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1. 发明申请

WO2022232178A1 COMPOSITIONS AND METHODS FOR TREATMENT OF OCULAR DISEASE ASSOCIATED WITH ANGIOGENESIS 审中-公开
公开(公告)号：WO2022232178A1
公开(公告)日：2022-11-03
申请号：PCT/US2022/026395
申请日：2022-04-26
申请人： 4D MOLECULAR THERAPEUTICS INC.
发明人： BURNS, Christian , CALTON, Melissa , LEONG, Meredith , SZYMANSKI, Paul
IPC分类号： C12N15/86
摘要： The present disclosure provides compositions and methods for the treatment of ocular diseases associated with angiogenesis, particularly wet age-related macular degeneration.

2. 发明申请

WO2022051294A1 CODON OPTIMIZED REP1 GENES AND USES THEREOF 审中-公开
公开(公告)号：WO2022051294A1
公开(公告)日：2022-03-10
申请号：PCT/US2021/048510
申请日：2021-08-31
申请人： 4D MOLECULAR THERAPEUTICS INC.
发明人： KIRN, David H. , KOTTERMAN, Melissa A. , FRANCIS, Peter
IPC分类号： A61K35/76 , A61K48/00 , A61P27/02 , C12N15/86
摘要： The present disclosure provides codon optimized nucleotide sequences encoding human REP1, vectors, and host cells comprising codon optimized REP1 sequences, and methods of treating retinal disorders such as choroideremia comprising administering to the subject a codon optimized sequence encoding human REP1.

3. 发明申请

WO2021222094A1 CODON OPTIMIZED GLA GENES AND USES THEREOF 审中-公开
公开(公告)号：WO2021222094A1
公开(公告)日：2021-11-04
申请号：PCT/US2021/029146
申请日：2021-04-26
申请人： 4D MOLECULAR THERAPEUTICS INC.
发明人： KIRN, David, H. , KOTTERMAN, Melissa , FRANCIS, Peter , SCHAFFER, David , SZYMANSKI, Paul , WHITTLESEY, Kevin
IPC分类号： A61K35/761 , A61K38/47 , A61K48/00
摘要： The present disclosure provides codon optimized nucleotide sequences encoding human alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.

4. 发明申请

WO2022051232A1 CODON OPTIMIZED RPGRORF 15 GENES AND USES THEREOF 审中-公开
公开(公告)号：WO2022051232A1
公开(公告)日：2022-03-10
申请号：PCT/US2021/048267
申请日：2021-08-30
申请人： 4D MOLECULAR THERAPEUTICS INC.
发明人： KIRN, David, H. , KOTTERMAN, Melissa, A. , SCHAFFER, David , FRANCIS, Peter
IPC分类号： A61K9/127 , A61K9/14 , A61K9/50 , A61K38/00 , C07K14/47 , C12N15/88
摘要： The present disclosure provides codon optimized RPGRorfl5 sequences, vectors, and host cells comprising codon optimized RPGRorfl5 sequences, and methods of treating retinal disorders such as XLRP comprising administering to the subject a codon optimized RPGRorfl5 sequence.

5. 发明申请

WO2021222148A2 ADENO-ASSOCIATED VARIANTS, FORMULATIONS AND METHODS FOR PULMONARY DELIVERY 审中-公开
公开(公告)号：WO2021222148A2
公开(公告)日：2021-11-04
申请号：PCT/US2021/029253
申请日：2021-04-26
申请人： 4D MOLECULAR THERAPEUTICS INC.
发明人： KOTTERMAN, Melissa , FRANCIS, Peter , CALTON, Melissa , GONZALES, Johnny , CROZE, Roxanne , SCHMITT, Christopher
IPC分类号： C12N15/864 , C07K14/47 , C07K14/015 , A61K48/00 , A61K38/17 , A61K38/16
摘要： The present disclosure provides a variant AAV capsid protein that confers tropism to lung cells and recombinant adeno-associated viruses comprising the variant AAV and pharmaceutical compositions comprising same and their use in the delivery of heterologous nucleic acids to lung cells for the treatment of pulmonary disorders.

6. 发明申请

WO2005089106A3 DEGRADABLE NANOPARTICLES 审中-公开
标题翻译：可降解的纳米颗粒
公开(公告)号：WO2005089106A3
公开(公告)日：2007-05-31
申请号：PCT/US2005006054
申请日：2005-02-28
申请人： MOLECULAR THERAPEUTICS INC , REDDY G RAMACHANDRA , ERATHODIYIL NANDANAN
发明人： REDDY G RAMACHANDRA , ERATHODIYIL NANDANAN
IPC分类号： A61K8/02 , A01N43/04 , A61K31/7072 , A61K38/17 , A61K49/00
CPC分类号： A61K31/7072 , A61K41/0071 , A61K47/62 , A61K47/6933 , A61K49/0021 , A61K49/0093 , A61K49/1854 , A61K49/1881 , B82Y5/00
摘要： The present invention relates to polymeric nanoparticles, particularly useful in drug and agent delivery, as well as for imaging and diagnosis. The polymeric nanoparticles of the present invention comprise cross-linkers that, when degraded, leave simple linear polymeric molecules that can be excreted by the body. The present invention also relates to methods of producing the polymeric nanoparticles of the present invention, and methods of using them in drug and agent delivery, as well as imaging and diagnosis.
摘要翻译：本发明涉及聚合物纳米颗粒，特别适用于药物和药剂递送，以及成像和诊断。本发明的聚合物纳米颗粒包含交联剂，当其降解时，留下可被身体排泄的简单的线性聚合物分子。本发明还涉及制备本发明的聚合物纳米颗粒的方法，以及将其用于药物和药剂递送以及成像和诊断中的方法。

