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Engineering Accuracy in CRISPR

Technologynetworks | April 16, 2019

Biomedical engineers at Duke University have developed a method for improving the accuracy of the CRISPR genome editing technology by an average of 50-fold. They believe it can be easily translated to any of the editing technology's continually expanding formats. The approach adds a short tail to the guide RNA which is used to identify a sequence of DNA for editing. This added tail folds back and binds onto itself, creating a "lock" that can only be undone by the targeted DNA sequence. The study appears online on April 15 in the journal Nature Biotechnology.  "CRISPR is generally incredibly accurate, but there are examples that have shown off-target activity, so there's been broad interest across the field in increasing specificity," said Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering at Duke. "But the solutions proposed thus far cannot be easily translated between different CRISPR systems." CRISPR/Cas9 is a defense system that bacteria use to target and cleave the DNA of invading viruses. While the first version of CRISPR technology engineered to work in human cells originated from a bacteria called Streptococcus pyogenes, many more bacteria species carry other versions. Scientists in the field have spent years looking for new CRISPR systems with desirable properties and are constantly adding to the CRISPR arsenal. For example, some systems are smaller and better able to fit inside of a viral vector to deliver to human cells for gene therapy. But no matter their individual abilities, all have produced unwanted genetic edits at times.

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Mission Bio Launches Tapestri v3 to Rapidly Accelerate Rare Cell Detection Applications for Translational Research and Precision Therapeutics

PRNewswire | June 01, 2023

Mission Bio, Inc., the pioneer in high-throughput single-cell DNA and multi-omics analysis, announced breakthrough improvements to the Tapestri® Platform and its core chemistry that enable highly confident detection of rare cells for a range of applications. With Tapestri® v3 chemistry, researchers can discover tiny numbers of single cells that, until now, easily escaped detection and influenced disease in invisible ways. At the same time, drug developers can use the new capabilities for a more complete understanding of their advanced therapies, potentially leading to safer, more effective medicines. The new Tapestri® v3 chemistry increases the number of cells captured per sample by up to four times compared to the prior chemistry, a notable enhancement. With enhanced cell capture, the Tapestri® Platform can more reliably detect rare cells, opening incredible new possibilities like improving the assessment of measurable residual disease (MRD), a key metric used increasingly in clinical settings to estimate the risk of relapse with certain cancers. In the case of MRD in hematological cancers, rare subclonal variants are commonly missed by bulk NGS due to its averaging effect, hindering the detection of relapse-driving clones that potentially offer new therapeutic targets. With Tapestri®'s expanded capabilities, new integrated multi-omics tools like the Tapestri® scMRD Assay for Acute Myeloid Leukemia (AML) will offer clinicians additional therapeutic insights, rather than providing a binary 'yes or no' answer to the presence of residual disease. A team of investigators from Memorial Sloan Kettering Cancer Center (MSK) using the assay has reported a high sensitivity of 0.01% limit of detection in data presented at Mission Bio's Tapestri® scMRD for AML Summit last year. Tapestri®'s new capabilities also hold promise for cell and gene therapy developers looking to improve quality assessment throughout the therapy development process. Powered by Tapestri® v3 chemistry, the increase in cell throughput means Tapestri® Genome Editing Solution can measure gene editing outcomes at single-cell resolution – even for very low-frequency events like translocations, which can have significant effects on the safety of the therapy. Tapestri® v3 is the latest example of Mission Bio's continued focus on customer-centric product and service development. The company has recently implemented additional quality control measurements including design and development, release, and documentation processes compliant with the ISO 9001 standard. "Our customers' success is at the forefront of our mind," said Adam Abate, PhD, Co-founder and Interim Chief Executive Officer of Mission Bio. "Researchers and drug developers are demanding ever-greater sensitivity and highly robust products to effectively progress their research or advanced therapeutic program, and we are committed to serving our customers and helping them achieve their goals." The new v3 reagents will be available for shipping starting in mid-June. About Mission Bio Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others.

