Critical Path Institute

“Jeff jacob and shaun kirkpatrick have a business relationship.”

“Form 990 is prepared by an external cpa firm using information provided by the organization. The form is reviewed by the organization's cfo, president/coo, ceo, the board audit, finance, and risk committee, and the board of directors prior to being finalized.”

“All members of the board of directors and all officers of the organization are required to fill out a conflict of interest form annually. The results are compiled and reviewed by the board's audit, finance, and risk committee. Any actual or perceived conflicts that have the potential to bias any discussions or decisions by the board are discussed by the board. Any director with a real or potential conflict will recuse themselves from any discussion or decision where the conflict has a bearing.”

“A board of directors compensation committee meets and reviews executive compensation annually. Periodically, the committee requests a compensation analysis to determine if employee compensation has remained within market range or if adjustments are required. This information is then used as a basis for the compensation committee's recommendation to the board of directors. An overall salary program for employees must be approved by the board of directors compensation committee before implementation. All employee salary increases must be approved by the president/coo.”

“Articles of incorporation can be found on the arizona corporation commission website. The conflict of interest policy and the financial statements (via the annual report) are posted on the institute's website.”

“Cp-rnd was founded in september 2022 pursuant to the act for als in order to develop collaborations that advance treatments to improve the lives of those affected by rare neurodegenerative diseases, including als. In the first two years of the project, cp-rnd has achieved important milestones regarding the focus on patient-focused drug development, data collection and aggregation, and drug development tool solutions to address the gaps in drug development for rare neurodegenerative diseases. Cp-rnd also established a pwle working group in coordination with accelerating medicine partnership (amp in fnih) to give and receive feedback on als-related projects. Cp-rnd has initiated working groups focused on clinical outcome assessments (coas), digital health technology tools and data in order collaborate with key opinion leaders to more clearly identify and address gaps in drug development. Upon recommendation by the fda, cp-rnd completed a thorough evaluation of the common coas used in als clinical trials against the fda's roadmap for developing patient-focused outcome measures and made recommendations on methods to improve or develop coas for key concepts important to pwle with als. Cp-rnd has researched over 70 large databases, with a priority listing created and shared with nih, fnih and fda. Cp-rnd, in partnership with rdca-dap, is working to create an als data knowledge portal containing prioritized datasets that can be standardized and used by stakeholders to advance research in als including the development of drug development tools. Of the priority listing created, three data sets have been received and are in various stages of curation and aggregation. Progress for data dictionary collection and data share agreements remain ongoing with the remaining data sets identified.”

“Rdca-dap is an fda-funded initiative that provides a centralized and standardized infrastructure to support and accelerate rare disease characterization with the goal of accelerating therapy development. The rdca-dap effort started in 2019 and launched the public interface of the platform in september 2021. To date, the platform hosts over 80 rare disease data sets representing over 41 disease states. There are now 642 platform users and 43 active workspaces, where users actively leverage the rdca-dap database to inform rare disease research and drug development activities. Active agreements currently include 42 data contribution agreements, 8 nord data contribution coversheets, 3 cords registry data contribution coversheets, and 34 task force agreements. Rdca-dap leveraged friedreich's ataxia database to develop a clinical trial simulation tool to inform trial design in fa. The tool entered regulatory submission to the office of clinical pharmacology in september 2024. Three task forces structured as small scale public private partnerships were launched in 2023 and 2024 to leverage the database and advance drug development solutions. The progressive supranuclear palsy and limb-girdle muscular dystrophy task forces are advancing disease progression modeling to inform trial design, and the rare mitochondrial diseases task force is focused on data aggregation to develop recommendations aimed at enhancing real world data collection and best practices. 2023 marked the release of critical path ontology (cpont) application, which facilitates mapping and linking between different standardized data vocabularies. In addition, a publication was published in the journal of pharmacokinetics and pharmacodynamics (barrett js et al., 2023). Lastly, the program made a significant impact on professional development in 2024, with eight webinars held as part of its professional development series, reaching 772 registrants and 37,344 attendees.”

“Critical path institute's translational therapeutics accelerator (trxa), launched in 2022, is designed to assist academic investigators in overcoming the drug development "valley of death" by providing strategic funding and mentorship. Through its bridging research and innovation in drug development grants (bridge) program, investigators are supported by trxa in defining and implementing effective pathways for advancing promising therapeutic candidates from the laboratory toward clinical evaluation. In 2024, trxa supported four (4) drug development projects with a total of $1,612,597 in grant funding. In addition to funding, grantees received the following: - strategic and tactical expertise in drug discovery and development, including considerations in regulatory science and preclinical strategy. - resources and hands-on guidance to build comprehensive data packages that strengthen the value and attractiveness of therapeutic candidates for partnership with biotechnology or pharmaceutical companies. - support in engagement of contract research organizations (cros) to conduct key discovery-phase experiments and validate academic findings, ensuring data robustness and reproducibility. In addition, through its 2024 request for proposals, trxa identified seven (7) new projects for bridge awards, totaling $2.4 million, to be funded in the 2025 calendar year.”