γ-globin

Achieved in vivo preclinical proof of concept of hematopoietic stem and progenitor cell editing by utilizing Editas Medicine’s proprietary targeted LNP as a key step forward toward developing a novel in vivo treatment for sickle cell disease and beta thalassemia Initiated process to partner or out-license reni-cel to focus resources on in vivo pipeline development Company to present data and discuss strategic update in a Company-sponsored Webinar today at 8:00 a.m. ET CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today announced its achievement of in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin (HbF) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells. The Company observed high levels of editing of the HBG1/2 promoter, leveraging our clinically validated upregulation strategy, utilizing a novel and Editas-proprietary targeted lipid nanoparticle (tLNP) formulation for extrahepatic tissue delivery. The Company is also providing business development and financial updates, including that it initiated a process to partner or out-license reni-cel. “Achieving in vivo preclinical proof of concept in a desired, extrahepatic target cell type, delivered utilizing a proprietary Editas LNP that works outside the liver, puts us on a clear path to develop a potentially first- and best-in-class in vivo gene edited medicine for the treatment of sickle cell disease and beta thalassemia,” said Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. “I am delighted with our concrete progress on in vivo functional upregulation as we drive towards our long-term vision to be a leader in in vivo programmable gene editing, specifically achieving in vivo preclinical proof of concept ahead of schedule.” Dr. O’Neill added, “Simultaneously, we are laser focused on the optimal use of capital as we continue development of reni-cel, invest in our in vivo pipeline and vision, and map to the future. As part of that focus, we frequently evaluate opportunities to most efficiently advance reni-cel, a potentially best-in-class cell therapy for the treatment of sickle cell disease and beta thalassemia, towards commercialization and ultimately deliver it to patients in need. We believe that the best option for both patients and our shareholders is for us to seek an alternative such as a global partner or out-licensing, which would allow for further development and ultimately commercialization of reni-cel with or by another party and would allow us to substantially reduce spend in 2025.” In Vivo Proof of Concept Data Highlights Achieved highly competitive level of in vivo hematopoietic stem and progenitor cell (HSPC) editing utilizing a novel, Editas-proprietary targeted lipid nanoparticle (t-LNP) for extrahepatic tissue delivery in a humanized mouse model, mice engrafted with human hematopoietic stem cells.Enabled an editing level of 29% in HSPCs after a single dose by utilizing a hematopoietic stem cell targeting strategy and tLNP formulation optimization.Further demonstrated that editing with the Company’s proprietary tLNP formulation resulted in the functional outcome of HbF induction, indicated by the presence of HbF expressing human red blood cells (on average 20%) that populate in the host by one month. This level of in vivo editing in a humanized mouse model after a single dose constitutes a highly competitive dataset relative to data in the public domain for the development of an in vivo medicine for sickle cell disease and beta thalassemia. Ex Vivo Hemoglobinopathies Reni-cel (renizgamglogene autogedtemcel, previously EDIT-301) for Severe Sickle Cell Disease (SCD) On-track to present a substantive clinical data set of sickle cell patients with considerable clinical follow-up in the RUBY trial at the American Society of Hematology (ASH) Annual Meeting and Exposition, December 7-10, 2024.As previously disclosed, the Company has completed enrollment of the adolescent and adult cohorts of the Phase 1/2/3 RUBY trial for SCD.Manufacturing drug product for the initial adolescent cohort patients.The Company continues to dose adult patients in the RUBY trial and has dosed 28 patients to date. Reni-cel for Transfusion-dependent Beta Thalassemia (TDT) On-track to present additional clinical data from the EdiTHAL trial by year-end 2024.The Company completed enrollment of the adult cohort of the EdiTHAL trial for TDT and continues to dose patients. Other Corporate Highlights On October 3, 2024, Editas Medicine announced the sale of certain future license fees and other payments owed to the Company under its Cas9 license agreement with Vertex Pharmaceuticals to a wholly owned subsidiary of DRI Healthcare Trust (DRI) for an upfront cash payment of $57 million. The upfront cash payment brings non-dilutive capital to Editas Medicine, helping enable further pipeline development and related strategic priorities.The Company ended the third quarter 2024 with approximately $265 million of cash, cash equivalents, and marketable securities, or approximately $320 million following receipt of the upfront cash payment