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SMART BIOELECTRONIC IMPLANTS FOR CONTROLLED DELIVERY OF THERAPEUTIC PROTEINS IN VIVO AND ITS APPLICATION IN LONG-TERM TREATMENT OF HEMOPHILIA A

Award Number: R01EB031992
ORGANIZATION: NATIONAL INSTITUTE Of BIOMEDICAL IMAGING & BIOENGINEERING
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 05/01/2022
PERIOD OF PERFORMANCE END DATE: 01/31/2026

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SMART BIOELECTRONIC IMPLANTS FOR CONTROLLED DELIVERY OF THERAPEUTIC PROTEINS IN VIVO AND ITS APPLICATION IN LONG-TERM TREATMENT OF HEMOPHILIA A - Cell-based therapies, where naturally or artificially engineered cells secreting therapeutic proteins are grafted onto the body to act as biological drug factories, are an attractive approach for long-term treatment of chronic diseases such as hemophilia, diabetes and liver disorders. However, ‘off the shelf’ therapeutic cells are immunogenic to the host and must be protected from the host immune system. Cell-encapsulation has emerged as an attractive strategy to transplant these cells without chronic immunosuppression. Here, cells are placed in an immune-isolating device which physically separates the cells from the components of the immune system while providing access to oxygen and nutrients. Retrievable macroscale cell- encapsulation devices (macrodevice), are attractive in this context as they provide a safer path to clinical translation. Unfortunately, a standalone macrodevice that remains functional in humans over long-periods (>6 months) is yet to be realized due to two core challenges: 1) a foreign-body reaction to the implanted device causing inflammation and fibrosis, and 2) inadequate supply of oxygen and nutrients to the encapsulated cells. Here, we propose to build on several promising recent advances in biomaterials design, microfabrication, bioelectronics and cell engineering from our team to develop an advanced “smart” macrodevice platform with integrated electronic components which overcomes the major limitations of current device designs. First, we will develop an engineered cell line which is amenable to long term encapsulation and suitable for clinical translation. Landing pads within these cells will ensure stable transgene expression, allowing for broad control of therapeutic protein secretion (Aim 1). Separately, we will develop a bioelectronic macrodevice as a platform for long- term transplant of these cells in vivo. Our device will incorporate novel membranes with uniform/controlled pore-sizes and enhanced oxygen transport properties. In parallel, we will develop new surface coating techniques to minimize fibrosis and ensure long- term graft survival. We will integrate proton exchange membranes and optoelectronic components to allow a) in-situ oxygen generation, and b) optical gene activation to allow for triggerable control of protein production by the encapsulated cells (Aim 2). Finally, we will test the device in B6 mice using a model protein (SEAP) to test for long term survival of cells and external control of protein delivery. We will develop the device as a platform to delivery of Factor VIII for the treatment of Hemophilia A (Aim 3) as a model disease. If successful, the platform will represent a qualitative technological advancement in the field of cell therapy.


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 = $0 )
2026	2025	MASSACHUSETTS INSTITUTE OF TECHNOLOGY	77 MASSACHUSETTS AVE	CAMBRIDGE	MA	02139	MIDDLESEX	USA	Discovery and Applied Research for Technological Innovations to Improve Human Health	000	4	5/22/2026	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2025 ( Subtotal = $604,673 )
2025	2025	MASSACHUSETTS INSTITUTE OF TECHNOLOGY	77 MASSACHUSETTS AVE	CAMBRIDGE	MA	02139	MIDDLESEX	USA	Discovery and Applied Research for Technological Innovations to Improve Human Health	001	4	7/24/2025	NON-COMPETING CONTINUATION	$60,467
2025	2025	MASSACHUSETTS INSTITUTE OF TECHNOLOGY	77 MASSACHUSETTS AVE	CAMBRIDGE	MA	02139	MIDDLESEX	USA	Discovery and Applied Research for Technological Innovations to Improve Human Health	000	4	1/16/2025	NON-COMPETING CONTINUATION	$544,206
														Subtotal = $604,673

Issue Date FY: 2024 ( Subtotal = $592,580 )
2024	2024	MASSACHUSETTS INSTITUTE OF TECHNOLOGY	77 MASSACHUSETTS AVE	CAMBRIDGE	MA	02139	MIDDLESEX	USA	Discovery and Applied Research for Technological Innovations to Improve Human Health	000	3	1/23/2024	NON-COMPETING CONTINUATION	$544,206
2024	2024	MASSACHUSETTS INSTITUTE OF TECHNOLOGY	77 MASSACHUSETTS AVE	CAMBRIDGE	MA	02139	MIDDLESEX	USA	Discovery and Applied Research for Technological Innovations to Improve Human Health	001	3	5/27/2024	NON-COMPETING CONTINUATION	$48,374
														Subtotal = $592,580

Issue Date FY: 2023 ( Subtotal = $604,673 )
2023	2023	MASSACHUSETTS INSTITUTE OF TECHNOLOGY	77 MASSACHUSETTS AVE	CAMBRIDGE	MA	02139	MIDDLESEX	USA	Discovery and Applied Research for Technological Innovations to Improve Human Health	000	2	1/26/2023	NON-COMPETING CONTINUATION	$604,673
														Subtotal = $604,673

Issue Date FY: 2022 ( Subtotal = $604,673 )
2022	2022	MASSACHUSETTS INSTITUTE OF TECHNOLOGY	77 MASSACHUSETTS AVE	CAMBRIDGE	MA	02139	MIDDLESEX	USA	Discovery and Applied Research for Technological Innovations to Improve Human Health	000	1	4/29/2022	NEW	$604,673
														Subtotal = $604,673

Grand Total All Awards = $2,406,599

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SMART BIOELECTRONIC IMPLANTS FOR CONTROLLED DELIVERY OF THERAPEUTIC PROTEINS IN VIVO AND ITS APPLICATION IN LONG-TERM TREATMENT OF HEMOPHILIA A

Award Number: R01EB031992

ORGANIZATION: NATIONAL INSTITUTE Of BIOMEDICAL IMAGING & BIOENGINEERING

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 05/01/2022

PERIOD OF PERFORMANCE END DATE: 01/31/2026

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