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2024
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P/S
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Downloadable software in the nature of a mobile application for enabling users to enter, access, ... |
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P/S
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Pharmaceutical preparations for the prevention and treatment of cardiovascular diseases and disea... |
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Invention
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Integrated continuous manufacturing of therapeutic protein drug substances.
Provided herein are ... |
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Invention
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Analytical ultracentrifugation for characterization of recombinant viral particles.
Provided her... |
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Invention
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Methods of controlling the formation of disulfide bonds in protein solutions.
Disclosed herein a... |
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Invention
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Substituted cyclic compounds and methods of treating phenylketonuria and other amino acidurias. T... |
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Invention
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Process for preparing 4-(3,3-difluoro-2,2-dimethyl-propanoyl)-3,5-dihydro-2h-pyrido[3,4-f][l,4]ox... |
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Invention
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Sleep-wake classification using machine learning. Methods, systems, and apparatus, including comp... |
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Invention
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Treatment of multiple sclerosis with anti-cd40l antibodies.
The present invention relates to tre... |
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Invention
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Treatment of multiple sclerosis with anti-cd40l antibodies. The present invention relates to trea... |
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Invention
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Fluid transfer device. A fluid transfer device includes a body having a first connecting portion ... |
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Invention
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Predicting motor function scores in pompe disease treatment. A prediction method for predicting a... |
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Invention
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Drug formulations of 4-(3,3-difluoro-2,2-dimethyl-propanoyl) -3,5-dihydro-2h-pyrido[3,4-f][1,4]ox... |
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Invention
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Isoxazolidines as ripk1 inhibitors and use thereof. 14141414,14222222, or a pharmaceutically acce... |
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Invention
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Isoxazolidines as ripk1 inhibitors and use thereof. 1236127103144143456166)alkyl group or a halog... |
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Invention
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Isoxazolidines as ripk1 inhibitors and use thereof. 14141414,142 14321414221414363636366)heterocy... |
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Invention
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Perfusion culturing methods and uses thereof.
Provided herein are methods of culturing a mammali... |
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Invention
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Parkinson's tremor classification using machine learning. Methods, systems, and apparatus, includ... |
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Invention
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Marker for acid sphingomyelinase disorders and uses thereof.
The present disclosure provides met... |
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Invention
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Methods of improving raav production.
This disclosure relates to methods and compositions for sc... |
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Invention
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Methods of improving raav production. This disclosure relates to methods and compositions for sca... |
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Invention
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Humanized anti-complement factor bb antibodies and uses thereof.
Provided herein are humanized a... |
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Invention
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Targeted gene therapy for dm-1 myotonic dystrophy.
Provided herein are RNAi molecules for treati... |
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Invention
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Scfv-fc dimers that bind transforming growth factor-beta1 with high affinity, avidity and specifi... |
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Invention
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Targeted gene therapy for dm-1 myotonic dystrophy. e.g., e.g., rAAV), viral particles, and pharma... |
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Invention
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Devices for cell lysis and methods of use thereof. Provided herein are methods for isolating intr... |
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Invention
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Gene therapy for neurodegenerative disorders.
Compositions and methods for treating disorders af... |
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Invention
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Treatment of dry age-related macular degeneration.
The present disclosure provides gene therapy ... |
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Invention
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Treatment of dry age-related macular degeneration. The present disclosure provides gene therapy t... |
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Invention
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Nbd1 modulators and methods of using the same. The present disclosure includes, among other thing... |
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Invention
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Indole compounds and methods of use.
The invention relates to heterocyclic compounds, pharmaceut... |
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Invention
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Automated screening for acid sphingomyelinase deficiency. Methods, systems, and apparatus, includ... |
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Invention
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Hsc-specific antibody conjugated lipid nanoparticles and uses thereof. Provided are ionizable cat... |
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Invention
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Assays for detecting the presence or amount of an anti-drug antibody.
Methods and kits for detec... |
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Invention
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Drug formulations of 4-(3,3-difluoro-2,2-dimethyl-propanoyl)-3,5-dihydro-2h-pyrido [3,4-f] [1,4] ... |
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Invention
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Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients.
The inventio... |
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Invention
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Recombinant aav vectors expressing osteoprotective genes, including has2 and lubricin, useful in ... |
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Invention
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Colony stimulating factor-1 receptor (csf-1r) inhibitors.
Compounds of the formulas
Compounds ... |
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2023
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Invention
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Gene editing of deep intronic mutations.
Provided herein are compositions, methods, kits, and vi... |
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Invention
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Anti-pd-1×4-1bb binding proteins.
Anti-4-1BB and anti-PD-1 binding proteins, as well as bispecif... |
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Invention
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Anti‑pd‑1×4‑1bb binding proteins. Anti 4 1BB and anti PD 1 binding proteins, as well as bispecifi... |
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Invention
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Drug formulations of (r)-l-(l-acryloylpiperidin-3-yl)-4-amino-3-(4- phenoxyphenyl)-1h-imidazo[4,5... |
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Invention
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Modified binding polypeptides for optimized drug conjugation.
Engineered antibodies that are sta... |
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P/S
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Pharmaceutical products for the prevention and treatment of immunological diseases; pharmaceutica... |
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P/S
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Pharmaceutical products for the prevention and treatment of immunological diseases, pharmaceutica... |
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Invention
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Tropomyosin-related kinase (trk) inhibitors.
Tropomyosin-related kinase inhibitors (Trk inhibito... |
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Invention
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Anti-complement factor bb antibodies and uses thereof.
The present disclosure provides anti-comp... |
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Invention
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Methods for purification of recombinant aav vectors.
Provided herein are methods for the purific... |
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Invention
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Pharmaceutical formulations of tropomyosin related kinase (trk) inhibitors.
Pharmaceutical formu... |
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P/S
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Pharmaceuticals for use in the treatment of lysosomal storage diseases |
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P/S
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Support services for patients and caregivers in the field of rare genetic diseases and disorders,... |
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P/S
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Pharmaceutical preparations for the treatment of autosomal dominant polycystic kidney disease; ph... |
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2022
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Invention
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Novel predictive biomarkers for secondary autoimmunity after lymphocyte depleting therapy.
The i... |
