BioScience Forum Gaining Momentum in Gene Therapy for Rare Diseases and Ocular Diseases-2017-07-19

Event Information
Event Name: 
BioScience Forum Gaining Momentum in Gene Therapy for Rare Diseases and Ocular Diseases
Event Date: 
07/19/2017 - 6:00pm
Event Location: 
The Holiday Inn, 275 S Airport Blvd, South San Francisco, CA 94080
Event Details
Event Type: 
Event Details: 

BioScience Forum, Wednesday Evening, July 19, 2017

Topic: "Gaining Momentum in Gene Therapy for Rare Diseases and Ocular Diseases”
Speaker: Amber Salzman, Ph.D., President and Chief Executive Officer, Adverum Biotechnologies
Date and time: Wednesday, July 19th, 2017
6:00 PM - networking
7:00 PM - dinner
8:00 PM - presentation
Location: The Holiday Inn, 275 S Airport Blvd, South San Francisco, CA 94080
$50 before 9PM, Monday, July 17th
$60 on-site
$40 full-time students pre-registration
$50 full-time students on-site
$3 service fee will be added to the pre-registration price
Details and registration at

Topic Description
With the discovery of novel adeno-associated virus (AAV) serotypes and the engineering of novel AAV capsids, gene therapy technology has made considerable progress in efficiently targeting certain tissues such as the liver and retina. In addition, gene therapy approaches to treat diseases are particularly attractive when standard of care consists of repeated frequent injections of recombinant proteins to prevent symptoms.

Adverum is developing novel therapies utilizing recent AAV technology for diseases with well established therapeutic pathways. Alpha-1 antitrypsin deficiency (A1AT) and Hereditary angioedema (HAE) are two diseases where patient’s hepatocytes do not express inhibitors of two distinct types of proteases which left uncontrolled are responsible for the development of the disease. AAV.rh10 is highly efficient at transferring DNA to hepatocytes with long lasting potential, so a single administration of an AAV vector with the appropriate transgene has the potential to treat the disease in a well tolerated way.

Wet Age related Macular Degeneration (wAMD) is currently treated with monthly or bimonthly intravitreally administered anti-VEGF recombinant proteins. Adverum’s proprietary AAV.7m8 vector has been shown in non human primates to transfer DNA to retinal tissue when delivered intravitreally, therefore providing the potential to treat wAMD with a single administration with a long lasting therapeutic effect. Preclinical proof of concept for each of these three diseases has been demonstrated. ADVM-043, ADVM-053, and ADVM-022 are now moving towards the clinic.

Speaker Bio
Amber Salzman, Ph.D. was appointed president and chief executive officer of Adverum in October 2016 after joining the company earlier in the year as president and chief operating officer after the merger of Annapurna Therapeutics with Avalanche Biotechnologies. Dr. Salzman was the president and chief executive officer of Annapurna Therapeutics between 2012 and 2016. Dr. Salzman has served in leadership roles at large pharmaceutical companies, small pharmaceutical companies, and in the rare disease community, including her role as chief executive officer of Cardiokine Inc. prior to its acquisition by Cornerstone Therapeutics, Inc. in 2011. Dr. Salzman had a 25-year career at GlaxoSmtihKline plc, where she served as a member of the R&D executive team, leading drug development projects and clinical trials in more than 30,000 patients worldwide.

Since 2001, Dr. Salzman has served as president of the Stop ALD Foundation, a patient-advocacy group seeking improvements in treatments for patients with adrenoleukodystrophy (ALD), and played a key role in developing a lentiviral gene therapy treatment for this disease.

Dr. Salzman received a B.A. in Computer Science from Temple University and a Ph.D. in Mathematics from Bryn Mawr College.