公开(公告)号：US11738047B2

公开(公告)日：2023-08-29

申请号：US16216774

申请日：2018-12-11

申请人： The Trustees of the University of Pennsylvania

发明人： Yangbing Zhao ,  Xiaojun Liu

IPC分类号： A61K35/17 ,  C07K19/00 ,  C07K14/725 ,  C12N5/10 ,  C07K14/705 ,  A61P35/00 ,  C12N15/62 ,  A61K38/00

CPC分类号： A61K35/17 ,  A61P35/00 ,  C07K14/7051 ,  C07K14/70596 ,  C07K19/00 ,  C12N5/10 ,  C12N15/62 ,  A61K38/00

摘要： The present disclosure provides modified immune cell (e.g., modified T cell) comprising an exogenous T cell receptor (TCR) having specificity for NY-ESO-1. The present disclosure provides modified immune cells or precursors thereof (e.g., modified T cells) comprising an exogenous TCR and a switch receptor. Gene edited modified cells are also provided, such that the expression of one or more of an endogenous T-cell receptor gene (e.g., TRAC, TRBC) or an endogenous immune checkpoint gene (e.g., PD-1 or TIM-3) is downregulated.

公开(公告)号：US20230255193A1

公开(公告)日：2023-08-17

申请号：US18302111

申请日：2023-04-18

申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

发明人： Matthew Winterton ,  Lawrence Scott Levin ,  Edward Cantu

IPC分类号： A01N1/02 ,  G09B23/28

CPC分类号： A01N1/0226 ,  A01N1/0247 ,  G09B23/285

摘要： Systems and methods for an ex vivo tissue perfusion apparatus are provided. In certain embodiments, the disclosed subject matter includes a container to store ex vivo tissue, a plurality of tubes containing a perfusate, and a pump which cycles the perfusate through the ex vivo tissue. The perfusion apparatus can be configured for mobility and can be transported to active emergency situations in order to perform mobile perfusion of ex vivo tissues. In certain embodiments, the discloses subject matter contemplates a method for performing mobile profusion of ex vivo tissue in emergency situations, for example, active warzones. In certain embodiments, the profusion apparatus can be used as part of a method of training for doctors in the reattachment of limbs or other extremities that have been amputated.

公开(公告)号：US11723989B2

公开(公告)日：2023-08-15

申请号：US16768547

申请日：2018-11-29

申请人： The Trustees of the University of Pennsylvania

发明人： James M. Wilson ,  Christian Hinderer ,  Juliette Hordeaux

IPC分类号： A61K35/76 ,  C12N7/00 ,  A61K48/00 ,  C12N15/86

CPC分类号： A61K48/0075 ,  A61K35/76 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143

摘要： Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5′ inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional human N-acetyl-alpha-glucosaminidase (hNAGLU), a regulatory sequence which direct expression of hNAGLU in a target cell, and an AAV 3′ ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIB.

公开(公告)号：US11718685B2

公开(公告)日：2023-08-08

申请号：US16417125

申请日：2019-05-20

申请人： The Trustees of the University of Pennsylvania ,  The Wistar Institute of Anatomy and Biology

发明人： Carl H. June ,  Ellen Pure ,  Liang-Chuan Wang ,  Steven Albelda ,  John Scholler

IPC分类号： C07K16/28 ,  C07K16/40 ,  C07K16/18 ,  A61K39/00 ,  A61K35/17 ,  C07K14/725 ,  C07K14/705 ,  C07K16/30

CPC分类号： C07K16/40 ,  A61K35/17 ,  A61K39/0011 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70521 ,  C07K14/70578 ,  C07K16/18 ,  C07K16/30 ,  C07K2317/00 ,  C07K2317/24 ,  C07K2317/51 ,  C07K2317/515 ,  C07K2317/622 ,  C07K2317/73 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/30 ,  C07K2319/70 ,  C12N2710/10343

摘要： The present invention provides compositions and methods for treating cancer in a human. The invention relates to targeting the stromal cell population in a tumor microenvironment. For example, in one embodiment, the invention provides a composition that is targeted to fibroblast activation protein (FAP). The invention includes a chimeric antigen receptor (CAR) which comprises an anti-FAP domain, a transmembrane domain, and a CD3zeta signaling domain.

公开(公告)号：US20230244006A1

公开(公告)日：2023-08-03

申请号：US18051624

申请日：2022-11-01

申请人： The Trustees of the University of Pennsylvania

发明人： Daeyeon Lee ,  Baek Min Kim

IPC分类号： G02B1/18 ,  C08J7/06 ,  G02B1/00

CPC分类号： G02B1/18 ,  C08J7/06 ,  G02B1/005 ,  C08J2325/06

摘要： Polymer-infiltrated nanoparticle films (PINFs) that have high volume fractions (>50 vol %) of nanoparticles (NPs) possess enhanced properties making them ideal for various applications. Capillary rise infiltration (CaRI) of polymer and solvent-driven infiltration of polymer (SIP) into pre-assembled NP films have emerged as versatile approaches to fabricate PINFs. Although these methods are ideal for fabricating PINFs with homogenous structure, several applications including separations and photonic/optical coatings would benefit from a method that enables scalable manufacturing of heterostructured (i.e., films with variation in structural properties such as porosity, composition, refractive indices etc.) PINFs. In this work, a new technique is developed for fabricating heterostructured PINFs with cavities based on CaRI. A bilayer composed of densely packed inorganic NP layer atop polymer NP layer is thermally annealed above the glass transition temperature of the polymer NP, which induces CaRI of the polymer into the interstices of the inorganic NP layer. Exploiting the difference in the sizes of the two particles, heterostructured double stack PINFs composed of a PINF and a layer with large cavities are produced at a moderate temperature (

公开(公告)号：US20230242612A1

公开(公告)日：2023-08-03

申请号：US18170695

申请日：2023-02-17

申请人： The Trustees of the University of Pennsylvania

发明人： Carl H. June ,  John Scholler ,  Avery D. Posey, Jr.

