Researchers from New York’s Mount Sinai have partnered with Theragene Pharmaceuticals to further develop a new airway-delivered gene therapy for pulmonary hypertension (PH). After showing promising results in animal studies, this new therapy will advance to clinical trials, which are expected to take place in the next two years.
PH is a rare disease characterized by high blood pressure in the blood vessels leading from the heart to the lungs. The condition is associated with an abnormal remodeling of blood vessels caused by abnormally high levels of calcium in vascular cells.
Mount Sinai researchers have previously reported that a molecule called sarcoplasmic reticulum calcium ATPase pump (SERCA2a), which is known to regulate calcium levels, could be a potential therapeutic target for PH treatment.
This led to the development of a new gene therapy based on the SERCA2a gene, in which the gene is delivered directly to the lungs through an aerosolized virus which acts as a vector, increasing levels of SERCA2a protein.
Results on this new gene therapy are found in a study, “Intratracheal Gene Delivery of SERCA2a Ameliorates Chronic Post-Capillary Pulmonary Hypertension: A Large Animal Model,” that appeared in the Journal of the American College of Cardiology.
Such gene therapy, after success in studies on rodents, also leads to improvements in heart and lung functions in a swine model, the study shows. Moreover, the therapy reduced or even reversed abnormal changes in cells.
“[PH] is a devastating disease, and our work in collaboration with many laboratories across the country has allowed us to identify a specific molecular target and use gene therapy to improve cardiovascular and lung parameters in experimental models of PH,” Roger Hajjar, senior author of the studies, said in a news release, adding that this gene therapy option represents a critical advancement in PH treatment.
“We are excited about the potential for SERCA2a gene therapy as a new modality in treating this serious disease,” concluded Theragene CEO Jon Berglin. “We look forward to develop and advance this promising product into the clinic.”