EIN 51-0169988

2023 Education Highlights

• Hosted the 36th National CF Education Conference as a hybrid event, where 28 speakers – including renowned experts in the field of CF, patient panelists, and CF researchers – presented the latest in research and clinical practice to over 100 in-person attendees and over 400 members of the global CF community virtually. Evaluations found that over 95% of participants improved their knowledge of CF and treatment options.

• Produced and posted 22 podcasts through our CF Community Voices podcast series, with over 3,400 combined views.

• Maintained our website as a dynamic resource – available in multiple languages – for the global CF community. In addition, CFRI engaged its 18,000+ Facebook, Twitter and Instagram followers with breaking news, resources, and inspiring stories.

• Distributed 28,000 copies of the CFRI Community newsletter to homes and health centers across the globe. Distributed 6,000 copies of CFRI Comunidad, CFRI’s Spanish-language newsletter, to CF centers nationwide.

• Provided updates on CF research, special events, advocacy efforts, and support programs through our weekly eNewsletter, distributed to over 15,000 subscribers.

• Distributed nearly 10,000 informational brochures about cystic fibrosis and CFRI’s services.

2023 Support Highlights

• Underwrote individual therapy sessions for nearly 100 individuals with CF and/or their immediate family members from 23 states, resulting in a statistically significant reduction in depression and anxiety symptoms.

• Offered free weekly online CF-specific wellness classes – including strength training, yoga, pilates, dance, flexibility and stretching – which were attended by over 100 people with CF and their family members.

• Hosted four retreats for our national and global CF community: a virtual CF Spring Retreat and hybrid Summer Retreat for adults with CF; and an in-person Spring Retreat and virtual Fall Retreat for mothers of children/adults with CF. All events provided resources, psychosocial support and connection to peers; evaluations showed measurable decreases in symptoms of depression and anxiety.

• Provided eight monthly virtual Support Groups facilitated by CF social workers: Adults with CF; caregivers of children with CF; parents/spouses/partners of adults with CF; people who lost a loved one to CF; adults with CF post-transplant; teens with CF; Spanish-speaking CF community members; adults with a late CF diagnosis. Participants were from across the U.S. and globe.

• Increased support for the Spanishspeaking CF community via print, social media, and website resources, including a Spanish language version of CFRI’s newsletter.

2023 Advocacy and Awareness Highlights

• Advanced awareness of the CF community’s racial and ethnic diversity through the creation of podcasts and films, informational sheets and resource guides in multiple languages; provided captioning and subtitles for conference attendees; made CFRI website accessible in multiple languages.

• Pursued efforts to create state Rare Disease Advisory Councils (RDACs) through coalition building with rare disease groups; sent multiple action alerts and partnered with other rare disease groups to engage our community on issues that impact the rare disease community.

• Engaged with other disease groups to advocate on potential threats to CF community access to therapies, including copay accumulator programs, surprise medical billing, and state Prescription Drug Affordability Review Boards (PDABs).

• Produced a CF Awareness and Advocacy video featuring members of the CF community to inspire greater engagement in advocacy activities.

• Created English/Spanish language informational fliers to raise awareness of the significantly higher rates of colorectal cancer among those with CF; released podcasts on this topic.

• Held over 40 virtual and in-person meetings with state and federal legislators to discuss issues impacting the health and quality of life for those with CF, including access to care.

• Participated in diverse activities to raise awareness of the discriminatory use of Quality Adjusted Life Year (QALY) to assess drug pricing and value for rare disease drugs. • Strengthened alliances with our CF organizational partners in the Cystic Fibrosis Engagement Network, with national rare disease organizations, disability groups, and with our partner organizations in the American Thoracic Society Public Advisory Roundtable.

2023 Research Highlights

In 2023, the following researchers conducted research with the support of CFRI:

Elizabeth Nash Memorial Fellowship Program

Paul Bollyky, MD, PhD (Principal Investigator), Kevin Qingquan Chen, PhD (Postdoctoral Fellow) – Stanford University Targeting Bacterial Resistance to Phage Therapy in Cystic Fibrosis

Matthew Porteus, MD, PhD (Principal Investigator), Anais Amaya Colina, PhD (Postdoctoral Fellow) – Stanford University A Hybrid Gene Correction Strategy for Cystic Fibrosis

New Horizons Program

Nadia Ameen, MD – Yale University School of Medicine Elucidating The Ion Transport Functions of CFTR High Expresser Cells (CHEs) And Its Relevance To Intestinal Disease In Cystic Fibrosis

Benjamin Chan, PhD – Yale University School of Medicine Optimization of Phage Therapy to Reduce PseudomonasInduced Inflammation in Cystic Fibrosis

Carlos Milla, MD – Stanford University Improving CF Airway Mucociliary Clearance: Toward Transition from Animals to Humans

Kenichi Okuda, MD, PhD – University of North Carolina at Chapel Hill Pathways Maintaining Basal Mucin and CFTR-Mediated Fluid Secretion in the Human Distal Airway

Zachary Sellers, MD, PhD – Stanford University Targeting IRBIT to Correct Bicarbonate Secretory Defects in Cystic Fibrosis

Katrine Whiteson, PhD – University of California Irvine Targeting Recalcitrant CF Pathogens with Phages, Antibiotics, And Small Molecule

Adjuvants Special Circumstances Grant

Beate Illek, PhD – University of California San Diego CFTR mRNA Delivery to CF Airways