CRISPR Therapeutics, at dealmaking 'middle ground,' seeks pipeline partners and new delivery tech

While most of the attention on CRISPR Therapeutics centers on the company’s Vertex-partnered cell therapy and looming FDA advisory committee meeting, Alex Harding, M.D., is cooking up the biotech's next wave of deals. 

The head of business development for the company, which is named after the Nobel Prize-winning gene editing technology, told Fierce Biotech that CRISPR is sitting at an interesting “middle ground” of partnership opportunities. The biotech's position is bolstered by the fact it has a pipeline that’s more than a dozen assets deep as well as a wallet filled with $1.8 billion in cash. 

“So we have money where we could go and do deals if we see something that's attractive to us, and then we also have a lot of assets that we could look to partner with pharma under the right terms,” Harding said on the sidelines of this year's Fierce Biotech Summit in Boston. 

Whereas most companies use the term “shots on goal” to describe the diversity of their pipeline, Harding thinks about it specifically with respect to improving the delivery of gene editing therapies. CRISPR has an internal group investigating how to use lipid nanoparticles outside of the liver along with conversations with “a lot of different companies” working on new delivery mechanisms. Harding said CRISPR is working with these potential partners to trial their technologies and “see how it's performing.” 

“If you crack delivery for cell type X, then there could be three, four, five programs that get generated,” he said. “The first step is delivery and then the gene editing, in some cases, is the part that we’ve already figured out.” 

It means that CRISPR has plenty on its R&D plate, and Harding sees the most opportunity for partnerships in its immuno-oncology and in vivo editing wings. The company has four clinical-stage, off-the-shelf CAR-Ts, two of which include updated gene edits that are meant to improve potency and limit cell exhaustion. 

While it helps for a potential partner to have a cell therapy background, it's not a prerequisite for a deal, said Harding, particularly since CRISPR has its own manufacturing capabilities. Companies that are looking to break into cell therapy have expressed interest beyond hematologic malignancies, he pointed out.

“So whether that's going into autoimmune CAR-T, which is an area that's gotten increasing interest, or whether it's going into solid tumors, we're hearing from pharmas that if we're able to get one of those things working, then they would have strong interest in getting into CAR-T as well,” he said.

Harding anticipates that CRISPR's in vivo pipeline will garner more attention once its first two assets reach the clinic, which is expected to happen over the next nine months. After that, "it's really something where we can keep churning out in vivo programs," he said. 

"We see a whole pipeline of in vivo programs coming right after the couple that we're getting into," he told Fierce. The biotech lists ongoing in vivo research programs for diseases like amyotrophic lateral sclerosis, Friedreich's ataxia and hemophilia A, among others. 

The push to find fresh partners comes as CRISPR is on the precipice of an FDA decision for its Vertex-partnered beta thalassemia and sickle cell treatment, exa-cel. The company has an advisory committee at the end of the month with an FDA decision penciled in for early December. 

Vertex and CRISPR’s tandem development effort has proven synergistic, with the two now working together on hypoimmune cell therapies for patients with Type 1 diabetes. Vertex's wholly-owned, clinical-stage T1D stem cell therapy, VX-880, has spurred insulin production in at least six patients in early-stage data, wowing investors.

Though Type 1 remains the core focus for now, Harding teased that CRISPR is also looking at ways to incorporate the vaunted GLP-1 mechanisms into this diabetes work. 

“It's probably too early to get into specifics there,” he said. “It's more exploratory.” 

Editor's note: This story was corrected to reflect that CRISPR and Vertex are not partners on VX-880.