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Design Therapeutics to Present Preclinical Data on its GeneTAC™ Small Molecule, DT-168, for the Treatment of Fuchs Endothelial Corneal Dystrophy

Globenewswire | April 25, 2023

Design Therapeutics, Inc. a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that preclinical data for the company’s novel GeneTAC™ small molecule, DT-168, an eye drop being developed for the treatment of Fuchs endothelial corneal dystrophy (FECD), will be presented during an oral session at the Association for Research in Vision and Ophthalmology 2023 Annual Meeting (ARVO 2023), which is being held in New Orleans from April 23-27, 2023. FECD is characterized by progressive corneal degeneration leading to vision loss and affects millions of people in the U.S. Approximately 75% of cases are caused by a mutation in the transcription factor 4 (TCF4) gene, consisting of a CTG trinucleotide repeat expansion that leads to the formation of pathogenic RNA foci in the nucleus and the mis-splicing of multiple transcripts. There are no disease-modifying therapies approved for FECD, and advanced cases generally require ocular surgery, including corneal transplant. DT-168 is designed to selectively target the expanded CTG repeats in the TCF4 gene to reduce RNA foci formation and mis-splicing. In preclinical studies, DT-168 reduced foci in patient-derived primary corneal endothelial cells (CECs) to levels seen in cells from healthy individuals with low nanomolar IC50 values. Treatment with DT-168 also significantly improved mis-splicing in patient-derived CECs across a panel of genes. Additionally, in animal studies DT-168 eye drops were well-tolerated after multiple doses and distributed throughout the cornea with micromolar levels of DT-168 observed in the cornea 24 hours after dosing. The preclinical data support the potential for DT-168 to address the most common genetic cause of FECD and support the continued development of DT-168 as a potential disease-modifying therapy. Design remains on-track to submit an Investigational New Drug application for DT-168 for the treatment of FECD in the second half of 2023. About Design Therapeutics Design Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC™ gene targeted chimera small molecules. The company’s GeneTAC™ molecules are designed to either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease. Design is currently evaluating its lead GeneTAC™ small molecule, DT-216, in an ongoing Phase 1 clinical trial in patients with Friedreich ataxia. The company is also advancing programs in Fuchs endothelial corneal dystrophy and myotonic dystrophy type-1.

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IPA's Subsidiary, BioStrand, Solves the Information Integration Dilemma (IID) for Systems Biology

Businesswire | May 31, 2023

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Mission Bio Launches Tapestri v3 to Rapidly Accelerate Rare Cell Detection Applications for Translational Research and Precision Therapeutics

PRNewswire | June 01, 2023

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Design Therapeutics to Present Preclinical Data on its GeneTAC™ Small Molecule, DT-168, for the Treatment of Fuchs Endothelial Corneal Dystrophy

Globenewswire | April 25, 2023

Design Therapeutics, Inc. a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that preclinical data for the company’s novel GeneTAC™ small molecule, DT-168, an eye drop being developed for the treatment of Fuchs endothelial corneal dystrophy (FECD), will be presented during an oral session at the Association for Research in Vision and Ophthalmology 2023 Annual Meeting (ARVO 2023), which is being held in New Orleans from April 23-27, 2023. FECD is characterized by progressive corneal degeneration leading to vision loss and affects millions of people in the U.S. Approximately 75% of cases are caused by a mutation in the transcription factor 4 (TCF4) gene, consisting of a CTG trinucleotide repeat expansion that leads to the formation of pathogenic RNA foci in the nucleus and the mis-splicing of multiple transcripts. There are no disease-modifying therapies approved for FECD, and advanced cases generally require ocular surgery, including corneal transplant. DT-168 is designed to selectively target the expanded CTG repeats in the TCF4 gene to reduce RNA foci formation and mis-splicing. In preclinical studies, DT-168 reduced foci in patient-derived primary corneal endothelial cells (CECs) to levels seen in cells from healthy individuals with low nanomolar IC50 values. Treatment with DT-168 also significantly improved mis-splicing in patient-derived CECs across a panel of genes. Additionally, in animal studies DT-168 eye drops were well-tolerated after multiple doses and distributed throughout the cornea with micromolar levels of DT-168 observed in the cornea 24 hours after dosing. The preclinical data support the potential for DT-168 to address the most common genetic cause of FECD and support the continued development of DT-168 as a potential disease-modifying therapy. Design remains on-track to submit an Investigational New Drug application for DT-168 for the treatment of FECD in the second half of 2023. About Design Therapeutics Design Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC™ gene targeted chimera small molecules. The company’s GeneTAC™ molecules are designed to either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease. Design is currently evaluating its lead GeneTAC™ small molecule, DT-216, in an ongoing Phase 1 clinical trial in patients with Friedreich ataxia. The company is also advancing programs in Fuchs endothelial corneal dystrophy and myotonic dystrophy type-1.

