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Cell and gene therapies offer a novel approach to treating, curing and ultimately preventing disease by changing the expression of a person’s genes or by providing new cells. Such therapies are still in the experimental stages, with most human clinical trials only in the early research stages, but there is certainly potential for revolutionary new treatments. To date, many factors have prevented researchers from developing successful gene and cell therapy products, such as finding an optimal gene delivery tool, having a complete understanding of gene function, and the problem of multigene disorders and environmental factors.

The promise of gene and cell therapies has yet to be realised and the FDA have not yet approved products in this field. However there is much research activity in this area and researchers are hopeful that the potential of gene and cell therapies and their viability will be proven within the next decade.

The main therapeutic areas of research at present, include cancer, diabetes, cardiovascular disease and HIV infection, with cancer leading the market due to the large number of products in clinical development and the lack of effective treatments.

This conference will examine the very latest techniques and developments in gene and cell therapies and evaluate the success of current projects.

A unique opportunity to learn from leading industry experts including:
Dr Wolfgang Buchinger, Head, Process Science, Boehringer Ingelheim
Prof Alan Kingsman, Chief Executive Officer, Oxford Biomedica
Dr Jaap Goudsmit, Chief Scientific Officer, Crucell
Prof Thomas Knittel, Vice President, Corporate & Medical Affairs, DeveloGen
Dr Barrie Carter, Executive Vice President & Chief Scientific Officer, Targeted Genetics
Dr Johanna Holldack, Chief Operating Officer, Medigene
Dr Robert Coffin, Chief Scientific Officer, Biovex
Dr Jeffrey Ostrove, President & Chief Operating Officer, Ceregene
Dr Steffen Panzner, Chief Executive Officer, Novosom
Dr Nigel Parker, Chief Executive Officer, Ark Therapeutics

Benefits of Attending
· POXVIRUS VECTORS: Discover poxvirus engineering for anti-cancer gene therapy
· ADENO-ASSOCIATED VIRUS VECTORS: Investigate AAV-based vaccination and gene therapy for lipoprotein disorders
· AIDS: Review gene therapy for AIDS using HIV-based lentiviral vectors
· CANCER: Assess the market outlook for gene therapies in cancer
· GENE DELIVERY: Understand intracellular delivery of plasmids and oligonucleotides via a systemic route
· IMMUNOTHERAPY: Consider the herpes simplex virus for oncolysis and cancer immunotherapy

"This is an excellent and timely meeting. This will be the next revolution in medicine, commanding markets of many tens of billions of dollars. The conference is a must for industry leaders and investors who want to get in before values soar"
Prof Alan Kingsman, Chief Executive Officer, Oxford Biomedica

Conference programme

8:30 Registration and Coffee

9:00 Chairman's Opening Remarks

Dr Robert Coffin

Dr Robert Coffin, Chief Scientific Officer, Biovex

9:10 DIFFICULTIES IN THE INDUSTRY

Dr Wolfgang Buchinger

Dr Wolfgang Buchinger, Head, Process Science, Boehringer Ingelheim

  • Scaleable process free of organic solvents and enzymes
  • Low cost of goods due to high yield and simple process steps
  • High cell density (HCD) fermentation
  • Scaleable, continuous, closed cell lysis systems combined with clarification
  • CIM-Convective interaction media for purification
  • 9:40 HSV VECTORS

    Prof Joseph Glorioso

    Prof Joseph Glorioso, Professor & Chairman, Molecular Genetics & Biochemistry & Director, Molecular Medicine Institute, University of Pittsburgh, School of Medicine

  • Herpes simplex virus is a human neurotropic virus that establishes life long latency in sensory neurons
  • HSV vectors have been engineered to be noncytotoxic, incapable of reactivation from latency and capable of long-term transgene expression, repeat dosing and tissue targeting
  • Protocols for safe manufacturing of HSV vectors have been established
  • Preclinical studies of treatment of chronic pain, peripheral neuropathy and malignant glioblastoma
  • 10:20 TREATING NEURODEGENERATIVE DISEASES WITH GENE THERAPY

