The first patient has been dosed in the third phase of the VIITAL study, which is intended to evaluate the effectiveness of EB-101. This medication is a gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEM), a skin condition that sees the progressive blistering of skin. Researchers are hopeful that this study will produce positive results, as it may provide a viable treatment for this disease.
About Recessive Dystrophic Epidermolysis Bullosa (RDEM)
Recessive dystrophic epidermolysis bullosa (RDEM) is a rare skin condition that sees painful skin blistering resulting from even slight bumps or knocks. This progressive condition is often fatal.
Deeper layers of both the external and internal skin are affected, meaning skin on the inside of the mouth and surface of the eyes form blisters. This means that eating, defecating, and seeing are all painful activities. Temporary blindness occurs due to the blisters on the eyes, and sight does not return until they heal. Scarring and contractures are other symptoms of this condition, reducing mobility and fusing the fingers and toes. A smaller mouth, known as microstomia, is another symptom. Affected individuals typically develop squamous cell carcinoma, a type of skin cancer.
The COL7A1 gene is mutated in those with RDEM. This gene is necessary for the creation of type VII collagen. Collagen is needed for the strength and structure of connective tissues, and it also stabilizes the skin. The mutation that results in RDEM causes a severe lack of collagen, which means that any trauma can cause the skin to separate. This separation leads to blisters. This condition is inherited in an autosomal recessive pattern, hence the name.
RDEM is diagnosed through a skin biopsy and transmission electron microscopy or immunofluorescent antibody-antigen mapping. Genetic testing is also available. If a diagnosis is confirmed, treatment consists of avoiding creating blisters and treating them when they occur. Affected individuals should avoid friction, use non-adhesive bandages or wrappings, keep the skin cool, and manage the blisters. Doctors should also monitor for cancer so that it may be treated quickly and effectively.
About EB-101
EB-101 was created by Abeona Therapeutics. It is an autologous, gene-corrected cell therapy. Researchers developed this drug to remove and fix the COL7A1 gene so that it can successfully produce collagen before transplanting them back into the patient. Past research has proven that EB-101 can treat the most challenging blisters for an extended period of time.
About the VIITAL Study
This study is currently in its third phase, with the first two showing that EB-101 is a safe method for “sustained and durable wound healing.” Researchers are hoping to replicate these results in the third phase of their study.
Phase III just saw the dosing of its first patient. Up to 15 participants will receive the medication in this randomized study, and it will span across multiple testing centers. Researchers will use EB-101 on one of the wounds on a participant and use the others to compare effectiveness. They will also assess secondary endpoints, such as patient reported outcomes and assessment of pain.
If the VIITAL study continues to provide positive results, it will be the first treatment of its kind to treat RDEM. Researchers hope that this drug is successful and gains FDA approval so that it can better the lives of those with RDEM.
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