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Minutes from June 12th 2013

Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy product

Date:

12th June, 2013

Speaker:

Prof. Terry Flotte
Dean, Provost, Executive Deputy Chancellor
University of Massachusetts Medical School

Members Present:

Mark Klempner
Aram Chobanian
Barbara Gilchrest
Mark Poznansky
Robert Sackstein
Thoru Pederson
Jeff Flier

Cocktails:

Conversation ranged from members’ plans for the summer to the Boston Marathon bombings. Also mentioned was the continuing late arrival of spring to Boston.

Seminar:

Terry Flotte was invited to speak at the MEC by Mark Klempner. Terry clearly and succinctly described the now 47 year history of the study of Adeno- associated virus biology and its application to gene therapy. He also described the unique attributes of the virus that made it suitable for usage for this treatment modality. He described both the field’s development and his involvement in the championing of this application of this technology to the treatment of patients with cystic fibrosis and alpha-1 anti-trypsin deficiency. He clearly explained how this involved both the ups and down of the early years and subsequent resurgence of the use of AAV vectors in clinical gene therapy trials. To this end, Terry pointed out how the AAV vector itself is now an approved commercial product in the form of Glybera which carries the lipoprotein lipase gene to treat patients with lipoprotein lipase deficiency at an estimated cost of around one million dollars per patient.

Post Seminar Discussion:

Terry’s  provocative presentation stimulated discussion about future clinical targets for AAV based gene therapy and the technical issues associated with maximizing transgene expression. Discussion was also met about the need to optimize levels of transgene expression and potential the immunological impact of persistent transgene expression and myositis associated with it. The immune consequences of this gene therapeutic approach are clearly very interesting.

The participants left the meeting at 8.35pm following a very pleasant and informative evening,
bidding a fond farewell to each other until next semester.

Respectfully submitted,

Mark Poznansky, Scribe