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| 24443 | N3C in Action | .not-last { margin-bottom: 20px; } The N3C is a partnership among the NCATS‑supported Clinical and Translational Science Awards (CTSA) Program hubs, the National Center for Data to Health (CD2H), and NIGMS‑supported Institutional Development Award Networks for Clinical and Translational Research (IDeA‑CTR), with overall stewardship by NCATS. February 2023 NIH RECOVER Research Identifies Potential Long COVID Disparities A study used electronic health record data from the N3C Data Enclave to examine patterns of more than 30,000 people diagnosed with long COVID. Researchers found that most patients in the study were White, female, non-Hispanic and likely to live in areas with low poverty and greater access to health care, suggesting that not all patients who have long COVID are being diagnosed. Learn more about the study. October 2022 N3C Effort to Speed COVID-19 Public Health Answers NCATS has launched a new effort to deliver fast, actionable analyses on pressing COVID-19 questions. The effort is called N3C Public Health Answers to Speed Tractable Results (PHASTR). It will leverage N3C’s collection of clinical data representing more than 16 million anonymized individuals. NCATS will post a series of timely questions about COVID-19 health outcomes that currently are not studied within the N3C. Individuals or teams then can submit proposals on how they would use the N3C Data Enclave to answer specific questions. The initial questions look at whether earlier findings on reinfection and long-term neurologic disorders among mostly white males are similar to findings from N3C’s more demographically diverse data set. NCATS will publish results from the analyses on the N3C Public Health Dashboards and disseminate them to public health officials. See more details, including FAQs. N3C PHASTR also is funded by the NIH National Institute of General Medical Sciences. N3C Data Reveal COVID-19 Hospitalization Increases Risk of Heart Failure Using data from more than half a million electronic health records in the N3C Data Enclave, researchers found that a greater percentage of people who had been hospitalized with COVID-19 developed heart failure than people who had been hospitalized without COVID-19. Learn more about this study. September 2022 Researchers Use N3C Data to Analyze Rural Versus Urban COVID-19 Hospitalization Rates Using electronic health records in the N3C Data Enclave, researchers discovered health disparities in COVID-19-related hospitalizations in rural communities compared to urban communities of the United States. The N3C allowed researchers to examine these findings more in-depth than in a traditional population-based study. Learn more about this research study and findings. May 2022 Study Finds Immune Dysfunction Is a Significant Risk Factor for COVID-19 Breakthrough Infection Researchers used data from the N3C Data Enclave to identify a link between COVID-19 breakthrough infections and compromised immune systems, including in individuals living with HIV or receiving immunosuppressant medications. A breakthrough infection is an infection that happens at least 14 days after full vaccination. The study also highlighted the importance of people staying up to date with vaccinations and providers recommending other risk-reduction steps. Learn more about the study. Scientists Identify Characteristics to Better Define Long COVID Long COVID is a condition marked by wide-ranging symptoms, including shortness of breath, fatigue, fever, headaches, “brain fog” and other neurological problems. Such symptoms can last for months or longer after an initial COVID-19 diagnosis. To get a better understanding of long COVID and the individuals who are likely to have it, NIH-supported researchers used machine learning techniques to analyze an unprecedented collection of de-identified electronic health records in NCATS’ N3C Data Enclave. Read the full news release. Researchers Use Cutting-Edge EHR Data Resource to Find Risk Factors for Severe COVID-19 in Children Using data from NCATS’ N3C Data Enclave, researchers studied electronic health records from more than 1 million patients ages 18 and younger who were tested for SARS-CoV-2. Of those children who tested positive, some developed a dangerous condition called multisystem inflammatory syndrome in children (MIS-C), in which some organs become inflamed. NCATS-supported researchers were able to find risk factors for the children with MIS-C. Doctors can account for these risk factors when making medical decisions. If pediatricians know which patients are most at risk, they can give those patients more aggressive treatments to prevent hospitalization and harm from COVID-19. Read the article. March 2022 N3C Study Ties Aspirin Use in Moderate COVID-19 to Lower Risk of Blood Clots and Death Giving aspirin to people hospitalized with moderate COVID-19 may reduce their risks of blood clots in the lung and of death, according to a large study of patient information from the N3C Data Enclave. Researchers examined health records from a racially and ethnically diverse group of 112,269 people in the United States with moderate COVID-19 that required a hospital stay. They compared people who didn’t receive aspirin when they entered the hospital with those who did. Patients given aspirin were 29% less likely to have a pulmonary embolism, in which a blood clot blocks an artery in the lung, and 14% less likely to die over the next 4 weeks. The reduced risks were most apparent in people given aspirin who were older than 60 years or who had at least one medical condition other than COVID-19. Learn more about the N3C-based findings on aspirin. February 2022 N3C Reveals COVID‑19 Hospitalization and Death Risks Among People with Type 2 Diabetes The N3C database has helped reveal that blood sugar levels may predict which people with type 2 diabetes and acute COVID‑19 infection are at greater risk of hospitalization or death. Researchers examined N3C patient health records to track outcomes for a diverse population of more than 39,000 people across the United States. Overall, they found that among people whose hemoglobin A1c levels — a measure of blood sugar — were above 7%, there were increased rates of hospitalization, ventilator use and death. Higher blood sugar levels posed a much greater risk of death for people who were Hispanic or Latino than for those who were Black or African American, white, Asian American, or Pacific Islander. Because people with diabetes can monitor their own hemoglobin A1c levels at home, they could use these measurements to better predict their own risks for COVID‑19 complications. Learn more about the N3C‑driven study findings. August 2021 N3C Database Reveals Increased COVID‑19 Mortality Risk in People with COPD Results from an N3C study showed that people with chronic obstructive pulmonary disease (COPD) were more likely to be hospitalized and die of COVID‑19 than people without COPD. Researchers examined patient records in the N3C Data Enclave. The researchers reviewed data from thousands of people who tested positive for COVID‑19 and had COPD. They compared these data with data from people who tested positive but did not have COPD. COPD is the most common chronic lung disease in the United States. It includes such conditions as emphysema and bronchitis. COPD disproportionately affects women and older adults. September 2020 Using Existing Partnerships to Build a National COVID‑19 Data Analytics Platform Investigators have collected huge amounts of COVID‑19 data, but accessing and combining that data in a useful way is challenging. To solve this problem, NCATS partnered with the CTSA Program hubs, the CD2H, and more than 70 research institutions to develop a centralized national data resource for COVID‑19 data: the N3C Data Enclave. The N3C has information from more than 4.9 million diverse patients with COVID‑19. That information is accessible through the N3C Cohort Explorer. The secure database helps researchers conduct studies related to COVID‑19 infection. More than 300 research projects are currently using N3C data. The researchers evaluated N3C data for trends. The data included vaccination status, COVID‑19 diagnoses, immune dysfunction diagnoses, comorbid conditions and demographics data. The N3C enclave is one of the largest collections of COVID‑19 clinical data in the United States. It was critical to this research effort. The data enclave eliminated the need for testing centers and widespread recruitment efforts. Such research components, among others, can pose challenges to studies. | Through N3C, NCATS and its researchers study health data to speed COVID-19 research and treatments. | N3C in Action | Through N3C, NCATS and its researchers study health data to speed COVID-19 research and treatments. | N3C in Action | ||||
