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Delivery of chemically modified PNA oligomers

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R41AI152613-01
Agency Tracking Number: R41AI152613
Amount: $299,999.00
Phase: Phase I
Program: STTR
Solicitation Topic Code: NIAID
Solicitation Number: PAR17-036
Timeline
Solicitation Year: 2017
Award Year: 2020
Award Start Date (Proposal Award Date): 2020-03-06
Award End Date (Contract End Date): 2022-02-28
Small Business Information
900 B W FARIS RD
Greenville, SC 29605-4255
United States
DUNS: 831389122
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: Yes
Principal Investigator
 DEV ARYA
 (864) 656-1106
 dparya@clemson.edu
Business Contact
 DEV ARYA
Phone: (864) 207-0002
Email: dev.arya@nubadllc.com
Research Institution
 CLEMSON UNIVERSITY
 
230 KAPPA STREET, STE. 200
CLEMSON, SC 29634-0001
United States

 Nonprofit college or university
Abstract

PROJECT SUMMARYRNA is a validated target for drug design, both as therapeutic and as a target.
Targeting specific RNA, such as rRNA which are involved in proliferation and survival of
bacteria is a promising approach. We are developing fast and low cost methods to
screen sequence-specific small molecules for novel anti-ribosomal activities. We will
construct sequence-specific chemically modified ribosomal targeting oligomers that can
be effectively delivered inside the cell, addressing the key objective of
PAR-17-036 (to generate new technologies and products for delivering nucleic
acids into cells and tissues for the purpose of treatment or prevention of
human disease). Complexes between ribosomal components will be exploited
as targets for small molecule drug libraries that-inactivate the ribosome.
NUBADs unique experimental approaches and technologies will allow us to target
ribosomal regions not previously explored for susceptibility against microbial
targets.The work proposed here, a multidisciplinary effort encompassing solid-phase
organic synthesis, oligonucleotide delivery, RNA targeted screening, antimicrobial
activity, and in vivo efficacy studies describes the development of sequence-specific cell
permeable binders of rRNA. The success of the proposed work would be a significant
addition to currently available ribosome-specific approaches in drug development. We
propose using a small rRNA target sequences to design conjugates that can be
employed to inhibit microbial growth, opening possibilities for developing sequence-
specific RNA targeted therapeutics.Health Relevance Statement.
The work proposed here, a multidisciplinary effort encompassing organic synthesis,
oligonucleotide (PNA) delivery, RNA targeted screening and in vivo efficacy studies,
describes the development of sequence-specific cell permeable binders of rRNA as
PNA based therapeutics. We propose using a small rRNA target sequences to design
conjugates that can be employed to inhibit microbial growth, opening possibilities for
developing sequence-specific RNA targeted therapeutics.

* Information listed above is at the time of submission. *

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