardiomyocyte proliferation is a potential new therapeutic strategy for myocardial regeneration and repair. Using our platform, we have uncovered novel therapeutic RNAi approaches that aid heart cell regeneration and proliferation. Patents, publications in progress; compounds validated in preclinical models.
Cardiotoxicity is a growing issue in Oncology: cardio-oncology is an emerging discipline focused predominantly on the detection and management of cancer treatment-induced cardiac dysfunction (cardiotoxicity). Around 20% of all cancer survivors will develop cardiovascular co-morbidities, with 1 in 10 cancer patients dying from cardiovascular complication, rather than cancer. In 2016, the European Society of Cardiology (ESC) established a task force of KOLs, raising the urgent need to evaluate the impact of anticancer drugs on cardiotoxicity linked morbidity and mortality. This led to the 2016 ESC position paper developed under auspices of the ESC committee for Practice Guidelines in the European Heart Journal, Zamorano et al. (2016).
Metabolic modulation and cardioprotection
Kearns-Sayre Syndrome (KSS) is a rare mitochondrial disease (7,400 – 22,000 patients in Europe) caused primarily by large-scale mitochondrial DNA deletions. Cardiac dysfunction is the principal clinical manifestation and primary cause of death. There are no current treatment options available that go beyond addressing some of the symptoms associated with KSS thus the unmet need is very high.