Autoimmune Hepatitis (AIH) & Primary Biliary Cirrhosis (PBC)

Size: px
Start display at page:

Download "Autoimmune Hepatitis (AIH) & Primary Biliary Cirrhosis (PBC)"

Transcription

1 ANANAS Liver

2 Autoimmune Hepatitis (AIH) & Primary Biliary Cirrhosis (PBC) Chronic rare Autoimmune Liver Diseases with unmet medical needs: 90% women PBC Therapy (UDCA or OCA) does not slow progression Strong Side effects 15% non respondent AIH Therapy (STEROIDS) Severe Side Effects: 5 % non respondent > 10 % stop therapy AIH-PBC overlap (10% of total) NO CURE Drug therapy FAILS Drug therapy WORKS Liver transplant or death year after diagnosis Life-long Severe side effects due to off-target drug effects

3 ANANAS-Liver A New solution for Autoimmune Liver Diseases without side effects Nanoparticle Biodegradable and biocompatible Releases drugs liver Works in animal model Low carrier immunogenicity and inflammatory One injection every two weeks

4 Benefits of ANANAS Liver NO more Systemic Drug SIDE EFFECTS STOPS DISEASES PROGRESSION INHIBITS FIBROSIS REDUCES AUTOIMMUNE MARKER SCALABLE

5 Market for Ananas Liver Nanoparticle Rx* PBC Non-respondent patients to Rx AIH Expanded Market 8,7 B 1,3 B PBC = 29k AIH = 25k 1,1 B Expanded Market 9,7 B Total PBC patients = 194K * Developed countries only Market Values have been calculated based on Cost of current Rx /yr Total AIH patients = 215K

6 Business model ANANAS: B2B with Pharma Technology Licensing $XX M Upfront and Milestones Access to Patent & Data on Validated Targets Sharing of R&D and Manufacturing Practices Royalty Fees 1-7% Depends on stage of development and market conditions

7 Competitors (drug delivery) 272 total companies, 21 nanoparticle based technologies, 5 addressing systemic or liver autoimmune disease, 0 in clinical or preclinical level or exploits a similar approach. NP Platform Biodegradable Liver Targeting No Off Target ANANAS Parvus Topas Arcturus Lipocure Phosphorex Protein based Iron SPION Iron SPION cationic lipds liposomes polymers a r r n.a. b b Immune cells & parenchina Immune cells Immune cells n.a. n.a. n.a. b b b r r r

8 Milestones achieved ANANAS platform Untreated ANANAS- Liver Start Cup& PNI prizes NanoChallange Prize NP platform consolidated Proof-of-Efficacy in AIH model IP protection-1 EU/USA IP protection-2 PCT TOT. INVESTMENTS ~ 500K business competition prizes, consultancieson other projects, revenues, personal In collaboration with

9 Milestones - Future History AIH and PBC Preclinical PoC Additional IP Preclinical GLP Phase I Clinical trial EXIT k (120k already committed by founders) 2023

10 Team Founders & Operations Advisory Board Davide Merlin CEO Business consultant, Strategy and business planning Margherita Morpurgo CSO Nanomedicine University of Padova Pietro Invernizzi, MD Hepatology Milano Bicocca Univ Giovanni Rizzo Business Advisor, Life Sciences, Start-Up Michel Bergh Business Advisor, Life Sciences, Intellectual property Paolo Bigini Mario Negri Institute Nanobiology Unit Head. Fabrizio Paganini CFO Fiscal and Tax Accountant Paolo Gubitta Business Organization & Investor Relations University of Padova Elisabetta Casarin CTO Nanotechnology & Colloidal Drug Delivery Mario Salmona Mario Negri Institute Head of Biochemistry Elena Pigato BS, Candidate Economics Chiara Tamburini BS candidate Economics

11 ANANAS-liver Extract of Scientific Data

12 IP portfolio Claims (in brief) ANANAS linked to drugs containing carbonyl groups through acid reversible hydrazone likers Claims (in brief) Application of the above to cancer, inflammatory and autoimmune diseases ANANAS general compoisition Application of the above for medical and diagnostic uses

13 ANANAS-Liver ANANAS-Hz-Dexametasone Healthy animal Patent pending The drug is liver only A LIVER Dexa Dexa_ANANAS AIH animal 13 WIP, In collaboration with Mario Negri Institute and Univ of Padova

14 ANANAS-Hz-Dex in a AIH model Fibrosis Effect on anti-cy2d6iggs (marker of AIH progression) AST/ALT (liver inflamation) Prevents No effect Free dexa (0.4 mg/kg) Minor reduction ANANAS-dexa (0.16 mg/kg dexa equiv.) Prevents Reduces/prevents build-up Major reduction ANANAS-dexa is more efficient than free dexa 14 WIP, Courtesy of Dr. Urs Christen (Univ Frankfurt) and Pietro Invernizzi

