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| 6540 | Pfizer’s CTI for NIH Researchers | NCATS led an innovative collaboration for the NIH with Pfizer’s Centers for Therapeutic Innovation (CTI) from 2014 to 2019. The CTI collaboration, which was governed by a steering committee, enabled NIH and Pfizer scientists to identify biologic compounds with activity in a pathway or target of interest and move these compounds through laboratory testing and into clinical evaluation. A cooperative research and development agreement (CRADA) collaboration with CTI included, among other things, access to Pfizer’s drug development enterprise and publishing rights. It was the first NIH-wide biologics initiative with a pharmaceutical partner that NCATS coordinated on behalf of all NIH intramural researchers. | Pfizer’s CTI for NIH researchers aims to bridge the gap between early scientific discovery and its translation into medicines through public-private collaborations. | /sites/default/files/woman-in-lab-900px.jpg | Pfizer’s CTI for NIH Researchers | Pfizer’s CTI for NIH researchers aims to bridge the gap between early scientific discovery and its translation into medicines through public-private collaborations. | /sites/default/files/woman-in-lab-900px.jpg | Pfizer’s CTI for NIH Researchers | ||
| 6517 | Frequently Asked Questions About the X02 Pre-Application | In collaboration with AstraZeneca and Janssen Research & Development, LLC, NCATS is seeking applications through its NIH-Industry Partnerships initiative to explore new treatments for patients, using existing experimental drugs or biologics. NCATS plans to commit an estimated $6 million to fund six to 10 awards to support partnerships between the pharmaceutical companies and the biomedical research community by making a selection of industry assets available to test ideas for new therapeutic uses. NIH’s National Institute of Dental and Craniofacial Research also is participating in these new opportunities. Interested researchers are asked to submit a letter of intent by March 17, 2017, and pre-applications are due April 17, 2017. Industry-Provided Assets How does NCATS solicit assets for inclusion in the New Therapeutic Uses program? Is this funding opportunity limited to the assets provided through the New Therapeutic Uses program, or can investigators propose other assets? If investigators have an asset they believe may have a new use but is not listed in the industry-provided assets tables for this program, can they apply? Will chemical structures for the proposed assets be available? There is more than one asset with the same target/mechanism of action on tables found on the Industry-Provided Assets page. Do potential grantees need to choose one in particular when submitting an X02 application? Could investigators have access to all listed assets under this announcement? Can an investigator submit an application requesting the collection of assets for preclinical studies, including screening? Could investigators apply separately for each asset on the list? Application Information Should applicants contact the pharmaceutical company before submitting an X02? What if two or more applicants submit X02 applications on the same asset for the same therapeutic indication? Are proposed new uses for this program limited to stand-alone interventions? Are applicants required to use the template confidential disclosure agreements (CDAs) and collaborative research agreements (CRAs) posted on the NCATS website? Pediatric Indications Are there any limitations on the use of assets in pediatric populations? What types of studies are acceptable for pediatric indications? Applicant Eligibility Can foreign applicants or companies apply to the program? Can investigators from small businesses and for-profit organizations apply for funding? Can an academic researcher and a biotech company collaborate to apply for the New Therapeutic Uses program? NIH Intramural Research Program (IRP) Are NIH IRP investigators eligible to apply for these funding opportunities? In what ways can an IRP investigator collaborate with an extramural investigator? Does the IRP program director/principal investigator need to be a tenured/tenure-track investigator? If a potential grantee has a dual appointment with NIH and an extramural research organization, which organization should be listed as the applicant organization? Budget/Funding What is the total budget limit for each application? Industry-Provided Assets How does NCATS solicit assets for inclusion in the New Therapeutic Uses program? NCATS invites prospective pharmaceutical companies to partner with NCATS to explore new indications for drug candidates (assets) across a broad range of human diseases. Requirements for pharmaceutical partner participation and asset selection criteria follow: Number of assets: At least three compounds will be contributed for the solicitation. See assets here: http://www.ncats.nih.gov/ntu/assets/current/ Asset characteristics Mechanism of action is known. Pharmacokinetics are suitable to explore the mechanism in a new indication. Phase I clinical trial has been completed – safety profile is understood. Assets currently in clinical development can be included. Major company responsibilities include: Provide asset information to be posted on the NCATS website. Provide preclinical and clinical supply for studies (both drug and placebo). Provide regulatory documents (i.e., cross reference letter or study reports) to enable a funded investigator to file an Investigational New Drug (IND) application in the U.S. in time to meet project timeline