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efficacy from toxicity over multiple iterations creating the next gene efficacy from toxicity over multiple iterations creating the next gene

efficacy from toxicity over multiple iterations creating the next gene - PDF document

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Uploaded On 2021-10-03

efficacy from toxicity over multiple iterations creating the next gene - PPT Presentation

110100100010000100000Concentration nggConcentration nggKey Tissue PMO QuantificationSkeletal muscle ID: 893861

delivery efficacy oligonucleotide disease efficacy delivery disease oligonucleotide muscle pepgen drive

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1 efficacy from toxicity over multiple ite
efficacy from toxicity over multiple iterations Ðcreating the next generation of delivery peptidesNew series of modified peptidesIdentify leads & iterateScreening for1. Efficacy in vivo2. Biodistribution3. Safety!PepGen EDOsSafe & EffectiveOUR EDOs WERE DEVELOPED THROUGH A DATA-DRIVE DESIGN PROCESSA novel enhanced delivery oligonucleotide (EDO) therapeutic demonstrates considerable potential in treating Duchenne muscular dystrophy Dr Ashling Holland, Dr Smita Gunnoo, Sam Ching, Rachel Johnson, Calum Irwin, Dr Sonia Bracegirdle, Dr Caroline GodfreyPepGen:BioEscalator, Innovation Building, Old Road Campus, Roosevelt Drive, Oxford, OX3 7FZ, United KingdomAn exciting step-change in oligonucleotide delivery with thepotential to transform the course of disease in DMDSuperior skeletal & cardiac muscle penetrance and efficacy, allowing critical disease phenotypes to be addressedA rapid path to the clinic: PepGen is on track to initiate first-in-human studies in 2022 110100100010000100000Concentration (ng/g) Concentration (ng/g)Key Tissue PMO QuantificationSkeletal muscle