15 Thank You

Supporting Information

Supporting Information Supporting Information Dexamethasone Conjugation to Biodegradable Avidin-Nucleic- Acid-Nano-Assemblies Promotes Selective Liver Targeting and Improves Therapeutic Efficacy in an Autoimmune Hepatitis Murine

More information

EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles

EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles BrainDTech team Dr. Fabio Bianco, PhD Chief Executive Officier PhD in Pharmacology, Degree in Biotech

More information

KPI Definition Comment Relates to Baseline Target

KPI Definition Comment Relates to Baseline Target IMI2 Key performance indicators (KPIs) Reporting on measuring and outcomes on the ten following Key Performance Indicators will be provided yearly as part of the IMI2 JU Annual Activity Reports for year

More information

Trubion Investor Presentation BioCentury NewsMakers in the Biotech Industry Conference September 6, 2007

Trubion Investor Presentation BioCentury NewsMakers in the Biotech Industry Conference September 6, 2007 Trubion Investor Presentation BioCentury NewsMakers in the Biotech Industry Conference September 6, 2007 Peter Thompson, M.D., FACP President, CEO and Chairman Trubion Pharmaceuticals, Inc. Safe Harbor

More information

Micar Innovation. Drug Discovery Factory for novel drug molecules

Micar Innovation. Drug Discovery Factory for novel drug molecules Problem There are so many incurable diseases around the world that need adequate novel compounds for the treatment, like: - Chronic pain still a pandemic in the 21 st century and affecting 1.5bn people

More information

Don Stewart, PhD President and CEO (416)

Don Stewart, PhD President and CEO (416) PlantForm Corporation Don Stewart, PhD President and CEO (416) 452 7242 don.stewart@plantformcorp.com www.plantformcorp.com PlantForm Opportunity Proprietary, plant-based manufacturing of monoclonal antibody

More information

Oral NT-KO-003 for the Treatment of Multiple Sclerosis

Oral NT-KO-003 for the Treatment of Multiple Sclerosis Oral for the Treatment of Multiple Sclerosis Madrid, 27 de noviembre de 2013 Content 1. The Company 2. The Product a) Target Indications b) Innovative mechanisms of action c) Differential features facing

More information

Company Report Daring to be different

Company Report Daring to be different Company Report 2010 Daring to be different Daring to be different Galapagos has continued to follow its own unique path and is now a leading international biotechnology company. In our 2010 report, we

More information

EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles

EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles BrainDTech Team Dr. Pietro Conti, MD, MBA President Medicine & MBA Founder/CEO several startups

More information

Can-Fite Presentation January 2015

Can-Fite Presentation January 2015 Can-Fite Presentation January 2015 1 Forward Looking Statement This presentation contains forward-looking statements, about Can-Fite s expectations, beliefs or intentions regarding, among other things,

More information

Achillion Reports Third Quarter 2017 Financial Results and Provides Update on Clinical Programs

Achillion Reports Third Quarter 2017 Financial Results and Provides Update on Clinical Programs November 1, 2017 Achillion Reports Third Quarter 2017 Financial Results and Provides Update on Clinical Programs Robust balance sheet to support global expansion of ACH-4471 clinical development program

More information

EOS: a Focussed Oncology Company Overview Forum della Ricerca e dell Innovazione Padova, May 16, 2008

EOS: a Focussed Oncology Company Overview Forum della Ricerca e dell Innovazione Padova, May 16, 2008 Growing and Nurturing Right Ideas EOS: a Focussed Oncology Company Overview Forum della Ricerca e dell Innovazione Padova, May 16, 2008 EOS S.p.A. (Ethical Oncology Science) Via Monte di Pietà 1/A, Milan,

More information

Successful Academia-Pharma Collaboration Drug Discovery and Clinical Development

Successful Academia-Pharma Collaboration Drug Discovery and Clinical Development Successful Academia-Pharma Collaboration Drug Discovery and Clinical Development Toshio MIYATA Tohoku University Graduate School of Medicine United Centers for Advanced Research and Translational Medicine

More information

KRISANI BIO SCIENCES PVT. LTD.

KRISANI BIO SCIENCES PVT. LTD. KRISANI BIO SCIENCES PVT. LTD. Flat B2, 2 nd Floor, Suryateja Apartments, Hindi Nagar, Punjagutta, Hyderabad, Telangana 500078. Email: kkr@krisanibio.com. URL: www.krisanibio.com Contact Number: +91 98494

More information

PlantForm Corporation

PlantForm Corporation PlantForm Corporation Annual General Meeting September 21, 2010 Dr. Don Stewart President and CEO +1 (416) 452 7242 don.stewart@plantformcorp.com PlantForm Corporation 2010 www.plantformcorp.com The Opportunity

More information

Market Analysis & Segmentation. Gary Phillips CEO Bioshares 29 July 2016

Market Analysis & Segmentation. Gary Phillips CEO Bioshares 29 July 2016 Market Analysis & Segmentation Gary Phillips CEO Bioshares 29 July 2016 1 Forward looking statement This document contains forward looking statements, including statements concerning Pharmaxis future financial

