Faraz Ali, MBA, on Adding Gene Editing Programs to Tenaya’s Cardiovascular Disease Pipeline

This is the second part of an interview with Faraz Ali, MBA. For the first part, click here.

“It's not that it's a shift away from gene transfer as much as we also saw opportunity [in gene editing] all along.”

Around 5 to 10 years ago, many companies and academic institutions began pursuing the development of genomic medicines for the treatment of a wide range of genetic diseases. At the time, the most practical way to go about this was with using adeno-associated virus vector capsids for a gene transfer approach. This approach was thought to mainly have potential specifically for diseases that originated from loss of function mutations in a specific, single gene as it allowed for the addition of functional copy of the gene to a patient’s genome. On the other hand, diseases that resulted from a gain of function mutation were unlikely to benefit from this approach, as AAV vector-based gene transfer simply inserts the functional copy of the gene at an imprecise point in a cell’s DNA. Several years later, the development of technology for precise gene editing approaches enabled researchers to go after diseases caused by gain of function mutations by directly editing the mutated gene in order to stop production of the mutant protein. Tenaya Therapeutics, a company focused on using genomic medicines to address both rare and common cardiovascular diseases, began work on gene transfer approaches to heart disease as early as 2018, but recently presented their preclinical work on a gene editing approach to heart disease at at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD.

At the conference, CGTLive® spoke with Faraz Ali, MBA, the chief executive officer of Tenaya, to learn more about the company’s decision to pursue gene editing. Ali clarified that the company is not making a shift away from gene transfer to gene editing, but rather that it always intended to pursue both approaches to genomic medicine based on what made sense for a particular indication. He also noted that the platform improvements the company has been making with regard to capsids, promoters, and manufacturing will be applicable to both its gene transfer and gene editing candidates.

Click here to view more coverage of the 2024 ASGCT Annual Meeting.