CELL AND GENE THERAPY

Tevard Biosciences and Zogenix Collaborates to Identify and Develop Novel tRNA-based Gene Therapies for Dravet Syndrome and other Genetic Epilepsies

Tevard Biosciences | December 04, 2020

Tevard Biosciences, a privately-held biotechnology organization pioneering tRNA-based gene therapies, and Zogenix, a worldwide biopharmaceutical organization creating and commercializing uncommon infection treatments, today reported that the organizations have gone into a cooperation to recognize and create novel tRNA-based quality treatments for Dravet syndrome and other genetic epilepsies.

Under the collaboration, Tevard will use its two interesting tRNA-based discovery platforms focused with respect to mRNA Stabilization and Nonsense Codon Suppression to find and advance novel drug candidates for the treatment of Dravet condition and other genetic epilepsies. Zogenix will additionally build up the up-and-comers through cutting edge preclinical examinations and clinical turn of events, and be responsible for overall commercialization.

Spotlight

In today’s increasingly connected world, health care researchers have access to previously unimaginable troves of data about the effects of healthcare interventions, including medicines. New technologies are allowing researchers to use so-called “big data” to investigate ways to better diagnose disease, predict which patients will benefit from which therapies, and observe the effects of medicines on a broader population than is possible during clinical trials.

Spotlight

In today’s increasingly connected world, health care researchers have access to previously unimaginable troves of data about the effects of healthcare interventions, including medicines. New technologies are allowing researchers to use so-called “big data” to investigate ways to better diagnose disease, predict which patients will benefit from which therapies, and observe the effects of medicines on a broader population than is possible during clinical trials.

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CELL AND GENE THERAPY

Biocytogen Completes a New Round of Financing Totaling Tens of Millions of Dollars

Biocytogen | June 23, 2021

Biocytogen, a worldwide biotechnology organization zeroed in on neutralizer drug innovative work (R&D) utilizing creative genetically designed creature models, today declared the fruitful culmination of another round of financing adding up to a huge number of dollars. The financing was together finished by Lake Bleu Capital, CPE, Octagon Capital and OrbiMed. Biocytogen Pharmaceuticals (Beijing) Co., Ltd. was set up in 2009. Utilizing its primary quality altering innovation, the organization autonomously created mouse stages for completely human neutralizer disclosure, RenMab® and RenLite®, that have free protected innovation rights. With the execution of these stages, Biocytogen has advanced from an agreement research association (CRO) into a global biotechnology organization with aptitude crossing the whole cycle of new drug R&D, including immune response drug target check, high-throughput single B cell counter acting agent revelation innovation, in vivo drug viability assessment utilizing adapted objective mice, and clinical turn of events. Genetic engineering of RenMab® and RenLite® mice has empowered the integration of Biocytogen's innovative qualities into a one of a kind enormous scope R&D plan for the revelation of helpful antibodies, a drive named the RenMice HiTS Platform. The HiTS Platform means the organization's obligation to finish high-productivity counter acting agent revelation and in vivo drug viability screening of in excess of 1,000 neutralizer targets in the following not many years, zeroing in on the disclosure of first-in-class and top tier drug targets. Through the ID of immune response drives that have been confirmed for adequacy in creature models, Biocytogen has set up co-improvement associations with in excess of twelve biotechnology and pharmaceutical organizations.

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MEDTECH

GP-write Partners with DNA Script to Accelerate DNA-Writing Technology and Accessibility

Genome Project-write | October 18, 2021

GP-write’s CAD is a one-stop shop for microbe, plant and animal genome writing and redesign. Its automated workflow allows users to rapidly upload a genome, redesign it and synthesize the new sequence. The tool enables researchers to directly order synthetic DNA or related products and services from GP-write’s affiliated members. DNA Script’s SYNTAX System, a benchtop DNA printer powered by their groundbreaking enzymatic DNA synthesis (EDS) technology, enables users to print sequences designed on GP-write’s CAD tool right in their lab. The first-of-its-kind DNA printer expedites workflows, making DNA writing as simple and efficient as next-generation sequencing. DNA Script will host a roundtable at the GP-write 5.0 conference on October 22 at 12:30 p.m. ET to engage attendees in a discussion centered on biosecurity as it relates to emerging technologies, including GP-write’s new CAD tool and DNA Script’s SYNTAX System. “We’re pleased to join GP-write and their industrial partners to drive innovation on the forefront of DNA printing technologies. Just as NGS, or DNA 'read,' and CRISPR, or DNA 'edit,' have brought significant advances to research and clinical care, we believe the broad accessibility of synthetic DNA printing, or DNA 'write,' offered by our SYNTAX System will be equally transformative and power the next bio-revolution.” Thomas Ybert, co-founder and CEO of DNA Script GP-write President and General Counsel, Amy Cayne Schwartz, notes that the organizations are partnering to work toward realizing “a shared vision of a future where writing genomes is facile, democratized and safely accessible.” Schwartz explains that “this will open up new frontiers for development of novel therapeutics and solutions for environmental health.” About Genome Project-write GP-write, conceived as a sequel to the Human Genome Project, applies lessons learned from HGP to pursue scientific exploration fully integrated with the development of genome engineering technologies. The primary goal of the project is to drive dramatic cost reductions and expedite whole-genome writing and redesign over the next decade, empowering researchers to uncover complex biological behavior and reprogram organisms to address defining global challenges in medicine, biotechnology and environmental health. About DNA Script Founded in 2014, DNA Script is a pioneering life sciences technology company developing a new, faster, more powerful and versatile way to design and manufacture nucleic acids. The company has developed an alternative to traditional DNA synthesis called Enzymatic DNA Synthesis, or EDS, allowing this technology to be accessible to labs with the first benchtop enzymatic synthesis instrument, the SYNTAX System. By putting DNA synthesis back in the lab, DNA Script aims to transform life sciences research through innovative technology that gives researchers unprecedented control and autonomy.

