公开(公告)号：WO2023037311A1

公开(公告)日：2023-03-16

申请号：PCT/IB2022/058516

申请日：2022-09-09

申请人： UNIVERSIDADE DE COIMBRA

发明人： DA SILVA FERREIRA, Lino ,  CARDOSO DUARTE RODRIGUES, Artur Filipe

IPC分类号： C12N15/88 ,  A61K9/51 ,  A61K47/69 ,  A61K41/00 ,  C12N9/00

摘要： Here polymeric nanoparticle formulations based on the combination of 3 monomers were identified lead candidates capable of efficient mRNA delivery, after evaluating 152 formulations by high-throughput screening using a reporter fibroblast model. Using in vitro and in vivo models, this formulation transfected fibroblasts much more effectively than other cell types populating the skin, with superior performance than lipid-based transfection agents in the delivery of Cas9 mRNA and guide RNA. This tropism could be explained by receptor-mediated endocytosis, involving CD26 and FAP, which are overexpressed in profibrotic fibroblasts. Structure-activity analysis revealed that efficient mRNA delivery required the combination of high buffering capacity and low mRNA binding affinity for rapid release upon endosomal escape. These nanoformulations may find multiple applications in the modulation of fibroblasts involved in fibrotic diseases of the skin, heart, liver, and lung, as well as in the microenvironment of epithelial tumors, and in cellular reprogramming.

公开(公告)号：WO2023027082A1

公开(公告)日：2023-03-02

申请号：PCT/JP2022/031768

申请日：2022-08-23

申请人： 積水化学工業株式会社

发明人： 譚　哲立

IPC分类号： C07K17/02 ,  A61K35/12 ,  A61K45/00 ,  A61K47/69 ,  A61P43/00 ,  C07K7/06 ,  C07K7/08 ,  C07K14/705 ,  C12N5/071 ,  C12N5/0735 ,  C12N5/0775 ,  C12N15/11 ,  C12Q1/24 ,  G01N33/53

摘要： 標的細胞により高濃度の有効成分を送達可能な、ハイブリッドリポソームエクソソーム等を提供することを課題とする。　ＣＤ６３、ＣＤ８１、及びＣＤ９から選ばれるエクソソームマーカータンパク質と、複合脂質の親水性部位にペプチドが結合している、ペプチド結合脂質誘導体と、を脂質二重層中に含む、ペプチド結合ハイブリッドリポソームエクソソームによれば、標的細胞のエンドソームに取り込まれた後のペプチド結合ハイブリッドリポソームエクソソームのエンドソーム脱出に関する付加的機能を付与することができ、以って標的細胞へのより高濃度の有効成分の送達が可能な、ドラッグデリバリーシステムのキャリアを提供できる。

公开(公告)号：WO2023007017A1

公开(公告)日：2023-02-02

申请号：PCT/EP2022/071475

申请日：2022-07-29

申请人： 4BASEBIO UK LTD ,  4BASEBIO, S.L.U.

发明人： WALKER, Amy ,  LANCKRIET, Heikki ,  PICHER, Ángel

IPC分类号： A61K49/00 ,  A61K47/50 ,  A61K47/69

摘要： Nanoparticles suitable for delivery of a linear DNA molecule (e.g. a closed linear DNA molecule) to a target cell are provided. Further provided are uses of the nanoparticles including the use of the nanoparticles for treating disease.