and milestones. Use template agreements that are negotiated with NCATS. See samples here: http://www.ncats.nih.gov/ntu/assets/agreements. Please email NewTherapeuticUses@mail.nih.gov if you work for a company that wishes to partner with NCATS. Is this funding opportunity limited to the assets provided through the New Therapeutic Uses program, or can investigators propose other assets? If investigators have an asset they believe may have a new use but is not listed in the industry-provided assets tables for this program, can they apply? This funding opportunity is limited to those assets provided by pharmaceutical company collaborators for the New Therapeutic Uses program through a Memorandum of Understanding with NIH. The program will not provide support for assets not listed in the tables on the Industry-Provided Assets page. NCATS encourages inquiries concerning this funding opportunity and welcomes the opportunity to answer questions from potential applicants. Institute or Center contacts can assist in directing investigators to other funding opportunity announcements (FOAs) to propose new therapeutic uses of other assets that might be of interest in specific disease areas. Will chemical structures for the proposed assets be available? Applicants who have meritorious X02 applications and are given the opportunity to submit a UG3/UH3 application will have access to any chemical structures and additional data after they sign a confidential disclosure agreement with the pharmaceutical partner. There is more than one asset with the same target/mechanism of action on tables found on the Industry-Provided Assets page. Do potential grantees need to choose one in particular when submitting an X02 application? No. In some cases, there will be sufficient information in the one-page summary charts for applicants to choose the most appropriate asset for the proposed study. In cases where the information provided on the NCATS website is not sufficient for an applicant to choose, he or she may simply identify the target/mechanism of action in the X02 application. X02 applicants who are notified of the opportunity to submit UG3/UH3 applications will receive company contact information to execute a confidential disclosure agreement with asset providers and obtain more information on the assets. To be responsive, the UG3/UH3 application must identify a single asset. Could investigators have access to all listed assets under this announcement? No. This program will not support screening the collection of assets to identify new uses. Can an investigator submit an application requesting the collection of assets for preclinical studies, including screening? No. This program does not support screening of the assets. The primary focus of applications should be on clinical trials (Phases I and II). If proposed, preclinical studies should be justified and tied to go/no-go decisions to test the asset in the patient population. Could investigators apply separately for each asset on the list? Applicants may submit more than one application, provided that each application is scientifically distinct. However, applicants should focus on one asset to develop biological evidence for the proposed new use. The initiative focuses on Phase II clinical trials with preclinical studies proposed if necessary to support the potential new use. Phase II clinical trials provide data on the relationship of dosing and response for the particular intended use (including trials on the impact of dose ranging on safety, biomarkers and proof of concept). Application Information Should applicants contact the pharmaceutical company before submitting an X02? No. Applicants should not contact the pharmaceutical companies before submitting the X02. Applicants whose X02 pre-applications are identified as being highly meritorious and relevant to NIH program priorities will be notified of the opportunity to submit UG3/UH3 applications. The notification will indicate the appropriate pharmaceutical company contact. However, applicants should work with their institutions in advance to discuss the conditions in the collaborative research agreement for the selected asset prior to submitting the X02 pre-application. What if two or more applicants submit X02 applications on the same asset for the same therapeutic indication? If two or more X02 applications are identified as being highly meritorious, these applicants will be notified of the opportunity to submit UG3/UH3 applications. Are proposed new uses for this program limited to stand-alone interventions? No. The program supports clinical studies/trials to develop new uses of the assets as stand-alone interventions or as add-ons to current treatments if there is no evidence of drug-to-drug interactions with the standard-of-care treatment. Are applicants required to use the template confidential disclosure agreements (CDAs) and collaborative research agreements (CRAs) posted on the NCATS website? In the X02 pre-application, applicants must include a letter of support from the appropriate institutional official, confirming the institution’s willingness to engage in the necessary negotiations with the pharmaceutical company. One of the barriers encountered in moving forward projects that involve the private sector and the academic sector or other collaborators is the time it takes to execute a CRA or equivalent document. In recognition of this barrier, template agreements have been developed to streamline interactions among the parties for the program. Applicants should use the agreements. X02 applicants should consider their willingness and the