IPC分类号： C07K14/725 ,  C07K16/28 ,  C07K16/30 ,  C07K14/705 ,  C12N15/85

CPC分类号： C07K14/7051 ,  C07K16/2803 ,  C07K16/30 ,  C07K14/705 ,  C07K14/70507 ,  C12N15/85 ,  C07K2319/02 ,  C07K2319/00

摘要： The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell expressing a CAR having an antigen binding domain, a transmembrane domain, a CD2 signaling domain, and a CD3 zeta signaling domain. The invention also includes incorporating CD2 into the CAR to alter the cytokine production of CAR-T cells in both negative and positive directions.

公开(公告)号：US20230233709A1

公开(公告)日：2023-07-27

申请号：US18061633

申请日：2022-12-05

申请人： The Trustees of the University of Pennsylvania

发明人： Jean Bennett ,  Jeannette Bennicelli ,  Junwei Sun ,  Ji Yun Song ,  Sergei Nikonov

IPC分类号： A61K48/00 ,  A61P27/02 ,  C12N15/86

CPC分类号： A61K48/005 ,  A61P27/02 ,  C12N15/86 ,  A61K48/0075 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2800/22

摘要： Compositions and methods are provided for treating Leber congenital amaurosis (LCA) in a subject. In one aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding Lebercilin. In another aspect, Lebercilin has an amino acid sequence of SEQ ID NO: 1. In yet another aspect, the nucleic acid molecule has a sequence of SEQ ID NO: 3 or a variant thereof. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.

公开(公告)号：US11709351B2

公开(公告)日：2023-07-25

申请号：US16763158

申请日：2018-11-06

申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

发明人： Seyyed Salili ,  Douglas Durian ,  Matthew Harrington

IPC分类号： G02B21/00 ,  G02B21/06 ,  G02B21/36

CPC分类号： G02B21/06 ,  G02B21/0076 ,  G02B21/36

摘要： Systems and methods for passive multi-directional illumination in light-sheet fluorescence imaging and microscopy are disclosed herein. An elliptical light shaping diffuser is placed in the illumination path between the source of a light-sheet and the illuminated sample. The light-sheet is diffused anisotropically along two directions perpendicular to its propagation direction, eliminating stripe artifacts in obtained images. The method includes converting a light-sheet into an elliptically diffuse light-sheet by passing it through an elliptical light shaping diffuser, illuminating a sample with the elliptically diffuse light-sheet. The system includes a light-sheet source, an elliptical light shaping diffuser adapted to convert the light-sheet into an elliptically diffuse light-sheet to illuminate the sample, typical microscopy optics and lenses, and image capturing elements.

公开(公告)号：US11701526B2

公开(公告)日：2023-07-18

申请号：US16818683

申请日：2020-03-13

申请人： The Trustees of the University of Pennsylvania

发明人： Alejandro Carabe-Fernandez ,  Alejandro Bertolet Reina

IPC分类号： A61N5/10 ,  G16H20/40 ,  G16H50/50

CPC分类号： A61N5/1071 ,  A61N5/1031 ,  G16H20/40 ,  G16H50/50 ,  A61N2005/1034 ,  A61N2005/1087

摘要： Methods and systems are described for determining quantities of interest for particle therapy. An example method can comprise determining one or more first functions indicative of spectral fluence associated with a particle therapy. The one or more first functions can be based on a plurality of simulations of a particle beam. The method can comprise determining, based on the one or more first functions and a second function associated with calculating a quantity, data indicative of the quantity. The method can comprise causing output of the data indicative of the quantity.

公开(公告)号：US20230210941A1

公开(公告)日：2023-07-06

申请号：US17998330

申请日：2021-05-11

申请人： The Trustees of the University of Pennsylvania

发明人： James M. Wilson ,  Juliette Hordeaux ,  Nathan Katz

IPC分类号： A61K38/16 ,  C12N15/86 ,  C12N9/24 ,  A61K9/00

CPC分类号： A61K38/162 ,  A61K9/0085 ,  C12N9/2402 ,  C12N15/86 ,  C12Y302/01046 ,  C12N2750/14122 ,  C12N2750/14143

摘要： A pharmaceutical composition formulated for delivery of a recombinant adeno-associated virus (rAAV) vector comprising an AAV capsid and a vector genome having human galactosylceramidase (GALC) coding sequence is provided. Also provided are 5 methods and uses of a pharmaceutical composition comprising a rAAV for the treatment of Krabbe disease.