Tome brings $213 Million to the Programmable Genome Editing Party

Tome Bioscience is developing genome-editing treatments that are based on the optimization of programmable additions via site-specific targeting elements (e.g., PASTE technology). This opens a possibility to create advanced integrative cell and gene therapies (CGTs), capable of correcting genes in vivo. Why is this a potentially significant company?

During Pharmaceutical Technology®’s September 2022 Drug Solutions Podcast titled How Pfizer Views Partnering and Investing for Emerging Therapies (1), Uwe Schoenbeck, senior vice-president and chief scientific officer for emerging science and innovation at Pfizer, was asked what he’d like to see if he could wave a magic wand to have just one more tool at his disposal, to which he replied: “Target delivery, not just for RNA but also for both small and large molecules, including oligonucleotide-based therapies. We believe if you are able to deliver your therapeutic modality to your target organ or target cell, this could bring a lot of additional advantages for patients. Allowing access to new indications where we currently do not have effective therapies, but even for existing [indications] it might be able to provide a safer, lower dose, approach ... Any kind of tropism that you could introduce, that would be selective, that could be dialed in and dialed out, as desired, we would be very interested in.”

Co-founded by PASTE developers Omar Abudayyeh, PhD, and Jonathan Gootenberg, PhD, former graduate students with Feng Zhang, PhD (Broad Institute), Tome says its programmable genomic integration (PGI) platform “is designed to enable the insertion of any DNA sequence of any size into any programmed genomic location. PGI encompasses a series of genome-editing approaches, each representing enhancements over PASTE” (2). Furthermore, going back to an earlier paper, Tome outlined its potential by saying, “Beyond direct correction of hereditary disease, gene insertion provides a promising avenue for cell therapies, and efficient integration of engineered transgenes, such as chimeric antigen receptors at specific loci, can produce improved therapeutic products in comparison to random integration … By providing efficient, multiplexed integration of transgenes in dividing and non-dividing cells and in animal models, the PASTE platform builds on fundamental developments in both integrase and CRISPR [clustered regularly interspaced short palindromic repeats] biology to expand the scope of genome editing and enable new applications across basic biology and therapeutics” (3).

References

1. Spivey, C. Drug Solutions Podcast: How Pfizer Views Partnering and Investing for Emerging Therapies. PharmTech.com, Sept. 6, 2022.
2. Philippidis, A. The Next Chapter in Genome Editing? Tome Emerges from Stealth with $213M. Genet. Eng. Biotechnol. News online. Dec.19, 2023.
3. Yarnall, M. T. N.; Ioannidi, E. I.; Schmitt-Ulms, C.; et al. Drag-and-Drop Genome Insertion of Large Sequences without Double-Strand DNA Cleavage using CRISPR-Directed Integrases. Nat Biotechnol. 2023, 41, 500–512. DOI: 10.1038/s41587-022-01527-4