Foundation for the National Institutes of Health

06/24/2026

We're excited to announce a significant milestone for the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC): completion of the pre-IND meeting with the FDA for the consortium's gene therapy program targeting NPHP5 retinal degeneration. This achievement marks an important step toward advancing a potential treatment for individuals living with this rare inherited retinal disease, which can cause severe vision loss beginning in childhood.

The rigorous dialogue with FDA provided valuable feedback on the development pathway and helps further de-risk the program as it progresses toward an Investigational New Drug (IND) application and first-in-human clinical studies. Early FDA engagement is a critical component of gene therapy development, helping ensure alignment on preclinical, manufacturing, and clinical considerations.

Reaching this important milestone with FDA reflects the power of collaboration. Thank you to the NPHP5 principal investigators, BGTC Consortium partners, subject matter experts, BGTC Coordination Center, and NCATS as IND sponsor, for your dedication, expertise, and unwavering commitment to this shared goal. Next steps include incorporating FDA feedback, completing remaining studies and manufacturing activities, submitting an IND application, and ultimately working toward initiation of a clinical trial.

Learn more about how pre-IND meetings are an important step in the regulatory pathway from this recent ASGCT Community Quick Take, created in collaboration with the BGTC: https://youtu.be/L6V7HvvrDRw?si=Q09vvTxSGkxJ--PL

For patient advocates and disease foundations working toward a gene...

06/16/2026

Living with Long COVID can be overwhelming, especially when it comes to understanding research timelines as we evaluate potential treatments. Join us for an informative RECOVER-TLC webinar to learn more about how clinical trials work, steps we take to ensure study participant safety, and how findings can impact clinical practice. This session will provide practical information, answer common questions, and empower you to make more informed decisions if you want to participate in a RECOVER-TLC clinical trial.

Join us on July 16 at 1 p.m. ET: https://fnih.zoom.us/webinar/register/WN_G2sv1jSRQyOL3PG85hQDFQ

06/11/2026

Sneak peek: Music can spark a memory, steady a step, or reach someone when words cannot. FNIH is preparing to launch a new initiative that will fund researchers examining the relationships between neuroscience, music-based therapeutic interventions, and patient care. FNIH’s Kevin A. Klock, COO and General Counsel, will be speaking about the new initiative at the 5th Annual LA County Arts and Health Week Summit this Friday, June 12, at 2:30 pm Pacific time.

Hosted by LA Opera and featuring acclaimed soprano and advocate for music and health Renée Fleming, the event begins at 10:00 am Pacific time and is free and open to all for in-person attendance. Register at LAOpera.org/Summit.

Learn more about the FNIH’s Music as Medicine Neuroscience Research Initiative at fnih.org/music.

06/04/2026

Team FNIH is inviting runners, walkers, and wheelchair athletes to join us for the DC Half Marathon & 5K — and if you sign up by June 5, you get early-bird swag. We also provide a training schedule and personalized fund-raising advice.

Whether you’re chasing a personal record, walking with friends, or rolling across the finish line, every mile helps support biomedical research. Through our partnerships, FNIH is making strides to advance gene therapies for rare diseases, more precise approaches for cancer, earlier Alzheimer's interventions, and more. We hope you will join us! https://fnih.org/team-fnih/

06/04/2026

The FNIH builds powerful research collaborations to tackle complex medical challenges – and that makes the Charles A. Sanders, MD, Partnership Award especially meaningful. This recognition honors the people and organizations that have made outstanding contributions in support of the mission of NIH. We are excited to announce the 2026 recipients:

🏆Dan Doctoroff, Target ALS founder and research advocate
🏆Biogen
🏆The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), one of NIH’s 27 Institutes and Centers

Recipients will be recognized at the 14th Annual FNIH Awards Ceremony on Oct. 28, 2026. Learn more: https://fnih.org/press-release/fnih-announces-recipients-sanders-partnership-award

Target ALS
Biogen
National Institute of Arthritis and Musculoskeletal and Skin Diseases

06/03/2026

We mourn the loss of Tony Villiotti, a patient ambassador for the FNIH and founder of Liver Education Advocates. After living through metabolic dysfunction-associated steatohepatitis (MASH), liver cirrhosis, liver cancer, and finally a lifesaving liver transplant, Tony turned his personal health journey into a passion for educating others about fatty liver disease, a condition that often has no symptoms until it progresses to an advanced stage.

