“When we started, the premise of this class of machinery of being programmable and useful for genetic medicine, and applicable to the human genome, had scant support,” Maltzahn says. Maltzahn adds that the company’s initial focus will be on grievous illness, several rare genetic disorders and oncology, before developing the platform to treat an even greater number of diseases. “In fact, if you’re trying to cure a disease, or prevent it from ever happening, you can make an argument that DNA is the rightful home for many of those therapies.” “DNA being the code of life as we know it, the opportunity to be able to make modifications with very high precision to a subset of cells in your body is going to be applicable to diseases in every therapeutic area,” Maltzahn says. Maltzahn says those with Huntington’s disease, a rare and incurable degenerative condition, are just one patient group that could benefit from gene writing therapies. The ability to correct errors in genes could see Tessera’s technology lead to cures for all manner of genetic disorders – including those that are rare and currently untreatable with existing genetic technologies. “Many of these have been honing the ability to be exceedingly efficient, to be exceedingly sequence specific, and to have very high fidelity, or low error rate, of inserting their code into new locations.” New hope for genetic disease treatment “And fortunately, the crucible of what nature has been looking for in this unique life form called the mobile genetic element, maps tightly into the unmet needs and technology attributes that the future of genetic medicine needs. “You could argue that mobile genetic elements are the tallest shoulders you could ever stand on to build a technology,” Maltzahn says. Recombination is where MGE DNA is directly exchanged between an origin site and a target site in the genome. Transposition involves cutting the DNA of an MGE out of the genomic origin site and pasting it into a target site in the genome. In retrotransposition, MGE DNA is transcribed from a genomic origin site and then reverse transcribed into a target site in the genome. Tessera is exploring three different ways that MGEs can alter genomes for therapeutic benefit. “ are sequences of DNA that encode machinery that can grab on to either a DNA or an RNA copy of their code or themselves, bring that to a new location in a genome, and write or paste that sequence into that site,” Maltzahn explains. MGEs are a type of genetic material that are found in all organisms and constitute around half of the human genome, and their ability to rewrite and move DNA within genomes means they hold great potential as a new class of gene engineering tool. Maltzahn says all biotechnologies are built “on the shoulders of Mother Nature” – and in looking to nature for the answer to gene writing, the team found mobile genetic elements (MGEs). “We believe it’s the unambiguous future of the field,” says Tessera CEO Geoffrey von Maltzahn, “and the key to unlocking extraordinary cures and therapeutics that have the potential to touch every therapeutic area in medicine.” The science of gene writing The company’s new gene engineering technology would allow therapeutic messages to be written into genomes, addressing diseases at their source. Massachusetts-based Tessera Therapeutics, on the other hand, is pioneering gene writing, a technique with the potential to cure practically any genetic disease. However, despite its vast therapeutic potential, the technology does have its limitations while CRISPR is useful for deleting problematic genes, it is less effective at replacing them, meaning that only certain inherited conditions can be treated this way. Please check your email to download the Report.
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