dedicated to achieving the commercial potential of...

Hear from 25+ Industry Leaders, Including:

Michael Linden VP, Gene TherapyPfizer

Lei Xu Chief General Medicine Branch 2, Division of Clinical Evaluation & Pharmacology/Toxicology, OTAT, CBER FDA

Lance WeedVP, US OperationsUniQure

Seng ChengGlobal Head of Research, Rare DiseasesSanofi

Sam WadsworthCSODimension Therapeutics

Sven Kili VP, Head of Cell & Gene Therapy Development GSK

Luk VandenbergheAssistant ProfessorHarvard Medical School

Chris MasonCSOAvroBio

Tara O’MearaVP, Clinical Development OperationsBluebird Bio

April 24 – 26, 2017, Boston, MA

Researched & Developed By:

Tel: +1 212 537 5898 | Email: [email protected]

www.genetherapy-conference.com

Dedicated to Achieving the Commercial Potential of Gene Therapies

• Optimize clinical development

• Navigate the regulatory landscape effectively

• Successfully scale-up manufacturing

• Execute effective pricing, reimbursement & commercialization strategies

This is a timely venue to connect and hear from the practitioners of this technology platform on the merits as well as remaining challenges associated with this therapeutic paradigm

Seng Cheng, Global Head of Research, Rare Diseases & Gene Therapy, Sanofi

Book Now & Save Up To $400

Event Partners

Gene Therapy for Rare Disorders

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Gene Therapy for Rare Disorders April 24 – 26, 2017, Boston, MA

Tel: +1 212 537 5898 Email: [email protected]

www.genetherapy-conference.com Gene Therapy for Rare Disorders

Understand Disease-Centric DesignVivian Choi, Shire will discuss how the requirements of the disease guide the design and route of administration, to target the right gene to the right tissue at the right time

Successfully Scale-Up ManufacturingOvercome the challenges involved in translating gene therapy development from academic to GMP commercial scale with insights from Lance Weed, UniQure

Manage Outsourcing & Contract InfrastructureImprove selection and ongoing interactions with CMOs and other contract service providers in the gene therapy space with Scott Burger, Advanced Cell & Gene Therapy

Investigate Pricing & Reimbursement ApproachesTim Miller, Abeona Therapeutics and Beth White, Pfizer will share their perspectives on how potentially transformative or curative therapeutics can be priced effectively

Improve Patient AdvocacyUnderstand the patient’s perspective with UniQure’s Director of Global Patient Advocacy, Daniel Leonard, who will discuss patient access to gene therapy treatments

Inform Future Commercial StrategyEmily Culme-Seymour, GSK and Adrien Lemoine, Orchard Therapeutics will discuss how mutually beneficial collaborations can be forged and maintained in the gene therapy space

Enhance Patient Recruitment & Trial DesignLearn how to improve the recruitment of patients for gene therapy trials in the rare disease space, with insights from Bluebird Bio’s Tara O’Meara

Select Appropriate EndpointsAniz Girach, NightstaRx will investigate which clinical endpoints are realistically measurable and how the regulators view these endpoints in the ophthalmology space

Navigate the Regulatory LandscapeUnderstand the critical regulatory frameworks that impact the clinical development of gene therapies in the rare disease field, with Lei Xu, FDA

Identify Unique CMC ConsiderationsRobert Baffi, BioMarin will guide you through the unique manufacturing, validation, quality control and analytical considerations for gene therapy products

Enhance Every Stage of Your Gene Therapy Development

Your Guide to Delivering Gene Therapies to MarketPromising early clinical results have reinvigorated the gene therapy field. After decades of unfulfilled potential, this space is gaining tangible momentum, particularly in treating a range of rare diseases.

Gene Therapy for Rare Disorders 2017 is the only conference devoted to overcoming the clinical, manufacturing, regulatory and commercialization challenges holding back progress in this field.

Bringing together leading experts from innovative biotechs, large pharma, academia, regulatory authorities and key service provider companies, this conference will provide a comprehensive insight

into the cutting edge progress of gene therapies combatting rare disease.

