At 4DMT, PATIENTS are at the center of everything we do. Everything we’re building is in service of better patient survival and quality of life—and we involve patients every step of the way. This focus on patients is essential to our success.
The voice of the patient is key within 4DMT. Our outreach to patients and their communities helps us understand their perspectives. We then incorporate patients’ goals in our drug development and patient support activities. To that end, through our patient advocacy collaborations we aim to achieve the following:
We’re proud of our strong relationships with our current patient advocacy partners. We’re also very open to additional opportunities—
if you are a patient or family member or represent a patient advocacy group and are interested in partnering, contact us here.
Our partnership with the Foundation Fighting Blindness (FFB) is focused on developing potentially breakthrough treatments—and possibly cures—for patients with inherited retinal diseases. We are developing drug candidates based on our proprietary AAV vectors which are optimized for intravitreal administration. The FFB brings a wide range of strengths to our collaboration including clinical expertise and networks of experienced physician-scientists, key opinion leaders, trial investigators and patients in need.
We have just announced a new agreement with the Cystic Fibrosis Foundation (CF Foundation) to develop precision gene medicines for cystic fibrosis (CF). The CF Foundation’s mission is to cure CF and to provide all people with the disease the opportunity to lead full, productive lives. Their investment builds upon previous work with 4DMT to advance new therapies for CF. Under this agreement, the CF Foundation will support the completion of IND-enabling research and development activities, and the planned Phase 1/2 clinical study of 4D-710, 4DMT’s wholly-owned product candidate for the aerosol treatment of CF lung disease. The Foundation also brings to us research and clinical expertise as well as a network of doctors, trial investigators, and patients that not only help inform product design, but also support clinical trial design and execution.
Our partnership with the National Fabry Disease Foundation (NFDF) is focused on developing a breakthrough treatment for patients with Fabry disease, a progressive and often life-threatening condition. NFDF is dedicated to supporting the Fabry disease community through multiple initiatives, including assisting with Fabry disease education, facilitating Fabry disease identification to improve Fabry disease recognition and diagnosis, providing various forms of assistance to individuals with Fabry disease, supporting Fabry disease research, and promoting advocacy for Fabry disease issues. Our partnership with the NFDF is key as we advance our lead product candidate into clinical stage trials and embark on a natural history study in patients with this devastating disease.
Our partnership with the Choroideremia Research Foundation (CRF) is focused on developing a transformative gene therapy product designed for intravitreal administration to treat choroideremia, a hereditary retinal degenerative disease caused by a mutation in the Rep-1 gene. The Foundation brings together grant support; clinical and regulatory expertise; and a network of doctors, trial investigators, patients and patient families. CRF resources and expertise are helping us plan and execute our clinical trials, including our natural history study.
We have received funding and scientific support from Cure Duchenne, whose mission is to save this generation of children and young adults with Duchenne muscular dystrophy, the most common form of muscular dystrophy. Cure Duchenne is helping to advance our Duchenne muscular dystrophy program.
We are working with the Fabry Support & Information Group (FSIG) as we develop a transformative gene therapy product to treat Fabry disease, a lysosomal storage disorder for which there are currently no adequate treatments. FSIG strives to support affected families and individuals, helping them lead fuller lives by raising awareness of Fabry disease and its symptoms, advocating for community needs, and leading the fight for the most effective treatments possible and eventually a cure. FSIG is providing 4DMT access to medical experts, researchers, and patients and families, helping us with both the development of our lead product candidate and our Fabry disease natural history study geared towards better understanding the wide-ranging impacts of this disease.
Have an event you think we’d be interested in supporting? Contact us here!
Rapid Research & Progress Series
An ongoing internal 4DMT series focused on bringing patients into our offices to share their perspectives. Our inaugural guest was singer/songwriter Olivia Davis, who navigates life with cystic fibrosis.
DISORDER: The Rare Disease Film Festival—Supporter
November 9–10, 2019, San Francisco, CA
National Fabry Disease Foundation’s Annual Conference—Sponsor
September 5–6, 2019, Greensboro, NC
Choroideremia Research Foundation’s 2018 Conference—Attendee
June 21–24, 2018, Irving, TX
Hear choroideremia patients and their loved ones explain—in their own words—the devastating impact of this debilitating eye disease. At 4DMT,
we understand how truly life-changing a treatment could be. We’re dedicating ourselves every single day to make that treatment a reality, giving the choroideremia community hope for a better future.
