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Preclinical Evaluation of Ancestral Coagulation Factor VIII

Award Number: R43HL135969
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 02/01/2017
PERIOD OF PERFORMANCE END DATE: 07/31/2018

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Preclinical Evaluation of Ancestral Coagulation Factor VIII - ABSTRACT The development of transformative therapeutics, including the possibility of a cure though gene therapy, is the sole mission of Expression Therapeutics and continues to be a major research and development activity in the overall $10 B USD hemophilia market space. However, progress in the field has been limited by significant hurdles including the size, complexity, instability, immunogenicity and biosynthetic inefficiency of coagulation factor VIII (FVIII). Through the study of existing vertebrate species and, even more recently, predicted ancestral FVIII variants, we have identified differentials in molecular, cellular, immunological and biochemical properties that are predicted to be pharmacologically beneficial. Based on these data, as well as existing knowledge of FVIII biosynthesis and mechanism of action, we have bioengineered a novel FVIII candidate, An-53, for both protein infusion and gene-based therapies. In the current proposal, we seek to compare the performance of An-53 to existing FVIII candidates including the hybrid human/porcine FVIII, ET3, and B domain deleted human FVIII in lentiviral vector and adeno-associated viral vector gene therapy platforms. Additionally, we will perform critical immunogenicity assessments that, together with the gene transfer performance data will facilitate lead candidate selection by Expression Therapeutics.


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: 2019 ( Subtotal = $0 )
2019	2017	EXPRESSION THERAPEUTICS LLC	1860 MONTREAL RD	TUCKER	GA	30084	DEKALB	USA	Blood Diseases and Resources Research	000	1	12/11/2018	NEW	$0
														Subtotal = $0

Issue Date FY: 2017 ( Subtotal = $224,976 )
2017	2017	EXPRESSION THERAPEUTICS LLC	1860 MONTREAL RD	TUCKER	GA	30084	DEKALB	USA	Blood Diseases and Resources Research	000	1	1/24/2017	NEW	$224,976
														Subtotal = $224,976

Grand Total All Awards = $224,976

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Preclinical Evaluation of Ancestral Coagulation Factor VIII

Award Number: R43HL135969

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 02/01/2017

PERIOD OF PERFORMANCE END DATE: 07/31/2018

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