SG11201910015SA - Gene therapy for neuronal ceroid lipofuscinoses - Google Patents

Gene therapy for neuronal ceroid lipofuscinoses

Info

Publication number
SG11201910015SA
SG11201910015SA SG11201910015SA SG11201910015SA SG 11201910015S A SG11201910015S A SG 11201910015SA SG 11201910015S A SG11201910015S A SG 11201910015SA SG 11201910015S A SG11201910015S A SG 11201910015SA
Authority
SG
Singapore
Prior art keywords
international
street
philadelphia
pct
aav
Prior art date
Application number
Other languages
English (en)
Inventor
James Wilson
Nathan Katz
Christian Hinderer
Juliette Hordeaux
Original Assignee
Univ Pennsylvania
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of SG11201910015SA publication Critical patent/SG11201910015SA/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485Exopeptidases (3.4.11-3.4.19)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/16011Herpesviridae
    • C12N2710/16111Cytomegalovirus, e.g. human herpesvirus 5
    • C12N2710/16141Use of virus, viral particle or viral elements as a vector
    • C12N2710/16143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009Tripeptidyl-peptidase I (3.4.14.9)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Dispersion Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SG11201910015S 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses SG11201910015SA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762504817P 2017-05-11 2017-05-11
US201762599816P 2017-12-18 2017-12-18
US201862652006P 2018-04-03 2018-04-03
PCT/US2018/032278 WO2018209205A1 (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses

Publications (1)

Publication Number Publication Date
SG11201910015SA true SG11201910015SA (en) 2019-11-28

Family

ID=64105028

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201912401QA SG10201912401QA (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses
SG11201910015S SG11201910015SA (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SG10201912401QA SG10201912401QA (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses

Country Status (11)

Country Link
US (2) US11591614B2 (enExample)
EP (1) EP3621612A4 (enExample)
JP (2) JP7273730B2 (enExample)
KR (1) KR102719222B1 (enExample)
AU (1) AU2018265531B2 (enExample)
BR (1) BR112019023303A2 (enExample)
CA (1) CA3061655A1 (enExample)
IL (2) IL316926A (enExample)
RU (1) RU2019139555A (enExample)
SG (2) SG10201912401QA (enExample)
WO (1) WO2018209205A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3209821A1 (en) 2013-05-15 2014-11-20 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
CN110198712A (zh) * 2016-11-04 2019-09-03 费城儿童医院 用于治疗神经退行性疾病的基因转移组合物、方法和用途
JP7449223B2 (ja) 2017-09-22 2024-03-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ii型を治療するための遺伝子療法
KR20210092755A (ko) * 2018-11-14 2021-07-26 리젠엑스바이오 인크. 신경원성 세로이드 리포푸신증에 대한 유전자 요법
MX2021006253A (es) * 2018-11-28 2021-09-21 Prevail Therapeutics Inc Terapias génicas para enfermedad neurodegenerativa.
WO2020210324A1 (en) * 2019-04-08 2020-10-15 The Children's Hospital Of Philadelphia Treatment of lysosomal storage disease in the eye through administration of aavs expressing tpp1
WO2020210633A1 (en) * 2019-04-12 2020-10-15 Encoded Therapeutics, Inc. Compositions and methods for administration of therapeutics
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
IL300409A (en) * 2020-08-10 2023-04-01 Prevail Therapeutics Inc Gene therapies for neurodegenerative disorders
MX2023008826A (es) * 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2025090598A1 (en) * 2023-10-23 2025-05-01 The Children's Hospital Of Philadelphia Aav vectors for treatment of cln2 disease