When I was 19 years old, my cystic fibrosis started getting serious, and I began to face the frightening reality of what it means to live with this disease. But I realized that I wasn’t alone in this feeling – my mother had been dealing with this reality since the day I was diagnosed. When I was too young to understand the gravity of my disease, my mother held it all for me. She held the fear, anxiety, and grief, with grace and humor and hope, and she always made me feel safe. For the first time, I started to reflect on how difficult that must have been – to raise a small child with such positivity and light, when there was a threat looming above our heads at all times. I can’t imagine the strength it must have taken to remain the brightest source of joy and comfort in those times when she felt helpless. As a child, when I was in the hospital for weeks at a time, my mom would pack suitcases large enough for me to fit in, and decorate the room so beautifully that no one would recognize it as a hospital. She would bring puzzles to pass the time, essential oils to combat the hospital smell, and enough books and CDs for a lifetime. We made pro and con lists about being in the hospital, and she helped me see that everything has a good side, if you pay enough attention to see it. Now that I am older, my mother can’t shield me from the fear. She is not so much my protector anymore – she is my comrade and my confidant as we navigate what it means to live with a life threatening illness. I am eternally grateful for everything this woman has given me and continues to teach me. This video is for my fiercest advocate and my best friend, my mother, Sara Pearson. Thank you so much to my talented friend Jenna Wallace for making this song and story come to life.
If you are a patient, caregiver or healthcare professional and are interested in receiving more information about 4DMT clinical trials, please fill out the form below. To read more about our clinical trials visit the links below.
An Open-label, Phase1/2 Trial of Gene Therapy 4D-310 in Adult Males with Classic Fabry Disease
View on clinicaltrials.gov.
Phase 1/2 Clinical Trial of 4D-125 for X-Linked Retinitis Pigmentosa (XLRP)
View on clinicaltrials.gov.
Phase 1 Clinical Trial of 4D-110: Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
View on clinicaltrials.gov.
Natural History Study of Choroideremia
View on clinicaltrials.gov.
Please check back as we are planning to initiate multiple studies in 2020. (You can also search “4D Molecular Therapeutics” on: clinicaltrials.gov.) Or, sign up using the form below and we’ll alert you when more information is available. (Your contact information will never be shared or sold.)
At 4D Molecular Therapeutics (4DMT), we are committed to developing safe, effective and transformative therapies for patients with rare diseases and limited or no treatment options. We understand the importance of bringing these therapies to the broadest group of patients as quickly as possible. We believe that the best path to potential regulatory approval, and subsequent access for the greatest number of patients, is by executing our clinical studies as efficiently as possible. Clinical studies collect the information necessary to understand the safety and efficacy of the potential therapy allowing for regulatory review and potential approval.
After careful deliberation, we have determined that at this point in time, we must focus our efforts on conducting our clinical trials as efficiently as possible. Therefore, participation in 4DMT clinical trials is the only way for patients to gain access to 4DMT’s investigational therapies currently early in development. We will continually evaluate the possibility of compassionate use, “Right to Try” and other expanded access programs.
Investigational drugs have not yet been approved by FDA or other regulatory agencies. Furthermore, investigational drugs may, or may not, be effective in the treatment of a condition, and use of the product may cause unexpected serious side effects.
We believe in the importance of collaborating closely with patients, their families, patient advocacy organizations, physicians, researchers and regulatory authorities to help achieve this goal.
Treating physicians, patients and/or caregivers interested in learning more about 4D Molecular Therapeutics’ investigational gene therapies currently undergoing clinical studies can find more information at www.clinicaltrials.gov.
For parents or patients interested in general information, please contact Patient Advocacy at info@4DMT.com and talk with your treating physician.
The list below is a starting point for people with diseases for which we are investigating potential therapies. Use these links to learn more about the condition and about available support and advocacy resources. Note these are all third-party (i.e. not 4DMT-sponsored) websites. Also note this list does not include all available resources.
A good source for disease information is MedlinePlus. Click or tap on the button below and then enter the disease you’re interested into the search bar. Also at the end of many of their fact sheets are listings of other reputable sources of information.
The Mayo Clinic is a similar source of information. Again, click or tap on the button below and then enter the disease you’re interested into the search bar.
Ophthalmology: XLRP, Choroideremia and Others