Andrew W Mercer

US Patent:

20220251567, Aug 11, 2022

Filed:

Jul 10, 2020

Appl. No.:

17/597351

Inventors:

- Paris, FR
- Marseille, FR
- Bordeaux, FR
- Rockville MD, US
Angélique PERET - Marseille, FR
Olivier DANOS - Rockville MD, US
Andrew MERCER - Rockville MD, US

International Classification:

C12N 15/113
A61P 25/08
C12N 7/00
C12N 15/86

Abstract:

The present disclosure relates to gene therapy targeting GluK2 subunit that can be used to inhibit epileptiform discharges. Short interfering RNA sequences against the human Grik2 gene sequence are described which are efficient in decreasing the expression of GluK2-containing KARs in neurons engineered to express the equivalent shRNA or miRNA. Using a tissue culture model of TLE, the examples remarkably demonstrate that viral expression of shRNA or miRNA inhibits the frequency of epileptiform discharges. Therefore, RNA therapeutics aimed at decreasing the expression of GluK2-containing KARs in neurons can remarkably prevent spontaneous epileptiform discharges in TLE. In particular, the present disclosure relates to a recombinant antisense oligonucleotide that targets a Grik2 mRNA. The present disclosure also relates to a method for treating epilepsy in a subject in need thereof, wherein the method comprises: administering an effective amount of a vector comprising an oligonucleotide encoding an inhibitory RNA that binds (e.g., hybridizes) specifically to Grik2 mRNA and inhibits expression of Grik2 in the subject.

US Patent:

20220186256, Jun 16, 2022

Filed:

Apr 2, 2020

Appl. No.:

17/600418

Inventors:

- Rockville MD, US
Ye Liu - Clarksville MD, US
Andrew Mercer - Poolesville MD, US
Samantha Yost - Rockville MD, US
Elad Firnberg - Baltimore MD, US

International Classification:

C12N 15/86
C07K 14/005

Abstract:

The present invention relates to recombinant adeno-associated viruses (rAAVs) having capsid proteins engineered to include amino acid sequences that confer and/or enhance desired properties. In particular, the invention provides engineered capsid proteins comprising peptide insertions from heterologous proteins inserted within or near variable region IV (VR-IV) of the virus capsid, such that the insertion is surface exposed on the AAV particle. The invention also provides capsid proteins that direct rAAVs to target tissues, in particular, capsid proteins comprising peptides derived from erythropoietin or dynein that are inserted into surface-exposed variable regions and that target rAAVs to retinal tissue and/or neural tissue, including the central nervous system, and deliver therapeutics for treating neurological and/or eye disorders.

US Patent:

20210393802, Dec 23, 2021

Filed:

Nov 13, 2019

Appl. No.:

17/292750

Inventors:

- Rockville MD, US
Subha Karumuthil Melethil - Germantown MD, US
Matthieu Pierre Guibert - Germantown MD, US
Andrew Christopher Mercer - Poolesville MD, US
Micheal James Carrell - Mt. Airy MD, US
Robert Thomas Stadelman - New Market MD, US
Franz Michael Gerner - Myersville MD, US
Nicholas Alexander Piers Sascha Buss - Rockville MD, US
Jared Bee - Gaithersburg MD, US
Tristan James Marshall - Potomac MD, US
Roberto DePaz - Bethesda MD, US

International Classification:

A61K 48/00
A61P 25/00

Abstract:

Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5′ inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3′ ITR. Also provided herein are methods of treating Batten disease comprising administering to a subject in need thereof the rAAV described herein via more than one route. Also provide herein are pharmaceutical compositions comprising the rAAV described herein and related methods of treating Batten disease.

US Patent:

20210147854, May 20, 2021

Filed:

Sep 24, 2020

Appl. No.:

17/031149

Inventors:

- La Jolla CA, US
Andrew Mercer - Poolesville MD, US
Brian M. Lamb - San Diego CA, US
Thomas Gaj - Solana Beach CA, US

International Classification:

C12N 15/62
C12N 9/16
C12N 9/22

Abstract:

Disclosed herein are chimeric polypeptides, including compositions thereof, expression vectors, and methods of use thereof, for the generation of transgenic cells, tissues, plants, and animals. The compositions, vectors, and methods of the present invention are also useful in gene therapy techniques.

US Patent:

20180230474, Aug 16, 2018

Filed:

Jan 23, 2018

Appl. No.:

15/878043

Inventors:

- La Jolla CA, US
Andrew Mercer - Poolesville MD, US
Brian M. Lamb - San Diego CA, US
Thomas Gaj - Solana Beach CA, US

International Classification:

C12N 15/62
C12N 9/22
C12N 9/16

Abstract:

Disclosed herein are chimeric polypeptides, including compositions thereof, expression vectors, and methods of use thereof, for the generation of transgenic cells, tissues, plants, and animals. The compositions, vectors, and methods of the present invention are also useful in gene therapy techniques.

US Patent:

20150284728, Oct 8, 2015

Filed:

Sep 4, 2013

Appl. No.:

14/425944

Inventors:

- La Jolla CA, US
Andrew Mercer - Poolesville MD, US
Brian M. Lamb - San Diego CA, US
Thomas Gaj - Solana Beach CA, US

International Classification:

C12N 15/62

Abstract:

Disclosed herein are chimeric polypeptides, including compositions thereof, expression vectors, and methods of use thereof, for the generation of transgenic cells, tissues, plants, and animals. The compositions, vectors, and methods of the present invention are also useful in gene therapy techniques. The invention provides a chimeric polypeptide. The polypeptide includes: a) a recombinase, nuclease or transcription factor, or fragment thereof; and b) a transcription activator-like effector (TALE) protein.