CRISPR Platform Brings C-to-U Base Editing to RNA

Genetic Engineering and Biotechnology News | July 30, 2019

A new tool in the CRISPR toolbox doubles the number of single-base RNA edits on researchers wish lists. The tool aptly named RNA Editing for Specific C-to-U Exchange (RESCUE) can be programmed to convert targeted cytosine bases into uridine bases, complementing existing RNA base editors, which convert targeted adenine bases into inosine bases. RESCUE, its creators declare, is a particularly welcome development because many of the transcript modifications within RESCUE’s purview correspond to protein modifications relevant to cell signaling and cancer-linked pathways. For example, RESCUE enables modulation of more posttranslational modifications, such as phosphorylation, glycosylation, and methylation, as well as expanded targeting of common catalytic residues, disease mutations, and protective alleles. RESCUE was developed by a team of scientists led by Feng Zhang, PhD, an investigator at the McGovern Institute and a core member of the Broad Institute of MIT and Harvard. Zhang and colleagues made use of a deactivated Cas13 to guide RESCUE to targeted cytosine bases on RNA transcripts. Also, the scientists used a novel, evolved, programmable enzyme to convert unwanted cytosine into uridine—thereby directing a change in the RNA instructions.

Spotlight

The new QPix 2.0 Software release enables measurement of clearing zones within a lawn of bacteria due to an inhibitor producing colony. The "Zone of Inhibition Detection" module provides a gallery view of the colonies and outputs several parameters such as colony size, halo diameter, compactness, etc.

Spotlight

The new QPix 2.0 Software release enables measurement of clearing zones within a lawn of bacteria due to an inhibitor producing colony. The "Zone of Inhibition Detection" module provides a gallery view of the colonies and outputs several parameters such as colony size, halo diameter, compactness, etc.

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CELL AND GENE THERAPY

Curi Bio Closes $10M Series A in Oversubscribed Round

Curi Bio Inc. | December 30, 2021

Curi Bio Inc., a leading developer of human stem cell-based platforms for drug discovery, announced the second closing of a $10 million oversubscribed Series A financing. New investors include UTC Investment and DS Asset Management, joining current Curi Bio investor and Series A lead Dynamk Capital. The investment will be used to scale Curi’s existing business and accelerate the development of its innovative engineered tissue analysis platforms, including its Mantarray™ platform. “Curi Bio is thrilled to partner with the distinguished teams at UTC Investment, DS Asset Management, and Dynamk Capital to fuel our next stage of growth. To discover new therapies requires human-relevant disease models. Curi is working to close the gap between preclinical results and clinical outcomes, not only in small molecule discovery, but also in frontier areas like next-generation genetic medicines and cell therapies.” Michael Cho, JD, CEO of Curi Bio With costs to develop a single new medicine now exceeding $2Bn, the need for more human-relevant disease models to improve translational efficiency in the drug development process has never been greater. Curi’s core platform – the Curi Engine™ – integrates human stem cells, tissue specific biosystems, and A.I./M.L.-enabled data analysis to accelerate the discovery and development of new therapeutics. With this three-pronged strategy — human cells, systems and data — Curi is rapidly becoming a market leader in creating high-fidelity models of human diseases for drug discovery, especially for striated muscle, including cardiac and skeletal muscle, and neuromuscular models. “Curi Bio’s technology platforms create significant value for pharma and biotech companies by accelerating discovery timelines and increasing the chances of success for new therapies in development,” said Dr. Gustavo Mahler, Managing Partner, Dynamk Capital. “We look forward to strong growth in Curi Bio’s customer portfolio.” Curi Bio’s core technologies and products include NanoSurface™ Plates for structural maturation, Cytostretcher™ cell-stretching instruments, and the Mantarray platform for contractility analysis. The Mantarray platform enables researchers to generate and analyze 3D engineered human muscle tissues, providing clinically relevant functional readouts, and reducing reliance on poorly predictive animal models. Curi also offers a suite of customized research services utilizing the Curi Engine, including new assay and model development and phenotypic screening. Curi Bio counts all of the top-ten global pharmaceutical companies among its clients, customers, and partners. About Curi Bio Curi Bio’s preclinical discovery platform combines human stem cells, systems, and data to accelerate the discovery of new medicines. The Curi Engine is a seamless, bioengineered platform that integrates human iPSC-derived cell models, tissue-specific biosystems, and A.I./M.L.-enabled phenotypic screening data. Curi’s suite of human stem cell-based products and services enable scientists to build more mature and predictive human iPSC-derived tissues—with a focus on cardiac, musculoskeletal, and neuromuscular models—for the discovery, safety testing, and efficacy testing of new drugs in development. The company’s proprietary technologies are supported by over 100 publications and 19 patents. By offering drug developers an integrated preclinical platform comprising highly predictive human stem cell models to generate clinically-relevant data, Curi is closing the gap between preclinical data and human results, accelerating the discovery and development of safer, more effective medicines.

