CRISPR Technique Refined for Potential Application in Muscular Dystrophy

Techonology Networks | January 09, 2019

The gene editing technique known as CRISPR is a revolutionary approach to treating inherited diseases. However, the tool has yet to be used to effectively treat long-term, chronic conditions. A research team led by Dongsheng Duan, Ph.D., at the University of Missouri School of Medicine has identified and overcome a barrier in CRISPR gene editing that may lay the foundation for sustained treatments using the technique.
CRISPR gene editing is inspired by the body's natural defensive ability to fend off viruses. The technology enables researchers to alter DNA sequences by cutting out and replacing a mutation in the genome, which has the potential to treat a variety of genetic diseases and conditions. Duan, along with his collaborators at MU, the National Center for Advancing Translational Sciences at the National Institutes of Health and Duke University, are studying how to harness CRISPR to treat Duchenne muscular dystrophy (DMD).
Children with DMD have a gene mutation that interrupts the production of a protein known as dystrophin. Without dystrophin, muscle cells become weaker and eventually die. Many children lose the ability to walk, and muscles essential for breathing and heart function ultimately stop working.

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Live cell imaging allows scientists to view the inner workings of cells in unprecedented detail, but keeping cells alive throughout the imaging process poses unique challenges not present for other imaging techniques.This illustrated infographic presents tips for preparing cells for live cell imaging and gives an overview of the live cell imaging market today. Learn how to optimize each step of the sample prep process to make sure you get the clearest picture possible.

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QIAGEN and Neuron23 Partner to Develop Next-Generation Sequencing Companion Diagnostic for Novel Parkinson’s Disease Drug

QIAGEN and Neuron23™ | September 15, 2022

QIAGEN and Neuron23 Inc., an early stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases, announced the signing of an agreement to develop a companion diagnostic for Neuron23’s brain penetrant leucine-rich repeat kinase inhibitor for Parkinson’s disease. Under the new Master Collaboration Agreement, QIAGEN will develop and validate a clinical trial assay that will detect a combination of biomarkers discovered by Neuron23 that together predict the responsiveness of Parkinson’s disease to a LRRK2 inhibitor. The partnership will support the clinical development of Neuron23’s drug candidate that is currently in the late stages of preclinical development. Subject to further clinical development, the agreement also covers options for the future development of additional companion diagnostics. Neuron23 joins a group of more than 25 leading pharmaceutical and biotechnology companies who have reached master collaboration agreements with QIAGEN to develop and commercialize companion diagnostic tests for their drug candidates – a deep pipeline of potential future products to advance precision medicine for the benefit of patients around the world. The assay for this collaboration will be integrated into a next-generation sequencing workflow that leverages QIAGEN’s Sample to Insight capabilities, including instrumentation, IVD sample preparation, library preparation and bioinformatics. The workflow is planned to be developed using the NextSeqTM 500 System as part of the NGS strategic collaboration between QIAGEN and Illumina. Based on Neuron23’s artificial intelligence (AI)-enabled drug discovery and biomarker platform, it will target a complex signature of 50 single nucleic polymorphisms for U.S. and European populations and eventually additional SNPs that are prevalent in Asian populations. “The collaboration with Neuron23 shows the rapid momentum precision medicine is gaining in disease areas outside oncology. Our expertise in blood- and NGS-based molecular testing from Sample to Insight will enable Neuron23 to run a clinical trial for a drug candidate