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1. 发明申请

WO2019126593A1 IMPROVED COMPOSITION AND METHODS FOR TREATMENT OF ORNITHINE TRANSCARBAMYLASE DEFICIENCY 审中-公开
公开(公告)号：WO2019126593A1
公开(公告)日：2019-06-27
申请号：PCT/US2018/066970
申请日：2018-12-20
申请人： TRANSLATE BIO, INC.
发明人： ASKEW, Kim , CHUNG, Jou-ku , DEROSA, Frank , HEARTLEIN, Michael , KARVE, Shrirang , MCCAULEY, Thomas , SMITH, Lianne , BARBIER, Ann, J.
IPC分类号： A61K38/45 , A61P3/00
CPC分类号： A61K48/005 , A61K9/0019 , A61K9/1272 , A61K38/45 , A61K47/6929 , A61K48/0025 , A61K48/0075 , A61K48/0083 , A61P3/00 , C12N15/52 , C12N15/85 , C12Y201/03003
摘要： The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

2. 发明申请

WO2019079242A1 TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) 审中-公开
公开(公告)号：WO2019079242A1
公开(公告)日：2019-04-25
申请号：PCT/US2018/056001
申请日：2018-10-16
申请人： VOYAGER THERAPEUTICS, INC.
发明人： SAH, Dinah Wen-Yee , PATZKE, Holger , CHEN, Qingmin , CARROLL, Jenna
IPC分类号： C12N15/113 , C12N15/864 , A61K31/713
CPC分类号： C12Y115/01001 , A61K48/005 , A61K48/0075 , A61P21/00 , C12N15/1137 , C12N15/86 , C12N2310/14 , C12N2330/51 , C12N2750/14143
摘要： The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.

3. 发明申请

WO2019006161A1 TREATMENT OF DEVELOPMENTAL SYNDROMES 审中-公开
公开(公告)号：WO2019006161A1
公开(公告)日：2019-01-03
申请号：PCT/US2018/040084
申请日：2018-06-28
申请人： OVID THERAPEUTICS INC.
发明人： DURING, Matthew
IPC分类号： C12N15/63 , C12N15/85 , C12N15/86 , C12N15/861 , A61P3/04 , A61P3/08 , A61P3/10 , A61B5/055
CPC分类号： A61K48/0016 , A61K31/7105 , A61K48/005 , A61K48/0075 , A61M37/0092 , A61P43/00 , C07K14/475 , C12N15/113 , C12N15/1136 , C12N15/62 , C12N2310/141 , C12N2750/14143
摘要： A method of treating a developmental syndrome in a patient in need thereof includes applying ultrasound to a target location in the patient's brain to enhance permeability of the patient's blood brain barrier at the target location and administering to the patient a vector encoding BDNF for delivery of BDNF to the target location, wherein the method provides improvement in at least one symptom of the developmental syndrome. Also provided is a method of treating a developmental syndrome in a patient in need thereof that includes administering to the patient an effective amount of a vector including (i) a constitutive promoter operatively linked to nucleic acid encoding BDNF, and (ii) a regulatory sequence including an AGRP promoter operatively linked to an interference RNA sequence, wherein the regulatory sequence down regulates expression of BDNF in response to BDNF induced physiological changes, and the method provides improvement in at least one symptom of the developmental syndrome.

4. 发明申请

WO2017136500A1 GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I 审中-公开
标题翻译：基因疗法治疗粘多糖1型
公开(公告)号：WO2017136500A1
公开(公告)日：2017-08-10
申请号：PCT/US2017/016133
申请日：2017-02-02
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： HINDERER, Christian , WILSON, James, M.
IPC分类号： C12N15/86 , A61K48/00 , C12N9/24 , A61K39/00
CPC分类号： C12N15/86 , A61K48/005 , A61K48/0075 , C12N9/2402 , C12N2750/14143 , C12N2830/15 , C12Y302/01076
摘要： A suspension useful for AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating MPSI and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
摘要翻译：本文提供了用于AAV9介导的鞘内/脑池内和/或全身递送含有hIDUA基因的表达盒的悬液。还提供了包含这些载体和组合物的方法和试剂盒，用于治疗MPSI以及与Hurler，Hurler-Scheie和Scheie综合征有关的症状。

