Treatment of cardiac arrhythmia via re-expression of TBX5 (gene therapy)
Cardiovascular disease is the number one cause of death worldwide. Scientists at the University Medical Center Göttingen developed a gene therapy (TBX5 re-expression) for use in the prevention and acute treatment of a heart disease and associated complications (e.g. cardiac arrhythmia and sudden cardiac death).
Pharmacologically controlled vector for CNS gene therapies
Gene therapies are irreversible and not controlable in case of side effects. We offer a pharmacologically controlled one-vector expression system of a therapeutic factor (i.e. GDNF) with zero background expression, based on mifepristone (Mfp)-Gene Switch system, for the therapy of neurological diseases.
Targeted Demethylating Gene Therapy of Fibrosis
All-in-one construct dCas9-TET3CD-(target gene)-sgRNA for targeted fibrosis therapy through demethylation of the genes RASAL1, LRFN2, KLOTHO (i.e. in heart, kidney, liver, lung, cancer). Normal re-expression of these genes has been achieved successfully in vitro and in vivo as well as a reduction of fibrosis.
Prevention and treatment of of Fibrosis and Chronic Injury progression
Prevention or treatment of Fibrosis and Organ Protection via ARNT regulation
1-vector otoferlin DFNB9 gene therapy
Due to the large OTOF size a one-vector delivery has remained challenging.Our technology: Gene-therapy of the otoferlin gene (OTOF) with overloaded AAV virus mediated delivery into the cochlea. In vivo proof-of-concept successfully achieved.
Leucodistrophy-Repositioning: Laquinimod for Zellweger-Syndrome
Zellweger Syndrome spectrum, an orphan disease, is a peroxisomal biogenesis disorder with no treatment currently available. We offer the use of the clinically known Laquinimod (developed for multiple sclerosis) for the therapy of Zellweger Syndrom, for which we achieved successfully an in vivo proof-of-concept.
Bio-engineered neuronal organoids from human stem cells
Scientists at the University Medical Center Göttingen, Germany developed a reproducible, robust and fully defined method for serum-free production of human bio-engineered neuronal organoids (BENOs) from stem cells. This new method will allow for reproducible production of oganoids with fully functional neuronal network activity.
Improved marker vaccine against classical swine fever
Scientists at the University of Veterinary Medicine Hanover developed a new marker vaccine for classical swine fever based on chimeric pestiviruses with improved DIVA (Differentiating Infected from Vaccinated Animals) properties. The used marker Erns is a chimeric sequence of two different and remotely related pestiviruses.
Prodrugs/drugs for a selective ADEPT/ADC tumor therapy
Scientists at the University of Göttingen developed new highly potent drugs (with an IC50 in the pico-molar range) as well as a selective tumor therapy through their prodrugs. Selectivity is achieved by antibody tumor targeting. The highly soluble prodrugs are activated into the cytotoxic drugs only at tumor site.
Humanized anti-CCR2 Antibody for the Therapy of MS and RA
Scientists of the University of Göttingen and of the University of Regensburg developed in collaboration a proprietary anti-CCR2 antibodies for the therapy of Multiple Sclerosis (MS) and potentially Rheumatoid Arthritis (RA).The lead candidate Doc-2 has been humanized in collaboration with the MRC Technologies. It targets the CCR2 receptor and modulates the autoimmune process through depletion of CCR2+ monocytes.
New therapy for amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease is one of the most common degenerative neuromuscular diseases worldwide and characterized by rapidly progressive weakness and muscle atrophy throughout the body. Scientists at the University of Göttingen developed a new neuroprotective therapeutic approach for ALS.
Therapy of Alzheimer's Disease with an antibody against oligomeric pyro-Glu-Abeta peptides
Scientists at the University of Göttingen developed a novel, proprietary antibody for the therapy and/or diagnosis of Alzheimer's Disease (AD) through the targeting of specifically pyro-Glu-Amyloid beta peptides.