    Dr Jeffrey Ostrove

    Dr Jeffrey Ostrove, President & Chief Operating Officer, Ceregene

  • Targets in the Alzheimer’s brain
  • The biology of nervous system growth factors
  • Preclinical animal models of cholinergic decline
  • Phase 1 clinical studies
  • Prospects for other neurodegenerative diseases
  • 11:00 Morning Coffee

    11:20 LENTIVECTORS AND THE CNS

    Dr Nicholas Mazarakis

    Dr Nicholas Mazarakis, Vice President, Neurotherapy , Oxford Biomedica

    12:00 CNS GENE THERAPY

    Dr Carl Rosenblad

    Dr Carl Rosenblad, Director, Preclinical Development, NsGene

  • Neurodegenerative disorders
  • Neurotrophic factors
  • Local delivery neurotrophic factors in the CNS
  • Encapsulated cell technology (ECT) for local delivery in CNS
  • Future directions
  • 12:40 Networking Lunch

    13:40 REGENERATIVE MEDICINE FOR THE TREATMENT OF DIABETES

    Prof Thomas Knittel

    Prof Thomas Knittel, Vice President, Corporate & Medical Affairs, DeveloGen

  • Regenerative medicine as a cure for disease
  • Stem cells: facts and visions
  • Diabetes mellitus: a target for regenerative medicine
  • Identification and validation of control genes for beta cell development
  • Turning stem cells into beta cells by the use of Pax-4, a developmental control gene
  • Functional analysis of Pax-4 differentiated beta cells in vitro and in diabetic mice
    Outlook
  • 14:20 LENTIVIRAL VECTORS IN THE CLINIC

    Dr Boro Dropulic

    Dr Boro Dropulic, Founder & Chief Scientific Officer, VIRxSYS

  • Highly efficient gene delivery into primary human cells
  • Lentiviral vector cGMP manufacturing and QC
  • Targeted expression of anti-HIV antisense in HIV infected cells
  • Long anti-HIV antisense RNA decreases HIV resistance
  • Clinical scale transduction of primary T cells
  • Initiation of a phase I clinical trial using HIV vectors
  • 15:00 EX-VIVO THERAPEUTIC MODALITY FOR HIV-1 WITH LENTIVIRUS VECTOR TARGETED RNAi

    Dr Omar Haffar

    Dr Omar Haffar, President, Chief Science Officer, International Therapeutics

  • RNAi
  • HIV-1 RNA directed short hairpin RNA (shRNA)
  • Ex-vivo expansion of T-lymphocytes
  • ShRNA expression
  • Clinical protocol
  • 15:40 Afternoon Tea

    16:00 TURNING TOOLS INTO DRUGS

    Dr Steffen Panzner

    Dr Steffen Panzner, Chief Executive Officer , Novosom

  • Delivery is the bottleneck in nucleic acid based therapies
  • Liposomes are well approved in vitro but lack function in vivo
  • Charge reversible liposomes (Smarticles) were developed at Novosom
  • Smarticles are functional in gene transfer or oligonucleotide transport in vivo
  • Targeting is achieved with different chemistries
  • Smarticles are safe and well tolerated upon repeated dosage
  • 16:40 NON-VIRAL GENE DELIVERY FOR THERAPEUTICS

    Dr Douglas Doerfler

    Dr Douglas Doerfler, Chief Executive Officer & President, MaxCyte

  • Enhancing Safety & Efficacy
  • Reducing Regulatory Risks
  • Closed Sterile System
  • 17:20 REGULATORY AFFAIRS IN GENE AND CELL THERAPY

    Dr Lincoln Tsang

    Dr Lincoln Tsang, Of Counsel, Arnold & Porter

  • Regulatory framework governing emerging technology products
  • Regulatory and legal issues
  • Technical requirements
  • 18:00 Chairman's Closing Remarks and Close of Day One