7. 发明申请

WO2006031720A3 D-METHIONINE FORMULATION WITH IMPROVED BIOPHARMACEUTICAL PROPERTIES 审中-公开
标题翻译：具有改善的生物医学性质的D-甲硫氨酸配方
公开(公告)号：WO2006031720A3
公开(公告)日：2006-05-11
申请号：PCT/US2005032399
申请日：2005-09-14
申请人： MOLECULAR THERAPEUTICS INC , SUNKARA PRASAD
发明人： SUNKARA PRASAD
IPC分类号： A61K9/10 , A61K31/198 , A61K47/34 , A61P1/04 , A61P25/00
CPC分类号： A61K9/0095 , A61K31/198 , A61K47/26
摘要： The present invention provides pharmaceutical suspensions of D-methionine in which the aqueous solubility of D-methionine is exceeded, thereby allowing oral administration of higher doses. The present invention also provides processes for preparing these suspensions. The present invention further provides methods for preventing, treating, or ameliorating oral mucositis, hearing loss due to chemotherapy, antibiotics and noise, neuronal damage due to various CNS disorders and injuries, and anthracycline toxicity.
摘要翻译：本发明提供D-甲硫氨酸的药物悬浮液，其中超过D-甲硫氨酸的水溶性，从而允许口服较高剂量。本发明还提供了制备这些悬浮液的方法。本发明还提供了预防，治疗或改善口腔粘膜炎的方法，由于化疗引起的听力损失，抗生素和噪音，由于各种CNS障碍和损伤引起的神经元损伤以及蒽环类毒素。

8. 发明申请

WO2005120489A3 TREATMENT OF CENTRAL NERVOUS SYSTEM DISORDERS OR INJURIES WITH D-METHIONINE 审中-公开
标题翻译：治疗中枢神经系统疾病或D型甲硫氨酸的损伤
公开(公告)号：WO2005120489A3
公开(公告)日：2006-04-06
申请号：PCT/US2005019462
申请日：2005-06-03
申请人： MOLECULAR THERAPEUTICS INC , SUNKARA PRASAD
发明人： SUNKARA PRASAD
IPC分类号： A61K31/198 , A01N31/00 , A01N25/00 , A61K31/095
CPC分类号： A61K31/198
摘要： The present invention relates to methods for preventing, treating, or ameliorating neuronal damage resulting from a central nervous system disorder or injury in an animal comprising administering to the animal D-methionine.
摘要翻译：本发明涉及用于预防，治疗或改善动物中的中枢神经系统紊乱或损伤引起的神经元损伤的方法，包括向动物D-甲硫氨酸施用。

9. 发明申请

WO2019060454A2 ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF 审中-公开
公开(公告)号：WO2019060454A2
公开(公告)日：2019-03-28
申请号：PCT/US2018/051812
申请日：2018-09-19
申请人： 4D MOLECULAR THERAPEUTICS INC.
发明人： KIRN, David H. , KOTTERMAN, Melissa , SCHAFFER, David
摘要： Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.

10. 发明申请

WO2017197355A2 ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF 审中-公开
标题翻译： ADENO相关病毒变体和使用它们的方法
公开(公告)号：WO2017197355A2
公开(公告)日：2017-11-16
申请号：PCT/US2017/032542
申请日：2017-05-12
申请人： 4D MOLECULAR THERAPEUTICS INC.
发明人： KIRN, David, H. , KOTTERMAN, Melissa , SCHAFFER, David
IPC分类号： A61K48/00 , C12N15/861
摘要： Provided herein are variant adeno-assoeiated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infect! vity of the retinal cells by an AA V virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.
摘要翻译：本文提供了相对于亲本AAV衣壳蛋白在氨基酸序列中具有一个或多个修饰的变体腺伴随病毒（AAV）衣壳蛋白，其当存在于AAV病毒体中时赋予增加的感染性的一种或多种类型的视网膜细胞与感染相比！由包含未修饰的亲本AAV衣壳蛋白的AA V病毒粒子对视网膜细胞的视野。还提供了包含如本文所述的变体AAV衣壳蛋白的重组AAV病毒体及其药物组合物，制备这些rAAV衣壳蛋白和病毒体的方法，以及在研究和临床实践中使用这些rAAV衣壳蛋白和病毒体的方法，例如在例如将核酸序列递送至视网膜的一个或多个细胞以治疗视网膜疾病和疾病。

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