that may have the potential to modify the course of an inexorable neurodegenerative disease in a genetically defined population.” Jonathan Arnold, Vice President, Head of Oncology and Precision Diagnostics, at QIAGEN “This collaboration combines the leading expertise of Neuron23 in drug discovery, data science, and machine learning with QIAGEN’s long-standing experience and global leadership in companion diagnostic development. QIAGEN’s blood-based test will help to identify patients with Parkinson’s disease who are likely to respond to Neuron23’s LRRK2 inhibitor. The development of a companion diagnostic identifying this sub-population of Parkinson’s disease patients will de-risk the clinical development of Neuron23’s LRRK2 inhibitor and help identify individuals who may benefit from this disease-modifying therapy. We are excited to be working with an industry leader on the first companion diagnostic developed for Parkinson’s disease,” said Nancy Stagliano, Ph.D., CEO of Neuron23. No laboratory tests are currently available for the diagnosis of non-genetic cases for Parkinson’s disease. Usually, the disease is diagnosed based on medical history and neurological examination. Although no cure currently exists for Parkinson’s disease, therapies are used to alleviate some symptoms. LRRK2 is a complex, multidomain protein found in neurons and many tissues and cell types throughout the body. Mutations in the LRRK2 gene are one of the most common causes of familial Parkinson’s disease and individuals who inherit gain of function mutations in LRRK2 are clearly at higher risk to develop the disease in later life. Additionally, there is emerging evidence that LRRK2 may play a role in a subset of the larger population of patients with non-familial Parkinson’s disease. Recent investigations have shown that small-molecule LRRK2 inhibitors can be neuroprotective, suggesting that therapies targeting LRRK2 could be beneficial in a larger population of patients. QIAGEN is a pioneer in precision medicine and the global leader in collaborations with pharmaceutical and biotechnology companies to co-develop companion diagnostics, which detect clinically relevant genetic abnormalities to provide insights that guide clinical decision-making in diseases such as cancer. QIAGEN has an unmatched depth and breadth of technologies from NGS to polymerase chain reaction and digital PCR for companion diagnostic development. QIAGEN has ten PCR based companion diagnostic indications that are FDA approved, including therascreen EGFR for non-small cell lung cancer, therascreen KRAS for colorectal cancer and NSCLC, therascreen FGFR for urothelial cancer, therascreen PIK3CA for breast cancer based on tissue or plasma samples and the therascreen BRAF kit for colorectal cancer. About QIAGEN QIAGEN N.V., a Netherlands-based holding company, is the leading global provider of Sample to Insight solutions that enable customers to gain valuable molecular insights from samples containing the building blocks of life. Our sample technologies isolate and process DNA, RNA and proteins from blood, tissue and other materials. Assay technologies make these biomolecules visible and ready for analysis. Bioinformatics software and knowledge bases interpret data to report relevant, actionable insights. Automation solutions tie these together in seamless and cost-effective workflows. QIAGEN provides solutions to more than 500,000 customers around the world in Molecular Diagnostics, Applied Testing, Pharma and Academia. As of June 30, 2022, QIAGEN employed more than 6,100 people in over 35 locations worldwide. About Neuron23™ Neuron23™ Inc. is an early stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases. Neuron23 combines recent advances in human genetics with a state-of-the-art artificial intelligence (AI)-enabled drug discovery and biomarker platform to advance therapeutics for devastating diseases. The Company’s focus areas are neurodegenerative diseases, neuroinflammatory diseases, and systemic autoimmune and inflammatory diseases. Founded in 2018, Neuron23 has assembled a world-class team of experts and entrepreneurs located in South San Francisco, CA.

