* Replaced mRNA-1706 in Sept. 2017
Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Influenza A virus subtype H10N8
Infectious Diseases
mRNA-1440
Lead
Moderna
Indication/ Target
Influenza H10
Formulation
In Licensed
GLP Toxicology
Yes
IND/CTA Filed
Yes
Phase 1
Fully enrolled
Phase 2
n/a
Funding
See More Info
About the Disease
Influenza, also known as the flu, is a contagious respiratory illness caused by influenza viruses. Two influenza A virus subtypes, H1N1 and H3N2, are currently in general circulation among people. A pandemic, or global outbreak, occurs when there is a genetic shift that causes a new influenza A virus to emerge and spread rapidly because people have little or no immunity to the new strain.
Our influenza vaccine program is focused on two influenza A virus strains, both with pandemic potential: H10N8 (mRNA 1440) and H7N9 (mRNA 1851).
Influenza A viruses are classified by subtypes based on two proteins on the virus’s surface: hemagglutinin (HA) and neuraminidase (NA). The H10N8 virus has the viral antigenic proteins H10 and NA 8.
Of our two pandemic flu vaccine programs, H10N8 has currently only infected three people in China in 2013 although two of them died. If the H10N8 was to become a pandemic the world would not currently have a vaccine.
Source: Centers for Disease Control and Prevention
Our H10N8 mRNA Vaccine (mRNA-1440)
Our investigational H10N8 vaccine is being developed to deliver mRNA encoding for the membrane-bound hemagglutinin 10 (H10) protein. The mRNA directs the cells to produce and express H10 on the cell surface, exactly as a native infection would do but without the ability to cause disease or spread. As a result the body’s immune system responds as if the actual virus is present. The immune system produces antibodies that have the potential to neutralize the virus and prevent infections in the event the vaccinated person is exposed to the actual H10N8 virus in the future.
Moderna strategically selected the H10N8 vaccine as one of its first two clinical development programs (the other, an H7N9 vaccine – mRNA-1851) in order to rapidly assess both the safety and efficacy of its mRNA platform in humans.
Because this strain is not circulating in the general population in Germany, where the trial is taking place, Moderna is able to study the efficacy of its vaccine technology in a naïve patient population. Antibodies present in subjects’ blood after treatment with mRNA-1851 are likely attributed to Moderna’s vaccine and not to active immunity as a result of previous exposure to the virus.
Studying mRNA vaccines for Influenza strains allows Moderna to measure vaccine efficacy against a well-understood endpoint, the hemagglutination inhibition assay, or HAI. HAI is used by U.S. FDA and World Health Organization (WHO) to measure how well antibodies bind to and inactivate an Influenza virus. Vaccines demonstrating titers of 1:40 are considered effective in reducing the risk for Influenza infection and are, thus, approved as seasonal flu vaccines.
Clinical Development
- Phase 1 dose-escalating, placebo-controlled, immunogenicity and safety trial in healthy adult volunteers
- Completed enrollment, with a total of 201 subjects enrolled. The study remains active, with subjects continuing to be followed
- Moderna plans to publish topline study findings in 2017 and complete findings in 2018 upon completion of the study and full data analysis
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mRNA-1851
Lead
Moderna
Indication/ Target
Influenza H7
Formulation
In Licensed
GLP Toxicology
Yes
IND/CTA Filed
Yes
Phase 1
Fully enrolled
Phase 2
n/a
Funding
See More Info
Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Influenza A virus subtype H7N9
About the Disease
Influenza, also known as the flu, is a contagious respiratory illness caused by influenza viruses. Two influenza A virus subtypes, H1N1 and H3N2, are currently in general circulation among people. A pandemic, or global outbreak, occurs when there is a genetic shift that causes a new influenza A virus to emerge and spread rapidly because people have little or no immunity to the new strain.
Our influenza vaccine program is focused on two influenza A virus strains, both with pandemic potential: H10N8 (mRNA-1440) and H7N9 (mRNA-1851).
Influenza A viruses are classified by subtypes based on two proteins on the virus’s surface: hemagglutinin (HA) and neuraminidase (NA). The H7N9 virus has the viral antigenic proteins H7 and NA 9.
H7N9 has a high potential of becoming a pandemic. More than 600 cases have been reported, to date, in China, with a mortality rate of approximately one in three infected people.
Source: Centers for Disease Control and Prevention
Our H7N9 mRNA Vaccine (mRNA-1851)
Our investigational H7N9 vaccine is being developed to deliver mRNA encoding for the membrane-bound hemagglutinin 7 (H7) protein. The mRNA directs the cells to produce and express H7 on the cell surface, much like a native infection would do but without the ability to cause disease and spread. As a results, the body’s immune system responds as if the actual virus is present. The immune system produces antibodies that have the potential to neutralize the virus and prevent infections in the event the vaccinated person is exposed to the actual H7N9 virus in the future.
Moderna strategically selected the H7N9 vaccine as one of its first two clinical development programs (the other, an H10N8 vaccine – mRNA-1440) in order to rapidly assess both the safety and efficacy of its mRNA platform in humans.
Because this strain is not circulating in the general population in the U.S., where the trial is taking place, Moderna is able to study the efficacy of its vaccine technology in a naïve patient population. Antibodies present in subjects’ blood after treatment with mRNA-1851 are likely attributed to Moderna’s vaccine and not to active immunity as a result of previous exposure to the virus.
Studying mRNA vaccines for Influenza strains allows Moderna to measure vaccine efficacy against a well-understood endpoint, the hemagglutination inhibition assay, or HAI. HAI is used by U.S. FDA and World Health Organization (WHO) to measure how well antibodies bind to and inactivate an Influenza virus. Vaccines demonstrating titers of 1:40 are considered effective in reducing the risk for Influenza infection and are, thus, approved as seasonal flu vaccines.
Clinical Development
A Phase 1 study of healthy volunteers is underway in the U.S., with 104 healthy volunteers dosed to date.