willingness of their institution to agree to the conditions in the appropriate CRA for the selected asset prior to submitting a pre-application. Investigators should work with the appropriate office within their organization to finalize the terms and conditions of the CDA and CRA for the selected asset prior to submission of a UG3/UH3 application. Use of the template agreements is not required. However, UG3/UH3 applications submitted without evidence of access to and the ability to work with the assets, such as evidence that a CRA or equivalent document has been executed, will be deemed incomplete and returned to the applicant without review. Pediatric Indications Are there any limitations on the use of assets in pediatric populations? Yes. In general, pediatric populations to be considered for this FOA refer to disease populations for which there is no adult equivalent and thus no adult population in which the drug could be tested prior to testing it in children. However, trials in pediatric or juvenile populations for indications that also have an adult population (e.g., type 2 diabetes, autism, osteoarthritis) may be considered if there is a strong scientific rationale that justifies why Phase II trials in the pediatric population are required even though an adult patient population exists (e.g., the target in the pediatric population may differ from that in the adult or treatment of children may reduce progression or severity of the disease). Assets that the pharmaceutical companies will consider for use in pediatric populations are listed in the Table of Assets for Pediatric Indications. Applicants must click on the asset code number in the first column of the table to obtain more detailed asset information. To determine the type(s) of pediatric diseases the pharmaceutical company will consider (e.g., only trials in pediatric populations for which there is no adult population; trials for diseases/conditions that have a pediatric and adult population, if the trials in a pediatric population are scientifically justified), open the asset of interest and refer to the “Additional Characteristics” row. Applicants exploring therapies for diseases that occur in children and adults should consider applying in response to the companion UG3/UH3 FOA focusing on adult populations. What types of studies are acceptable for pediatric indications? Applicants should refer to the Table of Assets for Pediatric Indications to determine if the asset will be considered by the pharmaceutical company for pediatric use. After clicking on the asset of choice in the first column of the table, refer to the "Additional Characteristics" row of the more detailed asset information chart. This row provides information on the types of pediatric indications that the pharmaceutical company will consider (e.g., only trials in pediatric populations for which there is no adult population; trials for diseases/conditions that have a pediatric and adult population, if the trials in a pediatric population are scientifically justified). Applicants must provide NIH with documentation of access to the asset and associated data needed for conducting the proposed preclinical studies and pediatric clinical trials and for filing an investigator-sponsored IND application to conduct the proposed clinical trials in a UG3/UH3 application (e.g., letter from the pharmaceutical company providing access to the asset for the indicated use). Applicant Eligibility Can foreign applicants or companies apply to the program? No. Foreign investigators and institutions are not eligible to apply. However, they may participate as subcontractors of an awarded U.S. institution or investigator. Foreign components, as defined in the NIH Grants Policy Statement, are allowed. Can investigators from small businesses and for-profit organizations apply for funding? Yes. Small businesses and for-profit organizations with more than 51 percent U.S. ownership are eligible to apply. Can an academic researcher and a biotech company collaborate to apply for the New Therapeutic Uses program? Yes. NIH encourages scientific collaboration to leverage additional expertise and resources. NIH Intramural Research Program (IRP) Are NIH IRP investigators eligible to apply for these funding opportunities? Yes. NIH IRP investigators can apply as program director(s)/principal investigator(s) (PDs/PIs), as multiple PDs/PIs in conjunction with extramural investigators, or as collaborators with extramural academic or biotechnology company investigators, pending the availability of their respective Institute's or Center's intramural funds to support the project. IRP investigators and laboratories cannot request extramural funds. An official letter from the IRP applicant’s scientific director that indicates approval of the IRP scientist's role as PD/PI or as collaborator in the project must be included as a letter of support in the submission of the X02 pre-application. In what ways can an IRP investigator collaborate with an extramural investigator? IRP investigators can collaborate with extramural investigators in a variety of ways: The IRP and extramural investigators can submit an application as multiple PDs/PIs; however, if the applicant institution is an NIH Institute or Center, funds may not be requested for an extramural component/collaborator. The IRP investigator can be a collaborator on an application submitted by an extramural investigator. However, the requests by NIH intramural scientists will be limited to the incremental costs required for participation. The IRP investigator can be the PD/PI on a consortium of the