WO2022040432A1 - Adeno-associated virus vectors for treatment of rett syndrome - Google Patents

Adeno-associated virus vectors for treatment of rett syndrome Download PDF

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Publication number
WO2022040432A1
WO2022040432A1 PCT/US2021/046699 US2021046699W WO2022040432A1 WO 2022040432 A1 WO2022040432 A1 WO 2022040432A1 US 2021046699 W US2021046699 W US 2021046699W WO 2022040432 A1 WO2022040432 A1 WO 2022040432A1
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Prior art keywords
nucleic acid
seq
sequence
aav
promoter
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Ceased
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PCT/US2021/046699
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English (en)
French (fr)
Inventor
Colin O'BANION
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Stridebio Inc
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Stridebio Inc
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Priority to US18/022,059 priority Critical patent/US20230304038A1/en
Priority to MX2023002016A priority patent/MX2023002016A/es
Priority to CN202180063852.3A priority patent/CN116745409A/zh
Priority to BR112023003145A priority patent/BR112023003145A2/pt
Priority to EP21766796.3A priority patent/EP4200408A1/en
Priority to JP2023512086A priority patent/JP2023542614A/ja
Priority to CA3189878A priority patent/CA3189878A1/en
Priority to AU2021328475A priority patent/AU2021328475A1/en
Priority to IL300728A priority patent/IL300728A/en
Priority to KR1020237008777A priority patent/KR20230068444A/ko
Application filed by Stridebio Inc filed Critical Stridebio Inc
Priority to IL300717A priority patent/IL300717A/en
Publication of WO2022040432A1 publication Critical patent/WO2022040432A1/en
Anticipated expiration legal-status Critical
Priority to CONC2023/0003074A priority patent/CO2023003074A2/es
Priority to JP2025259436A priority patent/JP2026034639A/ja
Ceased legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/61—Growth hormone [GH], i.e. somatotropin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Definitions

  • Rett Syndrome is a genetic neurological disorder manifesting at about 6 months to 18 months of age primarily in girls and women. Affecting about 1 in 8,500 females, Rett Syndrome is a rare disease. Patients with Rett Syndrome often exhibit a wide range of symptoms including language disability, reduced coordination, microcephaly, repetitive movements, seizures, scoliosis, and cognitive disability. While the life expectancy for women with Rett Syndrome is in the mid-40s, males with Rett Syndrome often die in infancy.
  • the neuronal immediate early gene is selected from the group consisting of the Arc gene, the c-fos gene, and the egr-1 gene.
  • the promoter comprises a minimal Arc gene promoter (ArcMin).
  • the AAV cassette comprises a brain-derived neurotrophic factor (BDNF) short 3’ UTR, or a BDNF long 3’ UTR.
  • BDNF brain-derived neurotrophic factor
  • the BDNF short 3’ UTR, or the BDNF long 3’ UTR is located between the Rett-syndrome-associated gene and the 3’ ITR.
  • the AAV cassette comprises a BDNF short 3’ UTR.
  • the BDNF short 3’ UTR comprises a nucleic acid sequence of SEQ ID NO: 8, or a sequence at least 90% identical thereto.
  • the AAV cassette comprises a BDNF long 3’ UTR.
  • the BDNF long 3’ UTR comprises a nucleic acid sequence of SEQ ID NO: 10, or a sequence at least 90% identical thereto.
  • the disclosure provides methods for expressing brain-derived neurotrophic factor (BDNF) in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of any one of the nucleic acids disclosed herein, any one of the plasmids disclosed herein, any one of the cells disclosed herein, or any one of the recombinant AAV vectors disclosed herein, or any one of the compositions disclosed herein.
  • BDNF brain-derived neurotrophic factor
  • the BDNF-associated disease is depression, obsessive- compulsive disorder, Alzheimer's disease, Huntington's disease, and dementia, anorexia nervosa, bulimia nervosa, schizophrenia, epilepsy, post-traumatic stress disorder, bipolar disease, Rett syndrome, major depressive disorder, or post-chemotherapy cognitive impairment.
  • FIG. 9C shows an AAV expression cassette, comprising the nucleic acid sequence of SEQ ID NO: 28, and comprising the following elements: the hSyn promoter (SEQ ID NO: 38), mScarlet gene (SEQ ID NO: 36), the bGH polyA signal (SEQ ID NO: 9) and a stuffer sequence (SEQ ID NO: 13), all of which are flanked by a 5’ ITR (SEQ ID NO: 1) and a 3’ ITR (SEQ ID NO: 2).
  • the Rett-syndrome-associated gene encodes brain-derived neurotrophic factor (BDNF).
  • BDNF is a neurotrophin that functions in supporting survival and growth of neurons, synaptic development, and plasticity.
  • BDNF expression is altered in Rett syndrome. Loss of function of bdnf results in phenotypes similar to loss of function of mecp2. j and BDNF function is affected upon loss of mecp2 function. Further details about BDNF function are provided in Eduardo E. Benarroch, Neurology Apr 2015, 84 (16) 1693-1704; Chang et al. Neuron.
  • the AAV expression cassette comprises SEQ ID NO: 1 (5’ ITR), SEQ ID NO: 16 (E-SARE), SEQ ID NO: 7 (BDNF), SEQ ID NO: 9 (bGH polyadenylation sequence) and SEQ ID NO: 2 (3’ ITR).
  • the AAV expression cassette comprises SEQ ID NO: 1 (5’ ITR), SEQ ID NO: 16 (E-SARE), SEQ ID NO: 7 (BDNF), SEQ ID NO: 8 (BDNF short 3’UTR), and SEQ ID NO: 2 (3’ ITR).
  • the AAV expression cassette comprises SEQ ID NO: 1 (5’ ITR), SEQ ID NO: 6 (hSARE-ArcMin), SEQ ID NO: 7 (BDNF), SEQ ID NO: 10 (BDNF long 3’UTR), and SEQ ID NO: 2 (3’ ITR).
  • the neurons are active neurons.
  • the level of expression of the Rett Syndrome-associated gene is higher in active neurons than in resting neurons of the body. In some embodiments, the expression of the Rett Syndrome-associated gene is not detectable in resting neurons.
  • the administration is by injection into the central nervous system.
  • the most suitable route in any given case will depend on the nature and severity of the condition being treated and/or prevented and on the nature of the particular vector that is being used.
  • Delivery to a target tissue can also be achieved by delivering a depot comprising the virus vector and/or capsid.
  • a depot comprising the virus vector and/or capsid is implanted into skeletal, cardiac and/or diaphragm muscle tissue or the tissue can be contacted with a film or other matrix comprising the virus vector and/or capsid.
  • FIG. 7 shows that the subcellular localization of mScarlet expressed from a constitutive promoter varies based on the 3’UTR region or polyadenylation signal of the expression cassette. While expression cassettes comprising the long 3’UTR and bGHpA resulted in mScarlet expression in the cell body and neurites, expression cassettes comprising the short 3’UTR resulted in mScarlet expression only in the cell body.
  • Embodiment 17 The nucleic acid of embodiment 16, wherein the promoter comprises a nucleic acid sequence of SEQ ID NO: 16, or a sequence at least 90% identical thereto.
  • Embodiment 27 The nucleic acid of any one of embodiments 1-26, wherein at least one of the 5’ ITR and the 3’ ITR is isolated or derived from the genome of AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12, AAVrh8, AAVrhlO, AAVrh32.33, AAVrh74, Avian AAV or Bovine AAV.
  • Embodiment 35 The nucleic acid of embodiment 34, wherein the BDNF long 3’ UTR comprises a nucleic acid sequence of SEQ ID NO: 10, or a sequence at least 90% identical thereto.