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mRNA MRK-1777
Lead
Merck
Indication/ Target
Undisclosed
Formulation
In Licensed
GLP Toxicology
Yes
IND/CTA Filed
Yes
Phase 1
Started: Nov ‘16
Phase 2
Funding
See More Info
Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Undisclosed
Partnered Program; Led by Merck
A Phase 1 clinical study is underway for mRNA MRK-1777, an mRNA-based vaccine for an undisclosed infectious disease.
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mRNA-1388
Lead
Moderna
Indication/ Target
Chikungunya
Formulation
In Licensed
GLP Toxicology
Yes
IND/CTA Filed
Yes
Phase 1
Started: Aug '17
Phase 2
Funding
DARPA
See More Info
Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Chikungunya Virus
About the Disease
Chikungunya virus, or CHIKV, is a type of alphavirus and is transmitted by mosquitos.
It typically causes mild fever and transient, or fleeting, joint pain. However, in approximately 15 percent of infected patients it can cause long-term, severe arthritis. CHIKV has historically has been limited to warmer climates in Asia and Africa, but recent cases have been identified in the Americas and Europe. This is likely due to an increase in global travel, as well as the onset of transmission by the Aedes albopictus mosquito. Previously, the Aedes aegypti mosquito typically transmitted the disease.
There are no treatment options or approved vaccines for CHIKV. Management of the infection from the virus has been limited to supportive care and avoidance of mosquito bites.
Our CHIKV mRNA Vaccine (mRNA-1388)
Our CHIKV vaccine delivers mRNA encoding for viral antigenic proteins associated with CHIKV. The mRNA directs cells to produce and express the proteins, closely mimicking a native viral infection and tricking the body into thinking the actual virus is present. The immune system produces antibodies against the viral antigenic proteins.
If the person is exposed to the actual Chikungunya virus in the future, the body recognizes it as foreign. Antibodies target the viral antigenic proteins, activating a robust immune response to target and destroy the Chikungunya virus, so it cannot cause an infection.
Clinical Development
GLP toxicology studies have been completed and an IND filed with the U.S. Food & Drug Administration.
A Phase 1 study is anticipated to begin enrolling healthy volunteers in 2017.
Funding
In 2015, Moderna extended an existing grant from the Defense Advanced Research Projects Agency (DARPA) to finance preclinical toxicology studies and the Phase I study of mRNA-1388. The extension builds on an original grant announced in 2013 to develop antibody-producing drugs to protect against a wide range of known and unknown emerging infectious diseases and engineered biological threats.
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mRNA-1325
Lead
Moderna
Indication/ Target
Zika
Formulation
In Licensed
GLP Toxicology
Yes
IND/CTA Filed
Yes
Phase 1
Started: Dec ‘16
Phase 2
Funding
DARPA, BARDA
See More Info
Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Zika Virus
About the Disease
Zika is a member of the flavivirus family, which also includes viruses such as Dengue Fever and Yellow Fever. A rapidly emerging pandemic with the potential for long-term health implications. Zika is transmitted by the Aedes mosquito and mother to fetus transmission as well as sexual transmission have also been confirmed. There is a causal link between Zika infection and at least two serious diseases: microcephaly and Guillain-Barré Syndrome (GBS).
Children born to mothers infected with Zika can develop microcephaly, a severe disease characterized by abnormally small heads and severe neurologic disabilities. GBS is an autoimmune disease that attacks the peripheral nervous system, leading to rapidly progressive and potentially life-threatening muscle weakness. GBS can lead to death caused by respiratory arrest if a patient is not ventilated.
As of November 2016, 48 countries and territories in the Americas have confirmed autochthonous, vector-borne transmission of the Zika virus, and five countries in the Americas have reported sexually transmitted Zika cases. 184 locally transmitted cases have been reported in South Florida, and 34,000 locally transmitted cases throughout the U.S. and its territories, with most cases in Puerto Rico.
There are no treatment options or approved vaccines for the Zika virus.
Our Zika mRNA Vaccine (mRNA-1325)
Our investigational Zika vaccine, mRNA-1325, is being developed to deliver mRNA encoding for viral antigenic proteins associated with the Zika virus. The mRNA directs cells to produce and express the proteins on the cell surface, much like a native infection would do but without the ability to cause disease or spread. As a result the body’s immune system responds as if the actual virus is present. The immune system produces antibodies that have the potential to neutralize the virus and prevent infections in the event the vaccinated person is exposed to the actual Zika virus in the future.
Our mRNA platform, research engine and early development engine enabled us to advance our Zika vaccine from initial discovery concept to first-in-human dosing in 12 months.
mRNA-1325 is one of two Zika mRNA vaccines we are developing. mRNA-1325, the earlier of our two Zika mRNA programs, utilizes an in-licensed formulation, while our second investigational Zika mRNA vaccine, mRNA-1706, utilizes V1GL, one of our novel, proprietary formulations.
Clinical Development
A Phase 1/2 clinical study of mRNA-1325 began enrolling healthy volunteers in December 2016.
Funding
In 2016, Moderna received a funding award of up to $125 million from the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), to accelerate development of our Zika mRNA vaccine. Moderna’s preclinical work for mRNA-1325 was funded through a grant from the Defense Advanced Research Projects Agency (DARPA).
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mRNA-1893*
Lead
Moderna
Indication/ Target
Zika
Formulation
V1GL
GLP Toxicology
No
IND/CTA Filed
No
Phase 1
Phase 2
Funding
See More Info
Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Zika virus
About the Disease
Zika is a member of the flavivirus family, which also includes viruses such as Dengue Fever and Yellow Fever. A rapidly emerging pandemic with the potential for long-term health implications. Zika is transmitted by the Aedes mosquito and mother to fetus transmission as well as sexual transmission have also been confirmed. There is a causal link between Zika infection and at least two serious diseases: microcephaly and Guillain-Barré Syndrome (GBS).
Children born to mothers infected with Zika can develop microcephaly, a severe disease characterized by abnormally small heads and severe neurologic disabilities. GBS is an autoimmune disease that attacks the peripheral nervous system, leading to rapidly progressive and potentially life-threatening muscle weakness. GBS can lead to death caused by respiratory arrest if a patient is not ventilated.