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Emergence Therapeutics Licenses Synaffix ADC Technology Platform in $360M Deal

Synaffix | September 07, 2022

Synaffix B.V. and Emergence Therapeutics AG announce that the Companies have entered into a licensing agreement of up to $360 million, providing Emergence access on a target-specific basis to Synaffix's proprietary antibody drug conjugate technologies comprising GlycoConnect™, HydraSpace™ and SYNtecan E™ linker-payload. Under the terms of the agreement, Synaffix will be eligible to receive up to $360 million in upfront and milestone payments plus royalties on commercial sales. The first program will commence at signature, with the option to expand the collaboration to additional targets. The agreement is the result of positive proof of concept data following a successful initial discovery research collaboration between the companies. Emergence will be responsible for the research, development, manufacturing and commercialization of the ADCs. Synaffix will be responsible for the manufacturing of components that are specifically related to its proprietary technologies. Jack Elands, Chief Executive Officer of Emergence Therapeutics, commented: "This transaction demonstrates our commitment to the development of our pipeline of first-in-class and/or best-in-class ADCs. Beyond our lead program ETx-22, a next generation Nectin-4 ADC, we are actively advancing further programs to develop treatments for high unmet-need cancers. We are impressed with the Synaffix technology and are pleased to select it as a key component to accelerate our pipeline of ADCs." "This marks an important milestone for Synaffix as this is the 10th ADC developer who has licensed our innovative ADC technology to develop proprietary ADCs for their own pipeline. By consolidating all essential technologies within Synaffix, we have repeatedly enabled the development of best-in-class and first-in-class ADCs under a single technology license agreement. We are thrilled that Emergence has selected Synaffix as its long-term ADC technology partner and look forward to working closely with Emergence as they build out their portfolio of ADC candidates." Peter van de Sande, Chief Executive Officer of Synaffix About Synaffix B.V. Synaffix B.V. is a biotechnology company that enables ADC product candidates using its clinical-stage, site-specific ADC technology platform. In addition to GlycoConnect™ and HydraSpace™ technology, the toxSYN™ linker-payload platform rounds out a fully complementary technology platform that enables any company with an antibody to develop proprietary best-in-class ADC products under a single license from Synaffix. The Synaffix platform enables a rapid timeline to clinic due to the established supply chain of technology components. Granted patents covering Synaffix' technology provide end-to-end protection of the manufacturing technology as well as the resulting products through at least 2035. The business model of Synaffix is target-specific technology out-licensing, as exemplified through its existing deals with ADC Therapeutics, Mersana Therapeutics, Shanghai Miracogen, Innovent Biologics, ProfoundBio, Kyowa Kirin, Genmab and Macrogenics. About Emergence Therapeutics AG Emergence Therapeutics is a European biopharmaceutical company developing novel antibody drug conjugates (ADC) to treat high-need cancers. Its lead program combines a highly specific antibody with optimized linker and payload technology to target Nectin-4 – an important target for a broad range of cancers which has been clinically validated as an ADC target by enfortumab vedotin, now approved for the treatment of urothelial cancers by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Emergence Therapeutics is also actively developing a pipeline of further first- or best-in-class ADCs driven by therapeutic need. Emergence is supported by a network of world-leading experts in immuno-oncology and a leading international investor syndicate. Emergence Therapeutics AG is based in Duisburg, Germany with a subsidiary in Marseille, France. For more information, please visit: www.emergencetx.com About The Synaffix ADC Platform Technology Synaffix' proprietary ADC technology platform consists of GlycoConnect™, HydraSpace™ and toxSYN™ technologies. Both GlycoConnect™ and HydraSpace™ are clinical-stage technologies that enable best-in-class ADCs with significantly enhanced efficacy and tolerability. GlycoConnect™ is a conjugation technology that exploits the native antibody glycan for site-specific and stable payload attachment and is tunable to DAR1, DAR2 or DAR4 formats. HydraSpace™ is a compact and highly polar spacer technology that is designed to further enhance therapeutic index, particularly with hydrophobic payloads. toxSYN™ is a linker-payload platform that spans key, validated MOAs for ADC product development, including SYNtecan E™ and SYNeamicin G™, among other linker-payloads.

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IPA’s Subsidiary BioStrand and BriaCell Announce Artificial Intelligence Collaboration and License Agreement

BriaCell Therapeutics Corp. | December 01, 2022

IPA an advanced biotherapeutic research and technology company, announced that BioStrand BV an AI in silico discovery subsidiary of IPA, has entered into a research collaboration and license agreement with BriaCell Therapeutics Corp. a clinical-stage biotechnology company specializing in targeted immunotherapies for cancer. The collaboration will leverage BioStrand’s LENSai™ software, built upon IPA’s proprietary HYFT™ Universal Fingerprint™ technologies, and will focus on in silico antibody discovery to generate relevant clinical molecules for potential development. Under the terms of the Agreement, BioStrand and BriaCell will collaborate on the design, discovery, and development of anti-cancer antibodies. Upon successful antibody discovery, BioStrand will receive an upfront payment of US$500,000, and will be eligible to receive future success-based development milestones, including those for the submission of Investigational New Drugs clinical milestone payments, and commercial royalties on net sales of products. Further terms are not disclosed. “We are very excited to begin this program with BriaCell and apply our technology to the development of biologics that may have a huge clinical impact. Shortening timelines, while also integrating as much information as possible upstream to improve the efficiency of the process, is extremely important in the development of highly targeted therapies. It is a step towards realizing our long-term vision of significantly advancing precision medicine.” Dr. Ingrid Brands, General Manager and co-founder of BioStrand “We believe that BioStrand’s revolutionary AI-powered technology, combined with its cutting-edge protein engineering platform, will allow us to design and discover potent anti-cancer therapeutics,” stated Miguel A. Lopez-Lago, PhD, Chief Scientific Officer of BriaCell. “This approach would complement BriaCell’s current immunotherapy pipeline of innovative anti-cancer therapeutics.” About BriaCell Therapeutics Corp. BriaCell is an immuno-oncology-focused biotechnology company developing targeted and effective approaches for the management of cancer. 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 Ltd. and ImmunoPrecise Antibodies B.V. The IPA Family is a biotherapeutic research and technology group that leverages systems biology, multi-omics modelling 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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