Mendelspod Podcast
Taking Gene Therapy to the Next Level: Rahul Kakkar of Tome

Taking Gene Therapy to the Next Level: Rahul Kakkar of Tome

Note: Today’s show is open to all thanks to the sponsorship of Elegen Bio. Elegen's proprietary cell-free DNA manufacturing technology is a game-changer for the development of mRNA vaccines as well as cell and gene therapies. Visit to order rapid, cell-free synthetic DNA now."

0:00 We need a leap forward if we’re going to cure and not just treat disease

5:17 How do you define “programmable?”

15:03 How do you minimize off-target effects?

18:25 History, current state of company

24:25 Goals and challenges

34:50 Looking ahead fifty years

There’s a new genome editing company that everyone is talking about this year.  Tome Biosciences came out of stealth in December, claiming the ability to insert DNA sequences of any size at any location across in vivo and ex vivo modalities. Their website says they’re “taking us into the final chapter of medicine.”

Tome’s CEO, Rahul Kakkar, joins us today on the program.  He’s a physician-scientist (cardiology) who has previously led a couple of biotech companies, Pandion Therapeutics and Corvidia Therapeutics, through initial funding and acquisition.  

Rahul says that even though we’ve seen incredible breakthroughs in the gene therapy arena, all of the current editing technologies are “profoundly limited.”  That includes the technology for the first CRISPR-based drug approval, Casgevy.

“Gene therapy today is putting a gene into a cell that has no relationship to the genome itself.  It’s quite unrefined.  From a clinical perspective, their impact is limited because their technology is limited.”

Why so limited, and what is this great advancement in editing technology that everyone is talking about?  Rahul says the tech is able to “reprogram” a cell.

What are the history and current state of the company and this new technology?  How are they dealing with the infamous “off-target effects?”  And what is the overall opportunity?

Rahul talks in grand terms.  He says there is a need for genomic-based therapies if we’re going to move medicine away from treating to curing disease.  

Mendelspod Podcast
Offering a front row seat to the Century of Biology, veteran podcast host Theral Timpson interviews the who's who in genomics and genomic medicine.