As of November 2016, 48 countries and territories in the Americas have confirmed autochthonous, vector-borne transmission of the Zika virus, and five countries in the Americas have reported sexually transmitted Zika cases. 184 locally transmitted cases have been reported in South Florida, and 34,000 locally transmitted cases throughout the U.S. and its territories, with most cases in Puerto Rico.
There are no treatment options or approved vaccines for the Zika virus.
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mRNA-1647
Lead
Moderna
Indication/ Target
CMV
Formulation
V1GL
GLP Toxicology
Yes
IND/CTA Filed
No
Phase 1
Phase 2
Funding
See More Info
Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Cytomegalovirus
About the Disease
Cytomegalovirus, or CMV, is a member of the herpes virus family. Often a person will not have any symptoms of viral infection. CMV leads to severe disease in two populations: newborns and transplant patients.
CMV is the most common cause of newborn disability, leading to deafness, microcephaly (small, not fully developed heads), vision loss and neurologic disabilities, among other serious complications. It is also the most frequent viral disease in transplant recipients, often leading to transplant failure.
There are currently no approved vaccines for CMV.
Our CMV mRNA Vaccine (mRNA-1647)
Our investigational CMV vaccine in development, mRNA-1647, combines six mRNAs encoding for six viral proteins, including five proteins that comprise the CMV gH Pentamer complex (gH, gL, UL128, UL130 and UL131A), as well as another CMV antigen, the herpesvirus glycoprotein (gB) protein. The mRNAs direct cells in the body to produce and express all six proteins on the cell surface, including the expression of gB and a fully formed Pentamer complex, or five proteins expressed as a single antigen, much like a native infection would do but without the ability to cause disease or spread. As a result, the body’s immune system responds as if the actual virus in present. The immune system produces antibodies against the Pentamer and gB that have the potential to neutralize the virus and prevent infections in the event the vaccinated person is exposed to the actual CMV virus in the future.
mRNA-1647 has been nominated as a development candidate and is progressing to GLP toxicology studies.
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mRNA-1653
Lead
Moderna
Indication/ Target
HMPV/PIV3
Formulation
V1GL
GLP Toxicology
Yes
IND/CTA Filed
No
Phase 1
Phase 2
Funding
See More Info
Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Diseases: HMPV / PIV3
About the Diseases
Human Metapneumovirus (HMPV) and Parainfluenza Virus 3 (PIV3) are both part of the paramyxovirus family.
Most children have been infected at least once with both viruses by the age of 5. The viruses typically cause mild respiratory illness, but can become severe in young children, the elderly and other immunocompromised adults. HMPV and PIV3 can lead to bronchitis, bronchiolitis, croup and pneumonia.
HMPV and PIV3 are the second and third most common causes, respectively, of lower respiratory hospitalizations in children, behind RSV. HMPV and PIV3 each cause 10 – 25% of respiratory infections in babies, and approximately 75,000 babies are hospitalized annually due to one or both of these viruses.
There are no approved vaccines for either HMPV or PIV3.
Our HMPV/PIV3 mRNA Vaccine (mRNA-1653)
Our investigational HMPV/PIV3 vaccine, mRNA-1653, is a multivalent vaccine, meaning it is designed to immunize against multiple pathogens, in this case HMPV and PIV3. We combine mRNA encoding for a viral antigenic protein associated with HMPV and mRNA encoding for a viral antigenic protein associated with PIV3 into a single vaccine. The mRNAs are delivered to the body and direct cells to produce and express both antigenic proteins on the cell surface, much like a native infection would do but without the ability to cause disease or spread. As a result the body’s immune system responds as if the actual virus is present. The immune system produces antibodies that have the potential to neutralize the viruses and prevent infections in the event the vaccinated person is exposed to HMPV or PIV3 in the future.
mRNA-1653 has been nominated as a development candidate and is progressing to GLP toxicology studies
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mRNA MRK-V213
Lead
Merck
Indication/ Target
Undisclosed
Formulation
Undisclosed
GLP Toxicology
No
IND/CTA Filed
No
Phase 1
Phase 2
Funding
See More Info
Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Undisclosed
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Immuno-Oncology
mRNA-4157
Lead
Moderna/ Merck
Indication/ Target
Personalized Cancer Vaccines
Formulation
V1GL
GLP Toxicology
Yes
IND/CTA Filed
Yes
Phase 1
Safe to Proceed to Clinic
Phase 2
Funding
See More Info
Modality: Therapeutic Vaccines | Therapeutic Area: Personalized Cancer Vaccine / Immuno-Oncology | Disease: Various Cancers
Partnered Program with Merck
About Immuno-Oncology
Immuno-oncology is a therapeutic area that involves harnessing the body’s immune system to kill cancer cells in the same way the immune system responds to and eradicates a viral, microbial, fungal or parasitic infection. Also often referred to as immunotherapy, immuno-oncology is an increasingly expanding area of cancer drug development. This is particularly due to the successful introduction and use over the past several years of one type of immunotherapy known as checkpoint inhibitors.
T cells are a key part of the immune system; they identify and mount an attack on both infections and cancers. Checkpoint inhibitors enable T cells to better recognize cancer cells as foreign invaders that may otherwise go undetected and, therefore, evade attack. Unfortunately, not all patients respond to treatment with checkpoint inhibitors.
Our mRNA-based, Personalized Cancer Vaccine (mRNA-4157)
We are developing an mRNA-based personalized cancer vaccine (PCV), mRNA-4157, with the potential to prime the immune system to recognize cancer cells and mount a strong, tailored response to each individual patient’s cancer. Utilizing our mRNA vaccine technology platform, we plan to first identify and then create a vaccine encoding for peptides containing unique mutations (i.e., neoantigens) present in each patient’s specific tumor. When injected in the body, the mRNA directs cells to produce and express these neoantigens. This, in turn, activates the immune system to better recognize and destroy the cancer cells. Our mRNA-based personalized cancer vaccine has the potential to improve clinical outcomes associated with checkpoint inhibitor therapies, including our partner Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab).
Moderna has developed a rapid cycle time, small-batch manufacturing technique that will uniquely allow us to supply vaccines tailored to individual patients within weeks.
mRNA-4157 has completed GLP toxicology studies.