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CELL AND GENE THERAPY

Ginkgo Bioworks Launches Cell Development Kit Service for Standardized, Low-Cost Entry to its Cell Programming Platform

ginkgo biowork | November 11, 2021

ginkgo biowork the leading horizontal platform for cell programming, announced the launch of its Cell Development Kits (CDKs) service. CDKs provide streamlined access to the company's platform for aspiring cell developers to program cells to tackle the biggest challenges facing society. The first CDKs to launch are focused on protein expression programs. The service provides developers access to the toolkit needed to get started developing commercial proteins, including pre-engineered host cells optimized for protein production, specialized equipment, automation capabilities, genetic engineering expertise, insights garnered from Ginkgo's codebase and the applicable infrastructure to design, build and test a custom microbe. Ginkgo's CDKs are designed to cut the cost of launching a cell program and speed up development timelines to build engineered microbes, for example, to determine whether a protein may be successfully and commercially produced. The service is also designed to enable customers to prototype their idea in phases, with each phase providing data that can inform the customer's project and business strategy. By simplifying the pathway for companies to get started on the Ginkgo platform with standard terms, a phased approach, low costs—starting at $100,000 for protein expression projects—and clear deliverables, the CDK can help derisk projects prior to full scale technical development. "We believe engineering biology holds tremendous potential to revolutionize industries and our customers are using it to develop solutions ranging from alternative food proteins and vaccine ingredients to biodegradable plastics and sustainable chemicals But biology is still complex and highly variable, making the barrier to entry too high for many of the product developers that are out there with an idea they want to bring to the world. By providing these researchers, entrepreneurs and dreamers with the resources to realize their proof of concept, we're able to support the synthetic biology ecosystem to keep pushing the envelope on innovation." Jason Kelly, CEO of Ginkgo Bioworks If you are a developer interested in learning more about being in the next batch of CDK projects to launch, reach out to the Ginkgo team. Ginkgo will also be hosting an informational virtual event, Grow with Ginkgo: You're a Cell Developer, for prospective cell developers that are interested in learning more about the CDK on Thursday, Nov. 11 from 10 a.m.-12 p.m. ET. About Ginkgo Bioworks Ginkgo is building a platform to enable customers to program cells as easily as we can program computers. The company's platform is enabling biotechnology applications across diverse markets, from food and agriculture to industrial chemicals to pharmaceuticals. Ginkgo has also actively supported a number of COVID-19 response efforts, including K-12 pooled testing, vaccine manufacturing optimization and therapeutics discovery. Forward-Looking Statements of Ginkgo Bioworks This press release contains certain forward-looking statements within the meaning of the federal securities laws, including statements regarding the launch and potential benefits of Ginkgo's "Cell Development Kits" (CDKs) service and the potential of Ginkgo's cell programming platform generally. These forward-looking statements generally are identified by the words "believe," "project," "potential," "expect," "anticipate," "estimate," "intend," "strategy," "future," "opportunity," "plan," "may," "should," "will," "would," "will be," "will continue," "will likely result," and similar expressions. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this document, including but not limited to: (i) the effect of the business combination with Soaring Eagle Acquisition Corp. ("Soaring Eagle") on Ginkgo's business relationships, performance, and business generally, (ii) risks that the business combination disrupts current plans of Ginkgo and potential difficulties in Ginkgo's employee retention, (iii) the outcome of any legal proceedings that may be instituted against Ginkgo related to its business combination with Soaring Eagle, (iv) volatility in the price of Ginkgo's securities now that it is a public company due to a variety of factors, including changes in the competitive and highly regulated industries in which Ginkgo plans to operate, variations in performance across competitors, changes in laws and regulations affecting Ginkgo's business and changes in the combined capital structure, (v) the ability to implement business plans, forecasts, and other expectations after the completion of the business combination, and identify and realize additional opportunities, and (vi) the risk of downturns in demand for products using synthetic biology. The foregoing list of factors is not exhaustive. You should carefully consider the foregoing factors and the other risks and uncertainties described in the "Risk Factors" section of Ginkgo's current report on Form 8-K filed with the U.S. Securities and Exchange Commission (the "SEC") on September 20, 2021 and other documents filed by Ginkgo from time to time with the SEC. These filings identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements, and Ginkgo assumes no obligation and does not intend to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise. Ginkgo does not give any assurance that it will achieve its expectations.

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CELL AND GENE THERAPY

Specific Biologics Inc. Announces Therapeutic Development Award from the Cystic Fibrosis Foundation

Specific Biologics Inc. | December 08, 2021

Specific Biologics Inc. an early-stage biotechnology company developing novel gene editing technologies, announced that it will receive more than US$527,000 to support the preclinical development of its Dualase™ gene editing platform to target a set of cystic fibrosis -causing nonsense mutations where patients currently have fewer therapeutic options available. "Incredible advancements in the treatment of cystic fibrosis have been made since the identification of genetic mutations in the CFTR gene as the cause of CF Recent developments in gene editing technologies are creating the potential to correct these causative mutations at their source. We are truly grateful for the support of this Cystic Fibrosis Foundation award which will allow us to apply our Dualase™ gene editing technology to several frequent CFTR nonsense mutations." Brent Stead, CEO of Specific Biologics Inc Dualase™ cuts DNA differently than existing gene editors. This feature enables the high frequency insertion of new sequences into precise locations in the human genome. One can think of gene editing like a word processor that can find a specific spelling mistake in billions of letters and replace it with a correction. Similarly, inside a cell, Dualase™ can find a specific spelling mistake in the genome and replace it with the corrected sequence. Specific will deliver its gene editing technology using an inhalable lipid nanoparticle carrier, which will aid in delivering the gene editing machinery inside the cells of the lung. It is believed that if the CFTR nonsense mutations are corrected in enough cells, a gene editing therapeutic could provide a long-lasting benefit for CF patients. The award will help fund preclinical testing in disease relevant models. About Specific Biologics Inc. Specific Biologics Inc. is a venture-backed early-stage biotechnology company on a mission to develop novel gene editing technologies to treat diseases through precision gene editing. Our two-site Dualase™ platform gene editors cut DNA in a way that optimally exploits the cell's naturally occurring DNA repair pathways. This enables two gene editing outcomes, precise DNA deletions to disrupt genes or increased repair to correct genes. Specific also develops lipid nanoparticles to deliver the gene editor to target cells and is developing a pipeline of Dualase™-based therapeutics in areas of high unmet medical need.

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