LUMC collaborates with French therapy developer HORAMA to develop gene therapy for genetic eye disease22 April 2020• NEWSITEM
The Leiden University Medical Center (LUMC) has exclusively licensed its recently developed gene therapy program to treat Inherited Retinal Dystrophy to French therapy developer HORAMA for further clinical development.
Inherited Retinal Dystrophy is a rare but devastating ophthalmic condition leading to blindness. A team of researchers at the LUMC, led by Dr. Jan Wijnholds, has developed a platform for candidate gene therapies for children with pathogenic CRB1 mutations, which are associated with the disease.
This LUMC project was initiallly (co-) funded by the Leiden Regenerative Medicine Platform (LRMP), an organization with the mission to bring new innovations in the field of regenerative medicine closer to the patient.
"We are glad we could contribute to the further development of this gene therapy and are very happy to see that HORAMA is committed to bring this technology to the clinic", says Paul Bilars, CEO of LRMP.
For more information about regenerative medicine program developments visit rm.lumc.nl.
HORAMA is aimed at developing gene therapy to treat a broad range of inherited disorders. Its focus is on Inherited Retinal Dystrophies with its lead clinical program targeting patients with PDE6B gene mutations, a condition which leads to progressive vision loss in children and adults ultimately leading to legal blindness.
Horama’s team is pushing the boundaries of gene therapy by advancing next generation delivery platforms that will improve effectiveness and coverage of gene transfer to address multiple diseases. For more information, please go to: www.horama.fr.
Inherited Retinal Dystrophies
Inherited Retinal Dystrophies (IRD) represent a diverse group of progressive visually debilitating diseases that can lead to blindness. In patients with an IRD, mutations in genes, which are critical to retinal function, lead to progressive, direct or indirect photoreceptor cell death and associated visual function losses.
IRDs are a genetically heterogeneous group of diseases, with over 260 genes identified to date, IRDs associated with pathogenic CRB1 gene mutations are among this heterogeneous group, similar to the autosomal recessive IRD associated with pathogenic PDE6B gene mutations.
CRB1 gene mutations are a major cause of early onset and delayed onset IRD. Proteins such as CRB1 and CRB2 are essential in the retina to maintain adhesion between photoreceptors and Müller glial cells. Loss of CRB function results in loss of photoreceptors and causes blindness.