4D Molecular Therpeutics

Who is 4DMT?

4D Molecular Therapeutics, or 4DMT, is a biotechnology company focused on developing advanced gene therapies for rare and common diseases.

Mission and Approach

4DMT’s mission is to develop innovative gene therapies that address the underlying causes of disease, with the goal of providing long-term benefits to patients.

The company uses a proprietary platform called Therapeutic Vector Evolution to create highly specialized viral vectors. These vectors are designed to deliver therapeutic genes efficiently to specific tissues, including the retina, while minimizing the risk of immune reactions.

By exploring multiple vector options, 4DMT aims to develop safe and effective gene therapies that can be tailored to different diseases. Their approach emphasizes precision, safety, and patient-centered research, supporting clinical trials such as 4D-110 for choroideremia.

Relevance to the CHM Community

The 4D-110 study adds to ongoing research exploring gene therapy for choroideremia. For patients and families, it helps provide information about what types of treatments are under investigation, how clinical trials are designed, and what early safety and efficacy signals are being studied.

Safety and Research Ethics

As with all gene therapies, 4DMT’s studies follow strict clinical guidelines to prioritize participant safety and careful monitoring.

Important note: Please note that any information about clinical trials, gene therapies, or genetic testing is for informational purposes only. The Choroideremia Research Foundation does not endorse any specific company, trial, or test. Patients should discuss questions with their healthcare provider.

4DMT Sponsored Study

Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

NCT: NCT04483440
URL: https://clinicaltrials.gov/study/NCT03496012
Intervention: BIOLOGICAL: Injection of AAV2-REP1 (10e11 vg)

NOTE: Enrollment for this study is now closed.

Technical Summary: This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).  This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single IVT injection of 4D-110 at three dose levels in male patients with genetically-confirmed CHM. 13 patients were enrolled and are currently being monitored. The study is estimated to be completed in 2027.

Lay Language Summary: This study is testing the safety and early signs of effectiveness of a gene therapy called 4D-110. The treatment is given as a single injection into the eye. Only males with genetically confirmed choroideremia are included. This is a Phase 1 study, which means the main goal is to understand whether the treatment is safe and how well people tolerate it. Researchers are also looking for any early changes or improvements in vision. Thirteen participants have enrolled and are being monitored. The study is expected to finish in 2027.