• Skip to primary navigation
  • Skip to main content
  • Skip to primary sidebar
  • Skip to footer
  • Advertise
  • Subscribe

Drug Delivery Business

  • Clinical Trials
  • Research & Development
  • Drug-Device Combinations
  • FDA
  • Pharmaceuticals
  • Policy

Avrobio launches gene therapy program for Gaucher disease

March 22, 2017 By Sarah Faulkner

Avrobio launches gene therapy program for Gaucher diseaseCambridge, Mass.-based Avrobio said today that it expanded its pipeline to include a preclinical program focused on a gene therapy for Gaucher disease.

The investigational gene therapy makes use of a patient’s blood stem cells, genetically modifying them with a fully functional copy of the faulty gene responsible for the glucosylceramide accumulation that is characteristic of Gaucher disease. Avrobio licensed the technology from Lund University’s Dr. Stefan Karlsson, but did not disclose the terms of the agreement.

“The addition of a gene therapy targeting a [lysosomal storage disorders] with compelling preclinical proof-of-concept is an excellent strategic fit for Avrobio,” president & CEO Geoff MacKay said in prepared remarks. “Our talented team of industry veterans enables us to accelerate the development of this program with the ultimate goal of benefiting Gaucher disease patients worldwide. IND-enabling activities are currently underway in preparation for a Phase 1/2 clinical study. We are excited to add this program to our portfolio as we continue to focus on building our pipeline of gene therapies to treat rare diseases.”

Gaucher disease is caused by a hereditary deficiency of an enzyme, glucocerebrosidase, and occurs in up to 1 in 40,000 live births, according to Avrobio. Traditional therapies include lifelong, expensive intravenous infusions of enzyme replacement therapy.

Avrobio said that it aims to eliminate the need for these infusions with its gene therapy.

Filed Under: Featured, Preclinical Trials, Research & Development, Stem Cells, Wall Street Beat Tagged With: Avrobio

IN CASE YOU MISSED IT

  • Omnipod 5 rollout for type 2 rolls on as Insulet pursues next-gen automation
  • LifePlus reports clinical validation of non-invasive glucose, blood pressure monitor
  • Abbott Diabetes head talks Medtronic partnership, exciting times ahead
  • PharmaSens, SiBionics innovatively combine tech in all-in-one insulin patch pump
  • Study backs Biolinq intradermal sensor in muscle loss prevention during GLP-1 therapy

Reader Interactions

Comments

  1. KAYEMBE says

    August 15, 2018 at 11:34 am

    Greetings
    I have a three years and 7 months old daughter with Gaucher Disease type 1, Her health is deteriorating and she is not on ERT because we can not afford . I am requesting to volunteer for your gene therapy trials .
    Please contact me to this email

    Kind regards.

Primary Sidebar

“ddb
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest news and trends happening now in drug delivery.

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.

Footer

Drug Delivery Business News Logo

MassDevice Medical NETWORK

MassDevice
DeviceTalks
Medical Tubing + Extrusion
Medical Design & Outsourcing
MedTech100 Index
Drug Discovery & Development
Pharmaceutical Processing World
Medical Design Sourcing
R&D World

DRUG DELIVERY BUSINESS NEWS

Subscribe to Drug Delivery’s E-Newsletter
Advertise with us
About
Contact us
Privacy
Listen to our Weekly Podcasts

Copyright © 2025 · WTWH Media LLC and its licensors. All rights reserved.
The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media.

Privacy Policy | RSS