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R44HL135965

Project Grant

Overview

Grant Description
Erythromer: Nanoscale Biosynthetic Red Cell Substitute - Research Summary/Abstract

Erythromer (EM) is a novel biosynthetic blood substitute developed to address the critically unmet need for emergency transfusion in situations where the use of banked red blood cells (RBCs) are either not available or undesirable. EM is a self-assembled lipid-oligomeric hybrid nanoparticle with a high per particle payload of hemoglobin (HB) and the allosteric modifier, RSR13.

EM is specifically designed to rectify failures of previous hemoglobin-based oxygen carriers, which do not preserve RBC physiology. The bio-inspired EM design surmounts previous obstacles by emulating RBC features: long-term stability, precise dynamically-responsive allosteric effector control of HB oxygen affinity, control of HB interaction with nitric oxide (NO), preventing vasospasm, and mitigation of hemoglobin oxidation by containment in the vascular compartment.

EM is designed for sterile lyophilization enabling extended shelf life at ambient conditions and offers cost-effective production at scale. Kalocyte has developed a pragmatic yet robust step-wise goal-oriented development plan for commercialization including meticulous evaluation of preclinical safety to support FIH dosing.

A systematic and rigorous in vitro, ex vivo, and animal model-based pre-clinical proof-of-concept strategy provides strong evidence supporting the premise for and feasibility of this proposal. Further, our commitment to characterize a comprehensive non-GLP pharmacokinetic and exploratory toxicology as well as an FDA vetted GLP toxicology plan for EM is elucidated.

To meet these milestones in a timely fashion, non-GLP dose-dependent pharmacokinetic studies of EM that probe tissue distribution, metabolism, and elimination, and exploratory toxicology studies will be conducted. Successful completion of these experimental studies will inform on pharmacokinetic behavior, potential dose-dependent safety signals, and product quantity needs, moving forward into GLP toxicology studies and eventually FIH dosing.

The described, comprehensive dose-dependent toxicology studies are tailored to meet FDA expectations that allow for dosing of EM in a Phase 1 clinical trial. This process requires escalating single dose toxicology study in rabbits expanded to 14 days for post-dose recovery. Parallel studies investigating cardiac electrophysiology, pulmonary hemodynamics, and systemic hemodynamics in a dog safety pharmacology study with an added toxicokinetic arm.

Completion of these IND-enabling studies provides necessary pre-clinical animal data qualifying Kalocyte to submit an IND package for EM. EM has the capability to dramatically transform care in situations where the adverse effects of stored RBCs exceed benefit and may enable novel efficacies. However, the most compelling use will be in settings where stored RBCs are unavailable or undesirable.
Awardee
Funding Goals
THE DIVISION OF BLOOD DISEASES AND RESOURCES SUPPORTS RESEARCH AND RESEARCH TRAINING ON THE PATHOPHYSIOLOGY, DIAGNOSIS, TREATMENT, AND PREVENTION OF NON-MALIGNANT BLOOD DISEASES, INCLUDING ANEMIAS, SICKLE CELL DISEASE, THALASSEMIA, LEUKOCYTE BIOLOGY, PRE-MALIGNANT PROCESSES SUCH AS MYELODYSPLASIA AND MYELOPROLIFERATIVE DISORDERS, HEMOPHILIA AND OTHER ABNORMALITIES OF HEMOSTASIS AND THROMBOSIS, AND IMMUNE DYSFUNCTION. FUNDING ENCOMPASSES A BROAD SPECTRUM OF HEMATOLOGIC INQUIRY, RANGING FROM STEM CELL BIOLOGY TO MEDICAL MANAGEMENT OF BLOOD DISEASES AND TO ASSURING THE ADEQUACY AND SAFETY OF THE NATION'S BLOOD SUPPLY. PROGRAMS ALSO SUPPORT THE DEVELOPMENT OF NOVEL CELL-BASED THERAPIES TO BRING THE EXPERTISE OF TRANSFUSION MEDICINE AND STEM CELL TECHNOLOGY TO THE REPAIR AND REGENERATION OF HUMAN TISSUES AND ORGANS. SMALL BUSINESS INNOVATION RESEARCH (SBIR) PROGRAM: TO STIMULATE TECHNOLOGICAL INNOVATION, USE SMALL BUSINESS TO MEET FEDERAL RESEARCH AND DEVELOPMENT NEEDS, FOSTER AND ENCOURAGE PARTICIPATION IN INNOVATION AND ENTREPRENEURSHIP BY SOCIALLY AND ECONOMICALLY DISADVANTAGED PERSONS, AND INCREASE PRIVATE-SECTOR COMMERCIALIZATION OF INNOVATIONS DERIVED FROM FEDERAL RESEARCH AND DEVELOPMENT FUNDING. SMALL BUSINESS TECHNOLOGY TRANSFER (STTR) PROGRAM: TO STIMULATE TECHNOLOGICAL INNOVATION, FOSTER TECHNOLOGY TRANSFER THROUGH COOPERATIVE R&D BETWEEN SMALL BUSINESSES AND RESEARCH INSTITUTIONS, AND INCREASE PRIVATE SECTOR COMMERCIALIZATION OF INNOVATIONS DERIVED FROM FEDERAL R&D.
Place of Performance
Baltimore, Maryland 212011514 United States
Geographic Scope
Single Zip Code
Analysis Notes
Amendment Since initial award the total obligations have increased 190% from $1,050,000 to $3,043,060.
Kalocyte was awarded Nanoscale Biosynthetic Red Cell Substitute: Erythromer Grant Proposal Project Grant R44HL135965 worth $3,043,060 from National Heart Lung and Blood Institute in May 2017 with work to be completed primarily in Baltimore Maryland United States. The grant has a duration of 9 years and was awarded through assistance program 93.837 Cardiovascular Diseases Research. The Project Grant was awarded through grant opportunity NHLBI SBIR Phase IIB Bridge Awards to Accelerate the Commercialization of Technologies for Heart, Lung, Blood, and Sleep Disorders and Diseases (R44 Clinical Trial Optional).

