Leaders in Pharmaceutical Business Intelligence (LPBI) Group

TODAY – 9:30 am – 10:15 am Opening Keynote: Brad Margus, CEO of Genome Bridge – MassBio Annual Meeting 2014, Royal Sonesta Hotel, Cambridge, MA

Reporter: Aviva Lev-Ari, PhD, RN

Conference Coverage for Scientific and Social Media

Just In the Introduction 9AM to 9:30AM

Geoff MacKay, CEO Organogenesis, Inc.

Need the Gov’t to offer value care, US pends 50% more on healthcare form other countries, need to endorse HealthCare Reform for that target, reduce cost by innovating approaches to reach efficiencies. MassBio in 4/2014 MORE most important in leading the Industry, role of MassBio, the Team is stronger then ever. Financially strong. New Chairman to MassBio, serial Entrepreneur, Robert K. Coughlin

Election of New Board Members.

Incoming Mass Bio Chair: Glenn Batchelder

• stock market like Biotech and Healthcare Sectors
• In his Millennium days: Story on a Patient  with multiple myeloma establishing the Foundation, Millennium drug: PS341 drug for multiple myeloma made a difference in life of patients.
• MA became the Leader in Biotech in the World – See Impact 2020 Report by MassBio

Introduction to Brad

A game changer, kids had A-T, progressive muscular genetic rare disease, as Father, took initiative for discoveries of therapies using Genomics. Introduction by Ariad Pharmaceuticals, Inc. who introduce Francis Collins, NIH Chair in Boston, 2 years ago.

Brad Margus, CEO, Genome Bridge, spin off Broad Institute – Opening Keynote

From Harvard Business School, to leverage buyout of Shrimping Company — LOW tech background.

Two sons diagnosed with Orphan immuno-disease A-T- Cerebellar atrophy, no impact on cognition only on muscular system.

Started to do Fund raising for non-profit, Parents organized  organization – Fund raising events, publicity, TV, Marathons, Running sponsorship raise awareness

A-T Clinical Center @ John Hopkins, St. Jude

Expand like by 6 years, quality of like in deterioration.

Research Tools used for A-T – SINGLE GENE DISEASE – Children’s Project:

• mimicking human disease in other phenotypes: piglets, monkeys
• educational material on Clinical trials
• Registries
• Research on Protein, DNA damage – discovering the GENE in Israel in 1998, Dr. Shilo
• 2010 numerous genes methylation, alternative splicing –
• No protein identified yet for a target drug
• drug screening project
• cerebral Spinal Fluid Biomarker Study
• Biology and Pathway of the disease – Keeping cells alive:
• growth Factors in Cerebellum,
• Stem cells –
• Circuitry of the Disease – DCNs Hyperactive?
• Movement disorder PET scan, Cerebellum is hyperactive
• Watching Treatment for mutations
• Exoskeletons – muscle, balance,
• Serendipity in discovery – every scientist

PERLEGEN in Silicon Valley – wafer tech for Human Genome to capture 10 million SNPs and analyze variation and drug responses

• GENETIC MARKERS were discovered – not clinical useful – YET!
• 70 patents
• rare variants very important
• cheaper sequence need

ENVOY diagnostics – neurological diseases w/ Rockefeller University, Nobel Laureate Collaborator

ENVOY 2012 sold for $120 Millions to Takeda

SEQUENCING methods evolving, only HUGE DBs of Human Genomes for comparison will lead to knowledge need for drug development

IDEA –>>> FUNDNG —>> C0llaboration —>> Illumina $1000 for One genome  —>>> ANALYSIS of BIG DATA —->>> Hospital do sequencing —->>>>> NEED to STORE DATA –>>> Eric Lander –>>>>Interoperable platform —>>>>>>> GenomeBridge – Non-Profit —>>>>> hosted at BROAD —>>>>> develop API, data generated, now tackle storage securely genomic data.

MA– best position from MIT’s Broad  to Longwood Ave — MINING GENOMIC DATA —>>>

Answer – self produced protein – look at millions of Genomes TWO COPIES of A-Y Genes mutated, need be dead but does well CORRECTION elsewhere in the Genome!!

END of Talk