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Asimov launches AAV Edge, a suite of AI models, host cells, and genetic tools for end-to-end gene therapy development
AsimovSep 18 2024 Asimov, the synthetic biology company advancing the design and production of therapeutics, today announced the launch of the AAV Edge System, a comprehensive suite of tools for adeno-associated viral (AAV) gene therapy design and manufacturing. The system provides gene therapy developers a single access point to an array of best-in-class tools to supercharge gene therapy development. While gene therapy holds significant promise for treating otherwise intractable diseases, the field is grappling with challenges in safety, efficacy, manufacturability, and cost. These issues are exacerbated by a fragmented ecosystem where key technologies are siloed across service providers, each offering disparate solutions. This fragmentation leads to suboptimal therapeutic development. Asimov's AAV Edge System addresses these challenges by providing an end-to-end platform that brings together several essential technologies, allowing developers to select the modules that best meet their design and production needs. The AAV Edge System offers a comprehensive suite of tools for both payload design and production: Payload design: The system includes artificial intelligence (AI)-designed, animal-validated tissue-specific promoters to enhance safety and efficacy; advanced DNA sequence optimization capabilities to boost expression levels in vivo; and tools to silence the gene of interest (GOI) during production to improve manufacturing performance by minimizing GOI toxicity. These proprietary genetic parts and design algorithms are accessible via Kernel, Asimov's computer-aided genetic design software. Production system: Today's launch introduces Asimov's transient transfection-based AAV manufacturing system -- the first in a planned series of releases for AAV Edge. This platform features a clonal, suspension-adapted, GMP-banked HEK293 host cell line; an optimized two-plasmid system compatible across capsid serotypes; and model-guided process development to improve bioreactor performance, achieving unconcentrated titers up to E12 viral genomes per milliliter (vg/mL). Our team has been on a roll - AAV Edge is our third launch in cell and gene therapy this year. The cost and safety of gene therapies is top of mind for many in the field, and we're driven to help our partners on both design and production to enable more of these powerful medicines to reach patients. This is Asimov's latest application in programming biology, made possible by leveraging AI, synthetic biology, and bioprocess engineering. There's more to come, and we're excited to keep pushing the envelope." Alec Nielsen, Co-founder and CEO, Asimov Asimov
News-Medical.net
Thu, 19 Sept, 4:00 AM UTC
Flagship Pioneering Unveils Mirai Bio to Provide Fully Integrated End-to-End Genetic Medicine Development Capabilities for the Biotech Industry By Investing.com
Mirai's machine intelligence-based, open platform unlocks delivery to any tissue and cell type through rapid, iterative learning of in vivo tested chemistries Co-optimization of cargo design and manufacturing ensure the best possible medicine and manufacturing strategy for each new program Company emerges with an initial commitment of from Flagship Pioneering , /PRNewswire/ -- Flagship Pioneering, the bioplatform innovation company, today unveiled , a company pioneering the first open end-to-end platform for the biotech industry to enable the co-creation of fully optimized genetic medicines. Mirai's machine intelligence-based, open platform uses proprietary data algorithms and machine intelligence to unlock delivery to any tissue and cell type, optimize cargo design, and facilitate manufacturing, accelerating genetic medicines toward successful clinical translation for the company's partners. Mirai plans to further advance its platform with Flagship's initial commitment of , aiming to enhance and accelerate genetic medicine development across a wide range of therapeutic areas and modalities. "We are in the age of information molecules, yet enormous technological challenges in the delivery, cargo design, and manufacturing of these molecules have hindered the speedy and full realization of their potential," said , Ph.D., Founding President of Mirai and Operating Partner at Flagship Pioneering. "We created Mirai to solve these key limitations through AI trained on high quantities of quality in vivo data. By applying machine intelligence to the design of every atom within the medicine and opening this platform to the entire industry, we will have vast collective data points rolling through our optimization loops, allowing a greater innovation advantage to benefit each partner on the Mirai platform." Mirai's cutting-edge platform is enabled by high throughput automation and in vivo multiplexing, generating vast amounts of data that fuel extensive optimization loops. The co-optimization of delivery, design, and manufacturing has the potential to enable a higher probability of success and a faster development timeline for any genetic medicine. , Executive Chair at Mirai and Growth Partner, Flagship Pioneering, added, "Every new company with a payload idea faces the same challenge of either internally building high-quality, cost intensive delivery, design, and manufacturing capabilities, or externally sourcing these components, which can be fragmented and suboptimal. Leveraging learnings from