extramural investigator’s application. Does the IRP program director/principal investigator need to be a tenured/tenure-track investigator? Yes. Tenure-track investigators can apply if they will be at NIH for the full duration of the project; they should have approximately four years left at NIH (or five years if a pediatric trial will be proposed) at the time of application because the submission, review and award process can take up to one year. Postdoctoral fellows, staff scientists and others are not eligible to apply. If a potential grantee has a dual appointment with NIH and an extramural research organization, which organization should be listed as the applicant organization? Choice of applicant institution depends on a variety of factors, including but not limited to the duration of the appointments, the specific terms of each appointment, the availability of funds at the NIH laboratory, the available resources of each organization and many other individual factors. Budget/Funding What is the total budget limit for each application? For the X02 pre-application phase, no budget should be proposed. No direct awards will result from the X02 pre-application. | Find answers to questions about the X02 Pre-Application process | FAQ About the X02 Pre-Application | Find answers to questions about the X02 Pre-Application process | FAQ About the X02 Pre-Application | ||||
| 6926 | Homepage | Our focus is to advance the science of translation, which is the process of turning observations into interventions to improve health. We work with researchers, the public and other stakeholder groups to design new approaches and technologies that ultimately will deliver more treatments to more people more quickly. Read more | ||||||||
| 6436 | Gene Therapy for the Treatment of Pompe Disease | Pompe disease is a rare, inherited metabolic disorder. Mutations in the acid alpha-glucosidase enzyme cause toxic buildup of cellular byproducts, leading to damage to multiple organs and tissues, particularly the muscles. Symptoms can appear within a few months of birth or may arise later in life. Affected babies commonly experience muscle weakness, poor muscle tone, and liver and heart defects that can lead to premature death in the first year of life. Later-onset forms of the disease can result in delayed motor skills and progressive muscle weakness, leading to breathing problems and respiratory failure. Enzyme replacement therapy is available, but outcomes vary. The lead collaborators have developed a gene therapy approach that aims to overcome the variability of current enzyme treatment. The purpose of this project is to support further preclinical development and enable clinical trials. Scientific Synopsis Pompe disease (also called acid maltase deficiency or glycogen storage disease type II) is a metabolic condition caused by a deficiency of acid alpha-glucosidase (GAA) enzyme activity in striated and smooth muscle. The GAA enzyme is active in the lysosomes, metabolizing glycogen into glucose. Mutations in the GAA gene prevent the enzyme from carrying out its normal function, leading to toxic accumulation of glycogen. Onset and severity of symptoms vary. More severe forms of the disease may arise within a few months after birth, marked by muscle weakness and poor muscle tone, failure to thrive, liver and heart defects, and serious breathing problems leading to premature death. Milder forms may arise in childhood, adolescence, or even adulthood, marked by progressive muscle weakness and breathing problems that can lead to respiratory failure. Recombinant human GAA as an enzyme replacement therapy (ERT) is available, but its effectiveness varies. The ERT has been shown to ameliorate muscular and cardiac symptoms and increase overall survival rates, but the short half-life of GAA protein and the formation of antibody responses in some patients limits its utility. The lead collaborators have developed an adeno-associated virus (AAV) vector to transduce the GAA gene into the liver, enabling more continuous secretion of GAA protein. Preclinical studies have demonstrated uptake of the secreted GAA protein in heart and skeletal muscle tissues, associated with clearance of excess glycogen and improved muscle function. This project takes a novel approach of delivering a low dose of AAV-GAA to the liver to increase immune tolerance to GAA in patients. Lead Collaborator Duke University, Durham, NC Dwight Koeberl, M.D., Ph.D. Public Health Impact Pompe disease is a genetic disorder that leads to premature death, typically from heart or respiratory failure. Although an enzyme replacement therapy is available, it is not appropriate for all patients. The ERT is costly, shows variable effectiveness in some patients, and requires frequent, lifelong administration. A gene therapy could yield a more consistently effective approach, potentially requiring only a single administration. Outcomes TRND scientists supported the manufacture of clinical-grade therapeutic vectors for use in the lead collaborator’s planned phase I trial in Pompe disease patients. During the collaboration with TRND, the AAV gene therapy technology was licensed by Asklepios BioPharmaceutical, Inc. AskBio formed a spin-out company, Actus Therapeutics, to continue clinical development and commercialization. The key preclinical support provided by TRND enabled the lead collaborator to successfully obtain funding for the phase I trial, which TRND co-funded through a cooperative agreement with the National Institute of Arthritis and Musculoskeletal and Skin Diseases. See ClinicalTrials.gov, NCT03533673. | Pompe disease is a genetic disorder that leads to premature death. TRND scientists are working on a gene therapy that could yield a more effective treatment. | Gene Therapy for the Treatment of Pompe Disease | Pompe disease is a genetic disorder that leads to premature death. TRND scientists are working on a gene therapy that could yield a more effective treatment. | Gene Therapy for the Treatment of Pompe Disease | ||||
