State of Possible Science Moment: From the CNS renaissance to radioligand therapy

May 13, 2024

By Ryan Boehm, Senior Director of Communications & Media Relations

Panelists during State of Possible Conference seated in white armchairs in front of a brilliant LED screen with the words, Science Moment: Rewriting What is Possible.
MassBio State of Possible Conference, April 24, 2024. (Photo/Reba Saldanha)

Following lunch is never an easy task for a conference panel, but that certainly wasn’t the case for the high-powered lineup that Chris Garabedian, CEO of Xontogeny, moderated at MassBio State of Possible Conference held in Cambridge in April.

You can watch the panel in its entirety by visiting MB Vision and logging in with your member credentials.

External innovation and the Commonwealth of Massachusetts

Emma Lees, a white woman wearing a scarf around her neck and blue blazer, speaking during a panel discussion and gesturing with her hands.
Emma Lees. (Photo/Reba Saldanha)

The backgrounds of the panelists certainly lent itself to a robust conversation about the interactions between biotech and pharmaceutical companies. Having experience in both sectors, Bristol Myers Squibb’s Emma Lees offered a unique perspective on enabling innovation. Bemoaning not always having the time “to do the right science” or doing it quickly enough to satisfy VCs, Emma appreciates pharma’s intentional efforts to seek out external innovation and, in BMS’s case, create ‘internal biotechs’ that can respond in real-time.

Combining “the nimbleness you get in biotech” with the ability of a “large company to pull in resources” quickly is how BMS is finding success and why, like many pharmaceutical companies, it has chosen the Boston area to build its R&D teams. “You see a lot of [pharmaceutical companies] relocating here because they are closer to where that early innovation is,” added moderator and former MassBio board member Chris Garabedian.

Jacob Petersen, a white man in a dark sports coat and white Oxford shirt, gesturing during a panel discussion at MassBio's State of Possible Conference.
Jacob Petersen. (Photo/Reba Saldanha)

Novo Nordisk’s Jacob Petersen concurred: “We’re pretty good in Denmark, but we’re not anywhere close to as good” as Boston and its biotechs, higher education institutions, and hospitals. Novo’s expansion here is critical to its patient-centric focus on diabetes and cardiovascular diseases, two of the world’s biggest killers, as well as on prevention and “keeping people from becoming future patients.”

Success in down years

Rapport Therapeutics did something rare in 2023: raise two large rounds of financing, a Series A and a Series B, within six months of each other. Looking beyond the headlines and the dollar amounts, said Abe Ceesay, CEO at Rapport, is where you can see what is driving investor support. Not only are funders seeing the drug at the end of the development tunnel, but they’re also actively looking for a team they can have confidence in—and Boston’s abundance of talent gives companies located here an advantage.

Stan Wang, co-founder and CEO of Thymmune Therapeutics, added that finding success right now is grounded in spotting the unmet needs in the ecosystem. And it goes beyond just technologies to what is truly a commercializable opportunity “to move the needle and transform patient care.” Being seen amongst today’s class of emerging startups, beyond the team you put together, or really part of that, is the team’s track record in executing.

Abe Ceesay, a black man wearing an open-collared blue Oxford shirt and plaid sport cost, speaking during a panel discussion and gesturing with both his hands.
Abe Ceesay. (Photo/Reba Saldanha)

Ceesay also elaborated on Rapport’s success by diving into their science. “There is truly a renaissance in CNS [central nervous system], and it’s a renaissance that is driven by patient need,” said Ceesay, mentioning specifically drivers like an aging population and the prevalence of depression and affective disorders. He attributes this resurgence to chemistry catching up with biology and investors being able to see the path to a drug.

The accessory proteins were identified, but it took chemistry a long time to answer the question, “Can you actually develop compounds that can modulate receptors through the accessory proteins?” Even today, Ceesay pointed out, you have to be disciplined in identifying the highest probability of success in science and quickly get to the definitive “go” / “no go” points through so-called “kill experiments.”

Rebooting the immune system and advances in oncology, obesity

Inquiring minds wanted to know how Stan Wang landed on the thymus as the core of the second successful company he has co-founded, Thymmune Therapeutics. For him, it was basically combining something already approved and making a difference for patients (cell therapy), something in the clinic showing safety and efficacy (induced pluripotent stem cells or iPSCs), and a new understanding of the fascinating biology of the thymus.

Stan Wang, an Asian man, speaking during a panel discussion, gesturing with one hands, and wearing a blue blazer with an open-collared Oxford shirt.
Stan Wang. (Photo/Reba Saldanha)

“If we can ultimately help every person reboot their immune system and better respond to all forms of vaccines, cancers, infections using an off-the-shelf cell therapy approach, what an impact we can have on human health across the board,” said Wang.

When prompted by Garabedian to speak to their own focus areas, Lees cited progress in oncology, while Petersen explained Novo Nordisk’s learnings around additional obesity-related indications.

“We are fortunately in the era when we are starting to talk about cures,” remarked Lees, pointing to CAR-T cells and what’s been done there to benefit patients. But the fact that some patients don’t respond to these treatments means that the industry must “continue to innovate on the modality side” to unlock the disruptive potential of antibody-drug conjugates and radioligand therapies.

For Petersen and Novo, it’s about being more deliberate up front in investigating other indications and co-morbidities that may be associated with healthy weight loss. It is “exhilarating and exciting” to think about what is possible when combining other drugs with weight loss drugs to address comorbidities directly.

What’s hot: AI, radioligand, and gene therapy

In a lightning round to wrap up the discussion, each panelist offered up their thoughts on what’s exciting (and to their chagrin, they couldn’t use their own company’s technologies). Ceesay cited the application of AI in neuroscience (“I think what AI is teaching us actually is that you can really study patient demographics to understand who are going to be the true responders to modalities.”). Wang piggybacked on Ceesay’s response elevating the intersection between AI/ML and therapeutic development.

On the pharma side, Lees again mentioned radioligand and the ability for researchers to know after just a handful of patients if the targeted agent has succeeded in getting to the tumor. For Petersen, it’s the promise of gene therapy moving from rare diseases to serious, chronic diseases – becoming a curative or preventive medicine for large patient populations.

See all MassBio News