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mRNA-5671
Lead
Moderna
Indication/ Target
KRAS
Formulation
V1GL
GLP Toxicology
Yes
IND/CTA Filed
No
Phase 1
Phase 2
Funding
See More Info
Modality: Therapeutic Vaccines | Therapeutic Area: Immuno-Oncology | Disease: KRAS
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mRNA-2416
Lead
Moderna
Indication/ Target
OX40L
Formulation
N1GL
GLP Toxicology
Yes
IND/CTA Filed
Yes
Phase 1
Started: Aug '17
Phase 2
Funding
See More Info
Modality: Intratumoral Immuno-Oncology | Therapeutic Area: Immuno-Oncology | Disease: Various Cancers
About Immuno-Oncology
Immuno-oncology is a therapeutic area that involves harnessing the body’s immune system to kill cancer cells in the same way the immune system targets a common infection. Also often referred to as immunotherapy, immuno-oncology is an increasingly expanding area of cancer treatment. This is particularly due to the successful introduction and use over the past several years of one type of immunotherapy known as checkpoint inhibitors.
T cells are part of the immune system; they identify and mount an attack on infections from viruses or bacteria, as well as cancer. Checkpoint inhibitors enable T cells to better recognize cancer cells as foreign invaders that may otherwise go undetected and, therefore, evade attack. Unfortunately, not all patients respond to treatment with checkpoint inhibitors.
Moderna’s immuno-oncology program focuses on mRNA drugs that may generate a potent immune response to cancers, alone or likely in combination with checkpoint inhibitors, as well as co-formulated combinations of mRNAs that may present important alternative immunotherapy treatment options for cancer patients.
Our mRNA OX40L intratumoral (iTu) Immunotherapy (mRNA-2416)
OX40 Ligand (or OX40L) is a co-stimulatory membrane-bound protein that enhances the expansion, function and survival of T cells to mount an attack against cancer cells. We are investigating the potential for local injection of mRNA encoding for the OX40L protein into a tumor (intratumoral injection). When delivered directly into a tumor, cells in the tumor may express the OX40 ligand protein on their surfaces, which, in turn, may lead to a stronger T cell attack against the tumor. Additionally, we are investigating whether mRNA-2416 has the potential to elicit an abscopal effect in metastatic cancer, in which localized injection into one tumor would lead not only to shrinking of that tumor but also shrinking of tumors elsewhere in the body.
Combining our OX40L iTu immunotherapy with a checkpoint inhibitor may improve outcomes from cancer therapy.
Clinical Development
We filed an IND for mRNA-2416 with the U.S. FDA in Dec. 2016.
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mRNA-2752
Lead
Moderna
Indication/ Target
OX40L/IL23/IL36
Formulation
N1GL
GLP Toxicology
No
IND/CTA Filed
No
Phase 1
Phase 2
Funding
See More Info
Modality: Intratumoral Immuno-Oncology | Therapeutic Area: Immuno-Oncology | Disease: OX40L/IL23/IL36
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mRNA-2905
Lead
Moderna/ AstraZeneca
Indication/ Target
IL-12
Formulation
N1GL
GLP Toxicology
Ongoing
IND/CTA Filed
No
Phase 1
Phase 2
Funding
See More Info
Modality: Intratumoral Immuno-Oncology | Therapeutic Area: Immuno-Oncology | Disease: Various Cancers
Partnered Program with AstraZeneca
About Immuno-Oncology
Immuno-oncology is a therapeutic area that involves harnessing the body’s immune system to kill cancer cells in the same way the immune system innately targets a common infection. Also often referred to as immunotherapy, immuno-oncology is an increasingly expanding area of cancer treatment. This is particularly due to the successful introduction and use over the past several years of one type of immunotherapy known as checkpoint inhibitors.
T cells are part of the immune system; they identify and mount an attack on infections from viruses or bacteria, as well as cancer. Checkpoint inhibitors enable T cells to better recognize cancer cells as foreign invaders that may otherwise go undetected and, therefore, evade attack. Unfortunately, not all patients respond to treatment with checkpoint inhibitors.
Moderna’s immuno-oncology program focuses on mRNA drugs that may improve response to checkpoint inhibitors, as well as co-formulated combinations of mRNAs that may present important alternative immunotherapy treatment options for cancer patients.
mRNA IL-12 intratumoral (iTu) Immunotherapy (mRNA-2905)
Interleukin 12, or IL-12, is a cytokine (a type of signaling protein) that activates the immune system after being released from cells. We are investigating the potential for local injection of mRNA encoding for the IL-12 protein into a tumor (intratumoral injection). When delivered directly into a tumor, cells in the tumor may express IL-12 at a high concentration in the local microenvironment, which, in turn, may lead to a stronger T cell attack against the tumor. By expressing IL-12 locally, systemic side effects that have previously been seen from delivery of IL-12 protein into the blood may be more manageable. Additionally, we are investigating whether mRNA-2905 has the potential to elicit an abscopal effect in metastatic cancer, in which localized injection into one tumor would lead not only to shrinking of that tumor but also shrinking of tumors elsewhere in the body.
Combining our investigational IL-12 iTu immunotherapy with a checkpoint inhibitor may improve outcomes from cancer therapy.
Clinical Development
Moderna led discovery efforts and is leading preclinical development.
AstraZeneca will oversee early clinical development (led by MedImmune).
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Cardiovascular Disease
mRNA AZD-8601
Lead
AstraZeneca
Indication/ Target
VEGF-A
Formulation
Citrate/Saline
GLP Toxicology
Yes
IND/CTA Filed
Yes
Phase 1
Fully enrolled
Phase 2
Funding
See More Info
Modality: Localized Therapeutics | Therapeutic Application: Cardiovascular Disease (CV) and other ischemic vascular diseases
Partnered Program; Led by AstraZeneca
About The Program
mRNA AZD-8601 – VEGF-A: mRNA AZD-8601 is an investigational mRNA-based therapy being developed by AstraZeneca that encodes for vascular endothelial growth factor-A (VEGF-A). Using mRNA to initiate a strong, local and transient surge of VEGF-A expression could help overcome challenges associated with previous approaches to regulate this protein in tissues. When directed via local tissue injection, VEGF-A mRNA may potentially lead to the creation of more blood vessels and improved blood supply. mRNA AZD-8601 could one day provide a unique regenerative treatment option for patients with heart failure or after a heart attack, as well as for diabetic wound healing and other ischemic vascular diseases.