Rather than replicating broad academic cell and gene therapy meetings, you will learn from in-depth case studies detailing how gene therapies can become commercially successful in ophthalmic, CNS, hepatic, muscular and many other rare disorders.

Join your colleagues on this collaborative platform to accelerate the progress of the next generation of gene therapies that show improved efficacy, enhanced safety and commercial viability.

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SpeakersAdrien LemoineVP, Business DevelopmentOrchard Therapeutics

Edmund PezallaConsultantEJ Pezalla Independent Consultant

Luk VandenbergheAssistant ProfessorHarvard Medical School

Aniz GirachCMONightstaRx

Casey Maguire Assistant Professor Harvard Medical School

Beth WhiteAVP, Rare Disease Commercial DevelopmentPfizer

Gopalan NarayananVP, Disruptive BiologicsVoisin Consulting

Al HawkinsCEOMilo Biotechnology

Eileen SawyerDirector, Global Medical AffairsUniQure

Michael LindenVP, Gene TherapyPfizer

Chris MasonCSOAvroBio

Lance WeedVP, US OperationsUniQure

Andrea SpezziCMOOrchard Therapeutics

Emily Culme-SeymourExternal Strategy Manager, Gene Therapy, Rare Diseases UnitGSK

Rajendra Kumar-SinghCo-Founder & VPHemera Biosciences

Daniel LeonardDirector, Global Patient AdvocacyUniQure

Lei XuChief, General Medicine Branch 2, Division of Clinical Evaluation & Pharmacology/Toxicology, OTAT, CBERFDA

Seng ChengGlobal Head of Research, Rare DiseasesSanofi

Victoria SluzkySenior Vice President, Quality & Process DevelopmentBioMarin Pharmaceuticals

Vivian ChoiAssociate Director, Head of Gene Therapy ResearchShire Pharmaceuticals

Tara O’MearaVP, Clinical Development OperationsBluebird Bio

Rune KjekenScientific Director, Advanced Therapies, CATEMA

Sven KiliVP, Head of Cell & Gene Therapy DevelopmentGSK

Sam WadsworthCSODimension Therapeutics

Tim MillerPresident & CEOAbeona Therapeutics

Scott BurgerPrincipal Advanced Cell & Gene Therapy




This will be an opportunity, in one place, to hear multiple perspectives on the near term future of gene therapy development and commercialization

Al Hawkins, Founder & CEO, Milo Biotechnology




As the gene therapy space advances and clinical investigations continue to produce promising results, uncertainty about the regulatory expectations, frameworks and available procedures in place needs to be understood. Attend this workshop to gain an in-depth understanding of the regulatory landscape at this pivotal time for the gene therapy field. Attendees will learn about:

• What the regulators are looking for in every stage of development – from manufacturing and preclinical investigations, through clinical development and reimbursement

• The availability of regulatory incentives for the development of gene therapies in the rare disease space

• In-depth case studies, guiding you through regulatory strategy and decision making throughout the development process

Understanding the Regulatory Landscape and Expectations of the Gene Therapy FieldTime: 9.00am – 12.00pm

Workshop leaderGopalan Narayanan, VP, Disruptive Biologics, Voisin Consulting

Workshop leaderCasey Maguire, Assistant Professor, Harvard Medical School

Workshop A

Gene therapies have made significant progress in the rare disease space, and as these complex products approach commercialization, effective use of contract services, particularly contract manufacturing, becomes more essential than ever before.

Attendees will learn about:

• The criteria you should be using to adequately assess potential partners to outsource projects to in the gene therapy space

• The broader contract infrastructure – understand how to improve interactions with CMOs, CROs, contract testing firms and contract shipping and transportation companies

• Key factors in choosing whether to develop in-house capabilities or outsource services to third parties’

Enhancing the Outsourcing Process: How to Select, Qualify, and Manage Contract Manufacturers and Other Contract Service Providers Effectively Time: 1.00pm – 4.00pm

Workshop D

Evidently, the use of AAV vectors has had a significant impact on the field, as demonstrated by their continued use in the pioneering clinical trials currently reaching maturity. However, these vectors still encounter significant challenges upon injection that impact on the efficacy and durability of transgene expression. Attend this workshop to get up to speed on the latest developments in vector technology.


