Spark Therapeutics (United States)

Recent papers from Spark Therapeutics (United States)

1. Industry Perspective on Translational and Clinical Pharmacology Aspects of Viral-based Gene Therapies and Vaccines—Key Considerations and Learnings From Approved ProductsThe AAPS Journal · 2026
2. AAV2 bypasses direct endosomal escape by using AAVR to access the trans-Golgi network en route to the nucleusJournal of Virology · 2026
3. Research agenda and related workforce requirements inspired by Lived Experience Experts and grounded in health equity for the Bleeding Disorders Research CollaborativeExpert Review of Hematology · 2026
4. Polymorph-Dependent Oxidation Activity of MnO2: Influence of Surface Water, Morphology, and Surface Area in Benzylic OxidationChemistry · 2026
5. Interlaboratory Comparison of RF Power Measurements Made by Automatic Power Measurement SoftwareMetrology · 2026
6. Enabling remuneration for patient authors of company-sponsored publications: approaches and case study examples aligned with Good Publication practiceCurrent Medical Research and Opinion · 2026
7. Draft genome sequences of two Pseudomonas species from Lake St. Clair (Ontario, Canada) encode multiple heavy metal resistance determinantsMicrobiology Resource Announcements · 2026
8. A phase 1, first-in-human, open-label, dose-escalation and -expansion study of gamitrinib, a first-in-class, mitochondria-directed inhibitor of the molecular chaperone heat shock protein-90 (Hsp90) in patients with advanced cancer.Journal of Clinical Oncology · 2026
9. Discovery, development, and characterization of SPY002 and SPY072, two novel extended half-life monoclonal antibodies targeting TL1A: in vitro properties, in vivo pharmacology, pharmacokinetics, and preclinical safetymAbs · 2026
10. Quantitative Imaging of Iron Dysregulation in Multiple System Atrophy.2026
11. Intravitreal AAV vector delivery induces integrin-dependent ocular inflammation, complement activation, and antiviral and DNA damage responsesFrontiers in Immunology · 2026
12. DNA delivered by lipid nanoparticles induces CD8+ T cell-dependent antitumor responses and enhances anti-PD-L1 therapy.2026
13. Joseph B. Nadol, Jr., MD (1943–2025)Otology & Neurotology · 2026
14. Spatial transcriptomics and single-nucleus RNA sequencing reveal rAAV2- and rAAV9-specific transduction signatures in the mouse liverGene Therapy · 2026
15. Limits of Correctional Health Reporting: Findings From a Nationwide Jail Mortality Survey (2019-2024).2026
16. Systemic Administration of AAV9-G266A with Focused Ultrasound Enables Robust Brain Transduction with Minimal Liver Transduction.2026
17. AAV NRF2 Gene Therapy Preserves Retinal Structure and Function in Rodent Models of Oxidative DamageMolecular Therapy · 2026
18. Expert Consensus on Characteristics, Etiology, and Management of Chorioretinal Atrophy in Patients Treated with Voretigene NeparvovecOphthalmology Science · 2026
19. Role of viral protein ratio in the structure and separation of empty and full adeno-associated virus capsids: A molecular dynamics studyMolecular Therapy Advances · 2025
20. Administration of barcoded AAV capsid library to the putamen of non-human primates identifies variants with efficient retrograde transportMolecular Therapy · 2025