ZATA PHARMACEUTICALS, INC. — Department of Health and Human Services SBIR Phase I: DESCRIPTION (provided by applicant): There is enormous potential of oligonucleotides (ON)

ZATA PHARMACEUTICALS, INC. — SBIR Phase I award from Department of Health and Human Services.

Amount
$363,842
Agency
Department of Health and Human Services · National Institutes of Health
Program / Phase
SBIR · Phase I
Solicitation
PA13-234
NAICS
Place of performance
MA
Period

Description

DESCRIPTION (provided by applicant): There is enormous potential of oligonucleotides (ON) as therapeutics, but the challenge remains how to effectively deliver ON into cells. Currently, there are no effective and reliable ways of delivery. Outer cell membranes resist the cellular uptake of charged ON, and charges-eliminating backbone modifications such as those in peptide nucleic acids (PNA) and methylphosphonates reduce but do not solve the problem, because such structural changes compromise their aqueoussolubility. Use of delivery vehicles (formulations), such as virus-based delivery systems, liposomes, nanoparticles and transporter chemical groups, have not solved this problem fully and are often associated with significant side effects. Development of optimal oligotherapy for the treatment of infectious and genetic diseases still remains unrealized. ZATA Pharmaceuticals, Inc. is developing a nucleic acid technology platform that will enable the synthesis of self-neutralizing ON with enhanced intrac