5. 发明申请

WO2017136202A1 INJECTION OF SINGLE-STRANDED OR SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS 9 INTO THE CEREBROSPINAL FLUID 审中-公开
标题翻译：单束或自补体腺相关病毒注入脑脊液
公开(公告)号：WO2017136202A1
公开(公告)日：2017-08-10
申请号：PCT/US2017/014914
申请日：2017-01-25
申请人： EMORY UNIVERSITY , REGENXBIO INC.
发明人： DONSANTE, Anthony , KOZARSKY, Karen
IPC分类号： C12N15/63 , C12N15/861 , A61K48/00
CPC分类号： C12N15/86 , A61K48/0075 , C12N2750/14143
摘要： It is disclosed herein that ssAAV and scAAV vectors of the same serotype administered by injection into the cerebrospinal fluid (CSF) via the intracerebroventricular (ICV) or intrathecal (cisternal or lumbar) route exhibit different cellular tropisms in the central nervous system. Thus, a subject can be treated by injection into the CSF of ssAAV or scAAV vector encoding a therapeutic protein, such as an ssAAV9 or scAAV9 vector. The therapeutic protein can be targeted to specific cells using these vectors. In some embodiments, scAAV9 is utilized to achieve superior transduction in the hippocampus, cerebellum and cerebral cortex where both neurons, particularly Purkinje neurons, and glial cells (such as astrocytes) are transduced. In other embodiments, ssAAV9 is utilized to minimize transduction of astrocytes. In further embodiments, an immunosuppressive agent is also administered to the subject.
摘要翻译：本文中公开了通过脑室内（ICV）或鞘内（脑池或腰椎）途径注射到脑脊液（CSF）中的相同血清型的ssAAV和scAAV载体显示不同的细胞向性中枢神经系统。因此，可以通过向CSF中注射编码治疗性蛋白质如ssAAV9或scAAV9载体的ssAAV或scAAV载体来治疗受试者。使用这些载体可将治疗性蛋白质靶向特定细胞。在一些实施方案中，利用scAAV9在海马，小脑和大脑皮层中实现优异的转导，其中神经元，特别是浦肯野神经元和神经胶质细胞（例如星形胶质细胞）都被转导。在其他实施方案中，利用ssAAV9来最小化星形胶质细胞的转导。在进一步的实施方案中，还将免疫抑制剂施用给受试者。

6. 发明申请

WO2016197121A1 COMPOSITIONS AND METHODS FOR TRANSIENT GENE THERAPY WITH ENHANCED STABILITY 审中-公开
标题翻译：用于具有增强稳定性的瞬时基因治疗的组合物和方法
公开(公告)号：WO2016197121A1
公开(公告)日：2016-12-08
申请号：PCT/US2016/036045
申请日：2016-06-06
申请人： DANA-FARBER CANCER INSTITUTE, INC.
发明人： GOLDBERG, Michael , CARMONA, Ellese
IPC分类号： C07K14/435 , C12N15/11 , C12N5/00
CPC分类号： A61K35/17 , A61K48/0075 , A61K48/0091 , A61P35/00 , C07K14/005 , C07K14/195 , C07K14/435 , C12N5/0636 , C12N15/111 , C12N2320/51
摘要： The present invention provides circularized RNA and methods of making and using same.
摘要翻译：本发明提供了循环的RNA及其制备和使用方法。

7. 发明申请

WO2016193431A1 ADENOASSOCIATED VIRUS VECTORS FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSES 审中-公开
标题翻译：用于治疗脑膜炎的ADENOASSOCIAV病毒载体
公开(公告)号：WO2016193431A1
公开(公告)日：2016-12-08
申请号：PCT/EP2016/062655
申请日：2016-06-03
申请人： LABORATORIOS DEL DR. ESTEVE, S.A. , UNIVERSITAT AUTÓNOMA DE BARCELONA
发明人： BOSCH TUBERT, Maria Fátima , AREBA HAURIGOT, Virginia , MOTAS MALLOL, Sandra
IPC分类号： C12N9/16 , C12N15/864 , A61K48/00
CPC分类号： A61K48/0066 , A61K9/0019 , A61K48/00 , A61K48/0016 , A61K48/0075 , A61P43/00 , C12N9/16 , C12N15/79 , C12N2750/14143 , C12N2800/22 , C12Y301/06013
摘要： The present invention provides new Adeno-associated virus-derivedvectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disordersand specially, for the treatment of mucopolysaccharidoses Type II.
摘要翻译：本发明提供了新的腺相关病毒衍生的医药和含有它们的用于治疗溶酶体储存障碍的药物组合物，特别是用于治疗II型粘多糖贮积症。

