TRAIN eNews is delivered directly to organizations that take part in The Research Acceleration and Innovation Network (TRAIN). TRAIN is an affinity network of foundations interested in taking a more strategic and entrepreneurial approach—what some refer to as “venture philanthropy”—to their role as funders of medical research. TRAIN convenes dozens of forward-thinking foundations to learn from each other and share their novel solutions with the rest of the medical research system. To learn more about becoming a TRAIN organization, visit the TRAIN application page.

In This Newsletter

News from FasterCures
TRAIN Organization Updates
News You Can Use

News From FasterCures

• FasterCures to Host the Research Partnership Maturity Model Interactive Workshop
  FasterCures will host the Research Partnership Maturity Model Interactive Workshop in Santa Monica, California, on Tuesday, March 21, 2023. This one-day workshop will convene patient organization leaders to discuss how they can utilize FasterCures’ Research Partnership Maturity Model to develop strategies for advancing research partnerships and catalyzing biomedical research. FasterCures will share updates and resources coming out of the workshop to our community.

• Upcoming Diversity, Equity, and Inclusion Webinar
  The FasterCures TRAIN webinar “Building More Diverse and Inclusive Patient Organizations and Communities” will be held on Monday, April 3 at 2pm ET. In this virtual discussion, we will hear from foundation leaders who are working to improve diversity, equity, and inclusion both within their organizations and in the work they do externally in research and care. More details and registration information will be sent via email to the TRAIN community soon.

• Apply to be a Mentor in the FasterCures and CZI Rare As One Mentorship Program
  In partnership with the Chan Zuckerberg Initiative (CZI) Rare As One (RAO) Program, FasterCures is coordinating mentorship opportunities to deepen relationships within the nonprofit disease-research community and support RAO organizations as they develop patient-driven research networks and make progress toward treatments and cures. Current program mentors have cited the value of learning collaboratively, expanding their network, and sharing their expertise and lessons learned with their community. Please note, this mentor opportunity is not restricted to those representing rare disease organizations. For additional details and to apply, fill out this questionnaire by April 4, 2023.

• FasterCures’ ‘Advances in Science’ January Webinar Explores Long COVID
  In the first FasterCures’ “Advances in Science” webinar of 2023, Robert W. Eisinger, PhD, acting deputy director for Division of Program Coordination, Planning, and Strategic Initiatives at the National Institutes of Health (NIH), hosted a conversation with guest speaker Gary H. Gibbons, MD, director of the National Heart, Lung, and Blood Institute, on “Building Community-Engaged Research Platforms to Advance Understanding of Long COVID.” View webinar recordings and sign up to receive updates on upcoming webinars in the series.

TRAIN Organization Updates

• Rare As One Cycle 1 Mentees Join TRAIN
  FasterCures is pleased to accept the cycle 1 participants of the CZI and FasterCures’ RAO Mentorship Program into TRAIN. We are excited to further engage with the RAO community and welcome them to this incredible network! Check out this LinkedIn post welcoming our newest members.

• TRAIN Welcomes SARC as New Member
  FasterCures is excited to announce our newest TRAIN member: the Sarcoma Alliance for Research through Collaboration (SARC). SARC is a nonprofit organization dedicated to the development and support of research for the prevention, treatment, and cure of sarcomas—a cancer of the bone and connective tissue of the body.

• Children’s Tumor Foundation Expands as Impact Investor in NF Research
  The Children’s Tumor Foundation (CTF) has announced its new role as an impact investor in a Phase 2b clinical trial at NFlection Therapeutics, a biotechnology company focused on the discovery and development of effective, targeted therapies for rare disorders. CTF has a diversified portfolio of neurofibromatosis (NF) drug development investments tackling all forms of NF, from early-stage investments in young investigators, to established academic initiatives, to a large-scale, open data collaborative.

• Friends of Cancer Research Relaunches Advocacy Training through ProgressforPatients.org
  The Friends of Cancer Research announced the relaunch of its impactful advocacy training through ProgressforPatients.org. The platform is free to use and strives to help patients, advocates, and caregivers acquire the necessary tools to communicate effectively with drug researchers, developers, and regulators. More than 650 patients, advocates, caregivers, and individuals have completed the advocacy education program.

News You Can Use

• First FDA-Approved Treatment for Friedreich’s Ataxia on Rare Disease Day
  On National Rare Disease Day, the US Food and Drug Administration (FDA) approved Reata Pharmaceuticals’ SKYCLARYS, a treatment for Friedreich’s ataxia (FA), for adults and adolescents aged 16 and older. This is the first FDA-approved drug treatment for the FA community. This approval was based on a single study, focusing on the same set of patients over time as opposed to new ones. The notion that patient-community collected natural history data can be regulatory-grade and pave the way for product approvals is crucial to the future of therapy development for the rare disease community.

• ICON Develops Toolkit Designed to Mitigate Common Risks in Rare Disease Clinical Trials
  ICON, in collaboration with the Rare Disease Advisory Committee, has developed a toolkit that includes four digital resources designed to mitigate frequent risks in rare disease clinical trials and to support new patient-centric practices that promote trial participation, helping sponsors save costs related to inefficiency, high trial dropout rates, protocol amendments, and inability to complete a trial on time. The four tools include a patient-centric protocol risk assessment tool, a rapid participation burden survey tool, a patient involvement value dossier, and “What To Ask When You’re Interested in a Clinical Trial: A Guide for Rare Disease Patients and Caregivers.”

• Newly Authorized NIH Office of Autoimmune Disease Research
  In December 2022, President Biden signed an omnibus federal spending bill that includes language to establish the Office of Autoimmune Disease Research under the Office of Research on Women’s Health within the NIH. The bill provides $10 million to establish the new office and directs the office to develop a strategic plan for an autoimmune research portfolio.

• The National Center for Advancing Translational Sciences Awards Funding to Shorten Diagnosis Timeline
  The National Center for Advancing Translational Sciences strives 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 diagnose people with rare diseases correctly. The three research projects are working to speed up the timeline using methods such as machine learning, genetic analyses, and medical evaluation.

• Global Genes Hosts RARE Drug Development Symposium 2023
  Global Genes will host the RARE Drug Development Symposium May 1–3, 2023, in Philadelphia, Pennsylvania. Hosted alongside the Orphan Disease Center of the University of Pennsylvania, the symposium will bring together rare disease advocates to acquire the knowledge, skills, and connections they need to advance therapy developments for their communities.

• Operation Warp Speed for Rare Diseases
  The Food and Drug Administration (FDA) has announced that the next iteration of Operation Warp Speed will be aimed at rare diseases. According to the director of the FDA’s Center for Biologics Evaluation and Research, Peter Marks, MD, PhD, the FDA will initiate a pilot for the program soon. The pilot intends to take a rare disease drug with promising results and allow the clinical development to happen in constant communication, with the sharing of preliminary results before submission of a New Drug Application or a Biologics License Application. The FDA hopes this will quicken the testing process for drugs of rare diseases with no alternatives and allow for the quicker resolution of issues.

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