semiconductors as a centralized resource model that fueled the rapid advancement of tech, we've developed a solution that's been hiding in plain sight: an open platform to unlock genetic medicine development. Through aggressive investment in our own capabilities and expertise, and by creating long-term partnerships with biotech and pharma leaders in the space, Mirai's hope is to become the partner of choice and help bring more medicines into the world than we would ever achieve alone." , Ph.D., Founding CEO of Mirai and , Flagship Pioneering remarked, "Machine intelligence is transforming drug discovery and development as we know it, harnessing immense amounts of data to drive tomorrow's greatest innovations. The design space of genetic medicines is effectively infinite, and Mirai is pioneering the next big leap forward for us all." has built an open platform driven by machine intelligence that solves the key limitations hindering today's genetic medicines " delivery, design, and manufacturing. The company's platform combines the generative design of precise delivery solutions co-optimized with best-in-class manufacturing to develop the future of genetic medicine. Founded by Flagship Pioneering in 2021, Mirai is led by experts with decades of collective experience driving innovation in genetic medicines. About Flagship Pioneering Flagship Pioneering invents and builds bioplatform companies, each with the potential for multiple products that transform human health or sustainability. Since its launch in 2000, Flagship has originated and fostered more than 100 scientific ventures, resulting in more than in aggregate value. To date, Flagship has deployed over in capital toward the founding and growth of its pioneering companies alongside more than of follow-on investments from other institutions. The current Flagship ecosystem comprises 40 companies, including¯Foghorn Therapeutics¯(NASDAQ: FHTX),¯Moderna¯(NASDAQ: MRNA),¯Omega Therapeutics¯(NASDAQ: OMGA),¯Sana Biotechnology¯(NASDAQ: SANA),¯Generate Biomedicines,¯Inari,¯Indigo Agriculture, and¯Tessera Therapeutics.
Investing.com UK
Thu, 26 Sept, 12:18 PM UTC
Shape Therapeutics Unveils Major Advances in AI-Powered Platform for Precision RNA Editing
Company's DeepREAD AI model and small RNA expression system represent major leaps forward in RNA editing efficiency and specificity Generative AI model, trained on millions of experimental datapoints, for the first time enables efficient design of ADAR guide RNAs for any target Enhanced U7 SmOPT expression system dramatically increases RNA editing efficiency at gene therapy-relevant dose levels SEATTLE, Oct. 03, 2024 (GLOBE NEWSWIRE) -- Shape Therapeutics, a leader in RNA-based gene therapy, today released two preprint manuscripts detailing major technological breakthroughs that underpin the company's therapeutic editing platform RNAfix®. RNAfix® uses engineered guide RNAs (gRNAs) delivered by Adeno-Associated Virus (AAV) to recruit the natural ADAR enzyme and enable editing of target sites in a patient's RNA. These new advances overcome longstanding challenges to harnessing ADAR for therapeutic RNA editing, positioning ShapeTX at the forefront of programmable RNA medicines. The first manuscript, "Generative Machine Learning of ADAR Substrates for Precise and Efficient RNA Editing," introduces DeepREAD (Deep learning for RNA Editing by ADAR Design), one of Shape's custom AI models for gRNA design. Trained on data from high-throughput screens of millions of RNA sequences, DeepREAD generates gRNAs that form highly engineered structures when bound to target RNA. These novel structures redirect ADAR's natural sequence preferences, enabling specific and efficient editing of virtually any target adenosine. "DeepREAD represents a significant advancement in RNA editing gRNA design," said Ron Hause, Ph.D., SVP and Head of AI at Shape Therapeutics. "Our diffusion-based model generates highly efficient and specific guide RNAs for any adenosine in the transcriptome over 10,000 times faster than previous methods, rapidly producing thousands of high quality designs in minutes that outperform existing heuristic design approaches. This dramatically accelerates the development of RNA editing therapeutics for a wide range of genetic disorders." The second manuscript, "A Novel Engineered U7 Small Nuclear RNA Scaffold Greatly Increases in vitro and in vivo ADAR-Mediated Programmable RNA Base Editing," provides a detailed look at Shape's advanced system for expressing therapeutic gRNAs inside patient cells. By borrowing principles from natural small RNAs and performing deep profiling and additional engineering to enhance efficiency, Shape's novel scaffold drives high expression of gRNAs at the precise sites in a cell where ADAR activity occurs, leading to unprecedented levels of RNA editing in vitro as well as in the brain of animals given a systemic injection of AAV-delivered gRNA. "Our enhanced U7 SmOPT expression system represents a huge advancement for the therapeutic application of RNA editing, particularly in areas like the central nervous system where AAV delivery is often constrained to low doses per cell," said Adrian W. Briggs, Ph.D., Chief Technology Officer at Shape Therapeutics. The advances described in the two new manuscripts provide a blueprint for a breakthrough gene therapy pipeline. "By combining AI-powered gRNA design with our optimized expression system, we can now precisely and efficiently edit RNA targets that were previously out of reach and at an efficiency unmatched by other