SBIR Details

Research Type
SBIR Phase II
Title
ErythroMer: Nanoscale BioSynthetic Red Cell Substitute
Abstract
Research Summary/Abstract ErythroMer (EM) is a novel biosynthetic blood substitute developed to address the critically unmet need for emergency transfusion in situations where the use of banked red blood cells (RBCs) are either not available or undesirable. EM is a self-assembled lipid-oligomeric hybrid nanoparticle with a high per particle payload of hemoglobin (Hb) and the allosteric modifier, RSR13. EM is specifically designed to rectify failures of previous hemoglobin-based oxygen carriers, which do not preserve RBC physiology. The bio-inspired EM design surmounts previous obstacles by emulating RBC features: long term stability, precise dynamically-responsive allosteric effector control of Hb oxygen affinity, control of Hb interaction with nitric oxide (NO), preventing vasospasm, and mitigation of hemoglobin oxidation by containment in the vascular compartment. EM is designed for sterile lyophilization enabling extended shelf life at ambient conditions and offers cost-effective production at scale. KaloCyte has developed a pragmatic yet robust step-wise goal oriented development plan for commercialization including meticulous evaluation of preclinical safety to support FIH dosing. A systematic and rigorous in vitro, ex vivo and animal model based pre-clinical proof-of-concept strategy provides strong evidence supporting the premise for and feasibility of this proposal. Further, our commitment to characterize a comprehensive non-GLP pharmacokinetic and exploratory toxicology as well as an FDA vetted GLP toxicology plan for EM is elucidated. To meet these milestones in a timely fashion, non-GLP dose dependent pharmacokinetic studies of EM that probe tissue distribution, metabolism, and elimination and exploratory toxicology studies will be conducted. Successful completion of these experimental studies will inform on pharmacokinetic behavior, potential dose dependent safety signals and product quantity needs, moving forward into GLP toxicology studies and eventually FIH dosing. The described, comprehensive dose dependent toxicology studies are tailored to meet FDA expectations that allows for dosing of EM in a Phase 1 clinical trial. This process requires, escalating single dose toxicology study in rabbits expanded to 14 days for post dose recovery. Parallel studies investigating cardiac electrophysiology, pulmonary hemodynamics, and systemic hemodynamics in a dog safety pharmacology study with an added toxicokinetic arm. Completion of these IND- enabling studies provides necessary pre-clinical animal data qualifying KaloCyte to submit an IND package for EM. EM has the capability to dramatically transform care in situations where the adverse effects of stored RBCs exceed benefit and may enable novel efficacies. However, the most compelling use will be in settings where stored RBCs are unavailable or undesirable. 1
Topic Code
NHLBI
Solicitation Number
HL23-009

Status
(Ongoing)

Last Modified 6/5/25

Period of Performance
5/11/17
Start Date
5/31/26
End Date
94.0% Complete

Funding Split
$3.0M
Federal Obligation
$0.0
Non-Federal Obligation
$3.0M
Total Obligated
100.0% Federal Funding
0.0% Non-Federal Funding

Activity Timeline

Interactive chart of timeline of amendments to R44HL135965

Transaction History

Modifications to R44HL135965

Additional Detail

Award ID FAIN
R44HL135965
SAI Number
R44HL135965-1886964540
Award ID URI
SAI UNAVAILABLE
Awardee Classifications
Small Business
Awarding Office
75NH00 NIH National Heart, Lung, and Blood Institute
Funding Office
75NH00 NIH National Heart, Lung, and Blood Institute
Awardee UEI
MJMKM31FM667
Awardee CAGE
7KNT0
Performance District
MD-07
Senators
Benjamin Cardin
Chris Van Hollen

Budget Funding

Federal Account Budget Subfunction Object Class Total Percentage
National Heart, Lung, and Blood Institute, National Institutes of Health, Health and Human Services (075-0872) Health research and training Grants, subsidies, and contributions (41.0) $1,050,000 100%
Modified: 6/5/25