| 23551 | Application Information for RFA-TR-22-012: Emergency Awards: HEAL Initiative-Limited Competition: HEAL Initiative Resource Centers for the Pain Management Effectiveness Research Network (ERN) (U24 Clinical Trial Not Allowed) | li { color: #000; } .panel-heading, p > strong, li > strong { color: #333; } .panel-body { padding: 0 15px; } h2 { font-size: 20px; } Please note: The primary source of all information regarding RFA-TR-22-012: Emergency Awards: HEAL Initiative-Limited Competition: HEAL Initiative Resource Centers for the Pain Management Effectiveness Research Network (ERN) (U24 Clinical Trial Not Allowed) is the funding opportunity itself and any notices (i.e., NOT-TR-22-018) linked therein. Eligibility See More Please clarify who is eligible to serve as a principal investigator (PI) for this funding opportunity announcement (FOA). Is the Trial Innovation Network (TIN) PI required to be listed as a PI? And, if so, is the TIN PI required to be listed as the contact PI or can another PI serve as the contact PI? Eligibility for this FOA is limited to current NCATS TIN recipients. The FOA does not limit who may serve as PI. However, there is a clear expectation that the program director(s) and/or PI(s) have expertise and experience conducting multicenter trials or analysis of clinical data to achieve the scientific objectives of the HEAL ERN, as well as significant experience for the type of HEAL Resource Center specified. Relevant text from RFA-TR-22-012: Eligibility to apply to this FOA is limited to current NCATS TIN recipients supported under RFA-TR-15-002 and RFA-TR-15-004. Page Limitations See More Please confirm that the total page limitations — as described in the SF424 application guide and the Table of Page Limits — must be followed exactly. For a U24 mechanism, the limitation is prescribed as 12 pages for the Research Strategy plus the Specific Aims. What is the total page limit, including the Specific Aims for this application? Correct: Applications may not exceed the prescribed limitations. The Research Strategy section is limited to 12 pages plus one page for the Specific Aims. Relevant text from RFA-TR-22-012: All page limitations described in the SF424 Application Guide and the Table of Page Limits must be followed. Budget/Funding See More When is the anticipated start date for the HEAL award? The earliest start date will be July 2022. The number of additional trials is unstated; we are planning to budget for bringing our existing HEAL ERN trials to completion and adding two trials that would start planning in Year 1. Is this appropriate? Yes. Please see the companion RFA (RFA-AT-22-005) to learn more about the possible two new trials that would begin planning in Year 1. Relevant text from RFA-TR-22-012: Years 1–5: The budget must include allocations to continue support for the trial currently funded through RFA-NS-20-028 and two additional HEAL ERN trials/studies estimated to begin the first planning phase in Year 1. Note: There is an expectation that annual budgets will decrease as enrolling studies are completed and that final budgets will depend on the number of new trials beginning the planning phase in Year 1. Please refer to the limitations as set in the funding opportunity announcement: Data Coordination Resource Center (DCRC) Year 1: Not to exceed $2 million direct costs; Total Direct Costs for 5 years not to exceed $8 million. Clinical Coordinating Resource Center (CCRC) Year 1: Not to exceed $2 million direct costs; Total Direct Costs for 5 years not to exceed $6.5 million. Statistical and Safety Resource Center (SSRC) Year 1: Not to exceed $1.5 million direct costs; Total Direct Costs for 5 years not to exceed $4.5 million. Recruitment Resource Center (RRC) Year 1: Not to exceed $750,000 direct costs; Total Direct Costs for 5 years not to exceed $2.5 million. For Years 2–5, there are no direct cost limits per year; however, the total direct costs for the five-year budget period cannot exceed the limitations prescribed above. Relevant text from RFA-TR-22-012: Years 1–5: The budget must include allocations to continue support for the trial currently funded through RFA-NS-20-028 and two additional HEAL ERN trials/studies estimated to begin the first planning phase in Year 1. Note: There is an expectation that annual budgets will decrease as enrolling studies are completed and that final budgets will depend upon the number of new trials beginning the planning phase in Year 1. Years 2–5: The budget also must include designated expenses in Years 2–5 to provide HEAL ERN Resource Center services for currently funded HEAL ERN clinical trials with active participant enrollment supported through RFA-NS-19-021. Budget Clarifications Current Trial Innovation Network (TIN) award unexpended balances will provide support for the four UH3 trials that were funded starting in fiscal year (FY) 2019 (SurgeryPal, POSITIVE, RESOLVE and SKOAP) through June 30, 2023. Starting July 1, 2023, these four trials will be supported by funds from the new U24 awards solicited by RFA-TR-22-012. Funds for support of TIN infrastructure for the Optimizing the Use of Ketamine to Reduce Chronic Postsurgical Pain study and two new sickle cell pain management trials will be provided by awards funded through RFA-TR-22-012, starting in FY 2022. PLEASE ALLOW ADEQUATE TIME TO REQUEST USE OF ANY UNOBLIGATED BALANCES FROM THE CURRENT TIN AWARDS. Research Plan See More The RFA lists a specific activity for the Clinical Coordinating Resource Center (CCRC), Data Coordination Resource Center (DCRC) and Statistical and Safety Resource Center (SSRC) to plan to act as the single Institutional Review Board (sIRB) for at least one ERN study. All three Resource Centers are currently supporting one sIRB, but only two additional trials are anticipated. Therefore, it makes sense for only the DCRC and CCRC to budget for an additional sIRB. All three Resource Centers would continue the sIRB support that we are already providing for existing studies. Is it acceptable that the SSRC not be required to plan for an additional sIRB? The CCRC, DCRC and SSRC should each present a plan to provide new and/or continuing sIRB support to HEAL ERN trials. There is an obvious intent for collaboration among the four Resource Centers if the award is made, and the four Resource Centers have been collaborating for several years to support HEAL ERN trials. Because applicants are supposed to demonstrate synergy, it seems sensible that grant proposals should contain “common items,” such as the anticipated timeline for trial activities in all seven trials and the summary information about ERN accomplishments to date. This could result in sections of our four proposals that are identical, facilitating review of the applications. However, we do not want to create a perception of inappropriate collusion that would be problematic. In the original HEAL supplement applications, the first several pages of our applications were identical, but these were reviewed administratively, not by a study