Tome Biosciences made a splash last week when it emerged from stealth mode with $213 million in funding.

The programmable genomic integration (PGI) company based in Watertown, Massachusetts, raised significant financing from investors like Andreessen Horowitz (a16z) Bio + Health, Arch Venture Partners, Fujifilm Corporation and others. 

Tome CEO Rahul Kakkar, MD says that the company is entering the public sphere with a host of data and ambitious ideas to revolutionize how care is delivered and moving the conversation from treatments to cures.

MM+M caught up with Kakkar to discuss the recent funding effort, Tome’s efforts to build upon the progress that’s already been made with CRISPR/Cas9 technology and why the Boston biotech scene has such momentum at its back.

This interview has been edited lightly for length and clarity.

MM+M: Can you give us a little background on the company?

Kakkar: Tome is a company founded on this concept of PGI. We do often get lumped in with other CRISPR/Cas9 companies and I can understand why. The Cas enzyme, in all its glory, is a portion of our technology. 

However, the capability that we afford those enzymes by putting it in a much wider enzymatic architecture enables this idea of manipulating DNA in a way that’s far more efficient and impactful than has been accomplished before. 

The easiest way to think about that is we can start to recode DNA for what it is, which is software. If a cell is effectively a very complex computer and DNA is the operating system, we haven’t had the tools to recode and patch the operating system. Every software has bugs, and unfortunately, nature has imparted bugs in our system, particularly to those who were born with devastating genetic diseases. 

For the first time, we can think about correcting those errors. In a way that looks like a drug can be developed, marketed and bring the word cure into our industry.

MM+M: What was the funding experience like and what was the response you got from discussions with potential investors?

Kakkar: It has been an interesting journey. Certainly, these sorts of big idea, big science platforms are expensive. They require multiple different teams, different expertise, all coming together under one roof in order to develop what is ostensibly the most complex drug product our industry has ever envisioned. That’s not cheap. 

It takes a certain caliber and phenotype of an investor to both see the vision, but also be able to back a company that is expensive to run. 

We were lucky to be founded by some of the firms that can embark on these multi-year, big idea journeys, specifically of Arch, a16z and GV, in particular. 

When we raised our first round in 2021, we were still on the tail end of the hype of the COVID-19 pandemic. Fast forward to late 2022 and early 2023, as we were closing our second round, the world was a different place, particularly with the Federal Reserve having raised interest rates over 18 months. 

To be able to have investors who understand the impact, the emergence of a new class of medicines, a new way of developing genomic-based therapies and having them continue to support you, irrespective of what’s happening in the macro environment, is quite compelling and I think it validates the science.

MM+M: What do you expect in 2024 given the potential for renewed macroeconomic challenges and the uncertainty of a presidential election cycle? 

Kakkar: As we continue to move now from discovery research into more research and development, we have identified two key areas we want to work on, both for the integrative gene therapy side of the business as well as the cell therapy. 

That’s what’s unique about Tome is that we don’t look like any other company out there from a pipeline standpoint. Our pipeline ranges from corrective genomic therapies to gene therapies for rare monogenic disease all the way through to potentially curative cell therapies for common disorders. 

We have this broad pipeline that, as we move from early discovery now into development, we can start moving into program nomination. And this doesn’t become cheaper, for sure. Still, even as the macro environment will continue to be challenging in 2024, particularly because interest rates are still high and we have an election coming up, this technology now is significantly more de-risked than it was in 2021. 

As our momentum towards the clinic picks up, that also means that our technology is more de-risked and is becoming clarified in terms of the kinds of drugs we can bring to the market eventually. Those sorts of stories, even if they are big idea stories, now have validation and can continue to generate investor interest even in challenging environments.

MM+M: What do you make of the momentum that’s in CRISPR/Cas9 technology space and specifically how it affects your company?

Kakkar: It’s a wonderful time to be working in the field of genomic therapies. 

Yes, there are ebbs and flows in our industry where certain platforms, certain ideas, certain therapeutic areas come en vogue and come out. 

When we came into the pandemic, if you look at some of the valuations of the CRISPR-based companies, they were astronomical, even though these companies at that point were either years away from a clinic or early in improving their technology. 

If you look at where the valuations of CRISPR-based technologies have gone for the last couple of years, they’ve come down much more than the general biotech indexes. That’s because these sorts of things are, again, expensive, high impact platform-based companies have fallen out of vogue. 

Still, there are winds gathering at our back, like the approvals of the first CRISPR-based therapies. There is also the rise of the biotech index showing the reemergence of interest in biotech in general. 

Both of those tailwinds are going to help us as we now come out of stealth mode, especially in 2024 as we bring more of our data into the public sphere, start talking about our pipeline and our specific programs. 

MM+M: What are your thoughts on the continued momentum around Boston as an emerging biotech hub?

Kakkar: From what I’ve seen over the last decade in my career since I finished cardiology fellowship at Mass General Hospital, there has been a self propelling inertia to the Boston biotech scene. It is vibrant, it is mature, it is self-catalyzing. 

There are multiple concentrations of what you need to create a vibrant biotech community from both some, you know, world class scientists and academic institutions to, you know, venture funding, as well as pharma. That exists in the Bay Area, that exists here in Boston and it exists in the Cambridge/London area as well. There are also some efforts in the Middle East to kind of try to recreate that as well. 

But there is something that has allowed Boston to self-catalyze a vibrant startup scene that I think is quite unique and I don’t think anyone has an answer. It’s why Tome decided to call Boston, specifically Watertown. In our facility, which is 90,000 square feet, almost all 140 employees are here on site. 

We tried to create a home base kind of culture. The reason for that is kind of what I was talking about before: this is such a complicated technology. PGI isn’t one enzymatic architecture. We have envisioned three different enzymatic architectures, all of which can manipulate large formats of DNA in different types of ways and each has its own advantage. 

As we think about building these multi-enzyme, multi-delivery modality complex drug products, it’s important that our scientists, by and large, are commingling and interacting. That they’re having those serendipitous interactions, not having barriers or walls between groups or functional expertise, which are only made more stark by Zoom. We’ve decided to call Boston our home, not from a virtual standpoint, but from a physical footprint.

To read a January 2024 article on Tome Biosciences’ acquisition of Replace Therapeutics, click here.