More information

ImmunoGen, Inc. Reports Fourth Quarter and Fiscal Year 2013 Financial Results and Provides Fiscal Year 2014 Financial Guidance and Corporate Update

ImmunoGen, Inc. Reports Fourth Quarter and Fiscal Year 2013 Financial Results and Provides Fiscal Year 2014 Financial Guidance and Corporate Update August 2, 2013 ImmunoGen, Inc. Reports Fourth Quarter and Fiscal Year 2013 Financial Results and Provides Fiscal Year 2014 Financial Guidance and Corporate Update Kadcyla sales off to strong start. Decision

More information

SCM Lifescience COMPANY PROFILE BUSINESS AREAS. [Address] 310, 366 Seohae-daero, Jung-gu, Incheon, Korea [Website]

SCM Lifescience COMPANY PROFILE BUSINESS AREAS. [Address] 310, 366 Seohae-daero, Jung-gu, Incheon, Korea [Website] SCM Lifescience [Address] 310, 366 Seohae-daero, Jung-gu, Incheon, Korea 22332 [Website] http://www.scmlifescience.com COMPANY PROFILE [Industry] SCM Lifescience is a research-based biopharmaceutical company

More information

Marina Biotech Monetizes UNA Intellectual Property Estate through Agreement with Arcturus Therapeutics

Marina Biotech Monetizes UNA Intellectual Property Estate through Agreement with Arcturus Therapeutics Marina Biotech Monetizes UNA Intellectual Property Estate through Agreement with Arcturus Therapeutics CAMBRIDGE, MA, August 14, 2013 Marina Biotech, Inc. (OTC Pink: MRNA): Assigns Unlocked Nucleobase

More information

For personal use only

For personal use only i-bodies a new class of protein therapeutics to treat human disease Biotech meets Broker: September 2016 Sam Cobb, CEO and Managing Director AdAlta Limited (ASX:1AD) s.cobb@adalta.com.au Disclaimer Investment

More information

I. Company Information

I. Company Information Please fill out this form as completely as possible. Note: There is no fee to apply. Please send additional materials such as your pitch deck and executive summary to resi@lifesciencenation.com for us

More information

REIMAGINING DRUG DEVELOPMENT:

REIMAGINING DRUG DEVELOPMENT: Biology Reconstructed REIMAGINING DRUG DEVELOPMENT: Accurate Disease Modeling To Drive Successful Therapies Julia Kirshner, CEO julia@zpredicta.com 1 SUCCESS RATES OF DRUG DEVELOPMENT ARE LOW, " PARTICULARLY

More information

i-bodies a new class of protein therapeutics to treat human disease

i-bodies a new class of protein therapeutics to treat human disease i-bodies a new class of protein therapeutics to treat human disease AGM November 2016 Sam Cobb, CEO and Managing Director AdAlta Limited (ASX:1AD) s.cobb@adalta.com.au Disclaimer Investment in AdAlta is

More information

The Spanish biotech sector Case: ADVANCELL

The Spanish biotech sector Case: ADVANCELL In vitro models, reprofiling and nanomedicine for unmet medical needs The Spanish biotech sector Case: ADVANCELL SPAIN-LUXEMBURG ECONOMIC SEMINAR April 18th, 2007 1 ELEVATOR PITCH Product oriented, low

More information

Selecta Biosciences Announces Third Quarter 2016 Financial Results and Provides Corporate Update

Selecta Biosciences Announces Third Quarter 2016 Financial Results and Provides Corporate Update Selecta Biosciences Announces Third Quarter 2016 Financial Results and Provides Corporate Update November 10, 2016 7:00 AM ET Phase 2 Trial of SEL-212 for Treatment of Gout Initiated SEL-212 Phase 1 Clinical

More information

I. Company Information

I. Company Information Please fill out this form as completely as possible. Note: There is no fee to apply. Please send additional materials such as your pitch deck and executive summary to resi@lifesciencenation.com for us

More information

Global Allied Pharmaceuticals, LLC. Innovation, Research, and Technology. Breaking the bounders, first in its class, thinking outside the box.