section. Is it acceptable for us to incorporate common identical components in our applications? Each application is evaluated individually and needs to contain within it all information needed for its review. Other Questions See More Will applicants be allowed to roll over their unexpended funds into this new award? No. The unexpended funds from the current Trial Innovation Network (TIN) awards cannot be transferred to this new award. Please note that any current TIN award unexpended HEAL supplement funds from previous years or Year 7 HEAL supplements must be expended by the end of the project period (June 30, 2023). For Year 7 funding from the current supplement, should applicants assume the amount is the amount quoted in our current Notice of Grant Award (NOGA) under Future Funding Commitments? This is on page 7 of 11 in my NOGA from Sept. 3, 2021. The expectation is that the Year 7 HEAL supplements for the current TIN awards will be the committed level stated in the Notice of Award minus any HEAL unobligated balances. The goal is to expend all HEAL supplement dollars obligated to the current TIN awards by the end of the project period (June 30, 2023). Therefore, NCATS will aim to utilize any remaining HEAL unobligated balances as an offset for the final Year 7 award. Will this award allow a cost extension if the trials in which successful applicants are serving are extended beyond their current award periods? Future potential extensions or cost extensions may be permitted per Section 8.1.2.13 of the NIH Grants Policy Statement. function toggleText(pn) { var elem = document.querySelector(pn); if (elem.textContent === 'See More') { elem.textContent = 'See Less'; } else { elem.textContent = 'See More'; } } | Application Information for RFA-TR-22-012: Emergency Awards | Application Information for RFA-TR-22-012: Emergency Awards | ||||||
| 23470 | Multidisciplinary Machine-Assisted, Genomic Analysis and Clinical Approaches to Shortening the Rare Diseases Diagnostic Odyssey | The Need for New Approaches to Diagnose Rare Diseases More Quickly For many people with rare diseases, getting a correct diagnosis can take years and lots of visits to different doctors. During this “diagnostic odyssey,” people with rare diseases might have unnecessary tests and procedures, receive the wrong diagnosis, and experience delays in getting effective care. This long timeline means that many people with rare diseases experience irreversible damage as the disease progresses. Some may miss points in time when interventions could help. To improve the lives of people with rare diseases, NCATS wants to find innovative ways to shorten the timeline for getting a correct diagnosis. To do this, it has awarded funding to support three research projects that will study new tools and approaches to make it easier to correctly diagnosis people with rare diseases. About the Research Each of the three research projects is exploring a different approach to speed up the timeline for a correct diagnosis. These approaches include machine learning, genetic analyses and medical evaluation. To be successful, the approaches must be easy to apply and used early in patient care by front-line health care providers. Funding for the awards comes in two phases. Research teams must meet goals in the first phase before they move on to the next. In the first phase, researchers must develop a strategy for using their proposed approach to make faster diagnoses and then test that strategy in a real-world setting. In the second phase, researchers will test their strategy with patients in a different health care setting that presents new challenges or obstacles. For more information, contact Alice Chen Grady, M.D. or Eric W.K. Sid, M.D., M.H.A. Multidisciplinary Machine-Assisted, Genomic Analysis and Clinical Approaches to Shortening the Rare Diseases Diagnostic Odyssey (UG3/UH3 Clinical Trial Optional) Principal Investigator(s) Year Awarded Institution Title Gelb, Bruce D.; Chen, Rong; Balwani, Manisha 2022 Icahn School of Medicine at Mount Sinai Using Electronic Medical Record Data to Shorten Diagnostic Odysseys for Rare Genetic Disorders in Children and Adults in Two New York City Health Care Settings Gropman, Andrea Lynne; Berger, Seth I.; Vilain, Eric J. 2022 Children’s Research Institute Machine-Assisted Interdisciplinary Approach for Early Clinical Evaluation of Neurodevelopmental Disorders Lalani, Seema R.; Lee, Brendan 2022 Baylor College of Medicine Virtual Platforms for Genetics Evaluation in the Medically Underserved Visit our rare diseases web page to find information about other rare diseases research. | Research funded by NCATS seeks to shorten the “diagnostic odyssey” for patients with rare diseases. | Multidisciplinary Approaches for Diagnosing Rare Diseases More Quickly | Research funded by NCATS seeks to shorten the “diagnostic odyssey” for patients with rare diseases. | Multidisciplinary Approaches for Diagnosing Rare Diseases More Quickly | ||||
| 23242 | About the National COVID Cohort Collaborative | .n3c-about { padding: 0; } .intro-block { margin: 20px 0; } .intro-block-left { padding: 0; } .card.card-feature { margin-top: 3px; } .card-feature .card-body { background-color: #E3EBED; padding: 20px; } .card-feature .card-header { background-color: #30787D; padding: 1.5rem 1.75rem; } .card-feature h4 { color: #30787D; font-weight: bold; } .card-feature img{ max-width:100px; } h3.card-title { color: #fff; margin-bottom: 0; font-weight: 600; font-size: 26px; margin: 0; letter-spacing: 1px; } .card-body ul { padding-left: 10px; } .video-container { position: relative; padding-bottom: 56.25%; padding-top: 30px; height: 0; overflow: hidden; } .video-container iframe, .video-container object, .video-container embed { position: absolute; top: 0; left: 0; width: 100%; height: 100%; } @media (max-width: 991px) { .card.card-feature { margin-top: 20px; } } /* Latest News block*/ .news { background-image: url(/files/map-bg1.jpg); background-size: contain; padding: 0; position: relative; margin-bottom: 5px; margin-top: 15px; background-repeat: no-repeat; } .news-text { padding: 1%; background-color: #e4e6f0; opacity: 1; margin: 40% 0% 0% 0%; } .news-text ul { padding-left: 20px; } .news h3 { font-size: 2.3rem; color: #FFF; letter-spacing: 2px; text-transform: uppercase; background-color: #d86905; display: inline-block; padding: 10px 30px; position: absolute; top: 91px; box-shadow: 3px 3px 6px 0 rgba(0, 0, 0, 0.15); z-index: 3; } @media (max-width: 1199px) { .news h3 { font-size: 1.8rem; } } @media (max-width: 767px) { .news h3 { font-size: 2.3rem; } } @media (min-width: 768px) { .news h3 { top: 0; } .news-text { margin: 0% 0% 0% 40%; } } .content-card { background:#e4e6f0; height:100%; overflow:auto; margin-top: 10px; } .content-card h4{ background:#662e6b; color:#fff; text-align:center; font-size:16px; padding:.5em; height:55px; margin:0px; } .content-card ul{ padding:1em 0.5em 1em 2em; } .n3c-table { margin-bottom:1em; } @media (min-width: 768px) { .n3c-table { display: flex; } .n3c-table .col-sm-3 { padding-left:10px; padding-right:10px; } } Updates from N3C National COVID Cohort Collaborative (N3C) Data Enclave More than 7 million COVID-19-positive patients and more than 22.5 billion rows of data are included in this enclave. Apply for N3C access today. The public-facing N3C Dashboards provide high-level information about the N3C cohort and N3C Data Enclave. More than 400 projects are underway using the enclave data to examine associations between COVID-19 patient outcomes and social determinants of health. View the current list of publications. The N3C Data Enclave has a library of more than 30 external data sets, including mortality, viral variance and environmental data, that can be linked to the clinical data. NCATS has launched the N3C Public Health Answers to Speed Tractable Results (PHASTR) to deliver fast, actionable analyses on pressing COVID-19 questions. The National COVID Cohort