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Pioneer

Curative  Therapies

Therapies proven to be safe and effective in clinical trials are great first steps to lower the risks of relapse and improve lives.  GJCF is proud to have played a leading role for this goal.  And there is more to do.  Beyond these breakthroughs, GJCF is now accelerating toward cures.  It will take two bold steps to cure NMOSD, MOGAD and related autoimmune diseases once and for all:  1) Tolerization to reset the immune system so that it no longer targets self tissues; and 2) Regeneration to restore functions of tissues damaged by previous relapses.  If we are not aiming for cures, we are not aiming high enough.

 

A decade ago, many believed that approved therapies would never be possible for a disease as rare as NMO.  We believed otherwise.  Guided by the revolutionary GJCF blueprint, there are now three therapies approved, and clinical trials are testing next-generation treatments for anti-AQP4, anti-MOG (MOGAD) and seronegative disease.  Today, many believe that cures for NMOSD, MOGAD and other autoimmune diseases will never be possible.  We believe otherwise.

Restore Immune Tolerance to End NMOSD/MOGAD Disease

The immune system protects the human body against many types of potential threats, including infection and cancer.  It is also a key to tissue repair, healing and growth.  To achieve these vital roles, the immune system must distinguish between healthy "self" tissue and unhealthy tissues, infection and cancer.  This immune system superpower is called immune tolerance.

The immune system provides immunity to infection and cancer, and supports healthy tissue growth & repair.  To do so it must constantly monitor all tissues in the body and respond in beneficial ways.  A key to this role is the ability to tell the difference between healthy cells and molecules, and those that are abnormal.  This capability is called immune tolerance.  In some people, immune tolerance breaks down to certain proteins (such as AQP4 or MOG) over time.  As a result, the immune system mistakes these proteins as abnormal, and targets inflammatory responses against them and the cells that express them (such as astrocytes and oligodendrocytes).

Loss of Immune Tolerance

For reasons that are not clearly understood, over time the immune system can mistake healthy "self" tissue as if it were unhealthy or foreign.  This process results in the loss of immune tolerance.  In NMOSD or MOGAD (MOG Antibody Disease), a loss of immune tolerance occurs to specific self proteins, AQP4 or MOG (Myelin oligodendrocyte glycoprotein).  As a result, the immune system sees cells displaying such proteins as foreign, and injures them.  Loss of immune tolerance is at the core of NMOSD and other autoimmune diseases.  Solving immune tolerance in one autoimmune disease is likely to unlock to door to solving all autoimmune diseases!

Restoring Immune Tolerance

Immune tolerization represents the process of restoring immune tolerance. Tolerization re-educates immune system cells so they no longer see self tissues as threats, turning off autoimmune activity. The immune cells most important in tolerization are T and B cells along with other antigen presenting cells.  If immune tolerance is restored immune suppressive therapies are not necessary, and the disease process is stopped.  This is our goal.  GJCF is working with academic, biotech and pharma partners on amazing new science that holds great promise to restore immune tolerance for cures.

 

GJCF is planning its 3rd Summit on NMOSD Tolerization in January of 2023.

Click here to learn more about this field-leading event.

Roadmap to Immune Tolerizing Cures.

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GJCF is Cureageous.™

Curing diseases as complex as NMOSD or MOGAD is not easy.  But if we do not aim for cures we are not aiming high enough.  As with the first approved therapies, GJCF is leading the charge for cures - we are Cureageous™!  Join our mission to cure NMOSD & MOGAD.

GJCF is iNMOtion to Cures

Bold new science, technology and clinical trials are already beginning to explore ways to restore immune tolerance in NMOSD or MOGAD. A few examples are shown here. Learn more about the GJCF mission for cures!

  • Capsules delivering autoantigens to immune system
  • Goal: help immune system to ignore AQP4 or MOG

  • Inverse vaccines delivered through smart skin patch
  • Goal: turn immune system down to AQP4 or MOG

  • Nanoparticles delivering novel tolerizing medicines
  • Goal: program immune tolerance to AQP4 or MOG
  • Stem or autologous immune cell-based therapeutics
  • Goal: re-educate or destroy autoreactive T / B cells
  • Self red blood cells made to display AQP4 or MOG
  • Goal: use a natural tolerizing method of the body

 

  • AQP4 or MOG peptides designed as immune decoys
  • Goal: turn off autoimmunity versus AQP4 or MOG

 

GJCF led in catalyzing the first-ever approved therapies to dramatically reduce risks of relapses for patients with NMOSD. These breakthroughs are saving lives—and they transformed neurology.

