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Investigating the Impact of Metabolic Disease States on the Pharmacodynamics and Toxicity of FDA-Approved Antisense Oligonucleotides Using an Integrated Liver-Kidney Model

Award Number: R43TR005374
ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 07/01/2026
PERIOD OF PERFORMANCE END DATE: 12/31/2026

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Investigating the Impact of Metabolic Disease States on the Pharmacodynamics and Toxicity of FDA-Approved Antisense Oligonucleotides Using an Integrated Liver-Kidney Model - Anti-sense oligonucleotide (ASO) drugs have shown great potential in the treatment of human diseases. However, the lack of therapeutic efficacy and intolerance of adverse drug reactions (ADRs) and toxicity have led to the termination of numerous ASO candidates in the development pipelines, as well as the discontinuation from the market after approval by the FDA. Hepatotoxicity and nephrotoxicity are two major concerns for the FDA-approved ASO drugs in the market as well as development of new ASO-based therapies. Significant knowledge gaps still exist which prevent us from better understanding the lack of effectiveness and existence of ADRs and toxicity. One of these gaps lies in how an ASO drug goes through absorption, distribution, metabolism, and excretion (ADME) in its target organs and cells. Both in vitro cellular models and in vivo animal models have been used to assess ADME and toxicity of ASO drugs; however, they have numerous limitations. While cellular models lack human complexity, animal models lack human-specificity. Microphysiological systems (MPSs) may provide a solution to overcome this unmet need by providing human specificity and complexity as an in vitro platform. Here, we propose using the Javelin Liver Tissue Chip Plus (LTC+) platform, which integrates human liver and kidney MPSs, to study distribution, metabolism, excretion, and toxicity profiles (hepato- and nephrotoxicity) of four FDA-approved ASO drugs.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $349,677 )
2026	2026	JAVELIN BIOTECH INC	900 MIDDLESEX TPKE BLDG 5	BILLERICA	MA	01821	MIDDLESEX	USA	National Center for Advancing Translational Sciences - Small Business	000	1	6/16/2026	NEW	$349,677
														Subtotal = $349,677

Grand Total All Awards = $349,677

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Investigating the Impact of Metabolic Disease States on the Pharmacodynamics and Toxicity of FDA-Approved Antisense Oligonucleotides Using an Integrated Liver-Kidney Model

Award Number: R43TR005374

ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 07/01/2026

PERIOD OF PERFORMANCE END DATE: 12/31/2026

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