Day 1 Agenda – March 27, 2019

8:45am – 9:00am: Welcome

9:00am – 9:30am: Keynote – State of the State of Possible: Where are we in the History of Biotech and What Lies Ahead?

• Jeremy Levin, Chairman & CEO, Ovid Therapeutics, Inc.

No way to predict the future: rich and diverse, existential moment

• impact of life expectancy
• FDA in 2018 – record of Dr. Gottlieb in approval of new drugs
• capital is flowing in LARGE amounts into innovations: new medical modalities: Gene therapy, immune oncology, RNA, CAR-T – 2500 clinical trials in immune oncology – product emerging as BIOSIMILARS
• cell therapy, gene therapy
• Health Insurers matters, consolidation, CIGNA partners with pharma
• Insulin is $400 patients that can’t pay — covenant: Health Care must be universal for Economic growth considerations
• Innovators part of the pie shrinks
• Drug Pricing shifts the political balance
• Bipartisan Pressure on the Pharma industry
• WHAT IS COMING

1. consolidations
2. massive innovations
3. innovations is essential – Financial burden
4. CNS innovations – Alzermer’s Disease: Epilepsy,
5. BIOSIMILARS: Crushing wave will create monopolies on OLD molecules PD!
6. Organizational changes: Patient centricity using AI and precision
7. Investors in Biotech in 2007 investors did not believe in immuno-oncology
8. Pricing controversy – access and affordalitiry of cost out of pocket
9. Bad democracy: if access is not to all
10. CEO’s compensation need be tied to product innovations not to drug pricing
11. IP Protection
12. BIOTECH – matters: Sustainability if patient is the purpose
13. Democracy is by all and for all

9:30am – 10:15am: ‘Fireside Chat’ on RNAi and CRISPR: What Can the Past Teach Us and What Opportunities Await?

Mike, Nikitas, Moderator

• John Maraganore, Chief Executive Officer, Alnylam

1. Tissue to benefit from RNA therapies only the beginning, not yet for cancer
2. FDA has amazing scientists to continue Dr. Scott Gottlieb’s legacy
3. Patients at the center and the Team move Science forward

Katrine Bosley, former Chief Executive Officer, Editas MedicineGenetic repair by gene deletions – as advances are achieved knowledge is shared cross projects on multiple indications

1. 1. regulators need to move faster as they see the innovations for tissue by gene medicine
   2. Dialog with FDA will continue Human germ line is NOT appropriate

10:15am – 10:30am: Possible Talk – Morgan Stanley

Possible Talk – The Possibilities of Gene Editing for Transforming the Practice of Medicine

Base Editing – Repair DNA

Creation of a new base editor – platform creation – platform to enable development – mammal cell lines

• Cytidine Base Editor: (CxG -> TxA)
• ApoE4 genetic factor fo rAlzheimer’s DIsease
• Adenine Base Editor: (AxT ->GxC??)
• Substrate specificity, delivery, product selectivity
• @ BEAM Base editing as a Human Therapy 0 genetic disease faulty gene expression

1. Gene correction,
2. Gene regulation,
3. Gene silencing,
4. Gene reprogramming

Treating genetic disease ONE LETTER AT A TIME

10:30am – 11:00am: Networking Break 

11:00am – 11:15am: Possible Talk

Possible Talk – Leveraging Artificial Intelligence to Revolutionize Drug Discovery

• Historical evolution of AI started on the 50s
• Is Pharma ready for AI?
• Publications on AI in Drug Discovery is th elargest growth in Publishing
• Pharma Corporations, AI conpanies, Tech corporations
• Potential contributions of AI to Human Biology

11:15am – 12:00pm: Solving the Value Equation to Fix our Broken Healthcare System & Ensure Patient Access