from DRI.The Company has engaged Moelis & Company LLC, a leading global independent investment bank, to lead the global process to partner or out-license reni-cel. In light of this webinar, the Company does not plan to host a conference call when it announces third quarter 2024 financial results next month. Webinar Presentation Details: The live and archived webcast of the Company’s webinar presentation will be accessible through this webcast link, or through the Events & Presentations page of the “Investors” section of the Company’s website. A replay of the webinar will be available upon conclusion of the webinar in the Investors section of the Editas Medicine website at https://www.editasmedicine.com/. About renizgamglogene autogedtemcel (reni-cel)Reni-cel, formerly known as EDIT-301, is an experimental gene editing medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Reni-cel consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by AsCas12a, a novel, proprietary, highly efficient, and specific gene editing nuclease. Red blood cells derived from reni-cel CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT. About Editas MedicineAs a clinical-stage gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com. Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Company’s intent to partner or out-license reni-cel and any benefits resulting therefrom, the initiation, timing, progress and results of the Company’s preclinical and clinical studies and its research and development programs, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, including presenting additional clinical data from the RUBY trial at the ASH Annual Meeting and Exposition and from the EdiTHAL trial by year-end 2024, the potential of, and expectations for, the Company’s product candidates, including any in vivo gene edited medicines the Company may develop, and the Company’s expectations regarding cash runway. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials, including the RUBY and EdiTHAL trials, and clinical development of the Company’s product candidates, including reni-cel; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from preclinical studies and clinical trials will be predictive of the final results of the study or trial or the results of any future clinical trials; expectations for regulatory approvals to conduct trials or to market products; and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise. This press release contains hyperlinks to information that is not deemed to be incorporated by reference in this press release.

Includes Twist RNA Exome, target enrichment for RNA and whole transcriptome sequencing SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Twist Bioscience Corporation (NASDAQ: TWST), a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, today announced the launch of a portfolio of RNA sequencing tools, which includes the Twist RNA Exome, Twist RNA Library Prep Kit and the Twist Ribosomal RNA (rRNA) & Hemoglobin (Globin) Depletion Kit, as well as custom target enrichment capabilities for RNA and whole transcriptome sequencing. This comprehensive suite of RNA tools can enable targeted or whole transcriptome research across fields including precision medicine, biomarker discovery and immuno-oncology research. “Twist’s RNA sequencing portfolio builds on our established NGS capabilities in genomic sequencing and methylation detection and expands into gene expression analysis, opening a new and significant market for Twist,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. “This offering allows our customers to leverage Twist’s leading target enrichment platform to discover and resolve low abundance RNA transcripts in the human transcriptome. Our RNA sequencing workflows are designed specifically for challenging samples. By enriching relevant transcripts, we can enable researchers to get high-quality data from a range of sample types, including damaged RNA.” Unlike the genome, which is relatively static, the transcriptome changes over time in response to varying disease states and drug therapies. Gene expression levels also differ across tissues and cell types. To fully understand this dynamic quality of RNA, researchers often sequence RNA samples from the same source at multiple time points, incurring additional costs and resource burdens. Each component of the Twist RNA sequencing portfolio is purpose-built to maximize data quality, minimize workflow inefficiency, decrease wasted sequencing reads, and support comprehensive transcriptomic profiling across even low-quality and low-input samples common in oncology. The Twist RNA sequencing portfolio provides end-to-end workflows that can be used to study total RNA and measure the whole transcriptome, protein coding sequences of messenger RNA (mRNA) or custom targets. These products can also be integrated into customers’ existing workflows. Twist RNA sequencing tools enable minimal hands-on time and increase the scale of samples sequenced through utilizing automation. They are compatible with a wide range of sample