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EPITOR THERAPEUTICS, INC.

EPITOR THERAPEUTICS, INC.

Company Details

Name:	EPITOR THERAPEUTICS, INC.
Jurisdiction:	New York
Legal type:	FOREIGN BUSINESS CORPORATION
Status:	Active
Date of registration:	16 Jun 2023 (2 years ago)
Entity Number:	6880660
ZIP code:	10021
County:	New York
Place of Formation:	Delaware
Foreign Legal Name:	EPITOR THERAPEUTICS, INC.
Address:	4 east 70th st.,, apt. 9a, NEW YORK, NY, United States, 10021

DOS Process Agent

Name	Role	Address
the corporation	DOS Process Agent	4 east 70th st.,, apt. 9a, NEW YORK, NY, United States, 10021

Form 5500 Series

Employer Identification Number (EIN):

923401493

Plan Year:

2023

Number Of Participants:

Sponsors Telephone Number:

3474954416

File:

View File

Filings

Filing Number	Date Filed	Type	Effective Date
230620002675	2023-06-16	APPLICATION OF AUTHORITY	2023-06-16

USAspending Awards / Financial Assistance

Date:

2024-09-25

Link:

View on USAspending

Awarding Agency Name:

Department of Health and Human Services

Transaction Description:

APPLICATION OF A NOVEL DCASNANO-BASED EPIGENETIC EDITING TO TREAT RETT SYNDROME - ABSTRACT RETT SYNDROME IS A DEVASTATING NEURODEVELOPMENTAL DISEASE CAUSED BY LOSS OF FUNCTION MUTATIONS OF THE MECP2 GENE, LOCATED ON THE X CHROMOSOME. AFFECTING NEARLY 20,000 GIRLS IN THE US AND EU, DISEASE MANIFESTATION IS DUE TO NEURONAL MOSAICISM CAUSED BY RANDOM X INACTIVATION: HALF RETT PATIENT NEURONS EXPRESS THE WILD TYPE (WT) GENE ON THE ACTIVE X CHROMOSOME (XA), WHILE THE OTHER HALF EXPRESS THE MUTATED (MT) MECP2 GENE, WITH THE WT MECP2 SILENCED ON THE INACTIVE X CHROMOSOME (XI). EPIGENOME EDITING USING CRISPR SYSTEMS IS A POTENTIAL TARGETED THERAPY ALLOWING REACTIVATION OF THE SILENCED BUT FUNCTIONAL MECP2 GENE ON XI. THE EPIGENETIC EDITOR IS COMPRISED OF A CATALYTICALLY DEAD CAS (DCAS) FUSED WITH EFFECTOR PROTEINS, SUCH AS THE DNA DEMETHYLASE TEN-ELEVEN TRANSLOCATION (TET) PROTEIN TO DEMETHYLATE AND THEREBY ACTIVATE TARGET GENES. EPITOR THERAPEUTICS IS DEVELOPING NOVEL EPIGENOME EDITING TOOLS TO TREAT RETT SYNDROME. OUR EDITOR COMBINES CASNANO, AN ULTRACOMPACT CRISPR PROTEIN RETAINING ROBUST DNA TARGETING, FUSED TO TETMINI, A MINIATURE YET POTENT TET DEMETHYLASE DOMAIN. COMPACT ENOUGH TO PACKAGE INTO A SINGLE ADENO- ASSOCIATED VIRUS (AAV), THIS EPIGENETIC EDITOR ENABLES TARGETED DELIVERY OF EPIGENOME EDITING MACHINERY TO THE BRAIN. OUR PROPOSED STRATEGY INCLUDES OPTIMIZING THE EPIGENETIC EDITOR COMPONENTS FOR DNA TARGETING ABILITY AND SPECIFICITY (AIM 1); ASSESSING IN VITRO TARGET VALIDATION IN RETT PATIENT IPSC-DERIVED NEURONS AND OFF-TARGET ANALYSIS IN HUMAN EMBRYONIC STEM CELLS (AIM 2); AND ASSESSING PHARMACOLOGY AND PHENOTYPIC EFFICACY OF THE AAV9 ENCAPSULATING DCASNANO-TETMINI EPIGENETIC EDITORS IN A RETT SYNDROME, FEMALE MECP2-HUMANIZED MOUSE MODEL (AIM 3). IF SUCCESSFUL, THIS WOULD REPRESENT THE FIRST EPIGENETIC EDITING TREATMENT FOR DISORDERS OF THE CNS, OVERCOMING KEY DELIVERY BARRIERS THAT HAVE BLOCKED DEVELOPMENT OF TARGETED EPIGENETIC THERAPIES.

Obligated Amount:

698459.00

Face Value Of Loan:

0.00

Total Face Value Of Loan:

0.00

Date of last update: 20 Mar 2025

Sources: New York Secretary of State