Date: 2015-04-23
Type of information: Initiation of development program
phase:
Announcement: initiation of development program
Company: Dimension Therapeutics (USA - MA)
Product:
Action
mechanism: gene therapy
Disease: ornithine transcarbamylase deficiency
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases
Country:
Trial details:
Latest
news: * On April 23, 2015, Dimension Therapeutics, a rare disease company advancing novel, liver-directed treatments for diverse genetic disorders, announced that the company is advancing programs to treat two rare diseases with often devastating outcomes for patients and their families – Ornithine Transcarbamylase (OTC) deficiency and Glycogen Storage Disease Type Ia (GSDIa). Both diseases, due to genetically inherited inborn errors of metabolism, usually present in the neonatal period or infancy, and can have grave effects on patients of all ages. For severe forms, there are few or no effective treatment options other than liver transplantation, leaving patients with significant unmet needs. Dimension is developing novel gene therapy approaches to replace the missing or faulty gene, correcting the underlying defect to restore metabolic function. Dimension looks forward to continuing the work previously championed by advocacy organizations, including The Children’s Fund for GSD Research, which has funded much of the existing preclinical work in GSDIa. Eric Crombez, MD, Dimension’s Chief Medical Officer, commented, “Dimension is developing and strengthening partnerships with the patient communities, families, and physicians focused on improving the lives of patients affected by OTC deficiency and GSDIa. We will ensure that patient organizations, as key partners, will play an active role in developing these clinical programs.” The company anticipates commencing clinical trials in both patient populations in 2016. Dimension is advancing therapies based on a next-generation AAV platform that has demonstrated early validation and compelling preclinical in vivo proof of concept across multiple disease states in leading academic research centers. Dimension’s current efforts focus on AAV8 and AAVrh10, two forms of AAV vectors that selectively target liver cells and have been optimized to deliver missing intact genes in diseases associated with the liver. Initial indications of positive net benefit of this approach in patients living with hemophilia B, plus demonstration of robust gene expression in multiple preclinical in vivo models and commercially viable novel CMC approaches, provide a strong foundation for Dimension’s proprietary programs. OTC deficiency, the most common urea cycle disorder, is caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build-up excessive levels of ammonia in their blood, potentially resulting in neurological deficits and other toxicities. Neonatal onset disease in males is severe and can be fatal at an early age. The greatest percentage of patients experience late-onset disease, representing a clinical spectrum of disease severity. It is estimated that more than 10,000 patients are affected by OTC deficiency worldwide. Currently, the only curative approach is liver transplantation.