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CELL AND GENE THERAPY

Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle

Astellas Pharma Inc. and Dyno Therapeutics, Inc. | December 02, 2021

Astellas Pharma Inc. and Dyno Therapeutics, Inc. announced an option and license agreement was signed on November 23 to develop next-generation adeno-associated virus vectors for gene therapy directed to skeletal and cardiac muscle using Dyno's CapsidMap™ platform. Dyno's CapsidMap platform represents a transformative approach applying in vivo experimental data and machine learning to create novel AAV capsids – the cell-targeting protein shells of viral vectors – designed to optimize tissue targeting and immune-evading properties, in addition to improving packaging capacity and manufacturability. Unlike traditional approaches, CapsidMap is uniquely well-suited to simultaneously optimize capsids for delivery across multiple organs, with the goal of enabling more effective whole-body treatment for many diseases. With the establishment of the Astellas Gene Therapies Center of Excellence following the 2020 acquisition of Audentes Therapeutics Inc., Astellas is a leader in genetic medicines, working alongside its world-renowned partners to build a portfolio of potentially life-changing gene therapies. This research collaboration combines Dyno's AI-powered AAV vector engineering capabilities with Astellas Gene Therapies global leadership in AAV-based pipeline assets. "Through our efforts in gene therapy and the Astellas Gene Therapies Center of Excellence, Astellas strives to identify, develop and deliver transformative gene-based therapies for patients with genetic diseases who currently have few or no effective treatment options. Our principal focus is on developing adeno-associated virus delivered therapies for the treatment of well-defined serious diseases. We are dedicated to delivering novel approaches and utilizing new technologies that can deliver transformational value for patients." Naoki Okamura, Chief Strategy Officer and Chief Financial Officer, Chief Business Officer at Astellas "We are so happy to be partnering with Astellas, a world leader in developing gene therapies. Dyno and Astellas each bring unique strengths to this collaboration, together enabling more rapid creation of new therapies for patients with great unmet need," said Dyno's CEO and co-founder Eric D. Kelsic, Ph.D. "This partnership demonstrates the flexibility of Dyno's platform to precisely design the delivery properties of gene therapy vectors towards multi-organ and disease-specific profiles, applying the scientific insights we are rapidly learning across all our partnered and internal vector engineering programs using CapsidMap." Under the terms of the agreement, Dyno will design novel AAV capsids with improved functional properties for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities, including manufacturing, of gene therapy product candidates using the novel capsids. Dyno will receive an $18 million upfront payment and be eligible to receive additional payments during the research phase of the collaboration as well as clinical and sales milestone payments and royalties for any resulting products. The aggregate potential value of future milestone and royalty payments to Dyno exceeds $235 million per product and over $1.6 billion in total value. About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. About Astellas Gene Therapies Astellas integrated its wholly owned subsidiary, Audentes Therapeutics, Inc. as of April 1, 2021 and established "Astellas Gene Therapies" within the organization as an Astellas Center of Excellence to develop genetic medicines with the potential to deliver transformative value for patients. Based on an innovative scientific approach and industry leading internal manufacturing capability and expertise, we are currently exploring three gene therapy modalities: gene replacement, exon skipping gene therapy, and vectorized RNA knockdown and hope to also advance additional Astellas gene therapy programs toward clinical investigation. We are based in San Francisco, with manufacturing and laboratory facilities in South San Francisco and Sanford, North Carolina. About Dyno Therapeutics Dyno Therapeutics is a pioneer in applying artificial intelligence and quantitative in vivo experiments to gene therapy. The company's proprietary CapsidMap™ platform rapidly discovers and systematically optimizes Adeno-Associated Virus capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts.

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