Clinical Development
A Phase 1 safety study is currently enrolling patients in Europe. This study is a randomized, single-blind, placebo-controlled, single ascending dose study in male patients with Type 2 diabetes mellitus, performed at a single study center. This study is an essential first step to proving the clinical value of mRNA VEGF-A expression in cardiometabolic disease.
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Rare Liver Diseases
mRNA-3704
Lead
Moderna
Indication/ Target
MMA
Formulation
N2GL
GLP Toxicology
No
IND/CTA Filed
No
Phase 1
Phase 2
Funding
See More Info
Modality: Liver Therapeutics | Therapeutic Area: Rare Liver Diseases | Disease: MMA
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| Development Candidate | Lead | Indication/ Target | Formulation | GLP Toxicology | IND/CTA Filed | PH 1 | PH 2 | Funding | |
|---|---|---|---|---|---|---|---|---|---|
| mRNA-1440 | Moderna | Influenza H10 | In Licensed | Yes | Yes |
Fully enrolled |
n/a |
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Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Influenza A virus subtype H10N8 About the DiseaseInfluenza, also known as the flu, is a contagious respiratory illness caused by influenza viruses. Two influenza A virus subtypes, H1N1 and H3N2, are currently in general circulation among people. A pandemic, or global outbreak, occurs when there is a genetic shift that causes a new influenza A virus to emerge and spread rapidly because people have little or no immunity to the new strain. Our influenza vaccine program is focused on two influenza A virus strains, both with pandemic potential: H10N8 (mRNA 1440) and H7N9 (mRNA 1851). Influenza A viruses are classified by subtypes based on two proteins on the virus’s surface: hemagglutinin (HA) and neuraminidase (NA). The H10N8 virus has the viral antigenic proteins H10 and NA 8. Of our two pandemic flu vaccine programs, H10N8 has currently only infected three people in China in 2013 although two of them died. If the H10N8 was to become a pandemic the world would not currently have a vaccine. Source: Centers for Disease Control and Prevention Our H10N8 mRNA Vaccine (mRNA-1440)Our investigational H10N8 vaccine is being developed to deliver mRNA encoding for the membrane-bound hemagglutinin 10 (H10) protein. The mRNA directs the cells to produce and express H10 on the cell surface, exactly as a native infection would do but without the ability to cause disease or spread. As a result the body’s immune system responds as if the actual virus is present. The immune system produces antibodies that have the potential to neutralize the virus and prevent infections in the event the vaccinated person is exposed to the actual H10N8 virus in the future. Moderna strategically selected the H10N8 vaccine as one of its first two clinical development programs (the other, an H7N9 vaccine – mRNA-1851) in order to rapidly assess both the safety and efficacy of its mRNA platform in humans. Because this strain is not circulating in the general population in Germany, where the trial is taking place, Moderna is able to study the efficacy of its vaccine technology in a naïve patient population. Antibodies present in subjects’ blood after treatment with mRNA-1851 are likely attributed to Moderna’s vaccine and not to active immunity as a result of previous exposure to the virus. Studying mRNA vaccines for Influenza strains allows Moderna to measure vaccine efficacy against a well-understood endpoint, the hemagglutination inhibition assay, or HAI. HAI is used by U.S. FDA and World Health Organization (WHO) to measure how well antibodies bind to and inactivate an Influenza virus. Vaccines demonstrating titers of 1:40 are considered effective in reducing the risk for Influenza infection and are, thus, approved as seasonal flu vaccines. Clinical Development- Phase 1 dose-escalating, placebo-controlled, immunogenicity and safety trial in healthy adult volunteers |
|
| mRNA-1851 | Moderna | Influenza H7 | In Licensed | Yes | Yes |
Fully enrolled |
n/a |
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Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Influenza A virus subtype H7N9 About the DiseaseInfluenza, also known as the flu, is a contagious respiratory illness caused by influenza viruses. Two influenza A virus subtypes, H1N1 and H3N2, are currently in general circulation among people. A pandemic, or global outbreak, occurs when there is a genetic shift that causes a new influenza A virus to emerge and spread rapidly because people have little or no immunity to the new strain. Our influenza vaccine program is focused on two influenza A virus strains, both with pandemic potential: H10N8 (mRNA-1440) and H7N9 (mRNA-1851). Influenza A viruses are classified by subtypes based on two proteins on the virus’s surface: hemagglutinin (HA) and neuraminidase (NA). The H7N9 virus has the viral antigenic proteins H7 and NA 9. H7N9 has a high potential of becoming a pandemic. More than 600 cases have been reported, to date, in China, with a mortality rate of approximately one in three infected people. Source: Centers for Disease Control and Prevention Our H7N9 mRNA Vaccine (mRNA-1851)Our investigational H7N9 vaccine is being developed to deliver mRNA encoding for the membrane-bound hemagglutinin 7 (H7) protein. The mRNA directs the cells to produce and express H7 on the cell surface, much like a native infection would do but without the ability to cause disease and spread. As a results, the body’s immune system responds as if the actual virus is present. The immune system produces antibodies that have the potential to neutralize the virus and prevent infections in the event the vaccinated person is exposed to the actual H7N9 virus in the future. Moderna strategically selected the H7N9 vaccine as one of its first two clinical development programs (the other, an H10N8 vaccine – mRNA-1440) in order to rapidly assess both the safety and efficacy of its mRNA platform in humans. Because this strain is not circulating in the general population in the U.S., where the trial is taking place, Moderna is able to study the efficacy of its vaccine technology in a naïve patient population. Antibodies present in subjects’ blood after treatment with mRNA-1851 are likely attributed to Moderna’s vaccine and not to active immunity as a result of previous exposure to the virus. Studying mRNA vaccines for Influenza strains allows Moderna to measure vaccine efficacy against a well-understood endpoint, the hemagglutination inhibition assay, or HAI. HAI is used by U.S. FDA and World Health Organization (WHO) to measure how well antibodies bind to and inactivate an Influenza virus. Vaccines demonstrating titers of 1:40 are considered effective in reducing the risk for Influenza infection and are, thus, approved as seasonal flu vaccines. Clinical DevelopmentA Phase 1 study of healthy volunteers is underway in the U.S., with 104 healthy volunteers dosed to date. |
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| mRNA MRK-1777 | Merck | Undisclosed | In Licensed | Yes | Yes |
