Alphabet, Inc., the parent company of Google, plans to develop a life-long gene therapy for heart disease, the leading cause of death for men and women in the U.S.

Attaining this lofty goal will be the job of Alphabet’s gene-editing start-up, Verve Therapeutics, and Google's life science start-up, Verily.

This month, Google's venture fund, GV, partnered with three other funds to launch Verve Therapeutics with $58.5 million in Series A funding. The company’s scientific founders include Dr. Sekar Kathiresan (CEO), Dr. Kiran Musunuru (chief scientific adviser) and Dr. J. Keith Joung (strategic adviser).

Based in Cambridge, Massachusetts, Verve will focus on using CRISPR and gene-editing techniques to treat coronary artery and cardiovascular disease.

Verve and its founders and investors believe they can use gene editing and therapies to create a new paradigm for treating coronary artery disease-modifying patients’ cells to protect against heart attacks and otherwise improve various risk factors such as triglycerides, cholesterol and blood vessels.

“We think we can confer resistance to heart attacks” that could last throughout the patient’s life, said Dr. Kathiresan.

Verve Therapeutics will begin by developing a gene therapy that should dramatically lower circulating levels of bad or LDL cholesterol for life following a single administration. On the other hand, Verily’s job will be to develop the first nanoparticle vehicle that can deliver Verve's gene therapy to specific cells in the liver. Both start-ups have their work cut out for them since gene therapy is a billion dollar gamble with no guaranteed financial returns.

Experts note that gene therapies are difficult to develop and dangerous to administer. For these reasons, biopharmaceutical companies such as Verve that develop them have always aimed at life-threatening rare diseases. Verve, however, has stunned the medical community and investors because it's focusing on heart disease where the financial returns are bound to be smaller.

“Gene editing has the potential to completely transform the treatment paradigm for the disease,” said Musunuru. “Preclinical studies conducted in the field, including work done in my lab, have shown the promise of gene editing to safely reduce cholesterol and other coronary artery disease risk factors.”

Verve has already validated the efficacy of gene-editing approaches, including CRISPR, to safely reduce cholesterol in the lab.

It’s already licensed CRISPR patents, including Cas9 and Cas12a (Cpf1) from the Broad Institute and Harvard University. It also has a strategic collaboration deal with Beam Therapeutics.

Under this deal, Verve gains exclusive access to Beam’s base editing, gene-editing, and delivery technologies for human therapeutic applications against cardiovascular targets. Once Phase I trials are completed, Beam can participate in future development and commercialization and share 50 percent of profits and losses for any product directed against those targets. They will also collaborate on delivery technologies.

Verve’s partnership with Verily will see it use the latter’s nanoparticle screening platform to develop and optimize new gene-editing delivery systems.

“Coronary artery disease is a true pandemic and a growing health crisis,” said Dr. Kathiresan.

“Our genetic understanding of coronary artery disease, combined with increasing sophistication of gene editing technologies, have aligned to create a transformative moment in the treatment of this disease. Verve was founded to turn the tide of coronary artery disease worldwide. Gene editing offers the possibility of introducing protective gene variants to adults at risk of the disease through a one-time therapy.”

RELATED STORIES