HeartWorks has created a one-of-a-kind platform where doctors, scientists, and engineers work together to develop new treatments for the most severe forms of congenital heart defects (CHD). By advancing research in genetics, stem cell biology, clinical trials, and cell-based manufacturing, we’re turning discoveries into real solutions.
To move treatments from the lab into clinical trials—faster and in collaboration with top medical institutions. We believe our therapies have the potential to become a cure, so children and adults with CHD can look forward to a future without it.
A database of members medical journey data to inform future clinical innovation and design of clinical trials which address the needs of the members.
We’re collecting a small skin biopsy sample to create patient-specific stem cells. These cells are made for promising regenerative cardiac therapies which you may be eligible for in the future.
Skin cells that are manufactured into contracting cardiac lineage to be delivered into the heart muscle to determine if it will strengthen the heart.
On November 22, 2022, Bri had open-heart surgery for a congenital heart defect that we discovered when she was 21 years old. This heart defect is called an anomalous origin
“I was diagnosed at birth with Ebsteins Anomaly! Where my tricuspid valve wasn’t formed properly in the womb, it’s a rare congenital heart defect that affects only 1 in every
“We learned of our son had Hypoplstic Left Heart at our 20 week ultrasound. We were devastated to learn he would have half a heart and need so many surgery
“Hi! My name is Lauren. I’m 36 years old (I’ll be 37 on August 25!) and was born with CHDs called: Tricuspid Atresia, Hypoplastic Right Ventricle, a Large VSD, and
“Cora was born in September 2020 with double inlet left ventricle, transposed great arteries, and coarctation of the aorta. She underwent the Norwood procedure when she was 4 days old.
“Luke was born in December of 2019 after a uncomplicated, normal pregnancy. His delivery was as smooth as could be. He came out pink with a full head of hair!
At HeartWorks, critical advances in medicine start with foundational research, paving the way for innovative and effective therapies. Our Research and Development (R&D) team is at the forefront of this journey, driving our efforts to enhance and refine our bioengineered cell therapies.
A central focus of our R&D team is the in-depth characterization of cells used in our cardiomyocyte-based therapies. We begin with patient skin cells, which are converted into induced pluripotent stem cells (iPSCs). These iPSCs are then selectively differentiated into functional cardiomyocytes, or heart muscle cells. This process is intricate and involves creating bioengineered cell products that include various sub-types of cardiomyocytes.
Our R&D team has developed advanced methods to analyze these cell sub-types, gaining insights into their molecular characteristics and clinical potential. In 2023, we introduced two key approaches to advance this work:
The ultimate goal of our genetic and electrical studies is to develop patient-specific cardiomyocyte preparations that optimize heart muscle function and enhance therapeutic outcomes. Through these advancements, HeartWorks is committed to pushing the boundaries of medical research and delivering cutting-edge solutions to improve patient care.
HeartWorks innovative bioengineered cell therapies rely heavily on our exceptional manufacturing team. This team is essential in executing the intricate, multi-step processes required to transform patient skin cells into induced pluripotent stem cells (iPSCs), expand these stem cells, and then differentiate them into functional cardiomyocytes (heart muscle cells) for our therapies.
Our manufacturing processes have evolved continuously, with the development of robust, FDA-approved procedures crucial for our clinical trials. The achievements of our manufacturing team in 2023 reflect substantial progress, as demonstrated by the dramatic increase in cardiomyocyte yield:
2015 | 2017 | 2019 | 2023 | future |
100K | 4M | 300M | 3B | ++ |
This impressive scaling of production capacity highlights the innovation, productivity, and quality of our manufacturing operations. In 2023, our team enhanced batch sizes by increasing the number of vessels used for each sample, proving to be more effective than altering vessel sizes or designs. Additionally, we optimized productivity through refined synchronization of manufacturing activities.
Looking ahead, as patient numbers and clinical trials increase, we plan to incorporate bioreactors. These advanced systems will provide optimized conditions for producing cardiomyocytes, further advancing our ability to meet growing demand and continue delivering groundbreaking therapies.
Our manufacturing team’s dedication is a cornerstone of HeartWorks’ success, enabling us to push the boundaries of medical research and provide cutting-edge treatments to patients.
Our Co-op program addresses key challenges in managing patient records by providing a centralized, patient-controlled database. This system securely aggregates medical records from all providers, if the patients choose, making it easier for CHD patients and their families to access and manage their health information for their care and for use by HeartWorks and potentially other researchers.
The Co-op will be a major contributor to the heart community and to HeartWorks transforming patient care for CHD patients and their families.
Become a member of The Co-op here.
Clinical Research includes all research involving human participants. It does not include secondary studies using existing biological specimens or data collected without identifiers or data that are publicly available. Clinical research is necessary for new discoveries and treatments options to become available to patients on a routine basis (become standard of care), with the goal of improving outcomes.
Clinical Trials are a type of clinical research study involving human participants assigned to an intervention in which the study is designed to evaluate the effect(s) of the intervention on the participant and the effect being evaluated is a health-related biomedical or behavioral outcome. This is synonymous with an Interventional Study by NIH definition.
Observational study are research studies in which researchers simply collect information (called data) from participants or look at data that was already collected. Often, researchers use observational studies to look at (observe) the different ways people behave and how it affects their health. Some observational studies use patient registries. A patient registry is an organized collection of data that patients agree to give. Researchers can use a patient registry to quickly access data provided by hundreds, or thousands, of similar patients.
A clinical trial sponsor is an individual, company, or organization that manages and funds a clinical trial. The sponsor is responsible for the trial’s design, conduct, and outcome. HeartWorks is the “Sponsor” of several clinical trials, listed above.
A clinical trial site (or clinical site) refers to a physical location, such as a hospital, research center, or medical facility, where the clinical trial activities are conducted. HeartWorks has established a international consortium which includes several of the top pediatric CHD care centers in North America, and plans are underway to identify similar partner medical centers to be sites for our adult CHD clinical trials.
There are a number of ways to identify clinical trials or research studies. Results may include both studies currently open to enrollment, not yet enrolling, or completed.
• https://clinicaltrials.gov/
• https://www.mayo.edu/research/clinical-trials