Now, GJCF is once again at the forefront of life-changing advances for those facing NMOSD.
Discoveries driven by CIRCLES and other GJCF-funded research have enabled us to strive for cures.
This is a bold challenge—but if we are not aiming for cures—we are not aiming high enough.

Cures Take Courage. We Are Cureageous.™️

Applying its revolutionary blueprint, GJCF united experts, technologies and real-world experience to accelerate a biotech and pharma focus on NMOSD tolerization. Today, dozens of companies are developing tolerization therapies that may help cure NMOSD & end life-long immunosuppression.

Our road to tolerizing cures begins with NMOSD and MOGAD—but it could lead to cures for all autoimmune diseases. And technologies aimed at cures for type-1 diabetes, MS, lupus and other autoimmune diseases may help find cures for NMOSD and MOGAD. That is the power of rare.

GJCF is planning its 3rd Summit on NMOSD Tolerization in January of 2023.

Click here to learn more about this field-leading event.


Below are example biotech & pharma companies developing innovative tolerization technologies that are currently on the GJCF radar screen for their potential to help cure NMOSD & MOGAD.

Company

Technology

Mechanism

Publication (sample)

Stage

Aditxt

Apoptotic DNA Immunotherapy

Auto-Ag-targeted PCD of autoreactive APCs / Tc

Suppression of T1D using apoptotic DNA therapy

Pre-Clinical

Allero Therapeutics

Auto-Ag Targeted TBPs to Oromucosa

Tolerigenic bacterial particles (TBPs) target auto-Ag

Treg recruitment via oral mucosa in Celiac Dz

Pre-Clinical & IND

AnTolRx

Auto-Ag-Specific Tolerigenic NPs

Auto-Ag targeting immune modulating NP plateform

Tolerigenic NPs suppress CNS autoimmunity

Pre-Clinical

Anokion

Auto-Ag-Specific Hepato-RBC Vax

Liver-RBC reticuloendothelial inverse tolerigenic vaccine

Engineered Ags for RBC binding deletes ARTc

Phase I/II & Pre-Clinical

Apitope

[Worg Pharma]

Engineered Auto-Ag Decoy Apitopes

Tc anergizing & Tr1 / FoxP3+ Treg inducing apitopes

Apitopes for ImmunoTx in Autoimm Disease

Phase IIa

Barcelona GAEM

Autologous DC Tolerization Ex Vivo

Auto-Ag peptide-loading induces tolerigenic DC

Tolerigenic DC generated in MS & NMOSD

Phase Ib

BioNTech

Auto-Ag Specific Ψ mRNA Vax

Auto-Ag presentation w/o 2nd signal induces tolerization

Inverse mRNA vaccine to treat MOG-induced EAE

Pre-Clinical

Cellerys

[ Novartis ]

Auto-Ag Peptide Coupled RBC

Exploit tolerigenic systems in MHC-pathway RBC tolerance

TBD

Phase I/II

Cue Biopharma

Affinity-attenuated IL-2 Variant

Modulatory costimulatory ligands targeting ARTc

Modulated Ag-specific Tc by Peptide-HLA Tx

Pre-Clinical

Cour Pharma

PGLA-Auto-Ag Nanoparticle

Target hepatic APC for tolerigenic reprogramming

TAK-101 NPs induce

Ag-specific tolerance

Phase II

Diamyd Medical

Auto-Ag Specific

LN / Oral Vaccines

Intralymphatic vax targeting Auto-Ag / APC / Tc Crosstalk

GAD-alum immunoTx in HLA-DR3-DQ2 T1D

Phase I / II

EVOQ Therapeutics

[ Amgen ]

Auto-Ag-Targeted Nanodisc Vaccine

High-efficiency targeting of auto-Ag to APC for tolerization

High-density lipoprotein multifxnal nanoparticles

Phase I & Pre-Clinical

Idogen AB

Autologous Dc Tolerization Ex Vivo

Tolerigenic DC reprogramming for Auto-Ag specific tolerance

Patents

Phase IIa & Pre-Clinical

IgGenix, Inc.