Global Allied Pharmaceuticals, LLC. Innovation, Research, and Technology. Breaking the bounders, first in its class, thinking outside the box. Global Allied Pharmaceuticals, LLC. Innovation, Research, and Technology. Breaking the bounders, first in its class, thinking outside the box. The purpose of this presentation Looking for Joint Venture

More information

A Machine-Learning Drug Design Platform Company BUSINESS OVERVIEW

A Machine-Learning Drug Design Platform Company BUSINESS OVERVIEW A Machine-Learning Drug Design Platform Company BUSINESS OVERVIEW Introduction Revolutionizing Drug Discovery and Design Through Artificial Intelligence The $1 trillion global pharmaceutical industry faces

More information

I. Company Information

I. Company Information Please fill out this form as completely as possible. Note: There is no fee to apply. Please send additional materials such as your pitch deck and executive summary to resi@lifesciencenation.com for us

More information

NSE Grantees Meeting December 2015

NSE Grantees Meeting December 2015 NSE Grantees Meeting December 2015 The Spherical Nucleic Acid (SNA) Nanoparticle Changes the Paradigm for Oligo Therapeutics Linear DNA SNA Each company limited by chemistry, modality, and tissue of interest

More information

Corporate Presentation OCTOBER 2018

Corporate Presentation OCTOBER 2018 Corporate Presentation OCTOBER 2018 This presentation contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated

More information

N4 Pharma Investor Presentation

N4 Pharma Investor Presentation N4 Pharma Investor Presentation November 2017 Introduction o Established in 2014 o Specialist pharmaceutical company with two divisions: o Reformulation of existing generic drugs and o Silica nanoparticle

More information

Innovative Non-Viral Gene Therapies for the Treatment of Ocular Diseases

Innovative Non-Viral Gene Therapies for the Treatment of Ocular Diseases Innovative Non-Viral Gene Therapies for the Treatment of Ocular Diseases Innovative Non-Viral Gene Therapies for the Treatment of Ocular Diseases Company Clinical stage company developing innovative non-viral

More information

LTX 109. BIO International Philadelphia 2015

LTX 109. BIO International Philadelphia 2015 LTX 109 BIO International Philadelphia 2015 Lytix Biopharma company background Established in 2003 to develop and commercialize medical applications of lytic peptides originating from the University of

More information

I. Company Information

I. Company Information If your company won (1st-3rd place) an Innovation Challenge since RESI JPM 2018, or were a finalist at RESI NYC 2018, you do not qualify to participate in the Innovation Challenge at RESI JPM 2019. Please

More information

Investor Presentation. Winter 2010

Investor Presentation. Winter 2010 Investor Presentation Winter 2010 In a few sentences... Glycoregimmune is developing sulfatide, a first-in-class therapeutic, for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou

More information

ProMetic Life Sciences Inc. Biotech Showcase San Francisco January, 2011

ProMetic Life Sciences Inc. Biotech Showcase San Francisco January, 2011 ProMetic Life Sciences Inc. Biotech Showcase San Francisco January, 2011 1 Forward Looking Statement / Copyright notice / Disclaimer Forward Looking Statement This presentation contains forward-looking

More information

Inventiva: acceleration of the clinical development activities and consolidation PRESS RELEASE of the financial position in 2016

Inventiva: acceleration of the clinical development activities and consolidation PRESS RELEASE of the financial position in 2016 COMMUNIQUE DE PRESSE Inventiva: acceleration of the clinical development activities and consolidation PRESS RELEASE of the financial position in 2016 Launch of IVA337 Phase IIb trial in NASH Initiation

More information

100M sufferers in US (2.2B worldwide) 65% of people ages NOCTURIA is the #1 urology complaint of men and women 55-84

100M sufferers in US (2.2B worldwide) 65% of people ages NOCTURIA is the #1 urology complaint of men and women 55-84 INVESTOR DECK PAXEROL for NOCTURIA 100M people in the US suffer from a problem for which we have a strong solution. The patents are issued. We are raising funds for EU launch and FDA trial. David A. Dill,

More information

Acquisition of Vtesse Inc. Increased Focus on Orphan and Pediatric Diseases

Acquisition of Vtesse Inc. Increased Focus on Orphan and Pediatric Diseases Acquisition of Vtesse Inc. Increased Focus on Orphan and Pediatric Diseases 1 Forward Looking Statement This presentation contains "forward-looking statements" as that term is defined in the Private Securities

More information

Programa Cooperación Farma-Biotech Neurociencias G79

Programa Cooperación Farma-Biotech Neurociencias G79 G79 A Neuroprotective Therapy Barcelona, 15 de febrero 2011 Content 1. The Company 2. The Product a) Therapeutic focus b) Innovative mechanisms of action c) Differential features facing the market d) Current

More information

Q Financial Results and Highlights

Q Financial Results and Highlights Q1 2018 Financial Results and Highlights May 4, 2018 On Today s Earnings Call Stanley Crooke, M.D., Ph.D. Chief Executive Officer and Chairman Beth Hougen Chief Financial Officer Sarah Boyce President

More information

novel drug molecule for the treatment of Chronic Pain

novel drug molecule for the treatment of Chronic Pain novel drug molecule for the treatment of Chronic Pain Pandemic Problem Chronic Pain need adequate non opioid novel drug molecule to treat Chronic pain still a pandemic in the 21st century Chronic pain

More information

Nanotechnology and Advanced Materials for more effective Healthcare

Nanotechnology and Advanced Materials for more effective Healthcare Nanotechnology and Advanced Materials for more effective Healthcare This challenge taps into the potential of nanotechnologies and advanced materials to enable more effective therapies and diagnostics

