Tome Biosciences’ Post

We’re excited to participate in panel and poster sessions at the National PKU Alliance's 2024 Conference, July 18-21. We look forward to sharing our PGI approach to PKU and hearing the latest updates in research alongside the PKU community. #npkua2024conference #PKU

How can we make CRISPR-based cures accessible to all? That's an essential question that regulators, therapeutic developers, and academics must address to ensure the best patient outcomes. Check out this editorial piece from Nature Portfolio to dive into this critical question. https://lnkd.in/gRrtE8Pe #CRISPR #GeneTherapy

We’re honored to be highlighted as an Editor’s Pick in Nature Biotechnology! Tome was founded by Omar Abudayyeh and Jonathan Gootenberg with the idea that programmable genomic integration could revolutionize gene and cell therapies. As our President and CEO, Rahul Kakkar, said: “We don’t often come across techniques that can fundamentally transform two very important areas of drug discovery and development.” Check out the full article here: https://lnkd.in/gbMDwzp2 #cellandgenetherapy

Our lead integrative gene therapy indication is Phenylketonuria (PKU). But what is PKU? And how can gene editing help patients in need? PKU is a liver disease caused by a lack of the enzyme PAH, which is essential for healthy phenylalanine metabolism. Without PAH, phenylalanine levels become too high, which can cause neurocognitive dysfunction and psychiatric disorders. Our approach to treating PKU is to insert a healthy copy of the PAH gene in its natural genomic location, which has the potential to cure PKU. To learn more about the PKU, see this review article: https://bit.ly/3Kt5yQ4 #PKU #GeneTherapy

In addition to our in vivo work, we shared our progress in ex vivo cell therapy at ASGCT showing efficient multiplex editing and integration of large DNA sequences with functional gene expression in iPSC-derived NK cells. An exciting achievement indeed! But what does that mean? In this post, we will break down this accomplishment and explain why the details matter. 1) Multiplexing Multiplexing means placing multiple DNA sequences simultaneously. Unlike traditional CRISPR methods, I-PGI is not dependent on double-stranded DNA breaks, meaning we can make multiple edits to the DNA all at once and rapidly create advanced cell therapies. 2) Large DNA Sequences The ability to insert large DNA sequences in a site-specific manner means we can insert healthy copies of genes in the place of broken ones or, in the case of cell therapy, insert multiple genes that help increase the cell's safety and effectiveness. 3) Functional Gene Expression This is the gold-standard assessment of #genetherapy. We achieved functional gene expression, meaning we inserted the gene and assessed the resulting protein's function, showing that it acts as intended. #GeneEditing #CellandGeneTherapy

Recent controversy in the gene editing community has garnered the question, "Distinctions without a Difference: The Puzzling Argument About PASSIGE vs PASTE?"   In this exciting Op-Ed from Genetic Engineering & Biotechnology News, we explore why this is an apples-to-oranges comparison and find that there are more pressing questions in the genetic medicine community, such as:   How can we transform academic discoveries into a new era of genomic therapies? And most critically, how will we use gene insertion to create medicine for patients in need? Spoiler: We will do so with I-PGI.   Read more in the full article: https://lnkd.in/exq42sqq   #GeneEditing

Our Bio-Ball team had a great day on the court today, all while supporting a great cause. Thank you to those who supported us and Special Olympics Massachusetts!

On our path towards developing a CD19/BCMA CAR-iNK cell for renal autoimmune diseases, we have licensed iPSC lines from FUJIFILM Cellular Dynamics, Inc. Our iNK cell was designed with the autoimmune patient population in mind, and we look forward to bringing it to the patients in need.

In case you missed the announcement at ASGCT, Tome shared our early, but exciting proof-of-concept data in non-human primates! Click through to explore our achievement of therapeutically relevant programmable genomic integration and protein expression in non-human primates. #GeneEditing #NHPs