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Beam Therapeutics Acquires Georgia Tech Spin Out, Seeks Non-Viral Gene Therapy Delivery Vehicles
Months from initiating its first human trial, Beam is covering all its bases for the big day. On 23rd February, it acquired Georgia Tech spinout, Guide Therapeutics, a company that develops tools used to deliver genetic medications into cells. The $120 million all-stock deal, which Beam completed yesterday, centers on lipid nanoparticle technology that Guide is building for efficient and far-reaching transport of gene-editing machinery. Guide could receive as much as $320 million more in Beam stocks if the technology fares in clinical trials and hits certain milestones.
When asked why to acquire Guide, Giuseppe Ciaramella, the CEO of Beam, elaborated, “Since our founding, Beam’s strategy has been to establish the leading, fully integrated platform for precision genetic medicine. We believe that the innovative scientists and technology at GuideTx will enable us to broaden the reach of gene editing even further. This investment represents a significant expansion of Beam’s ongoing investment in a full suite of innovative delivery technologies for genetic medicines and potential lifelong cures for a wide range of diseases.”
Guide’s LNP Technology
Unlike the conventional delivery vehicles that use inactivated adenoviruses, Guide is testing lipid nanoparticles (LNPs) to deliver gene-editing therapies. These are similar to the ones that Moderna and Pfizer are using for delivering their mRNA vaccines. But unlike those particles, Guide’s nanoparticles have DNA barcodes. “DNA barcodes are DNA sequences rationally designed to act as molecular tags for specific nanoparticles.”
This serves two purposes. First, “hundreds of nanoparticles can be screened simultaneously in a single experiment,” saving time on R&D and accelerating the discovery of new nanoparticles. Second, it allows researchers to study how these particles are distributed throughout a tissue or organ and how specific they are for the tissue of interest. According to Guide, its screening platform can perform drug-delivery experiments in vivo at a rate of 15,000 times higher than a traditional platform. With years of research, GuideTx has developed a broad repertoire of “lipids and lipid formulations that could accelerate novel delivery” of gene editing tools to tissues other than the liver.
“GuideTx’s capacity to execute rapid high throughput experiments can potentially identify the ideal delivery vehicle for reaching specific tissue types, which could lead to improved nonviral delivery technologies. This acquisition builds on our core strength with existing validated delivery strategies, such as LNP targeting of the liver, and expands our ability to explore new tissues and disease indications with our editing technologies,” said Giuseppe Ciaramella, Ph.D. President and Chief Scientific Officer of Beam.
Beam’s Gene Therapy Programs
Guide’s delivery technology is an addition to the delivery methods that Beam is already developing, including electroporation, adeno-associated viruses, and LNPs. The company is planning to pack these vehicles with the base editing therapies it is developing. Base editors are essentially CRISPRs with based editing enzymes attached to them.
The unique property of these editors is that they only change a single base in the DNA, making it a suitable therapy for all single mutation diseases, like Sickle cell disease. Beam currently has three IND-enabling base editing programs in the pipeline, including BEAM-101 for Sickle cell disease and beta-thalassemia, BEAM-102 for Sickle Cell Disease alone, and BEAM-201 for T-cell acute lymphoblastic leukemia. Although, at the moment, Beam will use electroporation to deliver these therapies, it may use in-house LNPs and Guide’s LNPs to target ocular and liver diseases later.
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