University of California

University of Pittsburgh

Centre National De La Recherche Scientifique

University of Pennsylvania

Institut National De La Sante Et De La Recherche Medicale

Université Pierre Et Marie Curie (paris 6)

Inserm

Leah C Byrne

David V Schaffer

James T Koerber

John G Flannery

Junwei Sun

Thierry Leveillard

Timothy P Day

Meike Visel

Ryan R Klimczak

Morgan V Fedorchak

José-Alain Sahel

Xin Mei

Emmanuelle Clerin-Lachapelle

Ram Fridlich

Bilge Esin Ozturk

Deniz Dalkara Mourot

Thérèse Cronin

Jeanette Bennicelli

William A Beltran

Jean Bennett

Disclosed herein are embodiments of a polymer-based implant and methods of making and using the same. The polymer-based implant comprises a polymer component and a therapeutic agent. In some embodiments, the polymer-based implant can be used to treat and/or prevent retinal diseases and/or retinopathies. The polymer-based implant exhibits physical properties that provide the ability to safely place the polymer-based implant in an ocular region without undesired diffusion and also to allow for controlled and timely release of the therapeutic agent to a desired region of the ocular region, such as the retina. In particular disclosed embodiments, the polymer-based implant can be used for safe and effective gene therapy.

Polymer-based implant for retinal therapy and methods of making and using the same

In one embodiment, the invention provides an Adeno-Associated Virus (AAV) comprising an exterior surface, which surface comprises one or more peptide tags that form a bond with a binding-partner, wherein the AAV is a live virus. In another embodiment, the invention provides a conjugate comprising at least one such AAV and at least one polypeptide comprising a first domain which is the binding-partner for the tag and a second domain, which is a bioactive polypeptide. In another embodiment, the invention provides a conjugate comprising at least one such AAV (first AAV) and at least one second AAV, which second AAV comprises a second exterior surface, which second exterior surface comprises at least one binding-partner for the tag or for a third linker molecule, wherein the at least one first AAV and the at least one second AAV are bound.

Conjugates comprising AAVs and CAS9 polypeptides

The present disclosure provides recombinant adeno-associated virus (AAV) virions with altered capsid protein, where the recombinant AAV (rAAV) virions exhibit greater ability to cross barriers between intravitreal fluid and retinal cells, and thus greater infectivity of a retinal cell compared to wild-type AAV, and where the rAAV virions comprise a heterologous nucleic acid. The present disclosure provides methods of delivering a gene product to a retinal cell in an individual.

Compositions and methods of treating ocular diseases

Growing community of inventors

San Francisco, CA, United States of America

Leah C Byrne