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Metagenomi Showcases AI-Driven Advancements in Gene Editing at Cold Spring Harbor Laboratory Conference
Metagenomi, a genetic medicines company, presents its AI-enabled Smart Editing platform advancements at the Cold Spring Harbor Laboratory CRISPR Frontiers Conference, highlighting progress in gene editing technology.
2 Sources
Fri, 30 Aug, 8:12 AM UTC
Pathos Expands Pipeline With Worldwide License of Phase 2-ready Program, a Brain-penetrant, PRMT5 Inhibitor
- (www.pathos.com), a biotechnology company focused on revolutionizing precision medicine in cancer by harnessing the power of machine learning to transform drug development, announced today the world-wide license of PRT811, a potent, selective, and orally bioavailable brain penetrant SAM-competitive PRMT5 inhibitor from . PRT811 (renamed P-500) was developed by and completed a Phase 1 trial in . The trial enrolled patients with solid tumors including high-grade glioma and uveal melanoma and has potential application in other indications with high unmet need. Out of 16 patients with high-grade glioma with isocitrate dehydrogenase mutations (IDH+) in the Phase 1 trial, two confirmed complete responses (CR) were observed. At last follow-up, 1 response is ongoing and has lasted 31.0 months while the duration of response for the second CR patient was 7.5 months. Additionally, 1 patient achieved an unconfirmed partial response (PR). In addition, out of 23 uveal melanoma patients (10 patients with splicing factor 3B subunit 1 (SF3B1) splicing mutations and 13 without an SF3B1 mutation), one confirmed PR (duration of response of 10 months) and a second unconfirmed PR were observed, both in patients SF3B1 mutations. In the entire safety population (N=86), the most common adverse events of any grade, with an incidence of >20% were nausea (60.5%), vomiting (46.5%) fatigue (36.0%), constipation (29.1%), and thrombocytopenia (24.4%), and were predominantly grade 1-2. The most common adverse events (grade ?3), occurring >5% were thrombocytopenia (9.3%), anemia (9.3%), and fatigue (5.8%). 'These results from Prelude's Phase 1 study are promising news for high-grade glioma patients and clinicians, who still have limited treatment options with chemotherapy and radiation that hasn't changed in decades. With our AI Platform, we aim to increase the already encouraging response rate of P-500 through a novel biomarker-driven strategy, ultimately bringing this medicine to patients as efficiently as possible,' said , Pathos CEO. 'Prelude's discovery engine has delivered a number of first- or best-in-class precision medicines including PRT811, a molecule that has shown early promise in the treatment of high-grade glioma. We are confident that Pathos AI, sharing our passion for precision medicine and commitment to serving cancer patients with high unmet need, is an ideal company to drive the development of this molecule forward for patients.' said , Prelude CBO. 'The resources from this transaction will support advancing Prelude's pipeline.' About P-500 P-500 (previously PRT811) is a selective, brain-penetrant small molecule inhibitor of protein arginine methyltransferase 5 (PRMT5) that has the potential to provide clinical benefit for patients with advanced solid tumors, including high-grade glioma and uveal melanoma. PRMT5 is an enzyme that adds methyl groups to proteins in cells using a molecule called S-adenosylmethionine (SAM) which regulates protein function and interactions. Several processes that support cancer cell growth and spread depend on PRMT5, making P-500 relevant not only to advanced high-grade glioma and uveal melanoma (in which objective responses to P-500 were observed in the Phase 1 clinical trial) but also to a number of other cancer indications. Preclinical studies have demonstrated PRMT5 inhibition can sensitize cancer cells to other treatments, expanding the application of P-500 to combination therapy in additional indications. About Pathos Pathos is a clinical-stage biotechnology company focused on re-engineering drug development, leveraging the power of AI technologies to bring precision medicines to market through partnership with biopharmaceutical companies. Pathos has raised to accelerate the development of precision medicines and to expand its platform, combining computational approaches across multimodal real-world data and patient-derived biological models. Additional information can be found at www.pathos.com.
Market Screener
Fri, 16 Aug, 12:07 PM UTC
Gilead Sciences Partners with Genesis Therapeutics for AI-Driven Drug Discovery
Gilead Sciences has entered into a strategic collaboration with Genesis Therapeutics to leverage artificial intelligence for drug discovery. The partnership aims to accelerate the development of novel therapies across multiple disease areas.
2 Sources
Wed, 11 Sept, 12:05 AM UTC
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
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