Overview

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:

  • Work to deepen our understanding of patient experiences, unmet medical needs, treatment preferences and clinically significant treatment outcomes
  • Collect the patient perspective on gene therapy and gene editing, including patient concerns and needs
  • Work to deepen our understanding of regulatory approval pathways and clinical endpoints
  • Improve patient awareness of the potential to enroll in our clinical trials
  • Develop relationships with disease-specific clinician-scientists for their expertise and guidance (i.e. key opinion leaders)
  • Bring patients hope for potential life-changing therapies

PATIENT ADVOCACY PARTNERS

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.

Patient Advocacy

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.

 

COMMUNITY OUTREACH/SUPPORT

Have an event you think we’d be interested in supporting? Contact us here!

UPCOMING/ONGOING EVENTS

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.

PAST EVENTS

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

PATIENT STORIES

CHOROIDEREMIA PATIENTS AND FAMILIES SHARE THEIR JOURNEYS

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.

CLINICAL TRIALS

ACTIVE CLINICAL TRIALS

Natural History Study of Choroideremia
View on clinicaltrials.gov.

UPCOMING CLINICAL TRIALS

Please check back frequently 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.

YES, I’D LIKE TO BE NOTIFIED ABOUT 4DMT CLINICAL TRIALS UPDATES!

RESOURCES

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.

GENERAL SOURCES OF INFORMATION

MedlinePlus
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.
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Mayo Clinic
The Mayo Clinic is a similar source of information. Click or tap the button below and enter the disease into the search bar.
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DISEASE-SPECIFIC INFORMATION

PARTNER WITH US!

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.