Collaborative (N3C) maintains one of the largest collections of clinical data related to COVID-19 symptoms and patient outcomes in the United States. With stewardship from NCATS, more than 75 institutions worked together to build this extensive database. Having access to a large, centralized data resource allows research teams to study COVID-19 and identify potential treatments as the pandemic evolves. Learn the facts about the N3C by downloading and sharing the N3C fact sheet (PDF - 382KB). On this page: What is the N3C? How does the N3C improve public health? What data does the N3C have and where does it come from? How does the N3C keep data secure and protect patient privacy? What is the N3C? The N3C is a partnership among many organizations to provide clinical data in close to real time to improve our knowledge of COVID-19 and potential treatment strategies. The N3C effort is centered on the following: Establishing a secure data repository (the N3C Data Enclave) for studying COVID-19-related data. Receiving existing patient data derived from electronic health records (EHRs) provided by participating U.S. health care sites. Providing operational support for researchers using the N3C Data Enclave to navigate in the N3C platform and collaborate on COVID-19 research. Ensuring research using the N3C Data Enclave follows the rules and expectations of NCATS and its partners to keep the data secure and protect patient privacy. Since September 2020, the N3C has made data accessible to more than 3,000 researchers and clinicians to study the progression of COVID-19, identify risk and protective factors, search for effective treatments, understand the long-term disease effects, and determine how best to care for those with the disease. N3C partners include the following: Health care providers that provide the data in the N3C, including NCATS Clinical and Translational Science Awards (CTSA) Program hubs and the institutions supported by the NIH National Institute of General Medical Sciences’ Institutional Development Award Networks for Clinical and Translational Research (IDeA-CTR). The National Center for Data to Health (CD2H), which guides and governs the collaborative science environment within the N3C and also serves as the CTSA Program’s informatics coordinating center. NCATS, which provides governance, oversight and the secure research platform — the N3C Data Enclave — to maintain and protect the data. The scientific community and research leaders with data science and clinical expertise who harmonize data so that it can be studied together and compared across the nation. Learn more about key partners: NCATS CTSA Program NIGMS IDeA-CTR CD2H How does the N3C improve public health? Identifying the Public Health Need Since the beginning of the pandemic, health care providers and researchers have worked diligently to understand the novel SARS-CoV-2 virus and the disease it causes, COVID-19. Our understanding of COVID-19 — its signs, symptoms and effective courses of treatment — at the start of the pandemic was very limited. As this disease spread rapidly through communities, cities and countries, many clinics and hospitals collected important patient health information. However, when data are collected at different institutions and in different formats, it is difficult to put them together in a way that helps researchers and doctors understand the characteristics of the disease. The medical and research communities urgently need large amounts of data to better understand COVID-19, including how it spreads, who is most at risk, which treatments help, and what the effects of the disease are, including long-term effects. Making COVID-19 Data More Available for Research In response to this urgent need, NCATS and its partners developed the N3C to collect existing EHR data from hospitals and clinics and to make these data available to researchers seeking to understand COVID-19. The N3C receives patient information from more than 60 health care institutions across the country. NCATS harmonizes data from these institutions into a single format and makes them available for researchers and clinicians inside the N3C Data Enclave so they can study COVID-19 and potential treatments as the pandemic evolves. The N3C Data Enclave is a secure, cloud-based research environment with a powerful analytics platform provided by NCATS, which serves as the steward of N3C’s data. Data cannot be removed from the N3C Data Enclave. Since the N3C Data Enclave opened to researchers in September 2020, researchers have used the data to improve our understanding of COVID-19 and health equity, diabetes, cancer, COVID-19 medications and chronic obstructive pulmonary disease. Researchers currently are studying HIV and COVID-19 risk, mortality rates in rural populations, long COVID and much more using the N3C Data Enclave. Learn more: Browse N3C Data Enclave research projects. See a list of publications related to the N3C. Explore the N3C public health data browser. What data does the N3C have and where does it come from? The N3C's data come from existing patient records at participating institutions. The N3C receives data derived from EHRs of people who were tested for COVID-19 or who had related symptoms. EHRs include such information as age, sex, height and weight, medical history, lab results, health issues, medications, and treatments. Participating partners and other collaborators provide data to the N3C after they execute a Data Transfer Agreement with NCATS. The N3C harmonizes the data and manages it in a way that maintains the data’s validity while protecting patient privacy. Participating institutions do not obtain consent from individual patients for the data they send to the N3C. The 1996 Health Insurance Portability and Accountability Act (HIPAA) allows medical and health care institutions to release data for research without obtaining an individual’s authorization if direct identifying information is removed and appropriate oversight and agreements are in place. Under the HIPAA Privacy Rule requirements, these institutions can release what is called a limited data set. This is what participating health sites send to the N3C. The data set is “limited” because it leaves out 16 types of direct identifying information about the patient and their relatives, employers, or household members — such as names, account numbers, telephone numbers, email addresses and social security numbers. A limited data set may include city, state, ZIP code and elements of dates. The limited data set that the N3C receives includes ZIP codes and dates of service because these are critical for tracking the progress of the pandemic over time and place. The N3C does not contain direct identifying information, and additional measures have been put in place to protect patient privacy. As a result, NCATS received a waiver of consent from an NIH Institutional Review Board, conforming to the Federal Policy for the Protection of Human Subjects (“Common Rule”). Learn more: Download the Data Transfer Agreement. See the institutions that have executed a data transfer agreement with NCATS. Read about the HIPAA Privacy Rule. See the full list of information excluded from limited data sets. How does the N3C keep data secure and protect patient privacy? NCATS knows that the data it receives represent people and, as the steward of the data, NCATS takes its responsibility for keeping those data safe very seriously. NCATS has taken a comprehensive approach to address the security of the N3C Data Enclave and to protect patient privacy. It has invested significant time, resources and effort to keep N3C data private and secure. NCATS follows all applicable policies and regulations, has integrated key privacy measures into the N3C Data Enclave and its governance processes, and performs security testing and monitoring of activity inside the N3C Data Enclave. It also requires researchers to, among other things, adhere to a code of