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Newsletter April 2017

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News: Interview with Daniel Leonard, Director, Global Patient Advocacy, UniQure
Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company that was known as a leader in patient centricity. In his current role Dan works primarily with hemophilia organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Daniel recently sat down with David Snowdon, Program Director of Gene Therapy for Rare Disorders to discuss his work in this space and how he sees the field evolving.
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News: AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Gaucher disease. This late-stage preclinical program becomes AVROBIO’s second therapy for lysosomal storage disorders (LSDs), following on the heels of the Company’s 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. Terms of the license agreement were not disclosed.
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Conferences
- 19 - 21 April 2017, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), Cardiff, Wales, UK
- 24 - 26 April 2017, Gene Therapy for Rare Disorders 2017, Boston, MA
- 3 - 6 May 2017,International Society for Cellular Therapy (ISCT) 23th Annual Meeting, London, UK
- 8 - 9 May 2017, CRISPR Europe Congress 2017, London, UK
- 10 - 11 May 2017, Genome Editing & Advances in Transgenic Technology USA Congress, Boston, MA
- 10 - 13 May 2017, American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting, Washington, DC
- 14 - 19 May 2017, Gordon Research Conference: Virusses & Cells, Lucca, Italy
- 17 - 19 May 2017, World Advanced Therapies and Regenerative Medicine Congress 2017, London, UK
- 24 - 26 May 2017, 10th Australasian Gene Therapy Society Meeting (AGCTS), Sydney, Australia
- 8 - 9 June 2017, 2nd Edition of Biorpocessing of Advance Cellular Therapies Congress, London, UK
- 26 - 27 June 2017, Cell Culture and Cell Therapy: Bioprocessing Conference, Philidelphia, PA
- 20 - 22 July 2017, The 23th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Okayama City, Japan
- 24 - 25 August 2017, Gene Therapy Bioproduction Conference, Boston, MA
- 31 August - 1 September 2017, 2nd International Conference on Nucleic Acids, Molecular Biology & Biologics Conference, Philadelphia, PA
- 27 - 28 September 2017, Annual Summit on Cell Therapy and Molecular Medicine, Chicago, Il
- 17 – 20 October 2017, XXV Congress of the European Society of Gene and Cell Therapy (ESGCT), Berlin, Germany
- 9 - 10 November 2017, 3rd Annual Genome Editing Congress, London, UK
- 9 - 10 November 2017, 3rd Annual Cell & Gene Therapy Congress, London, UK
- 15 - 17 November 2017, 16th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases, Sonoma, California

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