| 6435 | Therapy for Fuchs Endothelial Corneal Dystrophy | Fuchs endothelial corneal dystrophy (FECD) is a degenerative disease of the eye. The front surface of the eye, called the cornea, helps regulate vision by focusing light onto the lens. FECD affects the thin layer of cells at the back of the cornea, which progressively become damaged and die. As these cells are lost, the cornea retains excess fluid, resulting in loss of optical quality and decreased vision. Most patients are diagnosed with FECD only after significant numbers of corneal cells have been lost, and the only treatment for advanced disease is corneal transplantation. The lead collaborators have developed a growth factor therapy that aims to halt, and potentially reverse, the degeneration of endothelial cells. The purpose of this project is to support further preclinical development and enable clinical trials. Scientific Synopsis FECD is marked by progressive degeneration of the monolayer of endothelial cells on the inner surface of the cornea. Extracellular matrix accumulates between the corneal stroma and the endothelial layer at Descemet’s membrane, leading to corneal edema, loss of optical quality, and decreased vision. FECD is slowly progressive, and typically patients do not seek treatment until the endothelial layer is badly degenerated. Transplantation is the only current treatment. However, donor corneas are in limited supply, surgical complications can be significant, and transplants due to endothelial dystrophy have a higher long-term failure rate. A more optimal therapy would avoid the need for transplantation altogether. Fibroblast growth factors (FGFs) have been shown to stimulate proliferation and migration of human corneal endothelial cells in vitro and have the potential to be regenerative therapies in vivo. However, the application of wild-type FGFs as therapeutics is limited by poor stability and pharmacokinetics. The lead collaborators have developed an engineered FGF that has demonstrated improved stability and potency in preclinical studies. Lead Collaborator Trefoil Therapeutics, LLC, San Diego David Eveleth, Ph.D. Public Health Impact FECD is a progressive, degenerative disease of the eye that can only be treated by corneal transplantation. Although outcomes of transplantation are generally good, failure rates in FECD patients are significant. A non-surgical therapy that can halt the degeneration — and potentially regenerate lost endothelial cells — would be transformative for this population. Outcomes TRND scientists developed a production process for eFGF, made key reagents for the analysis of eFGF in vitro and in vivo, and completed GLP toxicology studies. As a result of TRND support, the collaborators successfully submitted an Investigational New Drug application to the Food and Drug Administration, allowing clinical evaluation to proceed. See ClinicalTrials.gov, NCT04520321, NCT04676737, and NCT04812067. | FECD is a degenerative disease of the eye that TRND scientists are working on a non-surgical therapy. | Therapy for Fuchs Endothelial Corneal Dystrophy | FECD is a degenerative disease of the eye that TRND scientists are working on a non-surgical therapy. | Therapy for Fuchs Endothelial Corneal Dystrophy | ||||
| 6434 | Gene Therapy for the Treatment of AADC Deficiency | Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, inherited disorder that typically appears within the first year of life. The AADC enzyme is necessary for the production of important chemical messengers in the brain and nervous system. Children with the condition experience a number of symptoms, including severe developmental delays, weak muscle tone, involuntary movements of the arms and legs, and painful seizures. Patients require lifelong care, and particularly severe cases can lead to death within the first decade of life. The lead collaborators have developed an approved gene therapy technology to restore AADC enzyme production in the brain. The purpose of this project was to correct the underlying molecular defect and support further development work to accelerate the approval of this life-saving therapy for patients. Scientific Synopsis AADC deficiency arises due to mutations in the dopa decarboxylase (DDC) gene. The AADC enzyme is responsible for the final step in the synthesis of the key neurotransmitters dopamine and serotonin, which are required for normal nervous system function. Symptoms and severity of disease can vary due to the specific underlying mutations that abrogate AADC enzyme function. Affected children commonly experience severe developmental delays, weak muscle tone, and involuntary movement of the limbs. Patients with severe forms of disease usually die before the age of 7, due to severe motor dysfunction, autonomic abnormalities, and secondary complications such as choking, hypoxia, aspiration, and pneumonia. Prior to development of a gene therapy for AADC, therapeutic practice was to increase dopamine levels through inhibition of monoamine oxidase (MAO) and the direct stimulation of dopamine receptors with dopamine agonists. However, these drug therapies provide little benefit to patients. The adeno-associated virus (AAV) human AADC gene therapy (AGIL-AADC) was aimed at correcting the underlying molecular defect that leads to disease. Work in animal models of disease and in clinical studies in Taiwan demonstrated promising safety and efficacy, including increased dopamine production, gains in motor and cognitive functions, and improved ability to interact with caregivers and engage in physical therapy. Lead Collaborator Agilis Biotherapeutics, Inc., Cambridge, MA Jodi Cook, Ph.D. Public Health Impact This debilitating disease strikes in childhood, affects multiple aspects of the nervous system, and requires lifelong palliative care and caregiver support. Its most severe forms can lead to premature death within the first decade of life. Upstaza™ is now the first approved disease-modifying treatment for AADC deficiency. Outcomes The TRND team retrieved and organized existing data from the three clinical trials conducted in Taiwan, conducting a rigorous statistical analysis. The team completed an End-of-Phase 2 meeting with the Food and Drug Administration (FDA) to discuss the existing data. FDA agreed that the clinical package, plus key preclinical safety, biodistribution, and chemistry, manufacturing and controls data developed by TRND, were sufficient for Agilis Biotherapeutics to proceed to filing a Biologics Licensing Application (BLA) for marketing approval in the U.S. without requiring additional bridging trials. In 2022, PTC Therapeutics, who acquired Agilis and AGIL-AADC in 2018, received market authorization by the European Commission for Upstaza™, the first approved disease-modifying treatment for AADC deficiency and the first marketed gene therapy directly infused into the brain. Subsequently, it was granted authorization by the United Kingdom’s Medicines and Healthcare Products Regulatory Agency. This TRND project is complete. | Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, inherited disorder. TRND scientists are conducting a number of activities to support product development for AGIL-AADC. | Gene Therapy for the Treatment of AADC Deficiency | Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, inherited disorder. TRND scientists are conducting a number of activities to support product development for AGIL-AADC. | Gene Therapy for the Treatment of AADC Deficiency | ||||
| 6431 | Work With the SCTL | The Stem Cell Translation Laboratory (SCTL) is a state-of-the-art research facility within NCATS’ Division of Preclinical Innovation that is dedicated to addressing the scientific and technological challenges in the induced pluripotent stem cell (iPSC) field. SCTL staff members work as a dynamic multidisciplinary team who apply their diverse scientific expertise to iPSC characterization and utilization. The SCTL features advanced equipment and resources not available to most laboratories, such as quantitative, high-throughput, small molecule screening; robotic automation of cell culture workflows; multiscale assay development; 3-D bioprinting; and integrated platforms to profile gene and protein expression and measure functional endpoints in standard cultures, as well as on the single cell level.Areas of InterestAs part of a collaborative network of scientists, the SCTL is leading multidisciplinary efforts for the scalable expansion, controlled differentiation and translational utilization of iPSCs. As one of its goals, the SCTL will develop efficient and standardized methods to produce mature cells from iPSCs meeting strict quality-control and reproducibility standards. Establishing advanced iPSC protocols will be guided by the discovery, validation and dissemination of small molecule reagents that can replace expensive recombinant proteins, xenogenic material and undefined media components in cell differentiation protocols.Who Can Apply?NCATS is particularly interested in applications from early stage scientists (tenure-track and recently tenured) from the NIH intramural and extramural communities (e.g., academic institutions, other government research agencies) and from industry/for-profit organizations (e.g., biotech companies, pharmaceutical organizations). Selected applicants will work in partnership with SCTL staff to address specific biological and technical questions that currently impede the translation of iPSC research into treatments. Applicants will not be granted direct access to SCTL equipment and innovative technologies or provided with direct funding. In addition, all protocols, drugs and other products generated by the SCTL will be disseminated to the scientific community.How to CollaborateThe SCTL is seeking new collaborations to help achieve common goals in iPSC biology in a faster and more coordinated fashion. Prospective collaborators are encouraged to connect with NCATS for timely notification when program updates are announced. Learn more about the proposal process. | Learn how to work with the NCATS Stem Cell Translation Laboratory. | /sites/default/files/stemcell-01.jpg | Work with the SCTL | Learn how to work with the NCATS Stem Cell Translation Laboratory. | /sites/default/files/stemcell-01.jpg | Work with the SCTL | ||