More information

March Company Introduction. Microbiome R&D and Business Collaboration Forum: Europe Myriam Golembo, VP Development

March Company Introduction. Microbiome R&D and Business Collaboration Forum: Europe Myriam Golembo, VP Development March 2018 Company Introduction Microbiome R&D and Business Collaboration Forum: Europe Myriam Golembo, VP Development Biomx At A Glance We are a microbiome drug discovery company developing customized

More information

Roche, Roche Molecular Diagnostics and more

Roche, Roche Molecular Diagnostics and more , Molecular and more [Monte Wetzel, PhD] Patients have questions. We provide answers. Group Clear focus on Healthcare Innovation with two strong pillars Pharma Pharma Genentech Chugai Molecular Professional

More information

University of California Center for Accelerated Innovation

University of California Center for Accelerated Innovation University of California Center for Accelerated Innovation MICHAEL PALAZZOLO April 6, 2015 Outline CAI overview and introduction RFA and important dates Selection process Pre-application Finding help 2

More information

First-in-Class Bispecific Antibodies For Cancer Immunotherapy

First-in-Class Bispecific Antibodies For Cancer Immunotherapy First-in-Class Bispecific Antibodies For Cancer Immunotherapy 2015 Company Highlights Vision Team Platform Pipeline Partners To dominate the bispecific antibody space in immunooncology through unique pipeline

More information

PATHWAY TO VALUE CREATION NASDAQ: BNTC ASX: BLT

PATHWAY TO VALUE CREATION NASDAQ: BNTC ASX: BLT PATHWAY TO VALUE CREATION NASDAQ: BNTC ASX: BLT Annual General Meeting Presentation 8 November 2018 Jerel A. Banks, M.D., Ph.D. Chief Executive Officer and Executive Chairman SAFE HARBOR STATEMENT This

More information

PATHWAY TO VALUE CREATION NASDAQ: BNTC ASX: BLT. For personal use only

PATHWAY TO VALUE CREATION NASDAQ: BNTC ASX: BLT. For personal use only PATHWAY TO VALUE CREATION NASDAQ: BNTC ASX: BLT Annual General Meeting Presentation 8 November 2018 Jerel A. Banks, M.D., Ph.D. Chief Executive Officer and Executive Chairman SAFE HARBOR STATEMENT This

More information

Immune Design Reports Third Quarter 2017 Financial Results and Provides Corporate Update

Immune Design Reports Third Quarter 2017 Financial Results and Provides Corporate Update November 1, 2017 Immune Design Reports Third Quarter 2017 Financial Results and Provides Corporate Update Company conference call at 1:30 p.m. PT today SEATTLE and SOUTH SAN FRANCISCO, Calif., Nov. 01,

More information

OCTIMET. Company Profile

OCTIMET. Company Profile OCTIMET Oncology NV (OCTIMET) is a privately held Belgian based life science company specialized in accelerating the development of oncology therapies by providing rapid clinical proof of concept through

More information

Antisense Therapeutics Ltd ASX:ANP January 2017

Antisense Therapeutics Ltd ASX:ANP January 2017 Antisense Therapeutics Ltd ASX:ANP January 2017 Forward Looking Statements This presentation contains forward-looking statements regarding the Company s business and the therapeutic and commercial potential

More information

Foresee Pharmaceuticals, Inc.

Foresee Pharmaceuticals, Inc. Foresee Pharmaceuticals, Inc. Rapid Development and Commercialization of a Proprietary Sustained-release Depot Formulation of Leuprolide for the Treatment of Advanced Prostate Cancer PNWBIO Meeting 10

More information

Advancing the Frontiers of mab mixtures

Advancing the Frontiers of mab mixtures Advancing the Frontiers of mab mixtures...unlocking the power of the immune system Symphogen Corporate Presentation June 216 Symphogen/1 Symphogen Overview Privately held company - 125 employees Headquarters

More information

INVESTOR PRESENTATION FULL YEAR 2007 RESULTS. February 2008

INVESTOR PRESENTATION FULL YEAR 2007 RESULTS. February 2008 INVESTOR PRESENTATION FULL YEAR 2007 RESULTS February 2008 FORWARD LOOKING STATEMENTS This presentation contains forward looking statements. The words believe, expect, anticipate, intend, will, may, would,

More information

Cortendo and Antisense Therapeutics Announce Licensing Agreement for ATL1103 for Acromegaly

Cortendo and Antisense Therapeutics Announce Licensing Agreement for ATL1103 for Acromegaly Cortendo and Antisense Therapeutics Announce Licensing Agreement for ATL1103 for Acromegaly May 14, 2015 Goteborg, Sweden and Trevose, Pa., USA and Victoria, Australia Cortendo AB (publ) [ticker: CORT

More information

Capital Raising Presentation January 2017

Capital Raising Presentation January 2017 Capital Raising Presentation January 2017 Disclaimer Some of the information in this presentation may refer to Dimerix Limited ( Dimerix or the Company ) based on information available to it as at the

