4D Molecular Therapeutics (4DMT), a California-based gene therapy company, has secured orphan drug designation for 4D-110 from the US Food and Drug Administration (FDA) for the treatment of choroideremia (CHM), an X-linked monogenic recessive disease.
Choroideremia, which results from the mutation of the REP-1 gene begins with reduced night vision and progressive loss of peripheral vision, ultimately leads to loss of visual acuity and blindness.
Peter Francis – SVP Clinical, Translational R&D Program Leader, Retina Therapeutic Area, commenting on the orphan drug designation for 4D-110 for the treatment of choroideremia said: “Our product for CHM patients, 4D-110, will be administered by intravitreal injection, and is designed to result in widespread expression of the REP-1 protein within cells in the retina.
“In contrast to other AAV gene therapy agents for CHM patients, which are administered by subretinal surgical injection, 4D-110 is designed to treat the entire retina, and to treat patients with all stages of the disease.”
In an on-going natural history study, 4D Molecular Therapeutics has enrolled over 50 choroideremia patients. The study is anticipated to support the rapid enrollment of its planned clinical studies and to give baseline characteristics for pre- and post-treatment analysis, said the gene therapy company, which is likely to launch the phase 1 trial next year.
The FDA Orphan drug designation for 4D-110 enables certain benefits such as market exclusivity following regulatory approval, if received along with exemption of FDA application fees and tax credits for qualified clinical trials.
In September 2018, 4D Molecular Therapeutics raised $90 million via a Series B financing round. The company is involved in the discovery and development of targeted, personalized and next-generation adeno-associated virus (AAV) gene therapy products intended to be used in patients with severe genetic disorders with high unmet medical need.