Genetic Rx
Date: Location: Cost:
December 7, 2017 Harvard Medical School - Joseph B. Martin Conference Center
77 Avenue Louis Pasteur
Boston, MA 02115
(617) 262-9600
Location >

Starting at $1950


Dress Code:
Business Attire


Genetic Rx - December 7, 2017 - DRAFT

Co-hosts: Tom Barnes, Louis Breton, Jim Burns, Geoff MacKay, Sandy Macrae, Ivana Magovcevic-Liebisch, Jack Reich, Sue Washer

Time Agenda
7:00-8:00 am

Registration and Networking Breakfast

8:00-8:15 am

Welcome and Opening Remarks

8:15-8:45 am

Gene Therapy: New Therapeutic Modalities
Sean Ainsworth
, Executive Director, Clinical Development, Allergan; Tom Barnes, CSO, Intellia; Michael Linden, VP, Gene Therapy; Head, Genetic Medicine Institute, Pfizer; Tim Miller, CEO & President, Abeona Therapeutics; Jack Reich, CEO & Co-Founder, Renova Therapeutics
Moderator: JC Gutierrez-Ramos, CEO, Synlogic Therapeutics

8:45-9:15 am

Gene Editing: Progress to the Clinic
Jim Burns, President & CEO, Casebia Therapeutics; André ChoulikaCEO, Cellectis; Sandy Macrae, President & CEO, Sangamo Therapeutics; Albert Seymour, CSO, Homology Medicines; Luhan Yang, Co-founder & CSO, eGenesis

9:15-9:45 am

Epigenetics: The Future of Genetic Modification

9:45-10:15 am

Next Generation RNA Technologies
Sudhir Agrawal, President, Research, Idera Pharmaceuticals; Thomas McCauley, CSO, RaNA Therapeutics; Robert Overell, President & CEO, PhaseRx; Joseph Payne, President & CEO, Arcturus Therapeutics
Moderator: Edward KayePresident, CEO & CMO, Sarepta Therapeutics

10:15-10:45 am

Networking Break

10:45-11:15 am

Targeting Rare and Ultra-Rare Disorders
Daniel de Boer, CEO, ProQR Therapeutics; Bill Lundberg, CSO, CRISPR Therapeutics; Reenie McCarthy, CEO, Stealth BioTherapeutics; Christopher Penland, VP, BioPharma Programs, Cystic Fibrosis Foundation
Moderator: Eric Crombez, CMO, Dimension Therapeutics

10:45-11:15 am

The First Step to Finding a Cure: Diagnostics

11:15-11:45 am

Patient Recruitment and Engagement
Kyle Brown, CEO & Founder, AltaVoice; Brian Culley, CEO, MAST Therapeutics; Barbara Handelin, CEO, BioPontis Alliance; David Herbert, Treasurer & Board Member, Myotonic Dystrophy Foundation
Moderator: Jimmy Lin, CSO, Natera

11:45-12:15 pm

Manufacturing and Commercialization: Strategies for Success
Tom Chalberg
, COO, Oncorus; Nicolas Koebel, SVP Business Operations, Orchard Therapeutics; Geoff MacKay, President & CEO, AvroBio; Nora YangDirector, Portfolio Management & Strategic Operations, NIH 
Moderator: Sue Washer, CEO, AGTC

12:15-1:15 pm

Networking Lunch

1:15-1:45 pm

Moving Beyond Rare Diseases: What are the Challenges?
PJ BrooksProgram Director, NIH; JC Gutierrez-RamosCEO, Synlogic Therapeutics; Alvin Shih, CEO, Enzyvant Sciences; Paula Soteropoulos, CEO, Akcea Therapeutics
Moderator: Belen Carrillo-RivasExecutive Director, WW Safety & Regulatory, Pfizer

1:45-2:15 pm

Genetic Approaches to Muscular Dystrophy
Edward KayePresident, CEO & CMO, Sarepta; Jak Knowles, Managing Director, CureDuchenne; Shane Kovacs, CFO, PTC Therapeutics; Michael Panzara, Franchise Lead, Neurology, WAVE Life Sciences; Jude Samulski, CSO & VP, Gene Therapy, Bamboo Therapeutics
Moderator: Greg LaRosa, VP, CSO, Pfizer

2:15-2:45 pm

Academia, Industry, & Venture Partnerships: The New Wave
Irene Abrams, Senior Director, Technology & Innovation Development, Boston Children’s Hospital; Manoj KrishnanAsst. Professor, Emerging Infectious Diseases, Duke University; Ivana Magovcevic-Liebisch, EVP, Axcella Health; Harald Petry, CSO, uniQure

2:45-3:05 pm

Fireside Chat

3:05-3:30 pm

Networking Break

3:30-4:00 pm

Regulatory Strategies for Gene Therapy Products
Tom Madden, President & CEO, Acuitas Therapeutics; John Maslowski, SVP, Scientific Affairs, Fibrocell; Manal Morsy, SVP, Head, Global Regulatory Affairs, Athersys
Moderator: Christian MeyerCMO, uniQure

4:00-4:30 pm

Defining the Value of Genetic Therapies
Stacy Coen, Head, Rare Disease BD&L, Sanofi Genzyme; Doug DanisonVP, Access Value & Evidence Strategy, bluebird bio; Francois Lebel, CMO, Ziopharm Oncology; Ed Pezallafmr National Medical Director, Pharmaceutical Policy & Strategy, Aetna; Jason Slingsby, Head, BD, Oxford Biomedica
Moderator: Louis BretonCEO & Co-Founder, Calimmune

4:30-5:00 pm

Attracting Investment into the Field
PJ Anand
, President & CEO, Alcyone Lifesciences; Susan Graf, CBO, Epizyme; 

5:00-5:30 pm

Industry Outlook for 2018: What Will the Key Drivers Be?
Bob Carey, CBO, Horizon Pharma; Michael Goettler, Global President, Rare Disease, Pfizer; Kei Kishimoto, CSO, Selecta Biosciences; Massimo Radaelli, Executive Chairman, Pint Pharma Group

5:30-5:40 pm

Presentation of the Innovation of the Year Award

5:40-6:30 pm

Cocktails and Hors d'oeuvres

Supporting Sponsors: (see all sponsors)