Started: Nov ‘16 |
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Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Undisclosed Partnered Program; Led by Merck A Phase 1 clinical study is underway for mRNA MRK-1777, an mRNA-based vaccine for an undisclosed infectious disease. |
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| mRNA-1388 | Moderna | Chikungunya | In Licensed | Yes | Yes |
Started: Aug '17 |
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DARPA |
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Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Chikungunya Virus About the DiseaseChikungunya virus, or CHIKV, is a type of alphavirus and is transmitted by mosquitos. It typically causes mild fever and transient, or fleeting, joint pain. However, in approximately 15 percent of infected patients it can cause long-term, severe arthritis. CHIKV has historically has been limited to warmer climates in Asia and Africa, but recent cases have been identified in the Americas and Europe. This is likely due to an increase in global travel, as well as the onset of transmission by the Aedes albopictus mosquito. Previously, the Aedes aegypti mosquito typically transmitted the disease. There are no treatment options or approved vaccines for CHIKV. Management of the infection from the virus has been limited to supportive care and avoidance of mosquito bites. Our CHIKV mRNA Vaccine (mRNA-1388)Our CHIKV vaccine delivers mRNA encoding for viral antigenic proteins associated with CHIKV. The mRNA directs cells to produce and express the proteins, closely mimicking a native viral infection and tricking the body into thinking the actual virus is present. The immune system produces antibodies against the viral antigenic proteins. If the person is exposed to the actual Chikungunya virus in the future, the body recognizes it as foreign. Antibodies target the viral antigenic proteins, activating a robust immune response to target and destroy the Chikungunya virus, so it cannot cause an infection. Clinical DevelopmentGLP toxicology studies have been completed and an IND filed with the U.S. Food & Drug Administration. FundingIn 2015, Moderna extended an existing grant from the Defense Advanced Research Projects Agency (DARPA) to finance preclinical toxicology studies and the Phase I study of mRNA-1388. The extension builds on an original grant announced in 2013 to develop antibody-producing drugs to protect against a wide range of known and unknown emerging infectious diseases and engineered biological threats. |
| mRNA-1325 | Moderna | Zika | In Licensed | Yes | Yes |
Started: Dec ‘16 |
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DARPA, BARDA |
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Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Zika Virus About the DiseaseZika is a member of the flavivirus family, which also includes viruses such as Dengue Fever and Yellow Fever. A rapidly emerging pandemic with the potential for long-term health implications. Zika is transmitted by the Aedes mosquito and mother to fetus transmission as well as sexual transmission have also been confirmed. There is a causal link between Zika infection and at least two serious diseases: microcephaly and Guillain-Barré Syndrome (GBS). Children born to mothers infected with Zika can develop microcephaly, a severe disease characterized by abnormally small heads and severe neurologic disabilities. GBS is an autoimmune disease that attacks the peripheral nervous system, leading to rapidly progressive and potentially life-threatening muscle weakness. GBS can lead to death caused by respiratory arrest if a patient is not ventilated. As of November 2016, 48 countries and territories in the Americas have confirmed autochthonous, vector-borne transmission of the Zika virus, and five countries in the Americas have reported sexually transmitted Zika cases. 184 locally transmitted cases have been reported in South Florida, and 34,000 locally transmitted cases throughout the U.S. and its territories, with most cases in Puerto Rico. There are no treatment options or approved vaccines for the Zika virus. Our Zika mRNA Vaccine (mRNA-1325)Our investigational Zika vaccine, mRNA-1325, is being developed to deliver mRNA encoding for viral antigenic proteins associated with the Zika virus. The mRNA directs cells to produce and express the proteins on the cell surface, much like a native infection would do but without the ability to cause disease or spread. As a result the body’s immune system responds as if the actual virus is present. The immune system produces antibodies that have the potential to neutralize the virus and prevent infections in the event the vaccinated person is exposed to the actual Zika virus in the future. Our mRNA platform, research engine and early development engine enabled us to advance our Zika vaccine from initial discovery concept to first-in-human dosing in 12 months. mRNA-1325 is one of two Zika mRNA vaccines we are developing. mRNA-1325, the earlier of our two Zika mRNA programs, utilizes an in-licensed formulation, while our second investigational Zika mRNA vaccine, mRNA-1706, utilizes V1GL, one of our novel, proprietary formulations. Clinical Development A Phase 1/2 clinical study of mRNA-1325 began enrolling healthy volunteers in December 2016. FundingIn 2016, Moderna received a funding award of up to $125 million from the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), to accelerate development of our Zika mRNA vaccine. Moderna’s preclinical work for mRNA-1325 was funded through a grant from the Defense Advanced Research Projects Agency (DARPA). |
| mRNA-1893* | Moderna | Zika | V1GL | No | No |
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Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Zika virus About the DiseaseZika is a member of the flavivirus family, which also includes viruses such as Dengue Fever and Yellow Fever. A rapidly emerging pandemic with the potential for long-term health implications. Zika is transmitted by the Aedes mosquito and mother to fetus transmission as well as sexual transmission have also been confirmed. There is a causal link between Zika infection and at least two serious diseases: microcephaly and Guillain-Barré Syndrome (GBS). Children born to mothers infected with Zika can develop microcephaly, a severe disease characterized by abnormally small heads and severe neurologic disabilities. GBS is an autoimmune disease that attacks the peripheral nervous system, leading to rapidly progressive and potentially life-threatening muscle weakness. GBS can lead to death caused by respiratory arrest if a patient is not ventilated. As of November 2016, 48 countries and territories in the Americas have confirmed autochthonous, vector-borne transmission of the Zika virus, and five countries in the Americas have reported sexually transmitted Zika cases. 184 locally transmitted cases have been reported in South Florida, and 34,000 locally transmitted cases throughout the U.S. and its territories, with most cases in Puerto Rico. There are no treatment options or approved vaccines for the Zika virus. |