Tony first became involved with the FNIH through the Biomarkers Consortium's NIMBLE project to validate noninvasive biomarkers for diagnosing and monitoring liver disease, with the goal of replacing painful, invasive liver biopsies. He was a strong advocate for developing clinical tools, such as blood tests, that could make it less risky and more convenient for patients to take steps toward early diagnoses and interventions. He subsequently contributed his lived experience and patient perspective to the design and implementation of the MASHTrack project, which is examining biomarkers that could predict outcomes for patients with MASH, so patients at higher risk could be brought into effective clinical trials.

The FNIH is grateful for Tony’s determination both to increase awareness and knowledge about liver disease in the broader community and to incorporate patient needs into research that will advance clinical care and treatment. We remember Tony for his eternally positive attitude in the face of personal hardship and his commitment to the patient community. His optimism, collaborative spirit, and steadfast belief that things can and should be better for patients enabled us to build much-needed programs to find solutions for people living with liver diseases. His impact will live on for decades to come.

Read more about Tony at

FNIH honors the legacy of Tony Villiotti, who passed away on May 28, 2026. We are grateful for his partnership and his liver disease advocacy.

06/03/2026

Will Tolentino, Director of Advancement Services, was originally drawn to work at FNIH because of our mission to accelerate biomedical innovation for people facing some of the toughest health challenges, including HIV. After being hired, he also committed to being a steady financial contributor himself. “Recurring gifts, no matter the size, can provide steady income to an organization and add up to a much bigger effect over time," he said.

Learn more about what motivates Will to give back.

Will Tolentino first learned about the FNIH back in 2009, when a friend alerted him to a job opening. At the time, he was working as a fundraising consultant and had previous experience as a development officer for charitable organizations. The mission of the FNIH — advancing biomedical research ....

05/29/2026

Every medical breakthrough starts with people willing to go the distance. This fall, Team FNIH is bringing together runners, walkers, and wheelchair athletes to move with purpose at the DC Half Marathon & 5K. By joining us, you can support a future where scientific discovery powers health solutions for patients who are waiting. Learn more at https://fnih.org/team-fnih/

05/28/2026

May is Osteoporosis Awareness and Prevention Month, which makes it a great time to talk about the recent FDA action that could accelerate the development of much-needed new treatments. Osteoporosis is a chronic condition that develops when bone mass decreases with aging, a process that is accelerated by menopause in women. In the United States, more than 10 million older adults have osteoporosis, and another 43 million are at high risk.

The American Society for Bone and Mineral Research

Bone Health and Osteoporosis Foundation

05/21/2026

In recognition of Women’s Health Month AND Osteoporosis Awareness and Prevention Month, we celebrate a recent FDA action that could accelerate the development of much-needed new treatments for osteoporosis, a common condition in older women that thins bones and can lead to painful fractures.

For many years, the development of novel osteoporosis therapies was hindered by the high cost and long timelines of studies that evaluate a drug’s effectiveness by measuring bone fracture rates. But in December, the FDA qualified a change in bone mineral density as a surrogate biomarker for fractures in post-menopausal women participating in osteoporosis drug trials. As a result, studies can be shorter and less costly – and may lead to new therapies more quickly.

A multi-sector partnership launched and managed by the FNIH’s Biomarkers Consortium generated the data and evidence for this qualification. Learn more: https://fnih.org/our-programs/biomarkers-consortium-bone-quality-project/