Date: 2018-08-01
Type of
information: Submission of a clinical trial application
phase: preclinical
Announcement:
Company: Adverum Biotechnologies (USA - CA)
Product: ADVM-053
Action
mechanism:
- gene therapy. ADVM-053 (AAVrh.10-C1EI) is designed as a potential single-administration treatment to provide sustained release of the C1 esterase inhibitor (“C1EI”) protein to eliminate protein level variability and prevent breakthrough angioedema attacks. In preclinical studies, a single intravenous administration of ADVM-053 increased C1EI protein expression above therapeutic levels.
Disease: hereditary angioedema (HAE)
Therapeutic
area: Rare diseases - Genetic diseases - Hematological diseases
Country:
Trial
details:
Latest
news:
- • On August 1, 2018, Adverum Biotechnologies announced the company plans to submit an IND indication to the FDA for its next-generation adeno-associated virus (AAV)-based therapy program ADVM-053 targeting hereditary angioedema (HAE) in the fourth quarter of 2018million annually per patient in the U.S. This treatment regimen can be burdensome for patients and their caregivers, and patients may still experience breakthrough edema attacks despite treatment.
Is
general: Yes
