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 companies also working on a stem cell therapy for patients with Type 1 diabetes that’s spurred insulin production in at least six patients in early-stage data, wowing investors. Vertex doubled down earlier this year, giving CRISPR $100 million upfront to add its gene editing expertise to hypoimmune cell therapies for patients with Type 1 diabetes.
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.”
![GMP mRNA](https://static.packgene.com/wp-content/uploads/2023/07/13.jpg)
Check out our AAV CDMO service to expedite your gene therapy research
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
Skyline Therapeutics Receives FDA Orphan Drug Designation for Gene Therapy for Retinitis Pigmentosa
BOSTON & SHANGHAI, September 2, 2024 – Skyline Therapeutics, a gene therapy company focused on innovative treatments for rare and severe diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SKG1108....
Novavax Receives Emergency Use Authorization for Updated Covid-19 Vaccine
Novavax has received emergency use authorization (EUA) from the U.S. Food and Drug Administration (FDA) for its updated Covid-19 vaccine, NVX-CoV2705, joining other vaccine manufacturers in addressing evolving virus variants. The EUA permits the use of Novavax's...
Turn Biotechnologies Reports Historic Skin Cell Rejuvenation Breakthroughs at ESDR this Week
Company will present data from its successful rejuvenation of fibroblasts and keratinocytes to influential gathering of international dermatologists MOUNTAIN VIEW, Calif., Sept. 4, 2024 /PRNewswire/ -- Turn Biotechnologies, a developer of novel mRNA medicines and...
H5 influenza vaccines: What needs to be done to reduce the risk of a pandemic
As the global threat of H5N1 influenza looms with outbreaks across species and continents including the U.S., three international vaccine and public health experts say it is time to fully resource and support a robust strategy to address this and future potential...
Related Services
![](https://static.packgene.com/wp-content/uploads/2023/01/9-1.png)
Plasmids GMP Services
![](https://static.packgene.com/wp-content/uploads/2023/01/10-2.png)
AAV GMP Services
![aav icon](https://static.packgene.com/wp-content/uploads/2023/01/9-1.png)