Date: 2017-03-22
Type of information: Licensing agreement
Compound: investigational gene therapy to treat Gaucher disease
Company: Avrobio (USA - MA) Dr. Stefan Karlsson at Lund University (Sweden)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: licensing
Action mechanism:
Disease: Gaucher disease
Details:
- • On March 22, 2017, Avrobio announced the expansion of its pipeline to Gaucher disease. This late-stage preclinical program becomes its second therapy for lysosomal storage disorders, following on the heels of tits Phase 1 Fabry program. The program for Gaucher disease was licensed from Dr. Stefan Karlsson at Lund University in Sweden, where preclinical proof-of-concept has been demonstrated. Avrobio will continue to collaborate with, Stefan Karlsson, MD, PhD, Professor of Molecular Medicine and Gene Therapy at Lund University in Sweden. Terms of the license agreement were not disclosed.
- IND-enabling activities are currently underway in preparation for a Phase 1/2 clinical study. The aim of Avrobio’s investigational gene therapy to treat Gaucher disease is to deliver lasting and meaningful benefits for patients. The patient’s blood stem cells are extracted and genetically modified by adding a new, fully functional copy of the faulty gene. The modified cells are then delivered back into the patient via a one-time infusion. A durable elevation of endogenous enzyme is expected, with the potential to significantly improve patient outcomes and eliminate burdensome and costly lifelong biweekly intravenous infusions of enzyme replacement therapy.
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Is general: Yes