• The degree to which the current suite of AAV vectors are able to affect the expression levels of a transgene at a therapeutic level and comparison with new vectors in preclinical development

• Next generation delivery technologies with improved tissue targeting and the ability to deliver larger genes to the target tissue

• Novel technologies which can evade pre-existing antibodies as well as avoid immune response complications associated with re-dosing

Discussing Novel Technologies with the Potential to Impact the Gene Therapy Field Time: 9.00am – 12.00pm

Workshop C

Multiple frameworks and plans have been proposed in the US and across different geographies, to address the concerns of payers regarding the cost of gene therapies. Attend this workshop to investigate the current reimbursement landscape and the ways in which it will need to adapt in the future.


• The complexities of pricing potentially curative therapeutics• Comparisons between proposed pricing and reimbursement

models • Differences between pricing structures in different

geographies worldwide

How will Gene Therapies be Reimbursed?Time: 1.00pm – 4.00pm

Workshop B

Workshop leaderScott Burger, Principal, Advanced Cell & Gene Therapy

Workshop leaderEdmund Pezalla, Consultant, EJ Pezalla Independent Consultant

Choose A or C

Choose B or D

Pre-Conference Workshop Day | Monday, April 24, 2017




Conference Day One | Tuesday, April 25, 2017

7.30 Registration, Coffee & Networking

Chris Mason, CSO, AvroBio 8.20 Chair’s Opening Remarks

Michael Linden, VP, Gene Therapy, Pfizer

8.30 Keynote: From Academic to Commercial Scale – Managing the Rapid Progression of Gene Therapies• Bridging the gap between academic proof-of-concept studies to commercialization of

gene therapy products

• Investigating aspects of the drug development path that differentiate gene therapies as therapeutics

• Understanding the need for coherent, broadly applicable, industry-wide standards to measure the determinants of bioactivity of gene therapies

Sam Wadsworth, CSO, Dimension Therapeutics

Seng Cheng, Global Head of Research, Rare Diseases, Sanofi

Sven Kili, VP, Head of Cell & Gene Therapy Development, GSK

9.00 Keynote Panel Discussion: The Future of Gene Therapies for Rare DiseaseGene therapy is gaining momentum, but how do we ensure this momentum is sustained and avoid the mistakes that have cost the field in the past? Pioneering biotech companies at the forefront of the field will open the conference by sharing their insights into the key developments that will affect change over the next year:

• Assess the relative progress of gene therapies in rare ocular, CNS, hepatic and muscular disorders

• Understand the key challenges standing in the way of pioneering gene therapy approvals

• How can lessons from the past be used to guide future success in this field?

• Learn about the critical nature of characterization and standardization – of manufacturing processes, clinical development, regulatory frameworks and pricing and reimbursement

• Investigate the factors you need to consider as gene therapies gear up for commercialization in the coming years – what steps can you take now to stay ahead of the curve?

Vivian Choi, Associate Director, Head of Gene Therapy Research, Shire Pharmaceuticals

10.00 Improve Decision Making Early in the Gene Therapy Drug Development Process to Enable Successful Progression Through the Clinic• Delivering the right gene to the right place at the right time with the right control: What

novel approaches can improve our ability to do this?

• Understanding the need of the disease and how this should influence the design and route of administration of the molecule

• Analyzing key decision points in the early development of gene therapies for ophthalmic, CNS and hematological rare disorders

10.30 Speed Networking & Morning Refreshments

Enhancing Manufacturing Practices in Readiness for Commercialization

Sam Wadsworth, CSO, Dimension Therapeutics

11.30 Overcoming the Capacity Bottleneck: Managing In-House & Outsourced Value Chains Effectively• In-house vs. contact manufacturing: Key considerations when evaluating future

commercial scale manufacturing

• How to identify and partner with key contract manufacturing organizations throughout the supply chain

• Managing relationships with contract providers: The importance of quality agreements

• Identifying strategies to deal with the Catch-22 manufacturing situation: What is the most effective way to manage scale up in the interim period before commercial scale manufacturing facilities are more readily available?




