4D Molecular Therapeutics’ 4D-110 receives FDA orphan drug designation for choroideremia

4D Molecular Therapeutics (4DMT), a prominent California-based gene therapy firm, has achieved a significant milestone with the US Food and Drug Administration (FDA) granting orphan drug designation to its gene therapy product, 4D-110. This designation is specifically for the treatment of choroideremia (CHM), an X-linked recessive genetic disorder that leads to progressive vision loss.

Choroideremia is caused by mutations in the REP-1 gene, which begins with diminished night vision and progressively impairs peripheral vision. If left untreated, it ultimately results in the complete loss of visual acuity and blindness. The disorder affects the retinal pigment epithelium, leading to a gradual degeneration of the retina.

4D-110: A Novel Approach to Treating Choroideremia

Peter Francis, Senior Vice President of Clinical and Translational Research and Development Program Leader at 4D Molecular Therapeutics, highlighted the innovative nature of 4D-110. Unlike other adeno-associated virus (AAV) gene therapy treatments that require subretinal surgical injections, 4D-110 is administered via an intravitreal injection. This method is designed to achieve widespread expression of the REP-1 protein across the entire retina, offering a potential treatment solution for patients at all stages of choroideremia.

In contrast to existing therapies, 4D-110 aims to address the entire retina rather than localized regions, potentially offering a more comprehensive approach to managing this debilitating condition.

Ongoing Research and Development

Currently, 4D Molecular Therapeutics is conducting a natural history study involving over 50 choroideremia patients. This study is crucial for establishing baseline characteristics and will support the rapid enrollment of participants in upcoming clinical trials. The company anticipates launching its Phase 1 trial for 4D-110 in the coming year.

Regulatory and Financial Support

The FDA’s orphan drug designation provides 4DMT with several benefits, including market exclusivity upon regulatory approval, exemption from FDA application fees, and tax credits for qualifying clinical trials. These incentives are designed to facilitate the development and commercialization of treatments for rare diseases.

In September 2018, 4D Molecular Therapeutics successfully raised $90 million in a Series B financing round. The company focuses on developing targeted and personalized AAV gene therapy products for severe genetic disorders with significant unmet medical needs.