公开(公告)号：WO2023001108A1

公开(公告)日：2023-01-26

申请号：PCT/CN2022/106270

申请日：2022-07-18

申请人： 成都赜灵生物医药科技有限公司

发明人： 陈俐娟

IPC分类号： A61K31/5377 ,  A61K9/08 ,  A61K47/69 ,  A61K47/18 ,  A61P35/00

摘要： 本发明提供了一种组蛋白去乙酰化酶抑制剂的制剂，包含组蛋白去乙酰化酶抑制剂和辅料；所述辅料为环糊精、精氨酸、葡甲胺中的至少一种或两种以上的组合；本发明制剂显著增加了组蛋白去乙酰化酶抑制剂在水中的溶解度，提高了稳定性，并保留了组蛋白去乙酰化酶抑制剂优异的抗肿瘤活性，拓宽了组蛋白去乙酰化酶抑制剂的应用，具有极高的临床价值。

公开(公告)号：WO2023286060A1

公开(公告)日：2023-01-19

申请号：PCT/IL2022/050753

申请日：2022-07-13

申请人： NANOCARRY THERAPEUTICS LTD.

发明人： POPOVTZER, Rachela ,  BETZER, Oshra ,  SAGIV, Yuval ,  MANDIL-LEVIN, Revital ,  ANTEBI, Adam A.

IPC分类号： A61K47/69 ,  A61K31/00 ,  A61K9/51 ,  C08G65/00 ,  A61K47/00 ,  A61P25/00 ,  A61K47/54

摘要： The present invention provides a BBB-permeable multifunctional system for the synchronized delivery of distinct active agents to the brain. The multifunctional system is based on an inorganic core particle which is conjugated through a first polymeric linker to a first active agent; through a second polymeric linker to a second active agent; and through a third polymeric linker to a brain-internalizing transporter moiety. Further provided are a process for preparation of the multifunctional system, pharmaceutical compositions comprising the multifunctional system and uses thereof in therapeutic and/or diagnostic methods.

公开(公告)号：WO2022261150A2

公开(公告)日：2022-12-15

申请号：PCT/US2022/032579

申请日：2022-06-07

申请人： SCRIBE THERAPEUTICS INC. ,  FERNANDES, Jason

发明人： HIGGINS, Sean ,  COLIN, Isabel ,  SPINNER, Hannah ,  GARDNER, Matthew ,  GOMBERG, Trent ,  VIJAYAKUMAR, Gayathri ,  DENNY, Sarah ,  STAAHL, Brett T. ,  ADIL, Maroof ,  OAKES, Benjamin ,  SIDORE, Angus ,  MAKHIJA, Suraj

IPC分类号： A61K47/69 ,  C07K14/16 ,  C12N15/11 ,  C12N9/22 ,  C12N9/50 ,  C12N7/02 ,  C07K14/005 ,  C12N15/111 ,  C12N2310/16 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2320/32 ,  C12N2740/11022 ,  C12N2740/12022 ,  C12N2740/13022 ,  C12N2740/14022 ,  C12N2740/15023 ,  C12N2740/16022 ,  C12N2740/16043 ,  C12N2740/16222 ,  C12N2760/20122 ,  C12N2760/20222 ,  C12N2795/18122 ,  C12N7/00

摘要： Provided herein are delivery particle systems (XDP) useful for the delivery of payloads of any type. In some embodiments, a XDP particle system with tropism for target cells of interest is used to deliver CRISPR/Cas polypeptides (e.g., CasX proteins) and guide nucleic acids (gNA), for the modification of nucleic acids in target cells. Also provided are methods of making and using such XDP to modify the nucleic acids in such cells.

公开(公告)号：WO2022260480A1

公开(公告)日：2022-12-15

申请号：PCT/KR2022/008229

申请日：2022-06-10

申请人： 주식회사 나이벡 ,  서울대학교산학협력단

发明人： 박윤정 ,  정종평 ,  이주연

IPC分类号： A61K48/00 ,  A61K47/64 ,  A61K47/54 ,  A61K47/69 ,  A61K31/7088 ,  A61K31/713 ,  A61P35/00 ,  A61P29/00 ,  A61K9/51