Victoria Sluzky, Senior Vice President, Quality & Process Development, BioMarin Pharmaceuticals

12.00 Investigating CMC Considerations for the Production of a Gene Therapy Product• Identifying unique manufacturing considerations for gene therapy products

• Understanding unique validation considerations for gene therapy products

• Learning about the unique quality and analytical considerations for gene therapy products

12.30 Lunch & Networking

Lance Weed, VP, US Operations, UniQure

2.00 Optimizing the Scalability of Gene Therapy Manufacturing• Moving from academic scale to GMP commercial scale manufacturing – how can the key

challenges be overcome?

• What requirements are regulators placing on commercial scale manufacturing and how can these be met?

• Improving the yield of vector produced by manufacturing processes: Case study examining higher yield required for gene therapies in the hemophilia space

Andrea Spezzi, CMO, Orchard Therapeutics

2.30 The Patient Pathway: Examining the Complex Patient Logistics Required to Progress Ex Vivo Gene Therapies• Insights into the patient journey from diagnosis to treatment and follow up

• Investigating the safety and efficacy of lentivirus ex vivo gene therapy for ADA-SCID

• Overcoming the challenge of rejection – pre-conditioning patients prior to ex vivo gene therapy

3.00 Afternoon Refreshments & Networking

Navigating the Complex Regulatory Landscape

Rune Kjeken, Scientific Director, Advanced Therapies, CAT, EMA

3.30 Gene Therapy for Rare Diseases: EMA Perspective• Through the maze: Navigating the EMA regulatory landscape

• What regulators want to see: Lessons from EMA scientific advices on Gene therapy products

• Looking Into the future: Identifying gaps in information regarding vector technologies and how the field can work to address these issues

Chris Mason, CSO, AvroBio

Rune Kjeken, Scientific Director, Advanced Therapies, CAT, EMAGopalan Narayanan, VP, Disruptive Biologics, Voisin Consulting

4.00 Panel Discussion: Cross-Industry Perspectives on Defining the Regulatory Course for Gene Therapies in Rare Disease• Learning about regulatory differences across diseases

• Manufacturing elements that impact on the regulatory environment

• Evaluating preclinical models and how the regulators view these

• Exploring clinical development and endpoints

• International perspective – examining differences between the European and US regulatory landscapes

Chris Mason, CSO, AvroBio 4.45 Chairman’s Closing Remarks




Conference Day Two | Wednesday, April 26, 2017

Chris Mason, CSO,AvroBio 8.20 Chair’s Opening Remarks

Lei Xu, Chief,General MedicineBranch 2, Divisionof ClinicalEvaluation &Pharmacology/Toxicology, OTAT,CBER, FDA

8.30 Clinical Development of Gene Therapies for Rare Disease: FDA Perspective• Overview of OTAT, CBER, FDA

• Assessing the statutory requirement for new drug/biologics approval

• Exploring regulatory pathways leading to approval

• Improving interactions with the FDA

• Evaluating the lessons learned from Spinraza

Improving the Clinical Development of Gene Therapy Approaches

Tara O’Meara, VP, Clinical Development Operations, Bluebird Bio

9.00 Overcoming Patient Recruitment & Trial Design Challenges to Accelerate Clinical Progression• Learn from innovative gene therapy trial designs in the rare disease space – structure,

patient selection, recruitment, retention and endpoints

• Optimize multi-site travel and identify centers of excellence to accelerate clinical development

• Overcome the logistical challenges involved in long term patient monitoring

Aniz Girach, CMO, NightstaRx

9.30 The Selection of Endpoints for Ophthalmology Gene Therapy Trials in the Rare Disease Space• What are the key endpoints being explored at the moment and in the future?

• What endpoints are realistically measurable in the timescale?

• What is the regulators’ feedback relating to these endpoints?