8. 发明申请

WO2016183593A2 PRENATAL THERAPY 审中-公开
标题翻译：临床治疗
公开(公告)号：WO2016183593A2
公开(公告)日：2016-11-17
申请号：PCT/US2016/032785
申请日：2016-05-16
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： WANG, Aijun , MIGUELINO, Maricel , POWELL, Jerry
IPC分类号： A61K48/00 , C12N15/86
CPC分类号： A61K48/0075 , A61K48/0083 , C12N15/86 , C12N2740/15043
摘要： Compositions and methods are provided to treat or prevent a genetic disease or disorder, e.g., Hemophilia A, in a subject by administering to the subject an effective amount of a polynucleotide encoding a nucleic acid encoding a functional Factor VIII polypeptide in a pre-term placenta-derived stem cell. The subject to be treated can be a fetus and the cell is a pre-term chorionic villus tissue derived stem cell (CSC) that has been isolated from the placenta before the baby is born.
摘要翻译：提供组合物和方法以通过向受试者施用有效量的编码在术前胎盘中编码功能性因子VIII多肽的核酸的多核苷酸来治疗或预防受试者中的遗传疾病或病症，例如血友病A 衍生的干细胞。待治疗的对象可以是胎儿，并且细胞是在婴儿出生之前已经从胎盘分离出的术前绒毛膜绒毛组织来源的干细胞（CSC）。

9. 发明申请

WO2016130591A3 ENHANCED DELIVERY OF VIRAL PARTICLES TO THE STRIATUM AND CORTEX 审中-公开
标题翻译：增强向STRIATUM和CORTEX递送病毒颗粒
公开(公告)号：WO2016130591A3
公开(公告)日：2016-11-10
申请号：PCT/US2016017210
申请日：2016-02-09
申请人： GENZYME CORP
发明人： STANEK LISA M , SHIHABUDDIN LAMYA
IPC分类号： C12N15/86 , A61K48/00
CPC分类号： C12N15/86 , A61K48/00 , A61K48/0075 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152
摘要： Provided herein are novel methods for delivering recombinant adeno-associated viral (rAAV) particles to the central nervous system of a mammal (e.g., a human). In aspects, the methods involve administering rAAV particles containing a heterologous nucleic acid to the striatum and causing expression of the heterologous nucleic acid in at least the cerebral cortex and the striatum of the mammal.
摘要翻译：本文提供了将重组腺相关病毒（rAAV）颗粒递送至哺乳动物（例如人）的中枢神经系统的新方法。在方面中，所述方法包括将含有异源核酸的rAAV颗粒施用于纹状体，并引起异源核酸在至少大脑皮质和哺乳动物纹状体中的表达。

10. 发明申请

WO2016122791A1 SPINAL SUBPIAL GENE DELIVERY SYSTEM 审中-公开
标题翻译：脊柱亚基基因递送系统
公开(公告)号：WO2016122791A1
公开(公告)日：2016-08-04
申请号：PCT/US2015/065704
申请日：2015-12-15
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： MARSALA, Martin
IPC分类号： A61M25/06 , A61K35/76 , C12N15/113
CPC分类号： A61K48/0075 , A61B17/3401 , A61K9/0085 , A61K35/00 , A61K47/36 , A61L29/041 , A61M25/0662 , A61M2210/1003 , C12N7/00 , C12N15/111 , C12N15/86 , C12N2310/11 , C12N2320/32 , C12N2330/51 , C12N2750/14143
摘要： Delivery devices, systems, and methods related thereto may be used in humans for spinal delivery of cells, drugs or vectors. Thus, the system enables subpial delivery, which leads to a near complete spinal parenchymal AAV9-mediated gene expression or ASO distribution in both white and grey matter.
摘要翻译：与人有关的递送装置，系统和方法可以用于细胞，药物或载体的脊柱递送。因此，该系统能够进行唾液分泌，这导致在白色和灰色物质中接近完全的脊髓实质AAV9介导的基因表达或ASO分布。

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