RARE Daily

Tome Biosciences Launches with $213 Million to Advance Programmable Genomic Integration Platform

December 12, 2023

Rare Daily Staff

Tome Biosciences raised $213 million in series A and B financing to advance development of genomic medicines based on programmable genomic integration, or PGI, which enables the insertion of any DNA sequence, of any size, into any programmed genomic location.

The funding comes from Andreessen Horowitz (a16z) Bio + Health, ARCH Venture Partners, GV, Longwood Fund, Polaris Partners, Bruker Corporation, FUJIFILM Corporation, Alexandria Venture Investments, and others.

“PGI represents the maturation of editing technologies, breaking current barriers in genomic medicines discovery,” said Rahul Kakkar, president and CEO of Tome Biosciences. “PGI is revolutionary in that we can finally reprogram the human genome with an elegance and efficiency previously unimaginable. For patients with rare monogenic diseases, PGI allows for potentially curative treatments with a single drug per disease regardless of genetic heterogeneity, and for patients with more common disorders, PGI allows for the creation of cell therapies at the speed of biologics discovery with a complexity that enables the potential for broad use across human medicine.”

The company said PGI, in-licensed from the Massachusetts Institute of Technology, combines the site-specificity of CRISPR/Cas9 with enzymes capable of inserting or writing sequences of DNA, including entire genes, without the need for double-strand DNA breaks. Tome’s most advanced PGI technology, called integrase-mediated PGI (I-PGI), utilizes proprietary integrases and is based on groundbreaking PASTE technology first discovered by Tome’s co-founders, Omar Abudayyeh and Jonathan Gootenberg, while at MIT as investigators.

Just as a word processor is capable of pasting text anywhere in a document, I-PGI can insert large DNA sequences anywhere in the genome with unprecedented precision. Thus far, I-PGI has demonstrated insertions of more than 30kb of genetic code with site-specificity in multiple different dividing and non-dividing cell types and can be multiplexed to enable complex cell engineering that will underpin the future development of cell therapies. Tome has an exclusive license to the core PASTE technology.

“The ability to control where we insert DNA sequences is a game-changer. It means the field can now move away from random integration and utilize any natural promoter in the human genome, enabling us to orchestrate the tissue location, timing, and amount of gene expression,” said John Finn, chief scientific officer.

The company initially plans to develop integrative gene therapies for monogenic liver diseases and cell therapies for autoimmune diseases.

Photo: Rahul Kakkar, president and CEO of Tome Biosciences

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