10.00 Morning Refreshments & Networking

Eileen Sawyer, Director, Global Medical Affairs, UniQure

11.00 Bridging Gaps Through Early Integration of the Internal Medical Affairs Function• Illustrating how medical affairs can contribute to the clinical development of gene

therapies

• Investigating how medical affairs can integrate multiple stakeholders to address the scientific needs of the community

• Building key external relationships to advance the program and prepare for future challenges

Chris Mason, CSO, AvroBio

Rajendra Kumar-Singh, Founder, Hemera Bioscience

Al Hawkins, CEO, Milo Biotechnology

11.30 Panel Discussion: What Does it Take to Succeed in the Gene Therapy Space?With an array of companies vying to establish themselves in the gene therapy space, this is your oportunity to learn from organizations with early success stories to tell, who will really pioneer the field over the next year.

• How can a small company establish itself in this rapidly evolving field?

• Taking on developmental and manufacturing challenges with limited resources – learn from the most innovative small biotechs and how they have achieved success in this field

• Investigating clinical case studies for gene therapies in rare disease

• Key clinical, regulatory and manufacturing considerations to pave the way to successful commercialization of gene therapies

12.30 Lunch & Networking




I’m looking forward to a meeting bringing minds together and rallying us behind our field’s banner in preparation of what should be a momentous year

Adrien Lemoine, VP, Business Development, Orchard Therapeutics

Patient Advocacy, Pricing & Reimbursement Considerations

Daniel Leonard, Director, Global Patient Advocacy, UniQure

2.00 Patient Advocacy in the Gene Therapy Space: A Case Study in Hemophilia• How early in the gene therapy development should companies begin their patient

advocacy efforts?

• What are some key considerations for patient advocacy that are unique to gene therapy?

• Gain transferrable insights from the hemophilia space that can enhance patient advocacy efforts in other disease areas

Tim Miller, President & CEO, Abeona Therapeutics

2.30 Analyzing how a Single Gene Therapy Intervention may be Priced Effectively for Different Rare Diseases• In an environment where the payers and market are accustomed to corrective therapies,

how can potentially transformative or curative therapeutics be priced?

• Establishing a reimbursement framework for gene therapies

• Investigating the global pricing and reimbursement environment for gene therapies – contrasting between US and European approaches

3.00 Afternoon Refreshments & Networking

Commercialization & Effective Route to Market Strategies

Beth White, AVP, Rare Disease Commercial Development, Pfizer

3.30 Funding & Investment to Further the Development of Gene Therapies• How can the industry encourage funding for this space?

• What are big pharma expectations for this type of therapy? How can we learn from past failures in the field to ensure funding is maintained and enhanced?

• What are the inhibitors for investment in this industry?

Emily Culme-Seymour, External Strategy Manager, Gene Therapy, Rare Diseases Unit, GSKAdrien Lemoine, VP, Business Development & Operations, Orchard TherapeuticsAl Hawkins, CEO, Milo Biotechnology

Luk Vandenberghe, Assistant Professor, Harvard Medical School

4.00 Panel Discussion: Analyzing the Evolution of Collaborations & Deal Structures Between Companies & Academia Pioneering the Gene Therapy Space• Investigating how the field has moved from acquisitions to a more collaborative ecosystem

• State of the gene therapy lancscape: Assessing past successes to inform future commercial strategies for companies in the gene therapy field

• How will interactions between pharma, biotech and academic organizations adjust to this rapidly evolving space?

Chris Mason, CSO,AvroBio 4.45 Chairman’s Closing Remarks

5.00 Close of Conference




Partnership Opportunities

Organizations Attending Include:

Contact

Tom Faulkner Commercial Manager


Partner With Us

Why PartnerGene Therapy for Rare Disorders 2017 is your fastest route to organizations prioritizing gene therapy development. This meeting will bring together decision makers from pharma and biotech companies who are committed to speeding up the development of their gene therapy programs.

This is your opportunity to influence industry thinking as the landscape continues to develop and capitalize on emerging areas where your solution can impact progress. Partnering with this meeting will ensure you are front of mind as decisions on strategy are being made for the next 12 months.

*Expected attendees for 2017 based on recent industry research

You’ll Meet People From...

Biopharma

Academic

Contract Service Provider

Other69%

15%

10%6%

Industry Breakdown

Very targeted meeting filled with KOLs and industry leaders in this exciting field.

