JPM24, Day 3: Gene editors talk business development; Roivant CEO has money to spend

We're off for another day of the J.P. Morgan Healthcare Conference 2024. The flood of licensing deals and M&A may have slowed, but there's still plenty of action to come as companies make pitches to their peers, investors and industry watchers.

We start off the day with a recap of the gene editors. 

Our Day 1 conference tracker is available here, and all the Day 2 action is wrapped here. Fierce Pharma’s take on the action so far can be found here and here.

Check out all the updates for day 3 below. 

Wednesday 4:38 p.m. ET Jan. 10

Sanofi CEO Paul Hudson did not explicitly rule out pursuing future business development centered on Pompe disease, despite the U.S. Federal Trade Commission’s recent challenge of the pharma company’s deal with Maze Therapeutics.

The north star for Sanofi remains exciting science, Hudson said at a Wednesday briefing at the J.P. Morgan Healthcare Conference.

“We will do what we always do, which is every time we see something that we think is good science, that will benefit patients, that we can do better than other people, we will try,” he said. Story


Wednesday 4:26 p.m. ET Jan. 10

Roivant's J.P. Morgan has a different vibe this year. After selling off Telavant to Roche for $7.1 billion last year, the company is flush with cash and not looking to raise funds, for once. 

"One of the things that we're often doing at J.P. Morgan is meeting with investors around raising money. We don't need to raise any money so we're still meeting with investors, but that has a different flavor to it," CEO Matt Gline said in an interview on the sidelines of the conference. 

So far, the CEO has been peppered with questions about how he plans to spend that capital, which sits at $7 billion. 

"And that's really what this conference is about for us, is meeting with prospective partners, Big Pharma, biotech companies, and trying to find our next opportunities now from a position of some pretty significant strength," Gline said.   


Wednesday 10:58 a.m. ET Jan. 10

Intellia Therapeutics Chief Medical Officer David Lebwohl, M.D., says the biotech's work to assure the FDA that its gene edits do not pass down to a patients' children, aka germline editing, is going well. 

"We feel that the off-target work we have done is satisfying them," Lebwohl said during a Tuesday presentation.

The FDA requested that Intellia conduct a new preclinical test on the impact that NTLA-2002, a CRISPR-based treatment in hereditary angioedema, may have on a developing fetus. Intellia has already cleared this hurdle for its NTLA-2001 program for transthyretin (ATTR) amyloidosis, Lebwohl said. 

So Intellia has launched a preclinical reproductive toxicity study, which Lebwohl said is not expected to have any meaningful surprises, based on earlier preclinical evidence. 

The company has benefited from a regenerative medicine advanced therapy designation in this regard, which has allowed more free communication with the FDA as the off-target possibilities are studied, according to Lebwohl.


Wednesday 10:36 a.m. ET Jan. 10

CRISPR Therapeutics, hot off the heels of the historic approval of sickle cell gene therapy Casgevy, doesn't need cash. But CEO Sam Kulkarni, Ph.D., says there's still room for business development in the gene editing biotech's future, and he's having a lot of meetings at J.P. Morgan that might one day blossom into those partnerships. 

"This is something that we've always been good at, is doing very good BD deals. Our partnership with Vertex has been an excellent one. We had other partnerships as well. The question now, is we have the money so we don't need the money for our programs. But I think it does make sense to look at potential partners because if all seven of our trials succeed, we may not have the bandwidth and capacity to prosecute all of them all the way to the finish, in which case we would start looking at potential partners," Kulkarni said during a Tuesday presentation.

And that could include Big Pharmas, who had tended to stay away from cell therapy but are now coming around to the table. 

"Interestingly, at this meeting here at J.P. Morgan, we had to rejigger our schedule because we ended up having a lot more BD meetings than we anticipated outside of investor meetings," Kulkarni said. "Because one of the things that people have been complaining about in cell and gene therapies is pharma is not jumping in, but in fact, I do think that pharma's interest in cell and gene therapies is increasing rapidly. Almost every pharma company now wants to have a cell and gene therapy strategy."

Another huge opportunity for pharma is the rapid expansion of CAR-T into auto-immune disease. 

"So we're continuing to have BD discussions. Nothing impending but I think we'll carefully think about what to partner and what not to partner based on how the data develop over the next three to four months," the CEO concluded. Story


Wednesday 9:15 a.m. ET Jan. 10

In response to a question about the long-standing CRISPR patent battle, Editas Medicine's Chief Financial Officer Erick Lucera said, "These are foundational patents, we believe everyone that's working in this area is going to have to have a conversation with us."

Just last month, Vertex Pharmaceuticals entered a deal worth up to $100 million—plus potential licensing fees—for rights to Editas’ Cas9 gene-editing tech, an agreement that encompasses freshly approved Casgevy.

In terms of different potential deals, the CFO said Editas "can be creative and structure something so we can have a win-win scenario." The scenario Lucera detailed yesterday during the company's presentation allows potential partners to use the gene-editing technology but also gives Editas either access to technology that other companies have via licensing deals or provides non-dilutive capital to continue to fund Editas' product development.