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PCT/US2021/046699 2020-08-19 2021-08-19 Adeno-associated virus vectors for treatment of rett syndrome Ceased WO2022040432A1 (en)

Priority Applications (13)

Application Number Priority Date Filing Date Title
IL300728A IL300728A (en) 2020-08-19 2021-08-19 Adeno-associated virus vectors for the treatment of Rett syndrome
CN202180063852.3A CN116745409A (zh) 2020-08-19 2021-08-19 用于治疗雷特综合征的腺相关病毒载体
BR112023003145A BR112023003145A2 (pt) 2020-08-19 2021-08-19 Vetores de vírus adenoassociado para tratamento da síndrome de rett
EP21766796.3A EP4200408A1 (en) 2020-08-19 2021-08-19 Adeno-associated virus vectors for treatment of rett syndrome
JP2023512086A JP2023542614A (ja) 2020-08-19 2021-08-19 レット症候群の治療のためのアデノ随伴ウイルスベクター
CA3189878A CA3189878A1 (en) 2020-08-19 2021-08-19 Adeno-associated virus vectors for treatment of rett syndrome
AU2021328475A AU2021328475A1 (en) 2020-08-19 2021-08-19 Adeno-associated virus vectors for treatment of Rett syndrome
US18/022,059 US20230304038A1 (en) 2020-08-19 2021-08-19 Adeno-associated virus vectors for treatment of rett syndrome
MX2023002016A MX2023002016A (es) 2020-08-19 2021-08-19 Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
KR1020237008777A KR20230068444A (ko) 2020-08-19 2021-08-19 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
IL300717A IL300717A (en) 2020-08-19 2021-08-19 ADENO-related virus vectors for the treatment of RETT syndrome
CONC2023/0003074A CO2023003074A2 (es) 2020-08-19 2023-03-14 Vectores de virus adenoasociados para el tratamiento del síndrome de rett
JP2025259436A JP2026034639A (ja) 2020-08-19 2025-12-17 レット症候群の治療のためのアデノ随伴ウイルスベクター

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US202063067668P 2020-08-19 2020-08-19
US63/067,668 2020-08-19

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KR (1) KR20230068444A (https=)
CN (1) CN116745409A (https=)
AR (1) AR123288A1 (https=)
AU (1) AU2021328475A1 (https=)
BR (1) BR112023003145A2 (https=)
CA (1) CA3189878A1 (https=)
CL (1) CL2023000493A1 (https=)
CO (1) CO2023003074A2 (https=)
IL (2) IL300728A (https=)
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WO2024079658A1 (en) * 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Bdnf gene therapy
WO2024079667A1 (en) * 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Nucleic acid regulatory elements for gene expression in the central nervous system and methods of use

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CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터

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