| 6430 | SCTL Proposal Process | Dopamine neurons derived from pluripotent stem cells. NCATS’ Stem Cell Translation Laboratory (SCTL) is seeking new collaborations to help achieve common goals in induced pluripotent stem cell (iPSC) biology in a faster and more coordinated fashion. Interested investigators may submit proposals on July 1, November 1 and March 1 to collaborate with SCTL throughout the year. General Proposal Process NCATS will assess submitted research proposals for scientific merit, technical feasibility, fit with available resources and alignment with SCTL programmatic goals. Steps include: Pre-proposal screening with SCTL staff to confirm eligibility as a collaborating entity and adequacy of the current data package. This process consists of submission of an abstract (no more than one page in 12-point Times New Roman) for the proposed project and a telephone discussion with SCTL staff. Send proposed project abstracts via email to Ilyas Singeç. View detailed proposal instructions (PDF - 160KB). An actionable existing data package. This means the applicant must: Prove that a protocol has already been tested using biological and technical replicates. Provide a complete list of reagents and other details. Submit a summary detailing the outcome of prior solid experimentation (e.g., a description of attempts to reproduce the protocol by different researchers in the same group or collaborators; information on optimization steps and troubleshooting carried out). View detailed proposal instructions (PDF - 160KB). Submission of a full proposal package. This includes a formal request (no more than five pages with data and figures, in 12-point Times New Roman) and specific supporting documents not to exceed two pages (e.g., publications, unpublished or preliminary data presented). Send proposal packages via email to Ilyas Singeç. Peer-reviewed, confidential scientific assessment of the submitted proposal by SCTL staff and advisory group members (composed of NIH staff with expertise in the proposal area). Decision-making criteria include: Strength of the data package Feasibility and the deliverables timeline Synergy with SCTL goals Translational sciences impact In-depth face-to-face meetings with potential collaborators and discussion of raw data under the protection of a confidential disclosure agreement. Agreement that all data (positive and negative results) and resources will be shared with the public. Considerations for SCTL Collaborators NCATS expects collaborative projects to have an emphasis on overcoming technological hurdles impeding the transition of iPSC research from “bench” to “bedside” (e.g., significant protocol improvement). These are not necessarily poor-quality protocols but rather those that are underdeveloped or have significant potential for improvement. For example, a collaborator intends to use an iPSC line in preclinical studies but can obtain only 50 percent purity following differentiation, when purity greater than 95 percent is needed. Alternatively, a researcher obtains well-characterized cells of sufficient quality and purity, but they are prohibitively expensive and time-consuming. In this scenario, the collaboration would focus on replacing expensive reagents with more defined and economic small molecule compounds, as well as shortening the duration of the cell differentiation protocol. In all cases, measurable parameters (deliverables, timelines and resources) would be agreed upon and implemented as a metric for informed decision making. Project Implementation Projects on protocol improvement and other technological hurdles will be initiated as soon as the review process is successfully completed. The SCTL team will conduct experiments to address the protocol improvement as defined in the research plan and will update collaborators on the progress made. NCATS SCTL staff will hold regular conference calls and in-person meetings for scientific exchange and progress evaluation. SCTL team members will rigorously test all steps of final protocols, including the use of biological and technical replicates; such testing will include repeat execution of the protocol by the same scientist, as well as execution of the protocol by a second team member to demonstrate transferability. NCATS will share final results via joint scientific publications, and complete protocol details will be included in a report on rigor and reproducibility testing accessible on the SCTL website. Additionally, the SCTL team will provide complete information on small molecule reagents to replace expensive recombinant proteins, xenogeneic material and undefined media components in cell differentiation protocols. Project Milestones Successful Completion A project will be deemed successful when the corresponding protocol improved by the SCTL team achieves the agreed-upon common goal(s). Upon completion, NCATS will disseminate all relevant data, protocol steps and newly discovered reagents as detailed in the above section. Project Termination NCATS will terminate a project if it fails to meet the protocol improvement goals set by the research plan and if the SCTL team has failed to make progress despite repeated attempts at optimization. Examples of failures to improve the protocol include but are not limited to: Failure to improve the desired-lineage cell type yield beyond the initial level; Failure of high-throughput screens to produce small molecules that improve yield or the duration of differentiation steps or to replace the key expensive reagent; and Failure to decrease variability in protocol performance. If project is terminated due to lack of significant protocol improvement, the joint team will make an effort to publish the negative results in order to inform the public on the roadblocks associated with the particular protocol. | Learn more about the general proposal process for the Stem Cell Translation Laboratory. | SCTL Proposal Process | Learn more about the general proposal process for the Stem Cell Translation Laboratory. | SCTL Proposal Process | ||||