conduct, sign an agreement with NCATS outlining terms and conditions for using the data, and take NIH information technology security training. The table below — showing the N3C’s four pillars of data protection — provides additional detail about the steps NCATS takes to keep data secure and protect patient privacy. Regulatory and Policy Data-contributing sites abide by the HIPAA Privacy Rule N3C research is subject to the Federal Policy for the Protection of Human Subjects in research ("Common Rule") Data are provided as a HIPAA-defined limited data set NIH IRB oversight and waiver of consent For COVID-19–related research only No genomic data No emergency public health authorities were used to obtain the data under these conditions Engaged in an NIH Tribal Consultation regarding use of American Indian and Alaska Native (AI/AN) data Privacy Measures Certificate of Confidentiality Data stay within the N3C Data Enclave: No download or capture of raw data Privacy Impact Assessment Review of project requests by the Data Access Committee Full five-digit ZIP codes will never be shown for AI/AN demographic data Security Testing and Monitoring Federal government–compliant enclave managed by NCATS Meets government security controls for cloud security and privacy Data encryption in transit and at rest, without exception Scheduled penetration testing Active monitoring and logging by NIH and HHS Auditing of activities in the N3C Data Enclave Researcher Responsibilities A user's organization signs a Data Use Agreement with NCATS for terms and conditions of use Users adhere to the N3C Data User Code of Conduct Required NIH IT security training Required Human Subjects Research Protection training Follow N3C’s Community Guiding Principles Users attest that they understand that use of AI/AN data and ZIP code information to make assumptions about Tribal affiliation is not valid, or permitted Learn more: See our Privacy and Security FAQs. Read the requirements researchers must meet to access N3C data. Read the N3C Data User Code of Conduct. Access the N3C Community Guiding Principles document. If you have questions about the N3C, please email NCATS_N3C@nih.gov. | The N3C is a partnership to create a national resource of COVID-19 clinical data to answer critical research questions. | About the National COVID Cohort Collaborative | The N3C is a partnership to create a national resource of COVID-19 clinical data to answer critical research questions. | About the National COVID Cohort Collaborative | ||||
| 23050 | Translational Science Principles | .colaborative { background-color: #eaedf2; padding: 0; margin-top: 40px; } .card-copy { padding: 4%; display: flex; } .image { padding: 0; } .copy { display: flex; margin: auto; } .card.card-feature { margin-top: 3px; } .card-feature .card-body { background-color: #E3EBED; padding: 20px; } .card-feature .card-header { background-color: #30787D; padding: 1.5rem 1.75rem; } .card-feature h4 { color: #30787D; font-weight: bold; } .card-feature img{ max-width:100px; } h3.card-title { color: #fff; margin-bottom: 0; font-weight: 600; font-size: 26px; margin: 0; letter-spacing: 1px; } .col-md-12 { padding-left: 0; padding-right: 0; } .card-body p { font-weight: bold; color: #30787D; font-size: 15px; text-decoration-color: #30787D; } .card-body ul li a { font-weight: normal; } #CTSA-COVID-Efforts.row { margin-left: 0; margin-right: 0; } .ckeditor-accordion-container { margin-top: 0; } section.row.research { margin-bottom: 40px; } .card.research-card.flex-fill { border: none; 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} } /* @media (max-width: 589px) { .ckeditor-accordion-container dl#accordion-2 dt:nth-of-type(2) a.ckeditor-accordion-toggler:before { top: 18px; } } */ @media (max-width: 638px) { .ckeditor-accordion-container dl#accordion-1 dt:nth-of-type(2) a.ckeditor-accordion-toggler:before { top: 18px; } } @media (min-width: 992px) { .research-card .card-img { height: 100px; overflow: hidden; } /* .ckeditor-accordion-container dl#accordion-1 dt:nth-of-type(2) a.ckeditor-accordion-toggler:before { top: 18px; } */ } @media (min-width: 1200px) { .research-card .card-img { height: 150px; overflow: hidden; } .ckeditor-accordion-container dl#accordion-1 dt:nth-of-type(2) a.ckeditor-accordion-toggler:before { top: 8px; } } @media (min-width: 768px) { .colaborative { display: flex; } } function load() { // console.log('on load'); $('.ckeditor-accordion-toggle').filter('.active').each(function(i) { $(this).removeClass('.active'); // console.log('on load'); }); } windows.onload = load; NCATS’ mission is to turn biomedical research discoveries into health solution — including diagnostics, treatments, and interventions — through the application of translational science.Translational science is the field that generates innovations that overcome longstanding bottlenecks and roadblocks to accelerate progress along the translational research pipeline. These include scientific, operational, financial and administrative innovations that transform the way that research is done.The NCATS Translational Science Principles offer eight principles to guide those designing and implementing translational science innovations. The Principles were generated by an NCATS committee with expertise across the translational spectrum, and in science, operations and administration. They were informed by in-depth case studies of three highly successful NCATS-led or -supported initiatives that span the translational continuum and pursue varied scientific goals. We identified commonalities in the approaches used across these initiatives that helped to advance translational progress.Click the image to view a larger version.Translational Science Principles×CloseThe NCATS Translational Science Principles also build upon scholarship identifying core competencies for translational science. This includes the Core Competencies in Clinical and Translational Research produced by the CTSA Education Core Competency Working Group, and the Characteristics of a Translational Scientist, produced by Translation Together.These Principles are intentionally broad, as they are designed to be relevant to research anywhere along the translational continuum. They do not prescribe particular strategies, but instead offer guiding principles to inspire those involved in the translational enterprise to develop strategies that are specific to their own research goals and aligned with these principles. In addition, they can guide not only researchers but everyone who enables, facilitates and supports translation, including those involved in science leadership, management, and administration, and patient and community engagement.We see the NCATS Translational Principles as a living product that will continue to be refined to reflect the community’s growing expertise in translational science. This webpage will be updated periodically to reflect these refinements.Explore the NCATS Translational Science Principles below.Prioritize Initiatives That Address Unmet NeedsFocus on pursuing scientific goals that address unmet scientific, patient or population health needs.Example approaches:Scientific Needs: Contribute to research advances in under-investigated areas of science or on scientific questions that present unique research challenges or disincentives (e.g., currently untreatable diseases; de-risking targets).Patient and Population Health Needs: Advance research to develop solutions for unmet patient and population health needs.Produce Generalizable Solutions for Common and Persistent ChallengesDevelop innovations that address persistent challenges to advancing translational progress that are found across multiple research initiatives or projects, or span research on multiple diseases or conditions.Example approaches:Across Multiple Projects or Initiatives: Advance research by identifying, developing and/or testing solutions to common bottlenecks or roadblocks that have stymied multiple projects. These