2337317
Project Grant
Overview
Grant Description
Sttr Phase I: Gex: An mRNA-based tolerogenic vaccine for viral-based gene therapies.
The broader impact of this Small Business Technology Transfer (STTR) Phase I project is to develop a scalable, relatively affordable, easily programmable and specific solution to the problem of unwanted immune response in cell and gene therapies, autoimmunity, and transplantation.
If successful, this could greatly expand the number of patients who are able to be treated by these types of therapies, including many suffering from rare diseases with little or no current treatment options.
The proposed project uses mRNA technology and novel anti-adjuvant technologies to train the immune response to ignore an antigen encoded in the delivered mRNAs.
This project focuses on adeno associated virus-based gene therapies, but the insight gained can be applied in other conditions when hyperactive immune response against self or foreign antigens creates unwanted result.
Current gene therapies face a number of challenges associated with immune response to delivery vehicles or the therapeutic proteins.
Achieving high and persistent level of therapeutic product expression has been a challenge, often leading to failure of therapies and necessitating redosing.
The proposed mRNA based tolerogenic vaccine will prepare the body to receive multiple doses of gene therapies and to achieve higher level of transgenes in light of body’s response to gene therapies.
This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the foundation's intellectual merit and broader impacts review criteria.
Subawards are planned for this award.
The broader impact of this Small Business Technology Transfer (STTR) Phase I project is to develop a scalable, relatively affordable, easily programmable and specific solution to the problem of unwanted immune response in cell and gene therapies, autoimmunity, and transplantation.
If successful, this could greatly expand the number of patients who are able to be treated by these types of therapies, including many suffering from rare diseases with little or no current treatment options.
The proposed project uses mRNA technology and novel anti-adjuvant technologies to train the immune response to ignore an antigen encoded in the delivered mRNAs.
This project focuses on adeno associated virus-based gene therapies, but the insight gained can be applied in other conditions when hyperactive immune response against self or foreign antigens creates unwanted result.
Current gene therapies face a number of challenges associated with immune response to delivery vehicles or the therapeutic proteins.
Achieving high and persistent level of therapeutic product expression has been a challenge, often leading to failure of therapies and necessitating redosing.
The proposed mRNA based tolerogenic vaccine will prepare the body to receive multiple doses of gene therapies and to achieve higher level of transgenes in light of body’s response to gene therapies.
This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the foundation's intellectual merit and broader impacts review criteria.
Subawards are planned for this award.
Awardee
Funding Goals
THE GOAL OF THIS FUNDING OPPORTUNITY, "NSF SMALL BUSINESS INNOVATION RESEARCH (SBIR)/ SMALL BUSINESS TECHNOLOGY TRANSFER (STTR) PROGRAMS PHASE I", IS IDENTIFIED IN THE LINK: HTTPS://WWW.NSF.GOV/PUBLICATIONS/PUB_SUMM.JSP?ODS_KEY=NSF23515
Grant Program (CFDA)
Awarding / Funding Agency
Place of Performance
Pittsburgh,
Pennsylvania
15213-2500
United States
Geographic Scope
Single Zip Code
Genexgen was awarded
Project Grant 2337317
worth $274,763
from National Science Foundation in September 2024 with work to be completed primarily in Pittsburgh Pennsylvania United States.
The grant
has a duration of 1 year and
was awarded through assistance program 47.084 NSF Technology, Innovation, and Partnerships.
The Project Grant was awarded through grant opportunity NSF Small Business Innovation Research / Small Business Technology Transfer Phase I Programs.
SBIR Details
Research Type
STTR Phase I
Title
STTR Phase I: GEX: an mRNA-based tolerogenic vaccine for viral-based gene therapies
Abstract
The broader impact of this Small Business Technology Transfer (STTR) Phase I project is to develop a scalable, relatively affordable, easily programmable and specific solution to the problem of unwanted immune response in cell and gene therapies, autoimmunity, and transplantation. If successful, this could greatly expand the number of patients who are able to be treated by these types of therapies, including many suffering from rare diseases with little or no current treatment options.
The proposed project uses mRNA technology and novel anti-adjuvant technologies, to train the immune response to ignore an antigen encoded in the delivered mRNAs. This project focuses on adeno associated virus-based gene therapies, but the insight gained can be applied in other conditions when hyperactive immune response against self or foreign antigens creates unwanted result. Current gene therapies face a number of challenges associated with immune response to delivery vehicles or the therapeutic proteins. Achieving high and persistent level of therapeutic product expression has been a challenge, often leading to failure of therapies and necessitating redosing. The proposed mRNA based tolerogenic vaccine, will prepare the body to receive multiple doses of gene therapies and to achieve higher level of transgenes in light of body’s response to gene therapies.
This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.
Topic Code
PT
Solicitation Number
NSF 23-515
Status
(Complete)
Last Modified 9/17/24
Period of Performance
9/1/24
Start Date
8/31/25
End Date
Funding Split
$274.8K
Federal Obligation
$0.0
Non-Federal Obligation
$274.8K
Total Obligated
Activity Timeline
Additional Detail
Award ID FAIN
2337317
SAI Number
None
Award ID URI
SAI EXEMPT
Awardee Classifications
Small Business
Awarding Office
491503 TRANSLATIONAL IMPACTS
Funding Office
491503 TRANSLATIONAL IMPACTS
Awardee UEI
JY3VPNHXH8D7
Awardee CAGE
96P11
Performance District
PA-12
Senators
Robert Casey
John Fetterman
John Fetterman
Modified: 9/17/24