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Ophthalmic Diseases

Focusing on Sight

Our Focus

Wet Age-Related Macular Degeneration (Wet AMD)

Neovascular age-related macular degeneration (wet AMD) is a type of age-related macular degeneration where abnormal blood vessels (choroidal neovascularization or CNV) grow into the macula, the central area of the retina. As a result, CNV causes swelling of and fluid retention in the retina, bleeding, scarring, and causes blurry vision and reduced ability to see small details. The ongoing leaking of the abnormal blood vessels is stimulated by vascular endothelial growth factor otherwise known as VEGF. This process distorts and can potentially destroy central vision and may progress to blindness without treatment. Wet AMD is typically treated with intravitreal anti-VEGF agents administered approximately every 4-12 weeks.

Diabetic Macular Edema (DME)

People who have type 1 or type 2 diabetes may develop a complication called diabetic retinopathy or damage to the small blood vessels of the eye’s retina that can lead to vision loss. Diabetic macular edema (DME), which is associated with diabetic retinopathy and progresses faster, occurs when fluid from these damaged blood vessels leaks into the macula — an area in the center of the retina that helps us see objects directly ahead of us — which causes swelling of the retina and is also associated with vision loss. DME is typically treated with intravitreal anti-VEGF agents administered approximately every 4-12 weeks.

X-Linked Retinitis Pigmentosa (XLRP)

X-Linked Retinitis Pigmentosa (XLRP) is a rare inherited X-linked recessive genetic disorder that causes progressive vision loss and blindness in boys and young men. There are currently no approved therapies for XLRP. Seventy percent of cases are caused by retinitis pigmentosa GTPase regulator (RPGR) gene mutations.

Choroideremia (CHM)

Choroideremia (CHM) is a monogenic blinding disease. No products are currently approved for the treatment of this disease. This X-linked, progressive degenerative disease of the retina and choroid is caused exclusively by mutations in the CHM gene that encodes for the REP1 protein.

Ophthalmology Clinical Trials

About 4D-150 for the treatment of wet AMD & DME

4D-150 is being evaluated in the currently-enrolling PRISM clinical trial, a Phase 1/2 dose-escalation and randomized, controlled, masked expansion study of intravitreal 4D-150 in adults with wet AMD. The primary endpoints of the study are safety and tolerability. Secondary endpoints include the number of supplemental aflibercept injections over 52 weeks, change from baseline in best corrected visual acuity (BCVA) over time and central subfield thickness (CST) measured by SD-OCT.

ClinicalTrials.gov

4D-150 will be evaluated in the SPECTRA clinical trial, a Phase 2 randomized, active-controlled, double-masked study of intravitreal 4D-150 genetic medicine in adults with DME. The primary endpoint of this study is the annualized number of aflibercept injections in the study eye. Secondary endpoints include change from baseline in BCVA, CST and percentage of subjects with improvement in diabetic retinopathy severity scale. Enrollment is anticipated to begin in Q3 2023.

About 4D-125 for the treatment of XLRP

4D-125 is being evaluated in the EXCEL clinical trial, a Phase 1/2 dose-escalation clinical trial in patients with XLRP related to mutations in the RPGR gene. The primary endpoint of this trial is to determine the safety and maximum-tolerated dose. Secondary endpoints include assessments of biological activity, including both visual field function and anatomical endpoints.

ClinicalTrials.gov

About 4D-110 for the treatment of Choroideremia

4D-110 is being evaluated in the CHORUS clinical trial, a Phase 1/2 dose-escalation clinical trial in patients with choroideremia related to mutations in the CHM gene. The primary endpoint of this trial is to determine the safety and maximum-tolerated dose. Secondary endpoints include assessments of biologic activity, including visual acuity, visual field function and anatomical endpoints.

ClinicalTrials.gov

Patient Advocacy Collaborators

Foundation Fighting Blindness

The urgent mission of the Foundation Fighting Blindness is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases

Learn More

Language courtesy of Foundation Fighting Blindness

Choroideremia Research Foundation

The Choroideremia Research Foundation (CRF) is the largest organization in the world focused on the search for a cure for choroideremia (CHM). Their mission is to raise funds in support of scientific research leading to a treatment or cure of choroideremia, a hereditary retinal-degenerative disease that causes blindness; to educate people affected by the disease; and to inform the public.

Learn More

Language courtesy of Choroideremia Research Foundation

About 4DMT

Our 4DMT genetic medicine engine holds the potential to generate transformative therapies that have the power to improve patient lives, or even cure disease.

Navigation

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5858 Horton St. Suite 455
Emeryville, CA 94608

(510) 505-2680
info@4dmt.com

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