More information

LA RICERCA TELETHON PER LE MALATTIE RARE TELETHON RESEARCH ON RARE DISEASES

LA RICERCA TELETHON PER LE MALATTIE RARE TELETHON RESEARCH ON RARE DISEASES LA RICERCA TELETHON PER LE MALATTIE RARE TELETHON RESEARCH ON RARE DISEASES Francesca Pasinelli Direttore Generale 3 Congresso Nazionale SIFaCT Roma, 9 ottobre 2015 About Telethon Fondazione Telethon is

More information

Drug Development: Why Does it Cost so Much? Lewis J. Smith, MD Professor of Medicine Director, Center for Clinical Research Associate VP for Research

Drug Development: Why Does it Cost so Much? Lewis J. Smith, MD Professor of Medicine Director, Center for Clinical Research Associate VP for Research Drug Development: Why Does it Cost so Much? Lewis J. Smith, MD Professor of Medicine Director, Center for Clinical Research Associate VP for Research Drug Development Process by which new chemical entities

More information

Rare diseases in the 7th EU Framework Programme for Research and Technological Development

Rare diseases in the 7th EU Framework Programme for Research and Technological Development Rare diseases in the 7th EU Framework Programme for Research and Technological Development Manuel Hallen, MD Head of Unit Medical & Public Health Research DG Research European Commission 1 The role of

More information

NanoAssemblr Benchtop. Develop Future Nanomedicines

NanoAssemblr Benchtop. Develop Future Nanomedicines NanoAssemblr Benchtop Develop Future Nanomedicines Develop the Future of Medicine Overcome key challenges in advancing nanomedicines: Challenges with conventional production methods Solutions with the

More information

Corporate Overview. October 1, 2018 Cantor Douglas Fambrough, CEO

Corporate Overview. October 1, 2018 Cantor Douglas Fambrough, CEO Corporate Overview October 1, 2018 Cantor Douglas Fambrough, CEO Forward-looking statements This information may contain projections and other forward looking statements regarding future events, including

More information

Introducing Immunomic Therapeutics Expanded Technology Platform: UNITETM

Introducing Immunomic Therapeutics Expanded Technology Platform: UNITETM Introducing Immunomic Therapeutics Expanded Technology Platform: UNITETM Immunomic Therapeutics is a privately-held, clinical stage biotech company developing modern DNA and RNA vaccines for allergic diseases

More information

ADVANCING SCIENTIFIC INNOVATION Together, the possibilities are endless.

ADVANCING SCIENTIFIC INNOVATION Together, the possibilities are endless. ADVANCING SCIENTIFIC INNOVATION Together, the possibilities are endless. COMMUNICATE INNOVATE EXPLORE COLLABORATE PARTNERSHIPS THAT BENEFIT YOU, US, AND ALL OF MANKIND Since Eli Lilly and Company was founded

More information

ARIKAYCE U.S. FDA Approval

ARIKAYCE U.S. FDA Approval S E P T E M B E R 2 8, 2 0 1 8 ARIKAYCE U.S. FDA Approval Forward Looking Statements This presentation contains forward-looking statements. "Forward-looking statements," as that term is defined in the

More information

Presidential Commission for the Study of Bioethical Issues 2 nd meeting September

Presidential Commission for the Study of Bioethical Issues 2 nd meeting September Presidential Commission for the Study of Bioethical Issues 2 nd meeting September 13 14 2010 David B. Weiner, Ph.D. Professor Department of Pathology and Laboratory Medicine University of Pennsylvania

More information

Jan Company Introduction. Microbiome Drug Development Europe Assaf Oron, CBO

Jan Company Introduction. Microbiome Drug Development Europe Assaf Oron, CBO Jan. 2018 Company Introduction Microbiome Drug Development Europe Assaf Oron, CBO Biomx At A Glance We are a microbiome drug discovery company developing customized phage therapies that target and destroy

More information

Sabrina Liu Amy Ma Thongchai Rattanaruengyot Anand Sharma. Presents ViroPharma Incorporated. Another Biotech? Why Harold & Kumar hate this

Sabrina Liu Amy Ma Thongchai Rattanaruengyot Anand Sharma. Presents ViroPharma Incorporated. Another Biotech? Why Harold & Kumar hate this Sabrina Liu Amy Ma Thongchai Rattanaruengyot Anand Sharma Presents ViroPharma Incorporated Another Biotech? Why Harold & Kumar hate this Biotechs are hard to understand. We can t project cash flows. They

More information

Antibody therapeutic approaches for cancer

Antibody therapeutic approaches for cancer Antibody therapeutic approaches for cancer Date2innovate Utrecht, March 21, 2014 Jan van de Winkel Forward Looking Statement This presentation contains forward looking statements. The words believe, expect,

