We are pleased to report new preclinical data demonstrating the ability of PM359 to correct a common disease-causing mutation of CGD. Jennifer Gori will present these findings at #ASGCT2024 this evening. Full details below. https://lnkd.in/euUeTEjb
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Chief Data Scientist @ Persistent | Leading Generative AI CoE | Author of “Keras to Kubernetes" | ex GE | 11 Patents
#artificialintelligence has already started accelerating #drugdiscovery from years to months. AI-driven drug discovery identified TNIK as an anti-fibrotic target, leading to INS018_055, a promising small-molecule inhibitor with anti-fibrotic and anti-inflammatory properties. This work by Insilico Medicine completed in 18 months, showcases the potential of AI in rapidly advancing preclinical candidates for fibrotic diseases. Detailed study: https://lnkd.in/dzMDRUQ8 Insilico's AI-Generated Drug in Human Trials: https://lnkd.in/duKD-Wx3
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Targeted drugs aim to pinpoint the exact location in the body where diseased tissue is located and where the medicine is required. The manifold benefits of administering a targeted drug include heightened efficacy, as the drug is meticulously designed for specificity, thereby reducing side effects, and minimizing damage to healthy tissue. Consequently, this approach enhances the patient's quality of life during treatment. Oligonucleotides (ONs), specifically designed short chains of DNA or RNA, have emerged as a crucial tool with immense potential in personalized medicine. These therapeutic ONs are already in use for conditions, such as certain types of muscular dystrophy and liver disease, which conventional drugs cannot address. Depending on the type, ONs can function by, preventing or changing the production of a protein in the cell, particularly beneficial in diseases caused by the overproduction of a specific protein. https://lnkd.in/ghtqZy7A
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We are delighted to participate in the ISPE Europe Annual conference on 16-18 April 2024 in Lisbon. We will present on the topic “Accommodating Multiple ATMP Modalities in the Same Facility”. The conference will explore themes like: * Digital Transformation * GAMP 5 * Good Engineering Practice and Efficient Investment Management * Learnings from GXP Inspections * Regulatory Trends and Drug availability *Our Speakers Alf Penfold, Technical Director - GMP/Regulatory Compliance and Jon Halling, Catapult Cell and Gene Therapy will speak at the event. *About CGT Catapult CGT Catapult collaborates with industry in the development, production and adoption of Advanced Therapy Medicinal Products (ATMPs). PM Group's Catapult facility design will describe a process that has been developed to accommodate multiple modalities within the same facility. This is starting to be used more widely within the industry. CGT Catapult highlights their compliance amidst multiple GXP inspections across various ATMP modalities. #ATMPmodalities #advancedtherapymedicinalproducts #CGT #cellgenetherapy #pmgroup CGT Catapult
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Advancements in translational research! Our point mutation mouse models are pivotal in preclinical drug discovery. Read more about our Coq2A252V model and its impact on CoQ deficiency syndrome. LINK: https://hubs.la/Q02yQFzt0 #translationalscience, #mousemodels, #genetargeting, #geneediting #drugdiscovery #ingenioustargeting
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Chief Business Officer at GPCR Therapeutics, Inc.
2moRewinding 20 years, my very first project was developing CGD gene therapy using a retroviral vector in collaboration with Seoul National University Hospital. It’s so lovely to see that there is a progress here. Best of luck to all of you!