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Nonclinical Efficacy and Toxicology Evaluation of a Human FVIII Gene Therapy Vector (TAK-754/SHP654) in Mice (ISTH 2019) - "Treatment with TAK-754/SHP654 was shown to be efficacious in mice at doses ≥1.2x10 12 cp/kg and the No-Observed-Adverse-Effect-Level in mice was considered to be 5x10 13 cp/kg, the highest dose tested."

Automated Immunofluorescence Analysis of Dystrophin and Other Markers in Mouse Muscle and Heart Sections (ASGCT 2022) - "After systemically treating Dup2 mice with our AAV9.U7.ACCA vector to induce U7snRNA-mediated skipping of exon 2, we show widespread restoration of dystrophin signal at the sarcolemma throughout whole sections from multiple ...

COVER: Clinical Study of the Safety and Immunogenicity of a Recombinant Viral Vector AAV5 (Adeno-Associated Virus Type 5 )-RBD (Receptor Binding Domain)-S Vaccine for the Prevention of Coronavirus Infection (COVID-19) (clinicaltrials.gov) - P1/2 | N=50 | Terminated | Sponsor: Biocad | N=160 50 | Trial completion date: Dec 2022 Apr 2022 ...

A novel AAV vector has been developed for the treatment of XLRS. AAV.SPR-hGRK1-hRS1 leads to therapeutic levels of RS1 in subretinally injected RS1KO mice, resulting in improvements in both retinal structure and function.

(ASCO 2019) - P2; "C consists of a dendritic cell-targeting lentiviral vector encoding NY-ESO-1 gene (LV305), and a TLR-4 agonist NY-ESO-1 recombinant protein plus GLA-SE (G305). Despite no major differences in PFS and OS between the treatment arms (Arm C+A had more advanced disease and more prior lines of chemotherapy), Arm A +C achieved PRs ...

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Laterally Spreading AAV.SPR-hRS1 Vector for Treatment of XLRS (A0341) - Apr 29, 2022 - Abstract #ARVO2022ARVO_1531; AAV.SPR delivered therapeutic levels of hRS1 to subretinally injected RS1KO mice, resulting in improvements in both retinal structure and function.

(PubMed, JAMA Ophthalmol) - "To use adaptive optics scanning light ophthalmoscopy (AOSLO) to assess the short-term integrity of the cone mosaic following subretinal injections of adeno-associated virus vector designed to deliver a functional version of the CHM gene (AAV2-hCHM) in patients with choroideremia...Minor foveal thinning observed ...

Moreover, we propose an integrated end-to-end neural network learning framework based on one complete encoder-decoder architecture transformer model: Transfer Text-to-Text Transformer (T5), by learning the embedding vector representation space of conditional molecular properties to encode and guide the vector representation of SMILES sequences ...

The observed safety and phenotypic correction support ongoing clinical study of SPK-8016 including optimization of the immunomodulatory regimen, with the potential for clinically meaningful FVIII activity at low vector doses."