More information

Vertex and Moderna Establish Exclusive Collaboration to Discover and Develop mrna Therapeutics for Cystic Fibrosis

Vertex and Moderna Establish Exclusive Collaboration to Discover and Develop mrna Therapeutics for Cystic Fibrosis Vertex and Moderna Establish Exclusive Collaboration to Discover and Develop mrna Therapeutics for Cystic Fibrosis -Collaboration to explore use of mrna Therapeutics to treat the underlying cause of CF

More information

Corporate Overview. Douglas Fambrough, President and CEO Cowen and Company 39 th Annual Health Care Conference March 12, DICERNA

Corporate Overview. Douglas Fambrough, President and CEO Cowen and Company 39 th Annual Health Care Conference March 12, DICERNA Corporate Overview Douglas Fambrough, President and CEO Cowen and Company 39 th Annual Health Care Conference March 12, 2019 Forward-looking Statements This information may contain projections and other

More information

Key Resources in Biomedical Product Development

Key Resources in Biomedical Product Development Key Resources in Biomedical Product Development Suguna Rachakonda - June 7, 2017 1 Business Model - Definition What does it mean to have a Business Model? A business model describes the rationale of how

More information

Investor Presentation. October 2018

Investor Presentation. October 2018 Investor Presentation October 2018 Forward Looking Statements This presentation contains forward-looking statements. "Forward-looking statements," as that term is defined in the Private Securities Litigation

More information

Agios Pharmaceuticals, Inc.

Agios Pharmaceuticals, Inc. Agios Pharmaceuticals, Inc. The people pictured here are some of the many friends and family of Agios employees affected by cancer. All of us at Agios are passionate about transforming patients lives.

More information

INITIATOR PHARMA: HALF-YEAR REPORT 2017

INITIATOR PHARMA: HALF-YEAR REPORT 2017 INITIATOR PHARMA: HALF-YEAR REPORT 2017 Financial Highlights Initiator Pharma A/S is a Danish registered company, and is reporting its financial situation in Danish kroner (DKK). Initiator Pharma A/S was

More information

Welcome to R&D Day! Christine Lindenboom VP, Investor Relations & Corporate Communications

Welcome to R&D Day! Christine Lindenboom VP, Investor Relations & Corporate Communications Welcome to R&D Day! Christine Lindenboom VP, Investor Relations & Corporate Communications 1 Alnylam Forward Looking Statements This presentation contains forward-looking statements, within the meaning

More information

SMTC1100: Progressing to Patient Clinical Trials

SMTC1100: Progressing to Patient Clinical Trials SMTC1100: Progressing to Patient Clinical Trials Action Duchenne Conference 9 th November 2013 Legal Disclaimer FORWARD LOOKING STATEMENTS This Document contains forward-looking statements. These statements

More information

NYSE American: PFNX Corporate Presentation

NYSE American: PFNX Corporate Presentation NYSE American: PFNX Corporate Presentation Pfenex Safe Harbor Statement This presentation, including the accompanying oral presentation (the Presentation ), includes forward-looking statements within the

More information

Presentation to J.P. Morgan 33rd Annual Healthcare Conference. Moshe Manor President and CEO January 15, 2015

Presentation to J.P. Morgan 33rd Annual Healthcare Conference. Moshe Manor President and CEO January 15, 2015 Presentation to J.P. Morgan 33rd Annual Healthcare Conference Moshe Manor President and CEO January 15, 2015 1 Note Regarding Forward-Looking Statements This presentation contains forward-looking statements

More information

Session 8: Marketing and Strategy (Practitioner) Athena Prib, RTTP Commercial Director, gemaker Chair of Marketing Committee, ATTP

Session 8: Marketing and Strategy (Practitioner) Athena Prib, RTTP Commercial Director, gemaker Chair of Marketing Committee, ATTP Session 8: Marketing and Strategy (Practitioner) Athena Prib, RTTP Commercial Director, gemaker Chair of Marketing Committee, ATTP Session 8: Marketing and Strategy (Practitioner) Marketing versus selling

More information

Bayer Pharma s High Tech Platform integrates technology experts worldwide establishing one of the leading drug discovery research platforms

Bayer Pharma s High Tech Platform integrates technology experts worldwide establishing one of the leading drug discovery research platforms Bayer Pharma s High Tech Platform integrates technology experts worldwide establishing one of the leading drug discovery research platforms Genomics Bioinformatics HTS Combinatorial chemistry Protein drugs

More information

Good start for external innovation with new business mix

Good start for external innovation with new business mix Good start for external innovation with new business mix Evotec AG, First Quarter 2018 Results, 09 May 2018 Forward-looking statement Information set forth in this presentation contains forward-looking

More information

First Quarter 2018 Financial Results. May 8, 2018

First Quarter 2018 Financial Results. May 8, 2018 First Quarter 2018 Financial Results May 8, 2018 Agenda Introduction Sarah Carmody, Senior Director of Investor Relations Corporate Update Craig Wheeler, President and Chief Executive Officer First Quarter

