![]() ![]() mRNA hairpins can be formed when two complementary sequences in a single mRNA molecule meet and bind together, after a folding or wrinkling of the molecule. In RNA, the secondary structure is the basic shape that the sequence of A, C, U, and G nucleotides form after they are linked in series, such a folding or curling of the nucleic acid strand. Hairpins are a common type of secondary structure in RNA molecules. The resulting structure looks like a loop or a U-shape. Photo courtesy of Scribe Therapeutics.A hairpin loop is an unpaired loop of messenger RNA (mRNA) that is created when an mRNA strand folds and forms base pairs with another section of the same strand. It allows us to truly bring all of these together to build new ways of modifying biology and applying it.”įeature photo of Scribe’s leadership team, from left: Benjamin Oakes, Brett Staahl, Svetlana Lucas, Jennifer Doudna and David Savage. I would like to build the right tools for the job. I don’t want to treat diseases with something meant for another disease. “We continue to build upon the model to make it better and better. “Engineering is a slow process, but each step gets you closer,” Oakes said. The new capital will be used to continue developing Scribe’s gene-editing system and its delivery technologies, such as how the therapies are delivered into the body to target where needed, as well as to advance a pipeline of therapeutics for neurodegeneration and other diseases. The therapy has the potential to halt and reverse the condition’s progression, the company said. In addition, this week, Intellia Therapeutics, a Cambridge, Massachusetts-based genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology, announced that the European Commission granted orphan drug designation for one of its candidates being developed to treat transthyretin amyloidosis. This rare condition impacts organs and tissues through the accumulation of misfolded transthyretin protein deposits. ![]() In 2020, Scribe began collaborating with Biogen as part of a $400 million research project to develop CRISPR-based therapies that address the underlying causes of Amyotrophic Lateral Sclerosis (ALS). The work can be both in vivo or ex vivo, meaning inside or outside an organism.ĬRISPR technology is also being used to develop treatments for unmet diseases. ![]() They include editing stem cells or T cells to create better stem cells, such as chimeric antigen receptor T cells, better known as CAR-Ts, for use in immunotherapy. Use cases for CRISPR depend on what the molecule is capable of, Oakes said. He added: “By accelerating the shift from an artisanal discovery approach to a fully industrialized one, the company is poised to fundamentally transform how we treat and manage genetic diseases at scale.” Use cases Meanwhile, Vijay Pande, general partner at Andreessen Horowitz, said in a statement that Scribe’s approach has “revolutionized industry expectations for blueprinting and creating CRISPR-based therapies.” “Scribe is applying deep, multidisciplinary engineering expertise to develop the custom tools and applications needed to confront genetic diseases,” said Aghazadeh in a written statement. Gordon, managing partner at OrbiMed Advisors, will join Scribe’s board of directors. To date, the company raised $120 million, which includes a $20 million Series A round in 2018 led by Andreessen Horowitz, according to Crunchbase data.Īs part of the financing, Behzad Aghazadeh, managing partner at Avoro Ventures and Avoro Capital Advisors, and Carl L. Rowe Price Associates, and funds managed by Wellington Management, RA Capital Management and Menlo Ventures. The syndicate also includes Perceptive Advisors, funds and accounts advised by T. “I was spending 90 hours a week testing, so you can see immediately why CRISPR would be impactful.” Treating diseaseĪvoro Ventures and an affiliated company, Avoro Capital Advisors, led the Series B financing and were joined by OrbiMed Advisors and Andreessen Horowitz. “I started in genome editing a decade ago, and the only way to do this was to build a library of molecules and test every one over and over again,” Oakes said. Scribe was founded “on paper” in 2017 by molecular engineers Benjamin Oakes, Brett Staahl and David Savage, as well as CRISPR co-inventor Jennifer Doudna, who won the 2020 Nobel Prize in chemistry, to create advanced technologies for CRISPR-based genetic medicine.ĬRISPR stands for “ clustered regularly interspaced short palindromic repeats” and enables researchers to more easily identify DNA sequences and modify gene function to develop in vivo therapies that treat the underlying cause of disease, Oakes told Crunchbase News. Molecular engineering company Scribe Therapeutics has completed an oversubscribed $100 million round of Series B financing to further develop its “CRISPR by design” platform, as well as advance its pipeline of genetic treatments.
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