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| mRNA-1647 | Moderna | CMV | V1GL | Yes | No |
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Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Cytomegalovirus About the DiseaseCytomegalovirus, or CMV, is a member of the herpes virus family. Often a person will not have any symptoms of viral infection. CMV leads to severe disease in two populations: newborns and transplant patients. CMV is the most common cause of newborn disability, leading to deafness, microcephaly (small, not fully developed heads), vision loss and neurologic disabilities, among other serious complications. It is also the most frequent viral disease in transplant recipients, often leading to transplant failure. There are currently no approved vaccines for CMV. Our CMV mRNA Vaccine (mRNA-1647)Our investigational CMV vaccine in development, mRNA-1647, combines six mRNAs encoding for six viral proteins, including five proteins that comprise the CMV gH Pentamer complex (gH, gL, UL128, UL130 and UL131A), as well as another CMV antigen, the herpesvirus glycoprotein (gB) protein. The mRNAs direct cells in the body to produce and express all six proteins on the cell surface, including the expression of gB and a fully formed Pentamer complex, or five proteins expressed as a single antigen, much like a native infection would do but without the ability to cause disease or spread. As a result, the body’s immune system responds as if the actual virus in present. The immune system produces antibodies against the Pentamer and gB that have the potential to neutralize the virus and prevent infections in the event the vaccinated person is exposed to the actual CMV virus in the future. mRNA-1647 has been nominated as a development candidate and is progressing to GLP toxicology studies. |
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| mRNA-1653 | Moderna | HMPV/PIV3 | V1GL | Yes | No |
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Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Diseases: HMPV / PIV3 About the DiseasesHuman Metapneumovirus (HMPV) and Parainfluenza Virus 3 (PIV3) are both part of the paramyxovirus family. Most children have been infected at least once with both viruses by the age of 5. The viruses typically cause mild respiratory illness, but can become severe in young children, the elderly and other immunocompromised adults. HMPV and PIV3 can lead to bronchitis, bronchiolitis, croup and pneumonia. HMPV and PIV3 are the second and third most common causes, respectively, of lower respiratory hospitalizations in children, behind RSV. HMPV and PIV3 each cause 10 – 25% of respiratory infections in babies, and approximately 75,000 babies are hospitalized annually due to one or both of these viruses. There are no approved vaccines for either HMPV or PIV3. Our HMPV/PIV3 mRNA Vaccine (mRNA-1653)Our investigational HMPV/PIV3 vaccine, mRNA-1653, is a multivalent vaccine, meaning it is designed to immunize against multiple pathogens, in this case HMPV and PIV3. We combine mRNA encoding for a viral antigenic protein associated with HMPV and mRNA encoding for a viral antigenic protein associated with PIV3 into a single vaccine. The mRNAs are delivered to the body and direct cells to produce and express both antigenic proteins on the cell surface, much like a native infection would do but without the ability to cause disease or spread. As a result the body’s immune system responds as if the actual virus is present. The immune system produces antibodies that have the potential to neutralize the viruses and prevent infections in the event the vaccinated person is exposed to HMPV or PIV3 in the future. mRNA-1653 has been nominated as a development candidate and is progressing to GLP toxicology studies |
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| mRNA MRK-V213 | Merck | Undisclosed | Undisclosed | No | No |
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Modality: Prophylactic Vaccines | Therapeutic Area: Infectious Diseases / Viral Vaccines | Disease: Undisclosed |
| Development Candidate | Lead | Indication/ Target | Formulation | GLP Toxicology | IND/CTA Filed | PH 1 | PH 2 | Funding | |
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| mRNA-4157 | Moderna/ Merck | Personalized Cancer Vaccines | V1GL | Yes | Yes |
Safe to Proceed to Clinic |
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Modality: Therapeutic Vaccines | Therapeutic Area: Personalized Cancer Vaccine / Immuno-Oncology | Disease: Various Cancers Partnered Program with Merck About Immuno-OncologyImmuno-oncology is a therapeutic area that involves harnessing the body’s immune system to kill cancer cells in the same way the immune system responds to and eradicates a viral, microbial, fungal or parasitic infection. Also often referred to as immunotherapy, immuno-oncology is an increasingly expanding area of cancer drug development. This is particularly due to the successful introduction and use over the past several years of one type of immunotherapy known as checkpoint inhibitors. T cells are a key part of the immune system; they identify and mount an attack on both infections and cancers. Checkpoint inhibitors enable T cells to better recognize cancer cells as foreign invaders that may otherwise go undetected and, therefore, evade attack. Unfortunately, not all patients respond to treatment with checkpoint inhibitors. Our mRNA-based, Personalized Cancer Vaccine (mRNA-4157)We are developing an mRNA-based personalized cancer vaccine (PCV), mRNA-4157, with the potential to prime the immune system to recognize cancer cells and mount a strong, tailored response to each individual patient’s cancer. Utilizing our mRNA vaccine technology platform, we plan to first identify and then create a vaccine encoding for peptides containing unique mutations (i.e., neoantigens) present in each patient’s specific tumor. When injected in the body, the mRNA directs cells to produce and express these neoantigens. This, in turn, activates the immune system to better recognize and destroy the cancer cells. Our mRNA-based personalized cancer vaccine has the potential to improve clinical outcomes associated with checkpoint inhibitor therapies, including our partner Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab). Moderna has developed a rapid cycle time, small-batch manufacturing technique that will uniquely allow us to supply vaccines tailored to individual patients within weeks. mRNA-4157 has completed GLP toxicology studies. |
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| mRNA-5671 | Moderna | KRAS | V1GL | Yes | No |
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Modality: Therapeutic Vaccines | Therapeutic Area: Immuno-Oncology | Disease: KRAS |
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| mRNA-2416 | Moderna | OX40L | N1GL | Yes | Yes |