inputs, including difficult or low-quality samples. RNA panel designs can also now incorporate Twist’s new exon-aware design algorithm, which reduces bias in detection and can provide an important tool for researchers studying rare gene transcripts that drive tumor biology. Twist Targeted RNA Sequencing Workflow Twist target enrichment for RNA leverages the performance, efficiency and sensitivity of Twist target enrichment for sequencing of RNA transcripts of interest across samples and species. It can be used to enrich relevant transcripts with the RNA Exome or in custom RNA panels. Twist RNA Exome The Twist RNA Exome with Twist’s exon-aware design algorithm enables targeted sequencing of the protein coding regions of the human transcriptome without bias toward specific combinations of exons. The Twist RNA Exome covers up to 99.9% of protein coding sequencing in Gencode and RefSeq databases. This coverage, along with the exon-aware design approach, could enable researchers to capture important transcripts including those in low input and degraded samples from formalin fixed paraffin embedded (FFPE) sections. Twist Whole Transcriptome Sequencing Workflow Twist Whole Transcriptome Sequencing can be used to discover novel transcripts, including low expressing genes and novel isoforms. It can be used with a variety of samples including whole blood, fresh and frozen tissue samples and low quality samples, such as FFPE samples. The Twist rRNA & Globin Depletion Kit can be used with this workflow to deplete rRNA and hemoglobin targets so that researchers can study the rest of the transcriptome. The full workflow can be completed in less than five hours with minimal hands-on time. For more information, please visit: About Twist Bioscience Corporation Twist Bioscience is a leading and rapidly growing synthetic biology and genomics company that has developed a disruptive DNA synthesis platform to industrialize the engineering of biology. The core of the platform is a proprietary technology that pioneers a new method of manufacturing synthetic DNA by “writing” DNA on a silicon chip. Twist is leveraging its unique technology to manufacture a broad range of synthetic DNA-based products, including synthetic genes, tools for next-generation sequencing (NGS) preparation, and antibody libraries for drug discovery and development. Twist is also pursuing longer-term opportunities in digital data storage in DNA and biologics drug discovery. Twist makes products for use across many industries including healthcare, industrial chemicals, agriculture and academic research. Follow us on Twitter | Facebook | LinkedIn | YouTube Twist Bioscience Legal Notice Regarding Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical facts contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the ability of Twist’s RNA sequencing portfolio to enable customers to discover and resolve low abundance RNA transcripts and get high quality data from a range of sample types; maximize data quality, minimize workflow efficiency, decrease wasted sequencing reads, and support comprehensive transcriptomic profiling; enable minimal hands-on time; and increase the scale of sequenced samples, and statements regarding the ability of Twist’s exon-aware algorithm to reduce bias in detection. Forward-looking statements involve known and unknown risks, uncertainties, and other important factors that may cause Twist Bioscience’s actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the ability to achieve the expected benefits of Twist Bioscience’s workforce reduction, transition of production to the Factory of the Future and reduced investments in DNA data storage; the ability to attract new customers and retain and grow sales from existing customers; the ability of Twist to achieve sufficient revenue to achieve or maintain positive cash flow from operations or profitability in any given period; risks and uncertainties of rapidly changing technologies and extensive competition in synthetic biology that could make the products Twist Bioscience is developing obsolete or non-competitive; uncertainties of the retention of significant customers; the ability of Twist Bioscience to successfully integrate acquired companies and to achieve expected benefits from acquisitions; supply chain and other disruptions caused by the COVID-19 pandemic or otherwise; risks of third party claims alleging infringement of patents and proprietary rights or seeking to invalidate Twist Bioscience’s patents or proprietary rights; and the risk that Twist Bioscience’s proprietary rights may be insufficient to protect its technologies. For a description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Twist Bioscience’s business in general, see Twist Bioscience’s risk factors set forth in Twist Bioscience’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 8, 2023 and subsequent filings with the SEC. Any forward-looking statements contained in this press release speak only as of the date hereof, and Twist Bioscience specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.