Our Clinical
Trials

Clinical Trial For

EB-101

RDEB (Recessive Dystrophic Epidermolysis Bullosa)

Active, not recruiting

Gene Transfer Clinical Trial for Recessive Dystrophic Epidermolysis Bullosa

This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen. The corrected cells will be transplanted back to the patient.

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Clinical Trial For

ABO-102

MPS IIIA (Sanfilippo A Syndrome)

Recruiting

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIA

Open-label, dose-escalation clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein

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Clinical Trial For

ABO-101

MPS IIIB (Sanfilippo B Syndrome)

Recruiting

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein

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Enrollment

For more information on enrollment into our current clinical trials, please visit clinicaltrials.gov for location information.

The safety and efficacy of the investigational use of this product has not been determined. There is no guarantee that the investigational use listed will be filed with and/or approved for marketing by a regulatory agency.