may be scientific, operational, or administrative in nature.Across Diseases or Conditions: Approach research challenges and develop solutions by seeking commonalities across research projects on a range of diseases or conditions.Organizational Environment: Enable development and testing of generalizable solutions through organizational policies, organizational structure, and shared resources.Emphasize Creativity and InnovationLeverage creativity and innovation in research design, conduct, and facilitating factors, with the goal of increasing the impact of the research.Example approaches:Research Design and Implementation: Pose innovative research questions and develop and implement innovations in research methods, technologies, and approaches that increase the impact of the research, as through pursuit of paradigm-changing goals, or innovations that are generalizable to advancing research across multiple initiatives, diseases and conditions.Research Processes and Structures: Develop and implement innovations in research team interactions, leadership and management, partnerships, and operations that facilitate and support the quality and impact of the research.Organizational Environment: Enable creativity and innovation through policies that encourage innovations and do not penalize failures.Leverage Cross-Disciplinary Team ScienceEngage team members with expertise across disciplines, fields, and professions to produce research that advances translation along the translational research continuum.Example approaches:Leverage Broad Expertise: Engage colleagues from across disciplines, fields, and professions to advance research along the translational continuum. This may involve leveraging scientific, administrative, financial and operational expertise.Integrate Knowledge: Integrate concepts, theories, methods, technologies, and approaches from the range of disciplines, fields, and professions that can contribute to advancing the research goals. Leverage knowledge integration to produce more holistic research designs and findings that are therefore more relevant to real-world applications.Organizational Environment: Enable team science via organizational policies, team leadership and management, shared instrumentation and space, and recognition and reward systems.Enhance the Efficiency and Speed of Translational ResearchImplement evidence-informed practices and scientific and operational innovations to accelerate the pace of translational research.Example approaches:Scientific Efficiencies: Develop and implement innovations in scientific approaches, methods and technologies that accelerate the pace of translational research.Collaboration Efficiencies: Implement evidence-informed practices to enhance the speed at which collaborations and teams form, develop a shared vision and goals, effectively communicate, and coordinate work tasks.Project Management Efficiencies: Implement milestone-based decision making to enable rapid agreement on go/no-go decisions, to enable resources to be used most efficiently.Organizational Environment: Reward efficiency, enable rapid failures and encourage redirection of resources to subsequent attempts.Utilize Boundary-Crossing PartnershipsLeverage collaborations across agencies and sectors and engage patients and communities in research to advance translational progress.Example approaches:Cross-Sectoral Partnerships: Form partnerships across government, universities, and industry to leverage varied expertise and resources to accelerate translational progress. Implement evidence-informed practices for effective cross-sectoral partnerships.Patient and Community Engagement: Involve impacted patients and communities as research collaborators to enable research advances across the translational continuum (e.g., via disease registries, clinical trials participation, intervention design). Implement evidence-informed practices for patient- and community-engaged research.Organizational Environment: Enable and incentivize boundary-crossing partnerships via leadership, policies, and recognition and reward systems.Use Bold and Rigorous Research ApproachesDevelop ambitious research questions and address them with rigorous and robust methods toward generating reproducible findings that contribute to advancing translation.Example approaches:Bold Scientific Approaches: Explore ambitious research goals that have the potential to produce major advances and/or paradigm shifts. These may be in areas of research that have been historically intractable or where there are high risks of failure.Rigor and Reproducibility: Employ rigorous and robust approaches to generate reproducible findings and high-quality FAIR (findable, accessible, interoperable, reusable) data that will enable the research to advance translational progress regardless of whether the initial research objective is met (e.g., learning from failures). To the maximum extent possible, disseminate all parameters utilized to conduct the research (e.g., materials, subjects), research methods and conditions, authentication of reagents and biological resources, data sets, metadata, analytic approaches and statistical tools used for experimentation and data interpretation, results and conclusions, to facilitate reproducibility and/or inform future study designs.Organizational Environments: Enable rigorous testing of bold, paradigm-challenging ideas, including high-risk high-reward opportunities. Encourage reporting of information necessary for reproducibility toward informing future studies.Prioritize Diversity, Equity, Inclusion, and Accessibility (DEIA)Leverage diversity, equity, inclusion, and accessibility to produce research outcomes that are relevant to the full diversity of the population.Example approaches:Research Priority Setting: Include diverse perspectives in research priority setting, such as through partnerships with diverse collaborators, so research investments represent diverse population and patient health needs.Scientific Approaches: Integrate DEIA into the development of research resources and design and implementation of studies, across the translational research continuum.Workforce and Operations: Advance DEIA in the scientific workforce to leverage maximum expertise. Implement best practices in outreach, recruitment and hiring, development, and retention to sustain a diverse and talented workforce.Additional ResourcesPublicationsAdvancing Education and Training through Application of the Translational Science PrinciplesFaupel-Badger JM, Vogel AL, Austin CP, Rutter JL. Advancing Translational Science Education [published online ahead of print, 2022 Aug 31]. Clin Transl Sci. 2022;10.1111/cts.13390. doi:10.1111/cts.13390)Translational Science Principles Enabling Team Science in NCATS’ Internal Research ProgramVogel AL, Knebel AR, Faupel-Badger JM, Portilla LM, Simeonov A. A Systems Approach to Enable Effective Team Science from the Internal Research Program of the National Center for Advancing Translational Sciences. Journal of Clinical and Translational Science. Cambridge University Press; 2021;5(1):e163.Teaching Translational Science PrinciplesFaupel-Badger JM, Vogel AL, Hussain SF, Austin CP, Hall MD, Ness E, Sanderson P, Terse PS, Xu X, Balakrishnan K, Patnaik S, Marugan JJ, Rudloff U, Ferrer M. Teaching Principles of Translational Science to a Broad Scientific Audience Using a Case Study Approach: A Pilot Course from the National Center for Advancing Translational Sciences. Journal of Clinical and Translational Science. Cambridge University Press; 2022:1–26.Evaluation of a Course Teaching Translational Science PrinciplesVogel AL, Hussain SF, Faupel-Badger JM. Evaluation of an Online Case Study-Based Course in Translational Science for a Broad Scientific Audience: Impacts on Students’ Knowledge, Attitudes, Planned Scientific Activities, and Career Goals. Journal of Clinical and Translational Science. Cambridge University Press; 2022;6(1):e82.Online Courses in