High-affinity tolerigenic Abs

Chimeric IgE/IgG4 Ab confer tolerigenesis to specific Ag

High-affinity Ag-specific Ab from IgE Bc transcripts

Pre-Clinical

Imcyse

Thio-Imotope

Modulating Peptide

Imotope-specific CD4 Tc target Auto-Ag reactive APCs

Thioreductase epitopes inhibit T1D in NOD mouse

Phase II

IASO Biothera

Autologous & Allo CARTc Therapy

CARTc targeting of CD19+ Bc using chimeric-Ag receptors

Targeting Ag-specific CD19+ autoreactive Bc

Phase I

Medeor Therapeutics

CD34+ Donor HSC

CD34+ Allo-HSC chimerism to induce tolerigenic paradigm

Mixed chimeric tolerance post-immunosuppression

Phase III

Novo Nordisk

Novel plasmid DNA targeted immunoTx

Targeted auto-Ag expression for tolerigenesis induction

Learning across diseases for tolerigenic cures

Phase I

Nykode Therapeutics

Ag/APC-Specific Vaccibody Platform

Ab-Targeting of Auto-Ag/BcR to APCs for Tolerization

Ab/Auto-Ag Plasmid Targeted to APC

Phase I & Pre-Clinical

Parvus Therapeutics

[ Genentech ]

Navacims (Auto-Ag MHC Nanoparticle)

Navacim-generated auto-Ag specific Treg induction

Reprogramming ARTc to Tr1 for Autoimmune Tx

Pre-Clinical

Pfizer

Small molecule inhibitor program

Targeted inhibition of key inflammation pathways

Immunology & Inflammation Pipeline

Phase I, II and III

Rubius Therapeutics

Modify Autologous RBC with Target Auto-Ag

Auto-Ag decorated RBCs for tolerigenic induction

Induction of Ag Specific Tolerance in T1D

Pre-Clinical

Selecta Bioscience

Auto-Ag specific ImmTOR NPs

ImmTOR nanoparticles target APCs induces Ag-specific Treg

Induction of Tolerigenic APCs by ImmTOR NPs

Phase I & Pre-Clinical

SQZ Biotech

Modify Autologous RBC with Auto-Ag

Multi-modal tolerigenesis via MHC-pathway RBC tolerance

Engineered RBC Auto-Ag Specific Tolerance

Pre-Clinical

Talaris Therapeutics

Facilitated FCR001 Allo-HSC Transplant

CD34+ / ab Tc-facilitated allogenic HSC transplant

Tolerance Induction by Facilitated HLA Chimera

Phase I & II

Toleranzia

Auto-Ag specific tolerogen peptides

Auto-Ag targeted tolerogens induce Treg tolerization

TBD

Pre-Clinical

Topas Therapeutics

[ Eli Lily ]

NP-Targeted

Auto-Ag Therapy

Liver sinusoidal endothelial cell targeted auto-Ag nanoparticle

Nanoparticle Targeting of Auto-Ag Inhibits CD8 Tc

Phase I & Pre-Clinical

Cures Take Courage . We Are Cureageous. TM

Note: information subject to change; see specific company website. Bracketed font denotes the parental or partner company.

Glossary

Ab, antibody; Ag, antigen; Allo-HSC, allogenic (donated) human stem cell; APC, antigen presenting cell; ARBc, autoreactive B cell; ARTc, autoreactive T cell; Auto-Ag, auto-antigen; Bc, B cell; CAR-Tc, chimeric antigen receptor T cell; CAAR-Tc, chimeric auto-antigen receptor T cell; CD4 Tc, CD4+ T cell; CD8 Tc, CD8+ T cell; CD19+, CD19 marker of B cells; Dc, dendritic cell; Dz, disease; DNA, deoxyribonucleic acid; EAE, experimental autoimmune encephalomyelitis; HLA, human leukocyte antigen; HSC, human stem cell; IgE, immunoglobulin E class; IgG, immunoglobulin G class; IL-2, interleukin-2; LN, lymph node; mRNA, messenger ribonucleic acid; MHC, major histocompatibility complex; MS, multiple sclerosis; NPs, nanoparticles; PCD, programmed cell death (apoptosis); RBC, red blood cell; RNA, ribonucleic acid; Tc, T cell; Tr1, Tr1 regulatory T cell; Treg, regulatory T cell; Tx, therapy; Vax, vaccine

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