Gene therapy developer Beam Therapeutics Inc. has acquired drug delivery specialist Guide Therapeutics Inc. in a deal valued at up to $440 million, adding yet another piece of the integrated platform CEO John Evans told BioWorld the company is striving to build. Guide's lipid nanoparticle (LNP) screening technology could help medicines developed by Beam and its partners reach new targets beyond the liver, an increasingly important endeavor for gene therapy developers like Beam, seeking to maximize the breadth of their growing portfolios. Terms of the deal included $120 million up front, plus up to $320 million in stock-based technology and product success milestone payments. Shares in Beam (NASDAQ:BEAM) fell 12.6% to $96.48 on Feb. 23.
Guide’s chairwoman and managing director of Biomatics Capital Partners, Julie Sunderland, said that integrating Guide’s LNPs “and high-throughput LNP discovery engine with Beam’s base editing, mRNA payloads and manufacturing capabilities will unlock the full potential of our platform to deliver the promise of genetic medicines to the greatest number of patients.” Guide was spun out of Georgia Tech in 2018 and backed by Biomatics, Greatpoint Ventures and GV.
Beam has already developed LNP delivery technology of its own alongside additional modalities in electroporation and adeno-associated viral-facilitated delivery. Yet, its team still saw substantial room for improvement as they sought to achieve the full potential of gene editing, Evans said. For LNPs, that meant leveraging new and different formulations of lipids that would allow for directing them to different target organs in the body.
"We were increasingly convinced that was going to be the case in the future, that we would want to go there. Guide had already started doing that work and made a lot of progress, and that's where the synergy for this deal comes from for us," Evans said.
What sets Guide's LNP technology apart, he said, is its capacity for high efficiency screening of LNP formulations. Using "DNA barcodes," sequences of DNA designed to act as molecular tags for specific nanoparticles, the approach allows for hundreds of nanoparticles to be screened simultaneously in a single experiment, "potentially generating in vivo drug delivery data at significantly greater rates compared to traditional experiments," the company said. Investigators then test each tissue of interest, looking for which LNP formulations got to which tissues.
Speaking to the direction of the gene editing field in general, Evans said LNPs had potential application for the in vivo delivery of gene editors to hematopoietic stem cells, T cells, muscle, CNS system, heart and lungs. Such a move could, for instance, allow Beam to move its sickle cell disease or other hematology programs from their current ex vivo mode, reliant on electroporation, to an in vivo mode using LNPs.
But while the acquisition of Guide has clear utility for Beam, Evans recognizes that it could also be a component of future technology partnerships. Already the company has out-licensed its base editing technology to Verve Therapeutics Inc., which is developing a potential medicine for heterozygous familial hypercholesterolemia, and Prime Medicine Inc., which is advancing a gene editing technology developed by one of Beam's co-founders, David Liu.
Beam, of course, has also developed a broad pipeline of its own, including programs for alpha-1 antitrypsin deficiency, glycogen storage disorder 1a, sickle cell disease, beta-thalassemia, acute lymphoblastic leukemia, acute myeloid leukemia and Stargardt disease, an inherited disorder of the retina. Like most companies in the gene editing space with the funds to do so, Beam is building its own manufacturing capabilities, something that Evans told J.P. Morgan analyst Eric Joseph in January would take the form of a 100,000-square-foot facility in North Carolina, due to be opened by 2023.
"Bigger picture," Joseph wrote on Tuesday, "with recent partner NHP data de-risking the lipid nanoparticle delivery of Beam’s base editor technology, combined with the establishment of the North Carolina cGMP manufacturing facility, we see the addition of in-house LNP capabilities as a clear positive for Beam, expanding the optionality of the platform and across the existing pipeline."