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 welcome opportunities to build additional relationships—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 received funding and scientific support from the Cystic Fibrosis Foundation, whose mission is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives. This partnership supports the discovery, engineering, and development of a 4DMT AAV gene therapy product designed to be optimized for aerosolized delivery to treat cystic fibrosis, a hereditary disease involving the lung and other tissues. The Foundation brings research and clinical expertise as well as a network of doctors, trial investigators, and patients that not only help inform product design, but also help support clinical trial design and execution.
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.
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.
Phase I Clinical Trial of 4D-110
If you are a patient, caregiver or healthcare professional and are interested in more information about this trial please contact us at the link below (see the Clinical Trial box for a patient to enter in their contact information) or visit clinicaltrials.gov
Natural History Study of Choroideremia
View on clinicaltrials.gov.
Please check back frequently as we are planning to initiate multiple studies in 2020. You can also:
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 the button below and enter the disease 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. Click or tap the button below and enter the disease into the search bar.
Lysosomal Storage Disorders: Fabry Disease
Lung Diseases: Cystic Fibrosis
Muscular Dystrophies: Duchenne Muscular Dystrophy
Ophthalmological Diseases: Retinitis Pigmentosa, Choroideremia and Others
Interested in partnering with us? Want to learn more about our current programs or community outreach events?
We’d love to hear from you! Email us here or fill out this form and we’ll get back to you shortly.