    8:30 Re-registration and Coffee

    9:00 Chairman's Opening Remarks

    Dr Nigel Parker

    Dr Nigel Parker, Chief Executive Officer, Ark Therapeutics

    9:10 ADENO-ASSOCIATED VIRUS (AAV)-BASED GENE THERAPY

    Dr Jan Boesen

    Dr Jan Boesen, Chief Executive Officer, AMT

  • LPL deficiency: the disease and market potential
  • Efficient gene delivery by AAV-1
  • Significant lowering of triglyceride levels after LPL gene delivery in LPL deficient animal models
  • Scaling of AAV production
  • Clinical development program outline
  • 9:40 MARKET OUTLOOK FOR GENE THERAPIES IN CANCER

    Andrew Paramore

    Andrew Paramore, Oncology Analyst, Decision Resources

  • Overview of gene therapy clinical trials (phase I, II and III)
  • Focus on gene therapy for specific cancers
  • Market size
  • Current therapies
  • Unmet need
  • Market opportunity for gene therapy
  • 10:20 LIVER METASTASIS OF COLON CANCER

    Dr Richard Rigg

    Dr Richard Rigg, Director of Process Development, MediGene

  • Modified herpes simplex virus
  • Genetically engineered to replicate in and kill cancer cells while sparing normal cells
  • Phase I study and further development
  • 11:00 Morning Coffee

    11:20 POXVIRUS ENGINEERING FOR ANTI-CANCER GENE THERAPY

    Dr Jean-Marc Balloul

    Dr Jean-Marc Balloul, Molecular Immunology Laboratory Head, Transgene

  • Non integrative
  • Non propagative
  • Low cost manufacturing and storage
  • Applications: cancer immunotherapy, suicide gene transfer, vaccinotherapy (HIV, malaria, dengue…)
  • 12:00 HERPES SIMPLEX VIRUS

    Dr Robert Coffin

    Dr Robert Coffin, Chief Scientific Officer, Biovex

  • Overview of Biovex’s oncolytic virus platform
  • Phase 1 clinical data with OncoVEX GM-CSF
  • HSV-mediated dendritic cell vaccines
  • ImmunoVEX tri-melan
  • 12:40 Networking Lunch

    14:00 HEAT ACTIVATED GENE THERAPY

    Dr Augustine Cheung

    Dr Augustine Cheung, President & Chief Executive Officer, Celsion

  • Gene control by focused heat energy
  • Heat shock promotor as gene switch
  • Heat activated tumor targeting drug and gene delivery
  • Development of heat activated antisense cancer repair inhibitor (CRI)
  • Use of CRI in cancer treatment
  • Preclinical evaluations
  • 14:40 ADENO-ASSOCIATED VIRUS VECTORS

    Dr Barrie Carter

    Dr Barrie Carter, Executive Vice President & Chief Scientific Officer, Targeted Genetics

  • AAV vectors mediate prolonged gene expression
  • Scalable commercializable manufacturing
  • An excellent safety profile in the clinic
  • An AAV vector for cystic fibrosis by delivery of CFTR to lung
  • An AAV vector for joint diseased by localized expression of a TNFa inhibitor
  • An AAV vector for a prohylactic vaccine for AIDS by intramuscular delivery
  • 15:20 ADENOVIRUSES AS VECTORS FOR EX VIVO VACCINATION AND GENE THERAPY

    Dr Jaap Goudsmit

    Dr Jaap Goudsmit, Chief Scientific Officer, Crucell

  • Difference in cell tropism of adenovirus serotypes
  • Rare adenovirus serotypes for ex vivo vaccination
  • Rare adenovirus serotypes for gene therapy
  • 16:00 Chairman's Closing Remarks followed by Afternoon Tea. Close of Conference

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    WHAT IS CPD?

    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

    ‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

    CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

    Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

    CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.

    CPD AND PROFESSIONAL INSTITUTES

    There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

    For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

    CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.

    TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

    Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

    ‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

    ‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

    The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

    As a formal provider of CPD certified activities, SMI Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.

    GLOBAL CPD

    Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

    CPD Certificates

    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@smi-online.co.uk

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