Mount Sinai, Theragene Form Partnership to Move Potential PH Gene Therapy to Clinical Development

Alice Melão, MSc avatar

by Alice Melão, MSc |

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New York’s Mount Sinai and Theragene Pharmaceuticals have joined efforts to move a novel investigational aerosol-delivered gene therapy to treat pulmonary hypertension (PH) into a clinical development program. The new therapy, if approved, will be the first to reverse the tissue damage caused by PH.

Clinical trials should begin in the next two years, said Dr. Roger J. Hajjar, director of the Cardiovascular Research Center at Mount Sinai’s Icahn School of Medicine.

“This 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,” Hajjar said in a news release. “We look forward to starting first-in-human studies using this approach in affected patients.”

This new gene therapy resulted from a previous study led by Hajjar, in which his team explored the therapeutic potential of the SERCA2a (sarcoplasmic reticulum Ca2+-ATPase 2a) gene for PH treatment. Using an aerosolized form of this gene combined with a viral vector — an engineered adeno-associated virus that carried the SERCA2a gene — the researchers could directly deliver it into the narrowed blood vessels in the lungs, and reverse some of their deficiencies.

“We are excited about the potential for SERCA2a gene therapy as a new modality in treating this serious disease,” said Jon Berglin, CEO of San Diego-based Theragene. “We look forward to develop and advance this promising product into the clinic.”

Researchers showed that increasing levels of the SERCA2a protein, through airway delivery of the SERCA2a gene prevents PH progression in rodent and swine models. The treatment improved the animals’ heart and lung functions, namely pulmonary artery pressure and vascular resistance, and also limited pulmonary blood vessel remodeling — a feature of PH.

“This represents another critical advancement in a potentially transformative therapeutic breakthrough by Mount Sinai scientists, demonstrating our commitment to improving health outcomes,” said Erik Lium, senior vice-president of Mount Sinai Innovation Partners.