editing platforms. This opens up exciting possibilities for treating a wide range of genetic disorders in the CNS and beyond," said Dr. Briggs. About Shape Therapeutics ShapeTX® is pioneering the field of programmable RNA medicines to repair the genetic causes of diseases. By merging innovations in AI and RNA technology to generate and analyze hundreds of billions of therapeutic possibilities, ShapeTX is developing breakthroughs in RNA editing, next-generation AAVs, and disruptive gene therapy manufacturing. The ShapeTX platform enables pharma innovators to design treatments across a wide range of diseases, including rare genetic disorders as well as debilitating conditions, such as Alzheimer's, Parkinson's, and many more. You can find us at shapetx.com and on LinkedIn and Twitter. info@shapetx.com Market News and Data brought to you by Benzinga APIs
Benzinga
Thu, 3 Oct, 10:07 PM UTC
RenaissThera Closes Seed Funding to Drive AI-Powered Therapies for Cardio-Metabolic Diseases
BENGALURU, India, Dec. 10, 2024 (GLOBE NEWSWIRE) -- RenaissThera Private Limited ("RenaissThera") a pioneering Bengaluru-based biotechnology company, has successfully closed its seed funding round backed by angel investors. This funding will accelerate the development of novel small-molecule therapies targeting GLP-1 and GIP receptors for Cardio-metabolic (CM) diseases, with an emphasis on diabetes and obesity. The company aims to address critical health challenges in underserved populations globally through affordable and transformative solutions. RenaissThera is revolutionizing the fight against cardio-metabolic diseases like diabetes and obesity through its AI-powered Innovation Platform, leveraging Generative AI (GenAI) and machine learning to rapidly identify and optimize small molecules for targeted therapies. Mr. Sudhir Nagarajan, Managing Director, said, "We thank all the investors in this round for their support and belief in our team and the promise of RenaissThera in delivering innovative medicines." With the successful close of its seed funding round, CEO Dr. Ramkesh Meena stated, "This milestone enables us to scale up and validate our early-stage pipeline in pre-clinical proof-of-concept studies and get ready for IND filing next year, propelling us closer to delivering life-changing, affordable therapies that address critical gaps in care." This innovative approach combines scientific rigor and efficiency to reduce development timelines and costs while improving patient outcomes worldwide. About RenaissThera. RenaissThera, based in Bengaluru, India is a biotechnology company dedicated to developing affordable, novel small-molecule targeted therapies leveraging AI platforms. Our goal is to innovate for underserved populations and bridge the gaps in novel therapy access worldwide. We focus on high-unmet-need areas of prevalent diseases in key therapy areas such as Cardio-metabolic including diabetes and obesity indications. Leveraging our Innovation Platform powered by GenAI tools, we identify small-molecule therapies for proven pathways, ensuring cutting-edge solutions for patients. For more information, visit www.renaissthera.com. Contact: Lakshmi Ramakrishna Email id - lramakrishna@renaissthera.com Mob. no. - +91- 9176940015 Market News and Data brought to you by Benzinga APIs
Benzinga
Tue, 10 Dec, 2:11 PM UTC
GitHub - Genesis-Embodied-AI/Genesis: A generative world for general-purpose robotics & embodied AI learning.
Genesis is a physics platform designed for general-purpose Robotics/Embodied AI/Physical AI applications. It is simultaneously multiple things: Powered by a universal physics engine re-designed and re-built from the ground up, Genesis integrates various physics solvers and their coupling into a unified framework. This core physics engine is further enhanced by a generative agent framework that operates at an upper level, aiming towards fully automated data generation for robotics and beyond. Note: Currently, we are open-sourcing the underlying physics engine and the simulation platform. Our generative framework is a modular system that incorporates many different generative modules, each handling a certain range of data modalities, routed by a high level agent. Some of the modules integrated existing papers and some are still under submission. Access to our generative feature will be gradually rolled out in the near future. If you are interested, feel free to explore more in the paper list below. Genesis aims to: Project Page: https://genesis-embodied-ai.github.io/ For the latest version, clone the repository and install locally: If you want to use Genesis from Docker, you can first build the Docker image as: Then you can run the examples inside the docker image (mounted to ): Comprehensive documentation is available in English, Chinese, and Japanese. This includes detailed installation steps, tutorials, and API references. The Genesis project is an open and collaborative effort. We welcome all forms of contributions from the community, including: The Genesis source code is licensed under Apache 2.0. Genesis's development has been made possible thanks to these open-source projects: Genesis is a large scale effort that integrates state-of-the-art technologies of various existing and on-going research work into a single system. Here we include a non-exhaustive list of all the papers that contributed to the Genesis project in one way or another: ... and many more on-going work. If you use Genesis in your research, please consider citing:
GitHub
Thu, 2 Jan, 12:02 AM UTC