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
WO1998044151A1 (en) 1997-04-01 1998-10-08 Glaxo Group Limited Method of nucleic acid amplification
NZ500546A (en) 1997-04-14 2001-09-28 Cell Genesys Inc Methods for increasing the production of high titre stocks of recombinant adeno-associated virus (AAV)
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
JP2002538770A (ja) 1998-11-10 2002-11-19 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル ウイルスベクターとその製造及び投与の方法
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
AU2001268149B2 (en) 2000-06-01 2005-08-18 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
ATE403013T1 (de) 2001-05-18 2008-08-15 Wisconsin Alumni Res Found Verfahren zur synthese von dna-sequenzen die photolabile linker verwenden
ES2258601T3 (es) 2001-11-13 2006-09-01 The Trustees Of The University Of Pennsylvania Un metodo para la identificacion de las secuencias desconocidas del virus adeno-asociado (vaa) y un kit para el metodo.
EP2359869B1 (en) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
US20080050357A1 (en) 2003-08-01 2008-02-28 Claes Gustafsson Systems and Methods for Antibody Engineering
EP3910063A1 (en) 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP4234687A2 (en) 2005-04-07 2023-08-30 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
WO2006132118A1 (ja) 2005-06-09 2006-12-14 Matsushita Electric Industrial Co., Ltd. 振幅誤差補償装置及び直交度誤差補償装置
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
CA3006395C (en) 2008-11-07 2022-05-31 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
WO2010071832A1 (en) * 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
MX342858B (es) 2010-03-29 2016-10-13 The Trustees Of The Univ Of Pennsylvania * Sistema de ablacion transgenica inducida farmacologicamente.
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
WO2012135857A1 (en) 2011-03-31 2012-10-04 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
LT2717893T (lt) 2011-06-08 2019-08-12 Translate Bio, Inc. Lipidų nanodalelių kompozicijos ir mrnr pristatymo būdai
EP2834260A4 (en) 2012-04-02 2016-08-10 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION OF MEMBRANE PROTEINS
US20150126589A1 (en) 2012-06-08 2015-05-07 Ethris Gmbh Pulmonary Delivery of Messenger RNA
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
WO2015013148A2 (en) * 2013-07-26 2015-01-29 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
EP3800260A1 (en) 2014-09-24 2021-04-07 City of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
BR112018008069A2 (pt) * 2015-10-23 2018-11-13 Univ Iowa Res Found métodos de tratamento de doenças neurodegenerati-vas usando terapia gênica para retardar o início e progressão da doença ao mesmo tempo em que fornece proteção cognitiva
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
CA3012195A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
FI3589730T3 (fi) 2017-02-28 2024-02-22 Univ Pennsylvania Kladin f adenoassosioitunut virus (aav) -vektori ja sen käyttötapoja

Also Published As

Publication number Publication date
RU2019139555A3 (enExample) 2021-10-12
IL316926A (en) 2025-01-01
AU2018265531A1 (en) 2019-11-14
EP3621612A4 (en) 2021-06-16
US20230383312A1 (en) 2023-11-30
AU2018265531B2 (en) 2024-07-11
CA3061655A1 (en) 2018-11-15
KR20200023280A (ko) 2020-03-04
IL270422B1 (en) 2024-12-01
SG10201912401QA (en) 2020-02-27
BR112019023303A2 (pt) 2020-06-16
KR102719222B1 (ko) 2024-10-21
IL270422A (en) 2020-01-30
EP3621612A1 (en) 2020-03-18
WO2018209205A9 (en) 2019-11-21
JP2020519292A (ja) 2020-07-02
JP7273730B2 (ja) 2023-05-15
US20200239908A1 (en) 2020-07-30
WO2018209205A1 (en) 2018-11-15
IL270422B2 (en) 2025-04-01
RU2019139555A (ru) 2021-06-11
JP2023099113A (ja) 2023-07-11
US12428653B2 (en) 2025-09-30
US11591614B2 (en) 2023-02-28

Similar Documents

Publication Publication Date Title
SG11201910015SA (en) Gene therapy for neuronal ceroid lipofuscinoses
SG11201907714UA (en) Adeno-associated virus (aav) clade f vector and uses therefor
SG11201811432WA (en) Rna for cancer therapy
SG11201907474UA (en) Compositions useful in treatment of spinal muscular atrophy
SG11201809029RA (en) Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
SG11201908575SA (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
SG11201909949XA (en) Targeted immunotolerance
SG11201811603WA (en) Novel adeno-associated virus capsid proteins
SG11201908023YA (en) T cell receptors and immune therapy using the same against prame positive cancers
SG11201809684YA (en) Adeno-associated virus variant capsids and methods of use thereof
SG11201811600PA (en) Pseudotyped oncolytic viral delivery of therapeutic polypeptides
SG11201906297QA (en) Nucleic acids encoding crispr-associated proteins and uses thereof
SG11201811338VA (en) A novel botulinum neurotoxin and its derivatives
SG11201809344QA (en) Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde
SG11201909777YA (en) Modulatory polynucleotides
SG11201906319XA (en) Compositions and methods for the depletion of cd137+ cells
SG11201810210UA (en) Engineered botulinum neurotoxins
SG11201804312RA (en) Microbubble-chemotherapeutic agent complex for sonodynamic therapy
SG11201804729RA (en) Methods and compositions for treating a serpinc1-associated disorder
SG11201901996UA (en) Formulations of ( r)-2-amino-3-phenylpropyl carbamate
SG11202000312UA (en) Encapsulated polynucleotides and methods of use
SG11201804673WA (en) Novel anti-claudin antibodies and methods of use
SG11201408679XA (en) Methods for treating tyrosine-kinase-inhibitor-resistant malignancies in patients with genetic polymorphisms or ahi1 dysregulations or mutations employing dianhydrogalactitol, diacetyldianhydrogalactitol, dibromodulcitol, or analogs or derivatives thereof
SG11201811244SA (en) Dual overlapping adeno-associated viral vector system for expressing abc4a
SG11201804070XA (en) Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a