A stroke kills brain cells by interrupting blood flow. The most common “ischemic stroke” is due to blockage in blood flow from a clot or narrowing in an artery. Brain…
Diabetes exacts a tremendous toll on patients, their families, and society in general. Autoimmune Type 1 diabetes, often called juvenile-onset diabetes, is caused by a person’s own immune system mistakenly…
Despite aggressive multimodal therapy and advances in imaging, surgical and radiation techniques, malignant brain tumors (high-grade gliomas) remain incurable, with survival often measured in months. Treatment failure is largely attributable…
Cancer is a major cause of human death worldwide. The vast majority of cancer patients suffer from solid tumors whose growth destroys vital organs. We propose to develop novel therapeutic…
A successful partnership, resulting in the creation of a Translating Center recognized as the leader in providing end-to-end preclinical development services (preclinical research, CMC, regulatory) to accelerate the availability of…
Therapeutic Candidate or Device RJB-0402 is a liver-targeting adeno-associated virus vector-based gene therapy that drives over expression of FGF21. Indication Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening…
Therapeutic Candidate or Device The therapeutic candidate to be studied under this proposal is a gene therapy product for the treatment of DOK7 Congenital Myasthenic Syndrome Indication The target indication…