More information

Leading the world in novel adult stem cell therapies Half-Year Financial Results

Leading the world in novel adult stem cell therapies Half-Year Financial Results Leading the world in novel adult stem cell therapies 2013 Half-Year Financial Results CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation, including any comments made during or following

More information

LAMP-based Vaccines. Revolutionary Technology to Treat Allergic Disease, Cancer, Infectious Disease

LAMP-based Vaccines. Revolutionary Technology to Treat Allergic Disease, Cancer, Infectious Disease LAMP-based Vaccines Revolutionary Technology to Treat Allergic Disease, Cancer, Infectious Disease Immunomic Therapeutics Update Presentation April 2014 For additional information, visit www.immunomix.com

More information

Conference call transcript for the March 2017 Quarterly Report

Conference call transcript for the March 2017 Quarterly Report ASX ANNOUNCEMENT Conference call transcript for the March 2017 Quarterly Report Sydney Australia, 31 May 2017: Benitec Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) today lodged its transcript

More information

Regulatory Market Update: What are the major changes and differences worldwide?

Regulatory Market Update: What are the major changes and differences worldwide? Regulatory Market Update: What are the major changes and differences worldwide? Marlene E. Haffner MD, MPH, CEO Haffner Associates Orphan Drug Summit 2015 Copenhagen, Denmark September 17, 2015 Orphan

More information

CL King Best Ideas Conference

CL King Best Ideas Conference Leadership in Surface Modification and Drug Delivery Phil Ankeny, Senior Vice President and CFO CL King Best Ideas Conference The Omni Berkshire Place Hotel September 19, 2007 Safe Harbor Statement Some

More information

Strategies for attracting healthcare venture capital

Strategies for attracting healthcare venture capital Beth Silverstein MS, RAC is the Director of SciLucent, LLC, a USbased management, regulatory and technical services consulting firm dedicated to helping healthcare product companies maximise the value

More information

Tavaborole demonstrates solid efficacy and safety in Phase III trials

Tavaborole demonstrates solid efficacy and safety in Phase III trials Anacor s Drug Tavaborole Should Receive FDA Approval On May 29, 2013, Anacor Pharmaceuticals (ANAC) announced successful completion of pre- NDA (New Drug Approval) communication with the U.S. Food and

More information

Q4 & Interim Full Year 2018 PRESENTATION February 13, 2019 Per Walday, CEO Ronny Skuggedal, CFO

Q4 & Interim Full Year 2018 PRESENTATION February 13, 2019 Per Walday, CEO Ronny Skuggedal, CFO Q4 & Interim Full Year 2018 PRESENTATION February 13, 2019 Per Walday, CEO Ronny Skuggedal, CFO 1 PCI BIOTECH Important notice and disclaimer This presentation may contain certain forward-looking statements

More information

Johan Christenson MD; PhD Partner Farmasidagene 2014 Oslo

Johan Christenson MD; PhD Partner Farmasidagene 2014 Oslo Johan Christenson MD; PhD Partner Farmasidagene 2014 Oslo 1 HealthCap Focuses on Breakthrough Therapeutics Significant resource base Partners with profound sector experience Six MD PhD s Extensive network

More information

Stem Cell Research: Identifying emerging high priority policy issues

Stem Cell Research: Identifying emerging high priority policy issues The state stem cell agency Stem Cell Research: Identifying emerging high priority policy issues Ellen G. Feigal, M.D. SVP, Research and Development National Cancer Policy Summit Washington, DC November

More information

Synthetic Biologics Reports Year End 2012 Financial Results

Synthetic Biologics Reports Year End 2012 Financial Results April 16, 2013 Synthetic Biologics Reports Year End 2012 Financial Results -- Strengthening Infectious Disease Portfolio to Include C. difficile, Pertussis and Acinetobacter Targets -- ROCKVILLE, Md.,

More information

Technology Transfer. Issues and Considerations

Technology Transfer. Issues and Considerations Technology Transfer Issues and Considerations Agenda The TTP Process Spin out vs. License Deal Sheets - License Terms Discussion Technology Transfer Process Primary Activities Technical & patent details

More information

Pascal Quiry March 2013

Pascal Quiry March 2013 Please send any questions on this case study to the author via the mail box on the web site www.vernimmen.com Pascal Quiry March 2013 This document may not be used, reproduced or sold without the authorisation

More information

Biopontis Alliance Rare Disease Foundation: an Innovative Model for Early Stage Rare Disease Therapy Financing and Development

Biopontis Alliance Rare Disease Foundation: an Innovative Model for Early Stage Rare Disease Therapy Financing and Development ECRD MAY 10 TH 2014 - SESSION 0303 INCENTIVES FOR A FAVOURABLE ECO-SYSTEM Biopontis Alliance Rare Disease Foundation: an Innovative Model for Early Stage Rare Disease Therapy Financing and Development

More information