摘要： 본 발명은 치료용으로 사용하려는 올리고뉴클레오티드를 세포 내로 전달하기 위한 전달체에 관한 것으로 세포 내에서 타겟 단백질의 억제하거나, 타겟 단백질의 발현을 촉진시키기 위해, 펩타이드-지질의 결합체를 제조하고, 올리고뉴클레오티드를 포함한 펩타이드-지질의 결합체로 구성된 나노입자 및 이를 포함하는 약학적 조성물에 관한 것이다. 본 발명에 따른 펩타이드-지질 결합체로 구성된 나노입자를 이용하여 세포 내로 올리고뉴클레오티드가 효과적으로 전달된 것을 확인하였다. 또한, 올리고뉴클레오티드에 의해 암을 유발하는 단백질의 발현을 감소시켰고, 암을 발생시킨 동물 모델에서 항암 효과가 있음을 확인함으로써, 상기 펩타이드-지질 결합체로 구성된 나노입자가 올리고뉴클레오티드의 전달에 효과적임을 확인할 수 있었다.

公开(公告)号：WO2022253950A1

公开(公告)日：2022-12-08

申请号：PCT/EP2022/065036

申请日：2022-06-02

申请人： TOPAS THERAPEUTICS GMBH

发明人： POHLNER, Johannes ,  DIGIGOW, Reinaldo ,  METZLER, Barbara ,  FLEISCHER, Sabine

IPC分类号： A61K47/69 ,  A61P37/00 ,  A61P37/08 ,  A61K47/6931 ,  A61K47/6933

摘要： The present invention provides nanoparticles comprising: (a) an amphiphilic polymer with a number average molecular weight (Mn) of 20,000 g/mol or less; and (b) a peptide that is covalently linked to the polymer, wherein the peptide comprises 8 to 50 amino acids, including an N-terminal linker sequence comprising at least one Arg amino acid residue and a sequence comprising an MHC binding sequence comprising a T cell receptor epitope. The present invention further comprises compositions comprising respective nanoparticles and a liquid or lyophilized carrier as well as nanoparticles and compositions of the invention for use in inducing tolerance to a therapeutic compound (protein, viral vector, lipid vesicle), an allergen or to an autoantigen or for treating an allergy, an autoimmune disease, an exogenous antigen (transplantation antigens, drugs) or a food intolerance.

公开(公告)号：WO2022246217A1

公开(公告)日：2022-11-24

申请号：PCT/US2022/030280

申请日：2022-05-20

申请人： NORTHWESTERN UNIVERSITY

发明人： MCMAHON, Kaylin, M. ,  THAXTON, C., Shad

IPC分类号： C40B20/04 ,  G01N33/53 ,  G01N33/68 ,  G01N33/92 ,  A61K47/64 ,  A61K47/69 ,  A61K48/00 ,  A61K38/00 ,  A61K39/00

摘要： Methods, devices and kits for detecting an antigen in a sample using synthetic nanoparticles in flow (e.g., vertical flow or lateral flow) are provided.

公开(公告)号：WO2022245032A1

公开(公告)日：2022-11-24

申请号：PCT/KR2022/006584

申请日：2022-05-09

申请人： 주식회사 디알나노

发明人： 한선미 ,  장준근 ,  이혜명 ,  이용덕

IPC分类号： A61K41/00 ,  A61K47/69 ,  A61K47/54 ,  A61K31/5415 ,  A61K9/00 ,  A61P29/00 ,  A61P31/00 ,  A61P31/16 ,  A61N5/06

摘要： 본 발명은 염증성 질환 치료용 광반응성 마이셀 복합체에 관한 것으로, 보다 상세하게는 (i) 메틸렌 블루 및 상기 메틸렌 블루에 이온 결합한 유기산을 포함하는 코어; 및 (ii) 지방산으로 이루어진 마이셀 형태로 상기 코어를 감싸는 쉘;을 포함하여 국소적으로 작용 가능하며 광 조사 시 상승적인 항균 및 항바이러스 효과를 갖는 코어-쉘 구조의 염증성 질환 또는 감염성 질환의 예방 또는 치료용 광반응성 마이셀 복합체를 제공한다.