Sarah Lewis

Pharmacokinetic and Pharmacodynamic Evaluation of Bidridistrogene Xeboparvovec in an Aged Murine Model of Limb-Girdle Muscular Dystrophy Type 2E/R4

Stephen H. Baine, Young-Eun Seo, Amber Kempton, Oliver C. Rogers et autres

Limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4) is an ultra-rare autosomal recessive disorder caused by mutations in SGCB , the gene that encodes for β-sarcoglycan (SGCB), a component of the dystrophin-associated protein complex that stabilizes muscle fibers during contractions. Bidridistrogene xeboparvovec is an …

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424P Micro-dystrophin expression and safety with delandistrogene moxeparvovec gene therapy for DMD in a broad population: phase 1B trial (ENDEAVOR)

Crystal M. Proud, Craig Mitchell Zaidman, Craig M. McDonald, John Day et autres

Hepatic stearoyl-CoA desaturase-1 deficiency induces fibrosis and hepatocellular carcinoma-related gene activation under a high carbohydrate low fat diet

Jayne-Norah Ntambi, Mugagga Kalyesubula, Dylan Cootway, Sarah Lewis et autres

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Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74

Rachael A. Potter, Ellyn L. Peterson, D. Griffin, Grace Cooper Olson et autres

Patients with pre-existing immunity to adeno-associated virus (AAV) are currently unable to receive systemic gene transfer therapies. In this nonhuman primate study, we investigated the impact of immunosuppression strategies on gene transfer therapy safety and efficacy and analyzed plasmapheresis as a potential …

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Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results

Jerry R. Mendell, Eric R. Pozsgai, Sarah Lewis, Danielle A. Griffin et autres

Abstract Limb-girdle muscular dystrophy 2E/R4 is caused by mutations in the β-sarcoglycan ( SGCB ) gene, leading to SGCB deficiency and consequent muscle loss. We developed a gene therapy approach based on functional replacement of the deficient SCB protein. Here we report …

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Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial

Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman, Linda Pax Lowes et autres

INTRODUCTION/AIMS: Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. Long-term delandistrogene moxeparvovec microdystrophin protein (a shortened dystrophin …

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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP ‐9001‐103 ( ENDEAVOR )

Craig Mitchell Zaidman, Crystal M. Proud, Craig M. McDonald, Kelly J. Lehman et autres

OBJECTIVE: Delandistrogene moxeparvovec is approved in the USA for the treatment of ambulatory patients (4-5 years) with Duchenne muscular dystrophy. ENDEAVOR (SRP-9001-103; NCT04626674) is a single-arm, open-label study to evaluate delandistrogene moxeparvovec micro-dystrophin expression, safety, and functional outcomes following administration of commercial …

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One-year Data from ENDEAVOR, a Phase 1b Trial of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD) (S48.003)

Craig Mitchell Zaidman, Crystal M. Proud, Craig M. McDonald, Stefanie Mason et autres

Objective: To evaluate expression and safety of intended commercial process delandistrogene moxeparvovec (SRP-9001) material in ENDEAVOR (NCT04626674). Background: Delandistrogene moxeparvovec is an investigational gene transfer therapy developed to address the root cause of Duchenne muscular dystrophy (DMD) through targeted skeletal and cardiac …

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Analysis of Vector Shedding Following Treatment with Delandistrogene Moxeparvovec (SRP-9001), an Investigational rAAVrh74-Based Gene Therapy for Duchenne Muscular Dystrophy (DMD) (P5-8.013)

Jyoti Dhar Malhotra, Elizabeth Smith, Sarah Lewis, Xiaolan Zhang et autres

Objective: To evaluate extent and magnitude of vector shedding following delandistrogene moxeparvovec (SRP-9001) administration. Background: Delandistrogene moxeparvovec is an investigational gene transfer therapy developed to address the root cause of DMD through targeted skeletal and cardiac muscle expression of SRP-9001 dystrophin protein, …

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Long-term Safety and Efficacy in Patients with DMD 4 Years Post-Treatment with Delandistrogene Moxeparvovec (SRP-9001) in a Phase 1/2a Study (P3-8.006)

J. Mendell, Zarife Sahenk, Kelly J. Lehman, Linda Pax Lowes et autres

To evaluate long-term safety and functional outcomes 4 years post-treatment with delandistrogene moxeparvovec (SRP-9001) in Study 101 (NCT03375164): a Phase 1/2a, single-dose, open-label clinical trial.

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Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model

Young-Eun Seo, Stephen H. Baine, Amber N. Kempton, Oliver C. Rogers et autres

Limb-girdle muscular dystrophy (LGMD) type 2C/R5 results from mutations in the γ-sarcoglycan ( SGCG ) gene and is characterized by muscle weakness and progressive wasting. Loss of functional γ-sarcoglycan protein in the dystrophin-associated protein complex destabilizes the sarcolemma, leading to eventual myofiber …

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Hepatic Oleate Regulates Insulin-like Growth Factor-Binding Protein 1 Partially through the mTORC1-FGF21 Axis during High-Carbohydrate Feeding

Lucas M. O’Neill, Yar Xin Phang, Zhaojin Liu, Sarah Lewis et autres

Stearoyl-CoA desaturase-1 (SCD1) catalyzes the rate-liming step of monounsaturated fatty acid biosynthesis and is a key regulator of systemic glucose metabolism. Mice harboring either a global (GKO) or liver-specific deletion (LKO) of Scd1 display enhanced insulin signaling and whole-body glucose uptake. Additionally, …

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