Principles of Translational ScienceThe NCATS Education Branch offers two case study–based courses in translational science principles taught by scientists who have led translational research at NIH, as well as additional experts in translational science. These courses are offered annually in partnership with the Foundation for Advanced Education in the Sciences.MEDI 501: Principles of Preclinical Translational ScienceThis course uses a case study of an NCATS-led promising preclinical drug discovery and development project to teach principles of translational science as applied to the preclinical space.MEDI 502: Translational Science in the COVID-19 PandemicThis course uses a series of case studies in COVID-19 research to teach principles of translational science as applied across the translational spectrum.Read more detailed descriptions of these courses or visit our Events page to see if they are currently enrolling.Case Studies in Translational ScienceThese video case studies highlight how translational science principles shape NCATS’ research programs and initiatives. The case studies cover drug discovery and development, the Toolkit for Patient-Focused Therapy Development, and the creation and development of tissue chips. Watch the playlist.NCATS’ Translational Science Principles Committee, which includes representatives from across the Center, contributed to the development of the principles. | Learn about principles underlying effective translational science. | Translational Science Principles | Learn about principles underlying effective translational science. | Translational Science Principles | ||||
| 22906 | The Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (BGTC) | The Bespoke Gene Therapy Consortium (BGTC) is part of the Accelerating Medicines Partnership® (AMP®) program, a public–private partnership among NIH, the U.S. Food and Drug Administration (FDA), multiple pharmaceutical and life sciences companies, and nonprofit and other organizations. The AMP program, which is managed by the Foundation for the NIH (FNIH), aims to improve current models for developing diagnostics and therapies. The BGTC is establishing platforms and standards to speed the development and delivery of customized or “bespoke” gene therapies that could treat millions of people affected by rare diseases, including diseases too rare to be of commercial interest. The BGTC is the first AMP initiative focused on rare diseases and the sixth AMP initiative overall. It also is the first to focus on a therapeutic platform. Rare Diseases and the Promise of Gene Therapy 3D rendering of genetic medicine with DNA isolated. (xsense/Shutterstock) Approximately 7,000 known diseases are characterized as “rare,” meaning each one affects fewer than 200,000 people in the United States. Collectively, rare diseases are hardly rare; 25–30 million people in the United States have rare diseases that diminish their quality of life and threaten their health. Moreover, they have significantly higher medical expenses, with medical care costing three to five times more for individuals with rare diseases than for individuals without rare diseases. Fewer than 10% of rare diseases have FDA-approved treatments. About 80% of rare diseases are caused by known alterations in a single gene. This common feature makes these diseases potential candidates for gene therapy, which entails replacing or correcting a defective gene. Developing gene therapies for rare diseases, however, is complex, time consuming and expensive. The gene therapy development process is hampered by a lack of access to proprietary tools and methods, a dearth of standards, and a one-disease-at-a-time approach. As of December 2021, only two rare diseases have an FDA-approved gene therapy. The Bespoke Gene Therapy Consortium Launched in October 2021, the BGTC will generate gene therapy resources that the research community can use to streamline gene therapy development for rare disorders, making the process more efficient and less costly. One of the BGTC’s goals is to improve the understanding of the basic biology of the harmless adeno‑associated virus (AAV), a common gene-delivery vehicle or vector. BGTC scientists will learn more about how AAVs carry genes to the correct place in cells, how those genes get into cells, and how the newly transported genes are turned on in the target cells. This information will help improve the effectiveness of AAV gene therapies. Another important BGTC goal is to improve the efficiency of both vector manufacturing and production quality control testing. The BGTC will achieve this by developing a standard and broadly applicable set of analytic tests that can be used to manufacture viral vectors. The BGTC clinical component aims to streamline the path from animal studies to human testing. Funded researchers will conduct between four and six clinical trials, with each trial focused on a different rare disease. None of the chosen diseases will have an existing gene therapy or one in development, but the diseases that are chosen will be well understood, and the funded scientists will have considerable experience studying each disease. In addition, the trials will employ different AAV vectors that already have been used in other trials. During the BGTC clinical trials, scientists will develop strategies for streamlining the regulatory processes for FDA approval of safe and effective gene therapies, and they will develop standardized approaches to preclinical testing (e.g., toxicology studies). NCATS’ Role NCATS is partnering with the FDA and FNIH, 10 NIH Institutes and Centers (ICs), and several pharmaceutical companies and nonprofit organizations to form the BGTC. NCATS, the lead NIH IC for BGTC, expects to contribute approximately $8 million of a total $39.5 million provided by participating NIH ICs. Together, NIH and private partners will contribute approximately $76 million over 5 years to support BGTC-funded projects. NCATS’ mission is to re-engineer the translational research process so new treatments and cures for diseases can be delivered to patients faster. Notably, NCATS focuses on the critical and unmet needs of people with rare diseases. The BGTC is one of several NCATS-led programs that apply the “many-diseases-at-a-time” approach to gene therapy development for rare diseases, including the Platform Vector Gene Therapy (PaVe-GT) pilot project. Both the PaVe-GT pilot project, which was launched in February 2019, and the BGTC work to improve the efficiency and accessibility of gene therapy development and delivery for rare diseases. These two efforts serve different but complementary goals, with the PaVe-GT pilot project using AAV vectors to develop gene therapies for four rare genetic diseases that differ from the four to six clinical studies that will be supported by the BGTC. The NCATS intramural laboratories will play an important role in the BGTC’s basic biology component. With expertise in preclinical drug development — including assay development, high-throughput screening, disease modeling, toxicity testing and more — NCATS researchers are poised to generate data that could lead to improved vector production and therapeutic gene activity. To learn more about the BGTC, including research opportunities, please visit the FNIH website or contact NCATS’ P.J. Brooks, Ph.D. Additional Links NCATS-supported rare diseases research initiatives Resources for people with rare diseases Division of Rare Diseases Research Innovation program contacts | The BGTC aims to optimize and streamline the gene therapy development process to treat rare diseases. | The AMP Bespoke Gene Therapy Consortium | The BGTC aims to optimize and streamline the gene therapy development process to treat rare diseases. | The AMP Bespoke Gene Therapy Consortium | ||||