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IPA's Subsidiary, BioStrand, Solves the Information Integration Dilemma (IID) for Systems Biology

Businesswire | May 31, 2023

ImmunoPrecise Antibodies Ltd. an AI-driven biotherapeutic research and technology company, announced that its subsidiary, BioStrand®, has successfully solved the Information Integration Dilemma (IID) by developing a unique technology design that enables their patented HYFT Technology to encapsulate and unify diverse data modalities - including syntactical (sequence) data, 3D structural data, unstructured scientific information (e.g. scientific literature), and beyond - into a singular, integrated framework. This breakthrough approach facilitates efficient data fusion, enabling a comprehensive analysis and interpretation of complex biological data. With HYFTs, BioStrand effectively addresses the IID, paving the way for quicker and more potent biological discoveries. This innovation has the potential to bring a paradigm shift in various domains including drug development, biomarker discovery, and the study of disease pathology, among others. The IID has long been a significant challenge for the biotechnology industry, as researchers and investors have grappled with the complexities of integrating various data types to gain meaningful insights. In the rapidly evolving field of biotechnology, data is generated from multiple sources such as scientific literature, experimental data, and genomic information. The integration of these different data types is critical for accelerating innovation and driving the discovery of novel therapeutics. However, traditional approaches have struggled to efficiently combine and analy ze this diverse data, often leading to incomplete or inaccurate conclusions. BioStrand's breakthrough approach leverages its patented HYFT® technology and LENSaiTM platform, which together enable the efficient analysis and understanding of complex biological data. By solving the IID, BioStrand has unlocked new possibilities for the discovery and development of novel therapeutics, which is expected to benefit both patients and the investor community. Dirk Van Hyfte, Co-Founder and Head of Innovation of BioStrand, said, "We are thrilled to have overcome the Information Integration Dilemma, which has long been a major hurdle in our industry. Our HYFT technology and LENSai platform are now poised to revolutionize the way researchers and AI-driven systems process and analyze complex biological data, ultimately leading to faster and more effective drug discovery and development." BioStrand's HYFT technology extracts unique patterns, known as Universal Fingerprint™ patterns, from the entire biosphere, and integrates them with various data sources, such as scientific papers and medical records. The resulting Knowledge Graph encompasses over 660 million HYFTs and more than 25 billion relations, providing a powerful resource for researchers and AI-driven analysis. The LENSai platform, powered by HYFT technology, takes advantage of the latest advancements in large language models (LLMs) to bridge the gap between syntax (sequences) and semantics (functions). This enables the platform to extract valuable insights from vast amounts of data, without the limitations of traditional LLMs. IPA's support of BioStrand's pioneering work reinforces its commitment to investing in cutting-edge biotechnology solutions with the potential to transform the industry. The successful resolution of the Information Integration Dilemma not only demonstrates the innovative nature of BioStrand's technology but also highlights the Company's dedication to improving the lives of patients worldwide. About ImmunoPrecise Antibodies Ltd ImmunoPrecise Antibodies Ltd. has several subsidiaries in North America and Europe including entities such as Talem Therapeutics LLC, BioStrand BV, ImmunoPrecise Antibodies (Canada) Ltd., and ImmunoPrecise Antibodies (Europe) B.V. The IPA Family is a biotherapeutic research and technology group that leverages systems biology, multi-omics modeling, and complex artificial intelligence systems to support its proprietary technologies in bioplatform-based antibody discovery. Services include highly specialized, full-continuum therapeutic biologics discovery, development, and out-licensing to support its business partners in their quest to discover and develop novel biologics against the most challenging targets.

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