Interius Biotherapeutics, Inc.

Intergalactic Therapeutics, Inc.

University of Pennsylvania

Ascidian Therapeutics, Inc.

Intergalactic Therapeutics, Inc. B

Philip R Johnson

Bruce C Schnepp

Ronnie M Russell

Jean Bennett

Junwei Sun

Krishna J Fisher

Scott J Dooley

Dominick Cerce

David Johnson

The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.

Trans-splicing molecules

Provided for herein are mutant VSV-G polypeptides, compositions comprising the same, and methods of using the same. Also provided for herein are polypeptides and compositions that bind to CD7 and uses thereof. Also provided for herein are polypeptides and compositions that bind to CD8 and uses thereof.

Pseudotyped viral particles, compositions comprising the same, and uses thereof

Provided for herein are viral particles comprising a heterologous viral glycoprotein and a targeting moiety, wherein the targeting moiety comprises a polypeptide comprising a formula of T-S, wherein T is a target binding domain and Sis a stalk portion. The stalk portion may comprise a variant Fe domain. The stalk portion may comprise a flexible polypeptide domain. The targeting moiety comprising the formula T-Smay be incorporated into a viral particle to assist with targeting such particles to a specific cell type. Also provided for herein are compositions comprising the same, and methods of using the same.

Mutated polypeptides, compositions comprising the same, and uses thereof

Provided herein are isolated DNA vectors comprising a heterologous gene, wherein the DNA vector is devoid of bacterial plasmid DNA and/or bacterial signatures, which can abrogate persistence in vivo. The invention also features pharmaceutical compositions (non-immunogenic pharmaceutical compositions) including the DNA vectors of the invention, which can be used for induction of long-term, episomal expression of a heterologous gene in a subject. The invention involves methods of treating a subject by administering the DNA vectors of the invention, including methods of treating disorders associated with a defect in a target gene.

Synthetic DNA vectors and methods of use

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Bryn Mawr, PA, United States of America

Philip R Johnson