| 22939 | Statement on NIH Study Testing Convalescent Plasma in Hospitalized Patients | Overall results show no benefit over placebo in delivering clinical improvementJanuary 11, 2022For people hospitalized with COVID-19, convalescent plasma taken from those who had recovered was not more effective than placebo in delivering clinical improvement 14 days and 28 days after treatment began.These findings are from a large Phase 3, randomized, placebo-controlled clinical trial funded by the National Institutes of Health (NIH). The Convalescent Plasma to Limit COVID-19 Complications in Hospitalized Patients (CONTAIN COVID-19) trial included data from 941 participants enrolled between April 2020 and March 2021 at 21 hospitals in Connecticut, Florida, Maryland, New York, Texas and Wisconsin. The primary outcome was clinical status based on the participant scores on the 11-point World Health Organization (WHO) Ordinal Scale for Clinical Improvement 14 days after randomization. The secondary outcome was clinical status on the same scale 28 days after randomization.Eligible participants in CONTAIN COVID-19 were adults 18 years or older who tested positive for SARS-CoV-2 infection, had been hospitalized for no more than three days or had experienced respiratory illness symptoms for no more than seven days and required noninvasive supplemental oxygen while hospitalized.While convalescent plasma did not meet the trial’s primary and secondary outcomes for clinical improvement, the researchers conducted additional statistical analyses because patient characteristics and COVID-19 treatments changed over the course of the 11‑month study. They sought to understand the effect of changes on convalescent plasma efficacy at different time points of trial enrollment. The results indicated possible clinical benefit in the early stage of the pandemic (April to June 2020), when participants received high-titer convalescent plasma without corticosteroids and remdesivir. During that period, patients receiving convalescent plasma were more likely to clear COVID-19 symptoms, be discharged from the hospital earlier and/or require less supplemental oxygen.Among the large, placebo-controlled study’s strengths was the highly diverse participant pool, which may make CONTAIN COVID-19’s findings generalizable across multiple population groups — 41% of study volunteers were female, 40% were Hispanic and 14% were non-Hispanic Black. The trial’s innovative statistical design also allowed near real-time monitoring of patient treatment information and faster assessment of therapeutic efficacy.Liise-anne Pirofski, M.D., and Hyunah Yoon, M.D., of Albert Einstein College of Medicine and Montefiore Medical Center, and Mila B. Ortigoza, M.D., Ph.D., of New York University Langone Health, led the multisite clinical trial. The study’s findings appear in JAMA Internal Medicine. NCATS oversaw the trial through its Clinical and Translational Science Awards Program research network. | NIH’s CONTAIN COVID-19 trial shows no benefit of convalescent plasma therapy over placebo in hospitalized patients. | /sites/default/files/cp_advisory_900x600.jpg | Statement on NIH Convalescent Plasma Study in Hospitalized Patients | NIH’s CONTAIN COVID-19 trial shows no benefit of convalescent plasma therapy over placebo in hospitalized patients. | /sites/default/files/cp_advisory_900x600_0.jpg | Statement on NIH Convalescent Plasma Study in Hospitalized Patients | ||
| 22627 | Gene Therapy and Gene Editing Conferences and Workshops | 2021 June Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery June 3, June 10 and June 17 Website • Agenda — Day 1 (PDF - 248KB) • Agenda — Day 2 (PDF - 258KB) • Agenda — Day 3 (PDF - 249KB) • VideoCast (Day 1) • VideoCast (Day 2) • VideoCast (Day 3) 2020 November Virtual Workshop on Systemic Immunogenicity Considerations for AAV-Mediated Gene Therapy Nov. 30–Dec. 1 Website • Agenda (PDF - 171KB) • VideoCast (Day 1) • VideoCast (Day 2) January Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies Jan. 28–29 Website • VideoCast (Day 1) • VideoCast (Day 2) 2019 June CNS Immunogenicity Considerations for AAV-Mediated Gene Therapy June 11 Website • Agenda (PDF - 164KB) • VideoCast 2018 August The Growing Promise of Gene Therapy Approaches to Rare Diseases Aug. 20–21 Website • Agenda (PDF - 168KB) • VideoCast (Day 1) • VideoCast (Day 2) | View past NCATS events relating to gene therapy, oligonucleotide and gene editing conferences and workshops. | Gene Therapy and Gene Editing Conferences and Workshops | View past NCATS events relating to gene therapy, oligonucleotide and gene editing conferences and workshops. | Gene Therapy and Gene Editing Conferences and Workshops | ||||
| 22624 | Gene Therapy and Gene Editing Programs | NCATS is involved with multiple programs for accelerating gene-targeted therapies, including gene therapy, antisense oligonucleotide therapy and gene editing. The approaches taken in these programs help speed the development of treatments for multiple rare diseases at a time. Gene-Targeted Therapy Programs 3D rendering of genetic medicine with DNA isolated. (xsense/Shutterstock) Bespoke Gene Therapy Consortium The Bespoke Gene Therapy Consortium (BGTC) is a public–private partnership that aims to develop platforms and standards that will speed the development and delivery of customized, or “bespoke,” gene therapies that could treat the millions of people affected by rare diseases. Platform Vector Gene Therapy The Platform Vector Gene Therapy (PaVe-GT) pilot project seeks to increase the efficiency of clinical trial startup by using the same gene delivery system and manufacturing methods for multiple rare disease gene therapies. Somatic Cell Genome Editing Program The Somatic Cell Genome Editing (SCGE) program is an NIH Common Fund program co-led by NCATS with the objective of accelerating the development of safer and more effective genome-editing therapeutics. Conferences and Workshops NCATS hosts a variety of gene therapy, oligonucleotide and gene editing conferences and workshops. View past events led by NCATS and collaborators. Contacts: Program Director Philip John (P.J.) Brooks, Ph.D. (301) 443-0513 Program Administrator Deanna Portero (301) 451-9968 | NCATS is involved in several programs to accelerate gene-targeted therapies and develop treatments for multiple rare diseases at the same time. | /sites/default/files/shutterstock_542582740_900x600_mirrored_3.jpg | Gene Therapy and Gene Editing Programs | NCATS is involved in several programs to accelerate gene-targeted therapies and develop treatments for multiple rare diseases at the same time. | /sites/default/files/shutterstock_542582740_900x600_mirrored_4.jpg | Gene Therapy and Gene Editing Programs | ||
| 22630 | Rare Diseases Research and Resources | .tabs-programs { margin: 40px 0; } #additional-info { background-color: #ccc; padding: 0; margin-top: 0; margin-bottom: 40px; } #additional-info > div > div { background-color: white; border: #006478 2px solid; padding: 20px; min-height: 200px; width: 49.5%; } #additional-info > div > div:nth-child(1) { margin-right: 0.5%; } #additional-info > div > div:nth-child(2) { margin-left: 0.5%; } .addinfo-title { margin-top: -35px; background-color: #006478; padding-left: 10px; color: white; } #featured-stories > #secondary-image > img:nth-child(1) { min-height: 312px; } NCATS is committed to using research to address the public health crisis presented by rare diseases. Speeding development of treatments for patients requires innovation in science and technology and engaging patients and their support organizations as essential partners. | NCATS is committed to using research to address the extraordinary public health issue presented by rare diseases. | /sites/default/files/2018_RDANR_Challenge_Graphic_900x400.jpeg | Rare Diseases Research and Resources | NCATS is committed to using research to address the extraordinary public health issue presented by rare diseases. | /sites/default/files/2018_RDANR_Challenge_Graphic_900x400_0.jpeg | Rare Diseases Research and Resources |
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