Margaux Hall, Moderator

• Erin Mistry, Managing Director Pricing and Access, Syneos Health

• Mike Bonney, Executive Chair, Kaleido Lifesciences

1. Payers need to change their system for each new product they will reimburse for – IT system change
2. Value based scheme are challenging due to data collections need by Payer to define contracts for VALUE definition and contract terms
3. Will the value change on the next year is more enrolled as users?
4. value-based constructs of therapeutics: population impact when patients start a given therapy – research shows that change in x yield favorable outcomes thus, approval of drug coverage by Payer  – ALWAYS have population effects
5. Payors can loose cohorts and gain other cohorts by incentives
6. Healthcare policies must be long term and not by Party election peril ti the Healthcare system
7. Show progress that business strategy bring scoring on outcomes
8. Saving life of patients, it is expensive, then it becomes free almost for developing countries, like vaccines and generics

• Tamar Thompson, Executive Director, State Government Affairs & Federal Payment, Bristol-Myers Squibb

1. pay less if pay cash without insurance vs pay insurance and pay co-pay — ?? affordability and gaining it fairly

12:00pm – 12:15pm: Possible Talk

Possible Talk: Opening Doors for the Future, a Patient’s Perspective

Make a change in life of Patients every day

12:15pm – 12:45pm: MassBio Presidential Address – Robert K. Coughlin, President and CEO, MassBio

12:45pm – 2:00pm: Lunch

2:00pm – 3:00pm: From Academia to Commercialization: What’s the Threshold of Data Needed to Start a New Company? [Science Track]

• Irene Abrams, Vice President, Technology Development and New Ventures, Boston Children’s Hospital
• Irena Ivanovska, Boston Site Head, Celdara Medical
• Issi Rozen, Chief Business Officer, Broad Institute

2:00pm – 3:00pm: Early Stage Funding in the Era of Venture Creation [Business Track]

• Todd Zion ex-Smart Cell, CEO

• Paula Ragan, CEO, X4Pharmaceuticals
• Rosemary Reilly, Partner, WilmerHale
• Ramani Varanasi, CEO, X-Biotix

3:00pm – 4:00pm: Addressing the Challenges of Large Population Diseases [Science Track]

• Calum MacRae, Vice Chair for Scientific Innovation, Brigham and Women’s Hospital, Harvard Medical School

3:00pm – 4:00pm: The Evolving Massachusetts Ecosystem: How Can we Continue to Sustain our Growth? [Business Track]

• Tanisha Sullivan, Associate General Counsel, Industrial Affairs, Sanofi Genzyme
• Doug Fambrough
• Chandra Ramanthan
• Johannes Fruehauf, Founder & CEO, LabCentral

4:00pm – 4:30pm: Networking Break 

4:30pm – 5:30pm: The State of Neurodegenerative Disease: Drug Targets & Alternative, Patient-Centric Clinical Endpoints [Science Track]

• Robert Alexander, Vice President and Head, Global Clinical Science Neuroscience, Takeda
• Joseph J. Higgins, MD FAAN, Vice President, Clinical Development, Huntington’s Disease Program Lead, Uniqure
• John Torous, Director, Digital Psychiatry Division, Beth Israel Deaconess Medical Center
• Sally Johnson, Biogen
• Gilmore O”Neill, Serepta
• Steve Wood – biomarkers (are LAB specific not regulated by FDA)
• Ecosystem will continue to make bets, concept of POOLING FUNDING for bedding in paralllel and develop in parallel several therapeutic targets
• Upside – investment will flow, Novertis and AztraZeneca joined funding on one development
• Placing bets on Neurological diseases
• neurogeneration of inherited diseases
• Earlier intervention is better
• target identification AUGMENTATION VS REMOVAL not clear what
• Biogen measuring SNIPS Partnerships Academia and Industry

4:30pm – 5:30pm: Addressing the Opioid Crisis [Business Track]

• Kerry Wentworth, Chief Regulatory Officer, Flexion Therapeutics

6:00pm – 9:00pm: State of Possible Celebration at the Museum of Science

Join us for drinks, food and networking at the Museum of Science!