PRODUCT PIPELINE
Clinical Trials
Innovative science is just the first step. Success is found in execution, and Humvira’s internal state-of the-art cell and gene manufacturing and production facility translates vision into reality. This is a significant step toward achieving our mission to deliver cell and gene therapies for people impacted by serious diseases.
RESEARCH AND DEVELOPMENT
At Humvira we are focused on treating rare diseases and serving patient communities through our pioneering work in gene therapy. Learn more about our products below.
DYSTROPHIC EPIDERMOLYSIS BULLOSA (DEB)
Auto-EB-x1 is an autologous, gene-corrected cell therapy for DEB that is caused by mutations in COL7A1. Treatment with Auto-EB-x1 involves using gene transfer to deliver COL7A1 genes into an DEB patient’s own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and skin function.
JUNCTIONAL EPIDERMOLYSIS BULLOSA
(JEB)
JEB is caused by mutations in three genes that jointly encode laminin-332 and in genes that encode collagen XVII and α6β4 integrins.
EB-Lam332 is an autologous, gene-corrected cell therapy for JEB that is caused by mutations in LAMA3, LAMB3 or LAMC2. Treatment with EB-Lam332 involves using gene transfer to deliver one of LAMA3, LAMB3 or LAMC2 genes into an JEB patient’s own skin cells (keratinocytes) and transplanting them back to the patient to enable normal heterotrimeric laminin-332 expression and skin function.
WHO IS ELIGIBLE TO PARTICIPATE IN THE STUDY?
The research project involves gene transfer into keratinocytes, which are the majority of the cells in the outer layer of skin. In this gene transfer trial we plan to biopsy some skin tissue, grow the cells in a skin cell culture (sterile dishes with special fluid that allows cells to grow and multiply) and then infect the cells with a virus that we have genetically engineered to insert the correct genes. The virus used is called a "retrovirus." The virus is made so that it only delivers the genes involved EB and it should not spread to other parts of the body. During the study we will check for growth of the virus.
After cells have received gene transfer, we will grow the cells in culture into a sheet of cells that look like a plastic film. We plan to graft the sheet to wounds. Grafting means we will take cells from the culture and stitch them to the patient's skin.
KEY CRITERIA FOR ELIGIBLE PATIENTS INCLUDE THE FOLLOWING
Diagnosis of EB
Age 10 years or older
Confirmed carrier parents