Started: Aug '17 |
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Modality: Intratumoral Immuno-Oncology | Therapeutic Area: Immuno-Oncology | Disease: Various Cancers About Immuno-OncologyImmuno-oncology is a therapeutic area that involves harnessing the body’s immune system to kill cancer cells in the same way the immune system targets a common infection. Also often referred to as immunotherapy, immuno-oncology is an increasingly expanding area of cancer treatment. This is particularly due to the successful introduction and use over the past several years of one type of immunotherapy known as checkpoint inhibitors. T cells are part of the immune system; they identify and mount an attack on infections from viruses or bacteria, as well as cancer. Checkpoint inhibitors enable T cells to better recognize cancer cells as foreign invaders that may otherwise go undetected and, therefore, evade attack. Unfortunately, not all patients respond to treatment with checkpoint inhibitors. Moderna’s immuno-oncology program focuses on mRNA drugs that may generate a potent immune response to cancers, alone or likely in combination with checkpoint inhibitors, as well as co-formulated combinations of mRNAs that may present important alternative immunotherapy treatment options for cancer patients. Our mRNA OX40L intratumoral (iTu) Immunotherapy (mRNA-2416)OX40 Ligand (or OX40L) is a co-stimulatory membrane-bound protein that enhances the expansion, function and survival of T cells to mount an attack against cancer cells. We are investigating the potential for local injection of mRNA encoding for the OX40L protein into a tumor (intratumoral injection). When delivered directly into a tumor, cells in the tumor may express the OX40 ligand protein on their surfaces, which, in turn, may lead to a stronger T cell attack against the tumor. Additionally, we are investigating whether mRNA-2416 has the potential to elicit an abscopal effect in metastatic cancer, in which localized injection into one tumor would lead not only to shrinking of that tumor but also shrinking of tumors elsewhere in the body. Combining our OX40L iTu immunotherapy with a checkpoint inhibitor may improve outcomes from cancer therapy. Clinical DevelopmentWe filed an IND for mRNA-2416 with the U.S. FDA in Dec. 2016. |
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| mRNA-2752 | Moderna | OX40L/IL23/IL36 | N1GL | No | No |
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Modality: Intratumoral Immuno-Oncology | Therapeutic Area: Immuno-Oncology | Disease: OX40L/IL23/IL36 |
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| mRNA-2905 | Moderna/ AstraZeneca | IL-12 | N1GL | Ongoing | No |
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Modality: Intratumoral Immuno-Oncology | Therapeutic Area: Immuno-Oncology | Disease: Various Cancers Partnered Program with AstraZeneca About Immuno-OncologyImmuno-oncology is a therapeutic area that involves harnessing the body’s immune system to kill cancer cells in the same way the immune system innately targets a common infection. Also often referred to as immunotherapy, immuno-oncology is an increasingly expanding area of cancer treatment. This is particularly due to the successful introduction and use over the past several years of one type of immunotherapy known as checkpoint inhibitors. T cells are part of the immune system; they identify and mount an attack on infections from viruses or bacteria, as well as cancer. Checkpoint inhibitors enable T cells to better recognize cancer cells as foreign invaders that may otherwise go undetected and, therefore, evade attack. Unfortunately, not all patients respond to treatment with checkpoint inhibitors. Moderna’s immuno-oncology program focuses on mRNA drugs that may improve response to checkpoint inhibitors, as well as co-formulated combinations of mRNAs that may present important alternative immunotherapy treatment options for cancer patients. mRNA IL-12 intratumoral (iTu) Immunotherapy (mRNA-2905)Interleukin 12, or IL-12, is a cytokine (a type of signaling protein) that activates the immune system after being released from cells. We are investigating the potential for local injection of mRNA encoding for the IL-12 protein into a tumor (intratumoral injection). When delivered directly into a tumor, cells in the tumor may express IL-12 at a high concentration in the local microenvironment, which, in turn, may lead to a stronger T cell attack against the tumor. By expressing IL-12 locally, systemic side effects that have previously been seen from delivery of IL-12 protein into the blood may be more manageable. Additionally, we are investigating whether mRNA-2905 has the potential to elicit an abscopal effect in metastatic cancer, in which localized injection into one tumor would lead not only to shrinking of that tumor but also shrinking of tumors elsewhere in the body. Combining our investigational IL-12 iTu immunotherapy with a checkpoint inhibitor may improve outcomes from cancer therapy. Clinical DevelopmentModerna led discovery efforts and is leading preclinical development. |
| Development Candidate | Lead | Indication/ Target | Formulation | GLP Toxicology | IND/CTA Filed | PH 1 | PH 2 | Funding | |
|---|---|---|---|---|---|---|---|---|---|
| mRNA AZD-8601 | AstraZeneca | VEGF-A | Citrate/Saline | Yes | Yes |
Fully enrolled |
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Modality: Localized Therapeutics | Therapeutic Application: Cardiovascular Disease (CV) and other ischemic vascular diseases Partnered Program; Led by AstraZeneca About The ProgrammRNA AZD-8601 – VEGF-A: mRNA AZD-8601 is an investigational mRNA-based therapy being developed by AstraZeneca that encodes for vascular endothelial growth factor-A (VEGF-A). Using mRNA to initiate a strong, local and transient surge of VEGF-A expression could help overcome challenges associated with previous approaches to regulate this protein in tissues. When directed via local tissue injection, VEGF-A mRNA may potentially lead to the creation of more blood vessels and improved blood supply. mRNA AZD-8601 could one day provide a unique regenerative treatment option for patients with heart failure or after a heart attack, as well as for diabetic wound healing and other ischemic vascular diseases. Clinical DevelopmentA Phase 1 safety study is currently enrolling patients in Europe. This study is a randomized, single-blind, placebo-controlled, single ascending dose study in male patients with Type 2 diabetes mellitus, performed at a single study center. This study is an essential first step to proving the clinical value of mRNA VEGF-A expression in cardiometabolic disease. |
| Development Candidate | Lead | Indication/ Target | Formulation | GLP Toxicology | IND/CTA Filed | PH 1 | PH 2 | Funding | |
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| mRNA-3704 | Moderna | MMA | N2GL | No | No |
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Modality: Liver Therapeutics | Therapeutic Area: Rare Liver Diseases | Disease: MMA |