Previous Partner in the Hanson Wade Cell & Gene Therapy Portfolio

Brammer BioBrammer Bio is a CDMO focused on providing process development, clinical, and commercial

supply of cell and gene therapy products, enabling the delivery of novel medicines and improving patient health. We have a highly skilled team with the development, manufacturing, analytical, and operational expertise from over 100 client projects that is required to tackle the challenges posed by these novel technologies. We can accelerate your product transition from the clinic to patients while focusing on meeting cGMP standards and commercial expectations. Brammer Bio has the expertise to support your gene and cell therapy projects to Phase III, commercial approval, and beyond.

www.brammerbio.com

Paragon BioservicesWorking as an outsourcing partner, and with a long-standing tradition of quality

and service, Paragon expands the capabilities of pharmaceutical companies, biotechnology companies and academic laboratories involved in identifying, developing and producing monoclonal antibodies, therapeutic proteins, disease biomarkers, vaccines and reagents for diagnostics. Paragon’s focus is the development and manufacturing of biopharmaceutical protein drugs. As a contract development and manufacturing organization (CDMO), Paragon is positioned to capitalize on industry trends—including consolidation in the pharmaceutical industry, all-encompassing “Programmatic Outsourcing”, and the looming imperative that is National Preparedness.

www.paragonbioservices.com

Exhibitor Exhibitor

Pricing

Register & Pay before Friday February 17, 2017

Register & Pay before Friday March 17, 2017 Standard Pricing

Gold PackageConference + 2 workshops

$3697 (save $400) $3797 (save $300) $3897 (save $200)

Silver PackageConference + 1 workshop

$3098 (save $300) $3198 (save $200) $3298 (save $100)

Bronze PackageConference only

$2499 (save $200) $2599 (save $100) $2699

Workshops (each) $699

PricingRegisterwww.genetherapy-conference.com/register Tel: +1 212 537 5898 Email: [email protected]: Hanson Wade 4th Floor, 52 Grosvenor Gardens, London, SW1W 0AU

• 10% discount – 3 delegates • 15% discount – 4 delegates • 20% discount – 5 +

*Please note that discounts are only valid when three or more delegates from one company book and pay at the same time.

Team Discounts* Top 3 Benefits of Attending

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Engage directly with industry leaders from biotech, pharma and the regulatory authorities in an intimate environment

Understand the clinical, regulatory and manufacturing steps that need to be taken to realize the commercial success of gene therapies

Immerse yourself in 3 days of in-depth talks and interactive sessions focusing specifically on gene therapies for rare disease

Venue

Sheraton Boston Hotel 39 Dalton StreetBostonMA, 02199USA

www.sheratonbostonhotel.com

Please note: Overnight accommodation and travel are not included in the registration fee

Full payment is due on registration. Cancellation and Substitution Policy: Cancellations must be received in writing. If the cancellation is received more than 14 days before the conference attendees will receive a full credit to a future conference. Cancellations received 14 days or less (including the four-teenth day) prior to the conference will be liable for the full fee. A substitution from the same organization can be made at any time.

Changes to Conference & Agenda: Hanson Wade reserves the right to postpone or cancel an event, to change the location or alter the advertised speakers. Hanson Wade is not responsible for any loss or damage or costs incurred as a result of substitution, alteration, postponement or cancellation of an event for any reason and including causes beyond its control including without limitation, acts of God, natural disasters, sabotage, accident, trade or industrial disputes, terrorism or hostilities.

Data Protection: The personal information shown and/or provided by you will be held in a database. It may be used to keep you up to date with developments in your industry. Sometimes your details may be obtained or made available to third parties for marketing purposes. If you do not wish your details to be used for this purpose, please write to: Database Manager, Hanson Wade, Suite A, 6 Honduras Street, London EC1Y 0TH

TERMS & CONDITIONS




I’m excited to come together with industry experts to discuss critical challenges. Learning from each other’s experiences will enable us as a field to more rapidly advance this potentially transformative technology

Eileen Sawyer, Director, Global Medical Affairs, UniQure

*Academics are entitled to a 40% discount off the Industry Pricing (Please note: No further discounts are applicable)

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