This gene-editing startup raised $ 315 million for a next-generation Crispr tool to cure rare diseases


The Crispr gene-editing technology may have been worthy of a Nobel Prize, but Andrew Anzalone was convinced he could make it even better. Often likened to a pair of molecular scissors, it allows scientists to cut DNA and rewrite the code of life, for example by turning off pathogenic genes. But the technology has some drawbacks – it breaks DNA’s double-stranded helix, which can lead to unwanted changes in other sections of the code. As a postdoctoral researcher in David Liu’s Broad Institute lab, Anzalone set out to build the equivalent of a Swiss Army Knife Crispr, a multifunctional gene-editing technology that can correct entire sequences of code without breaking the duplicate. propeller. “The idea was really to try to broaden the scope of what we can do with gene editing,” Anzalone explains of the invention called “master editing”.

The human genome is made up of 6 billion combinations of the four letters called bases: A, T, G and C. Bases are segments called genes, which contain the instructions for certain hereditary information. Crispr-Cas9 allows researchers to focus on a specific location in a gene and cut and add or remove genetic material. It is particularly good for inactivating or “killing” genes. But one of the drawbacks is the unwanted insertion and deletion of random pieces of code due to the breakdown of the DNA double helix, which can cause unintentional mutations in other parts of the genome. Other diseases, such as sickle cell anemia, are caused by an error in a single base pair. This is where basic editing, which can swap individual databases, comes in, although currently basic editors can only do 4 different permutations.

This is where the main edition comes in. This technology can perform all of these functions – insertions, deletions and the 12 different base shifts – and could potentially correct up to 89% of known mutations that can cause genetic disease. It is developed by Cambridge, Massachusetts-based biotech startup Prime Medicine. The company on Tuesday announced combined Series A and B funding of $ 315 million for the technology, which it describes using terms associated with word processing software. “We like to use the search-and-replace analogy, because the beauty of the Crispr system is that you can tell it where exactly in the genome to go. Our [prime editing] The system also tells him exactly how to fix it, and that’s really what makes it unique, ”says Anzalone, scientific co-founder and head of the leading publishing platform. The funding values ​​the under-2-year-old company at $ 1.2 billion, according to PitchBook. Prime declined to comment on the rating.

While $ 315 million may seem like a hefty sum for a company that didn’t really start operations until July 2020 when it hired former Merck and Rhythm Pharmaceuticals executive Keith Gottesdiener, the majority of investors are regular backers of Prime scientific co-founder David Liu. Although Liu was not involved in Prime’s day-to-day operations, he created several companies based on research conducted in his laboratory at the Broad Institute. F-Prime, Arch Venture Partners, GV (formerly Google Ventures), Cormorant Asset Management and Redmile Group were all investors in Beam Therapeutics, the core publishing company Liu co-founded and which went public in early 2020. Casdin Capital , GV and T. Rowe Price Associates invested in Editas Medicine, a gene editing company of which Liu is a scientific co-founder that went public in 2016. Other Prime investors include Newpath Partners, Moore Strategic Ventures, Public Sector Pension Investment Board and Samsara BioCapital.

Investor enthusiasm around Prime is driven by the breadth of core editing technology and the promise that it could theoretically be more powerful and accurate than other Crispr-based tools, leading to processing and editing. more personalized remedies, especially for rare diseases with very few patients. . “It had the potential to be what I might call a universal editor, which means you could read and write and correct multiple mutations,” says Stephen Knight, president and managing partner of F-Prime. “And it seemed both intellectually appealing but also revolutionary.” An early investor in Beam Therapeutics, Knight once again jumped at the opportunity to fund the commercialization of research from Liu’s lab.

“It won’t be many years before we actually try this in patients and hopefully make an amazing difference. ”

Keith Gottesdiener, Primary Medicine

EInitially, Prime and Beam entered into a licensing agreement under which Beam has the right to commercialize core editing applications for sickle cell disease, so as not to cannibalize its existing pipeline. The agreement also includes commercialization of the technology for other undisclosed terms. The two companies have also agreed to share their research and expertise, including the mechanics of manufacturing and delivery, to achieve a leading assembly in human trials as soon as possible, Prime CEO Gottesdiener said. As someone with extensive experience in bringing therapeutic products to market, the speed of advancements in gene editing is unprecedented, he says. “It’s not that long term goal, where we think about how our grandchildren are going to get it done,” he says. “It won’t be many years before we try this in patients and hopefully make an amazing difference. “

As for Prime’s own pipeline, choosing over 75,000 genetic mutations to find and correct also makes prioritization more difficult. Gottesdiener declined to name specific diseases, but provided broad categories that Prime is looking for, including drug discovery programs targeting hepatic, ocular and neuromuscular indications, as well as hematopoietic stem cells outside the body. A look at the 2019 Nature article published by Anzalone, Liu and his team, offers some potential clues for specific main editing applications, including sickle cell anemia, rare Tay-Sachs nervous disease and resistance to neuromuscular diseases linked to prions. The real holy grail – and one of Prime’s future aspirations – would be to use a single main editor to fix multiple mutations. “One of our hopes is that we can literally move up a chromosome – go from one point on one chromosome to another – and that we can correct every mutation in that particular gene very efficiently,” says Gottesdiener.

Gottesdiener wouldn’t provide specific milestones or timelines, but Prime already raised more money than Beam before it was made public. Positive data from studies in mice and monkeys has pushed Beam’s market cap from around $ 1 billion in mid-2020 to around $ 6.2 billion today. When Cambridge, Massachusetts-based Intellia Therapeutics released data last month indicating that it had successfully secured its Crispr-based treatment for a rare disease in six human patients, the action soared to more than 80% in one week, from $ 88 to $ 171 per share.

Despite the potential of the technology, the main assembly has yet to be demonstrated to work in people. So far, the highest level primary model organism edition has been tested in mice, and there is still some way to go before it reaches human trials, but the future potential is palpable. “Our goal is really to cure, stop or prevent genetic diseases,” says Gottesdiener. “It’s not always clear that by changing genetics you can go back and fix things that were already broken, so we can’t always promise that every change we make is a cure for an individual patient. . ” But, at a minimum, the diseases will not progress, he says, “and maybe one day we will get to these diseases soon enough before any damage actually occurs.”


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