| 6405 | Assay Guidance Workshop Agenda - February 2017 | Saturday, February 4, 2017 — 8:40 a.m. - 6:00 p.m. ET Walter E. Washington Convention Center, Room 152A, Washington, D.C. 20001, USA 8:40 - 9:00 a.m.: Robust or Go Bust: An Introduction to the Assay Guidance Manual G. Sitta Sittampalam, Ph.D., NCATS, NIH 9:10 - 9:55 a.m.: Treating Cells as Reagents to Design Reproducible Screening Assays Terry Riss, Ph.D., Promega Corporation 10:00 – 10:15 a.m.: Beverage Break 10:15 - 11:00 a.m: Strategies for Assay Selection and for the Development of Robust Biochemical Assays Nathan P. Coussens, Ph.D., NCATS, NIH 11:10 - 11:55 a.m.: Assay Interpretation: Studies in Mechanisms and Methods in Assay Interferences Douglas Auld, Ph.D., Novartis Institutes for BioMedical Research 12:00 - 12:50 p.m.: Lunch 12:50 - 1:35 p.m.: Biophysical Approaches to Small Molecule Discovery and Validation Michelle Arkin, Ph.D., University of California, San Francisco 1:45 - 2:30 p.m.: Basic Assay Statistics, Data Analysis and Rules of Thumb Thomas D.Y. Chung, Ph.D., Mayo Clinic 2:30 - 2:45 p.m.: Beverage Break 2:45 - 3:30 p.m.: Reproducibility and Differentiability of Compound Potency Results from Screening Assays in Drug Discovery V. Devanarayan, Ph.D., AbbVie, Inc. 3:40 - 4:25 p.m.: In Vitro Toxicological Testing in a qHTS Platform Menghang Xia, Ph.D., NCATS, NIH 4:35 - 5:20 p.m.: In Vitro Assessments of ADME Properties of Lead Compounds Xin Xu, Ph.D., NCATS, NIH 5:30 - 6:00 p.m.: Open Discussion Session Acknowledgements: Amy McGorry, Meghan Schofield, Steven Hamilton, James E. McGee, Steven D. Kahl, Jeffrey R. Weidner, Chris Austin, Nicholas Gelardi, Sandy Ismail, Terry LaMotte, Cindy McConnell, Anton Simeonov, and Geoff Spencer | The agenda from the February, 2017, Assay Guidance Workshop for High-Throughput Screening and Lead Discovery | Assay Guidance Workshop Agenda - February 2017 | The agenda from the February, 2017, Assay Guidance Workshop for High-Throughput Screening and Lead Discovery | Assay Guidance Workshop Agenda - February 2017 | ||||
| 6374 | NCATS Announces Funding Opportunities to Repurpose Existing Drugs Through Public-Private Partnerships | In collaboration with AstraZeneca and Janssen Research & Development, LLC, NCATS is seeking applications through its NIH-Industry Partnerships initiative to explore new treatments for patients, using existing experimental drugs or biologics. NCATS plans to commit an estimated $6 million to fund six to 10 awards to support partnerships between the pharmaceutical companies and the biomedical research community by making a selection of industry assets available to test ideas for new therapeutic uses. NIH’s National Institute of Dental and Craniofacial Research also is participating in these new opportunities. Through the NIH-Industry Partnerships initiative, which is part of NCATS’ Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program, the Center supports and advances research on a wide range of common and neglected diseases, from lymphangioleiomyomatosis (LAM) to diabetes. Interested researchers are asked to submit a letter of intent by March 17, 2017, and pre-applications are due April 17, 2017. “At Janssen, we believe in the power of public-private collaboration and are very pleased to participate in the New Therapeutics Uses program to maximize the potential of pharmaceutical compounds and ultimately to help improve public health,” said Husseini K. Manji, M.D., global therapeutic head for neuroscience at Janssen Research & Development, LLC. NCATS designed New Therapeutic Uses to foster approaches that improve the translational research process and accelerate the pace at which discoveries are turned into new preventions, treatments and cures for human diseases. A novel drug can take about 14 years and more than $1 billion to develop, with a failure rate exceeding 95 percent. This failure rate means there are many partially developed therapeutic assets that have already been clinically tested. These assets have cleared several key steps along the development path, thereby providing scientists with a strong starting point for contributing their expertise and, ultimately, accelerating the pace of therapeutics development. During the pilot phase of New Therapeutic Uses, NCATS tested the utility of its newly created template agreements, which proved to be effective in facilitating negotiations, enabling the research to begin more quickly. The template agreements reduced the time required to establish collaborations between industry and academia to as few as three months from the more typical nine months to one year. “AstraZeneca is pleased to continue its participation in the pioneering NCATS’ New Therapeutic Uses program,” said Craig D. Wegner, Ph.D., head of the Boston Emerging Innovations Unit at AstraZeneca’s Innovative Medicines & Early Development Biotech Unit. “Connecting academic researchers with industry compounds and scientists has the potential to advance medical research in the pursuit of novel therapies that might otherwise go unexplored.” Learn more about New Therapeutic Uses funding opportunities. Posted February 2017 | NCATS is seeking applications through its NIH-Industry Partnerships initiative to explore new treatments for experimental drugs. | /sites/default/files/NTU-partnerships-2017-900px.jpg | Funding Opportunities for Repurposing Existing Drugs | NCATS is seeking applications through its NIH-Industry Partnerships initiative to explore new treatments for experimental drugs. | /sites/default/files/NTU-partnerships-2017-900px.jpg | Funding Opportunities for Repurposing Existing Drugs |
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