公开(公告)号：US09803217B2

公开(公告)日：2017-10-31

申请号：US14881960

申请日：2015-10-13

Applicant: Novartis AG

Inventor： Vivian Choi ,  Chad Eric Bigelow ,  Thaddeus Peter Dryja ,  Seshidhar Reddy Police

IPC: C12N15/63 ,  C07H21/04 ,  C12N15/86 ,  A61K48/00 ,  A61K38/17 ,  C07K14/47 ,  C12N7/00 ,  C12N15/79

CPC classification number: C12N15/86 ,  A61K38/1709 ,  A61K48/005 ,  A61K48/0058 ,  C07H21/04 ,  C07K14/47 ,  C12N7/00 ,  C12N15/79 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2750/14343 ,  C12N2750/14345 ,  C12N2750/14371 ,  C12N2800/22 ,  C12N2810/6027 ,  C12N2810/85

Abstract: The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.

公开(公告)号：US10550404B2

公开(公告)日：2020-02-04

申请号：US15713021

申请日：2017-09-22

Applicant: Novartis AG

Inventor： Vivian Choi ,  Chad Eric Bigelow ,  Thaddeus Peter Dryja ,  Seshidhar Reddy Police

IPC: A61K31/70 ,  C12N15/63 ,  C07H21/04 ,  C12N15/86 ,  A61K48/00 ,  A61K38/17 ,  C07K14/47 ,  C12N7/00 ,  C12N15/79

Abstract: The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.

公开(公告)号：US10537648B2

公开(公告)日：2020-01-21

申请号：US15321025

申请日：2015-06-24

Applicant: NOVARTIS AG

Inventor： Joy Ghosh ,  Thaddeus Peter Dryja ,  Michael Roguska ,  Eric C. Carlson

IPC: A61K51/08 ,  A61K49/00 ,  A61K9/00

Abstract: The invention provides compositions for rendering a vitreous cavity visible during a surgical procedure to alleviate a structural disorder caused by the vitreous in an eye, and methods of using the compositions. The compositions are vitreous delineating agents that comprise a hyaluronan binding peptide linked to an optically detectable moiety. Such compositions can be in a formulation that may be a solution, a suspension, or an emulsion, and would be injected into the vitreous shortly prior to use. The composition may additionally contain a therapeutic agent, a diagnostic agent, or a chemosensing material, in use, the composition marks or delineates the vitreous by binding preferentially to the hyaluronan that permeates the vitreous humor and binding little or not at all to surrounding tissues such as the retina. The interface between the labelled vitreous humor and the non-labelled surrounding vital tissues produces a visible signal, thereby allowing a surgeon to clearly visualize the entire vitreous cavity and distinguish it from vital ocular structures. Use of the method improves the accuracy and safety of a vitrectomy and thus prevents suboptimal outcomes or the need for repeated procedures. The compositions comprising chemosensing material are useful as long-lasting biosensors, which when used in the vitreous enable repetitive, non-invasive, in vivo measurements of metabolites or pharmacologic agents in the vitreous of animals or humans.