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

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

Tissue to benefit from RNA therapies only the beginning, not yet for cancer
FDA has amazing scientists to continue Dr. Scott Gottlieb’s legacy
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. 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

Gene correction,
Gene regulation,
Gene silencing,
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

Payers need to change their system for each new product they will reimburse for – IT system change
Value based scheme are challenging due to data collections need by Payer to define contracts for VALUE definition and contract terms
Will the value change on the next year is more enrolled as users?
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
Payors can loose cohorts and gain other cohorts by incentives
Healthcare policies must be long term and not by Party election peril ti the Healthcare system
Show progress that business strategy bring scoring on outcomes
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

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!

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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.

9:30am – 10:15am: ‘Fireside Chat’ on RNAi and CRISPR: What Can the Past Teach Us and What Opportunities Await? Although researchers have been experimenting with gene therapy since the 1980s, 2018 was a major turning point in making these technologies a reality for patients. Not only was the first therapeutic using RNA interference (RNAi) approved last year, the first human clinical trials started for a CRISPR product. Come hear the two superstars of RNAi and CRISPR compare stories, lessons learned, thoughts on the future of gene editing, and more. – John Maraganore, Chief Executive Officer, Alnylam – Katrine Bosley, former Chief Executive Officer, Editas Medicine

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

10:30am – 11:00am: Networking Break

11:00am – 11:15am: Possible Talk

11:15am – 12:00pm: Solving the Value Equation to Fix our Broken Healthcare System & Ensure Patient Access FDA approval used to mean access – but that’s no longer the case. More insurers are restricting access to new drugs, all at the expense of patients, using the argument that they do not bring enough value to the healthcare system. But what does value really mean? Experts in pricing and market access offer thoughts on how we might solve the value equation to improve patient access while continuing to reward innovation. – Erin Mistry, Managing Director Pricing and Access, Syneos Health – Mike Bonney, Executive Chair, Kaleido Lifesciences – Tamar Thompson, Executive Director, State Government Affairs & Federal Payment, Bristol-Myers Squibb

12:00pm – 12:15pm: Possible Talk

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] In 2012, Nature famously published a review by a former Amgen head of oncology research that 10% of landmark studies could not be reproduced. Company founders and initial investors must be certain that the academic work underpinning the foundational science and licensed patents are grounded in a sufficiently robust body of multiple lines of evidence can be reproduced. Serial founders and prolific professors discuss how to be sure initial scientific results are both fundable and can be advanced to “pharma grade”. – 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] Over the past decade, new biotech company creation has shifted away from investing in entrepreneurs to the venture creation model, where venture investors themselves assess an unmet medical need and create a company to address it. Yet, companies continue to spawn outside the venture creation model. Come hear experts discuss how they’ve raised money outside of the venture creation model through new sources of capital. – 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] As we better understand the genetics of disease, we can increasingly treat the underlying disease mechanisms rather than the superficial disease etiology. Biomarker testing toward improved drug effectiveness in defined sub-groups have made this a reality. Unlike oncology, where this concept is now mainstream, how might precision medicine apply to large patient populations like cardiovascular and neurodegenerative diseases? This panel will discuss the challenges and opportunities for precision medicine in these widespread maladies. – 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] Massachusetts is the #1 life sciences cluster in the world, in large part due to the collaboration and partnership among academia, industry, and government. The vibrant startup scene, along with a wealth of talent, is keeping and attracting big pharma and investors to the area, creating a collaborative environment unlike anywhere else. Yet, major challenges face the industry’s continued success in Massachusetts, from workforce development to sky-high rents to traffic. Come hear Massachusetts leaders discuss how the ecosystem is evolving and what we must do to remain on top. – Tanisha Sullivan, Associate General Counsel, Industrial Affairs, Sanofi Genzyme – 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] Despite massive investments in research for diseases like Alzheimer’s and Parkinson’s, treatment options for patients are largely non-existent. ‘Soft’ clinical endpoints like Cognitive Impression scores are difficult to demonstrate statistical significance even when underlying biology is being modified. Industry and practicing clinicians share how they are introducing modern imaging and biomarker measurements as ‘harder’ endpoints to the FDA, along with including diverse patients in earlier trials to better predict large pivotal trials. – 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 4:30pm – 5:30pm: Addressing the Opioid Crisis [Business Track] Pain treatment remains a pressing need, but opioid abuse grew by >10%, killing nearly 50,000 Americans in 2018. Despite decades in the spotlight, how can policy and law enforcement, medical providers and the biopharma industry come together to reverse the trend?

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!