Rare Genes Partnering Forum (September 14, 2017, Irvine)

For an unusual conference, see Rare Genes Partnering and Investor Forum, a two day conference in Irvine California on September 14, 2017.  Website here.

Cut/paste of Agenda after the break.

• Wednesday, 09/13/17
•  

  3:00 PM  -  7:00 PM

  Registration & Information Desk

• Thursday, 09/14/17
•  

  8:00 AM  -  8:30 AM	Welcome by Co- Chairs - Setting the Stage Speakers: Alison Silva,  Brad Margus,  Michael Pistone
  8:35 AM  -  9:45 AM	Innovation Company Creation Models for Rare Diseases This discussion will explore the evolving landscape of rare disease drug discovery and development, with a focus on innovative investment strategies. We’ll identify some successes and pitfalls of new rare disease funding models, and concentrate on emerging drivers and trends in a field that has received a growing attention from pharmaceutical companies, venture investors, government agencies, and patient advocates. Speakers: Aileen Healy,  Alison Silva,  Art Pappas,  Bharatt Chowrira,  Jeremy Springhorn,  Kush Parmar
  8:45 AM  -  9:05 AM	Cures Within Reach
  9:15 AM  -  9:35 AM	Cure Duchenne
  9:45 AM  -  10:05 AM	Vencerex
  9:50 AM  -  10:30 AM	Overcoming the Development Challenges of a Rare Disease Therapeutic Speakers: Camilla Simpson,  Daniel Levine,  Jeff Sherman,  Matthew Howes,  Melanie Ivarsson,  Tanya Zaremba Fischer
  10:15 AM  -  10:35 AM	Immunogenix
  10:35 AM  -  11:15 AM	How to Ensure a Successful Commercial Launch of a Rare Disease Therapeutic Marketing rare disease therapies pose specific challenges because of the limited population of patients with these conditions. Drugmakers must educate patients, doctors, and payers about a disease and new therapy with which they may not be familiar. They must also justify what may seem like a high price tag for a new therapy. And, to make the economics of a rare disease therapy, they may need to have a commercial strategy that targets multiple international markets where many of the patients that may benefit from the drug may live. How can companies best find patients? Should they find a partner for international markets or build their own team? How should they prioritize markets and navigate the regulatory and reimbursement hurdles in different countries? What role can named patient programs play? What about the role of patient assistance programs? Speakers: Dan Bobear,  Daniel Levine,  Joe Nolan,  Molly Harper
  10:45 AM  -  11:05 AM	Zoetic
  11:15 AM  -  11:35 PM	Project Violet
  11:20 AM  -  12:30 PM	Alternatives to Standard Newcos: Spin-offs, Build-to-Buys, Joint Ventures and Alliances Increasingly, entrepreneurs and investors involved in forming rare disease-focused companies are seeking more innovative structures to translate scientific discoveries and create shareholder value rather than pursuing the traditional angel- and VC-backed financing of startups. This session will discuss the advantages and potential pitfalls of corporate spin-offs, strategic alliances and joint ventures, as well as build-to-buy models that include attractive, non-dilutive cash support combined with milestone-based options to acquire the entity. Speakers: Brad Margus,  Camille Samuels,  Chris Adams,  Dan Faga,  Neil Kumar,  Vern Norviel
  11:45 AM  -  12:05 PM	Think Genetic
  12:15 PM  -  12:35 PM	Portable Genomics
  12:40 PM  -  1:50 PM	Networking Lunch Sponsored by: Shire
  2:00 PM  -  2:20 PM	Children's Tumor Foundation
  2:00 PM  -  3:30 PM	Rare Disease Partnering Between Advocates, Academics, Startups and Global Pharmas Rare disease drug development is a team sport. This session will explore current and future partnering models between rare disease stakeholders including research institutions, industry, and patient organizations. Participants will discuss essential elements of successful partnering, how to align the differing interests of the participants, and how to overcome cultural differences to align everyone to work together towards common goals. Hear new ways that organizations are collaborating to discover, develop, and bring life-saving therapies to market. Speakers: Alvin Shih,  John Crowley,  Karen Erickson,  Michael Broxon,  Michael Pistone
  2:30 PM  -  2:50 PM	TREND Community
  3:00 PM  -  3:20 PM	3billion
  3:45 PM  -  5:00 PM	RARE Battle of the Brains In partnership with Scientist.com and Takeda Tak-celerator. Speakers: Jeremy Springhorn,  Kush Parmar
  5:30 PM  -  7:30 PM	Backyard BBQ Come out to unwind and network with fellow attendees at our Backyard BBQ. Enjoy food and drink while playing a variety of fun interactive games in this one-of-a-kind RARE space.