Pitch & Partner

Pitch & Partner, Wednesday 25 April 2018

last published: 23/Mar/18 19:55

Pitch & Partner, Thursday 26 April 2018



Katherine High

Keynote address: A new era in medicine- introducing gene therapy

  • What we’ve learned – lessons science has taught us as we create the path for the first gene therapy for an inherited disease in the U.S.
  • Staying true to our roots while preparing the patient, medical and policy maker communities for a new chapter in the retinal disorders textbook
  • Assessing the impact of potential one-time treatments like gene therapies for patients with rare diseases
Hans Bishop

Keynote address: Cell and gene therapy innovation and their role in the future of rare disease research

  • Current state of cell and gene therapies and why this field is becoming more and more significant 
  • Looking into scalability and centralized approaches for cell and gene therapy development and commercialization
  • Novel approaches and how these therapies benefit the continuum of research for rare diseases
Andre Choulika

Keynote address: Next- generation CAR T-cells to cure cancer - paving a new way for R&D, value-assessment and pricing and reimbursement of treatments for genetic diseases

  • Learnings from CAR-T development that can be applied to rare disease drug development 
  • Will CAR therapy pricing impact pricing schemes of other orphan drugs and gene therapies? 
  • Exploring aspects of these different therapeutic areas that share common goals, personalization and pricing challenges 
Panel discussion

Keynote CEO Panel: Gene therapy - a new paradigm for patients, new challenges for industry

  • Looking back at the commercial performance of the first generation of gene therapies- from proof of concept to the road to approval 
  • How will gene therapy innovation look in the next 10 years?
  • Considering potential new value propositions for patients, payers and the health care system
  • Strengthening clinical development, exploring novel technologies while maintaining safety- what needs to be improved? 


Pitch & Partner

Pitch & Partner

Pitch & Partner

Investment landscape & 10min Biotech Pitches

Brian Bronk
Pitch & Partner

VC Panel: Investing in the orphan drug space – derisking commercial models and driving biotechs to successful launches

  • Assessing advanced therapies commercial opportunities – and risks – as the new frontier of investment in rare disease companies
  • Type of work and diligence to obtain comfort with the ultimate market opportunity
  • Assessing barriers to entry including therapeutic modality and IP
  • Regulators role in incentivizing or impeding the ability to develop products both domestically and abroad. What can be different?
Alvin Shih
Pitch & Partner

Case study: Enzyvant’s approach to developing RVT-802, a novel tissue-based therapeutic for complete DiGeorge Syndrome

  • Leveraging expedited regulatory pathways, including breakthrough designation and Regenerative Medicine Advanced Therapy (RMAT) designation
  • Collaborating with academic partners
  • Fostering advocacy relationships and aligning corporate and patient priorities
  • Developing a commercial model for a transformative, one-time therapy in the pre-launch phase
Chandler Robinson
Pitch & Partner

Validive for the treatment of severe oral mucositis

Christer Rosen
Pitch & Partner

JOTROL™ for treatment of MELAS syndrome, Friedreich's ataxia and MPS I


Yann Le Cam

Lunch keynote address: Breakthrough therapies call for breakthrough processes to ensure sustainable patient access


2:30 ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min – attendees can select two roundtables to attend)

If you are interested in sponsoring a roundtable contact André Singer at andre.singer@terrapinn.com or +1 646 619 1797
Igor Lokot
Pitch & Partner

Temodex, a locally acting form of temozolomide, for treatment of glioblastoma

ROUNDTABLE 1: Pediatric trials - reducing the financial, logistical and emotional burden for patients and families participating in pediatric trials

Andrew McFadyen

ROUNDTABLE 2: Compassionate Use Programs - helping patients get access to medication, educating the community, physicians and investors to ensure safe access to experimental treatments

Scott Schliebner

ROUNDTABLE 3: Virtual Trials - enabling clinical trials to be more virtual, reducing distance times, overcoming geographic barriers and increasing data collection through the use of wearables

Jayne Gershkowitz

ROUNDTABLE 4: Expanded access - incorporating the patient advocacy perspective to develop a review process and a formalized procedure on expanded access

Joseph Musumeci

ROUNDTABLE 5: BD & Forecasting - pricing and access considerations and understanding how to build the patient forecast based on available epidemiological information

Yvette Venable

ROUNDTABLE 6: Strategy - patient engagement and pipeline building for the commercialization and access of rare disease products

Francesca Cook

ROUNDTABLE 7: Gene therapy – new payment and financing models are needed to facilitate access

Michael Murphy

ROUNDTABLE 8: Clinical development – When the sum is greater than the parts through academic, pharmaceutical and CRO collaboration in orphan disease drug development

Robert Donnell

ROUNDTABLE 9: Managed Access Programs - How MAPs (pre-commercial supply to patients with unmet medical needs) can be a valuable component of launch strategy for orphan drugs

Samantha Parker

ROUNDTABLE 10: Gene therapy - Optimizing gene therapy clinical development through education, collaboration, patient input and involvement in advisory boards

Stephen Franklin
Pitch & Partner

SFX-01 for the treatment of subarachnoid haemorrhage

Philip J. Brooks

ROUNDTABLE 11: Scientific innovation - platform approaches to evaluate multiple genetic diseases at the same time

David Lapidus

ROUNDTABLE 12: Epidemiology and forecasting - using epidemiological data to determine market viability, niche populations and commercial differentiation

Betsy Ricketts

ROUNDTABLE 13: Government affairs - developing a company’s footprint and contributions to impact policy, reimbursement and patient engagement

Anne Cropp

Roundtable 15: Expanded Access - drug development strategy considerations for open label extension or expanded access protocols

Ari Brettman

ROUNDTABLE 16: VC funding – successfully addressing investors interests and challenges when assessing companies of all sizes to guarantee funding of orphan drug biotechs

Trista Morrison

ROUNDTABLE 17: Disease awareness - best practices and tips for smaller companies to tell a corporate or rare disease story and raise awareness

Francesca Mcbride

ROUNDTABLE 18: Facility design – critical aspects to consider for clinical and commercial production of cell and gene therapy products

Francesca Mcbride, Director Regulatory Compliance, Jacobs
Saira Bates
Pitch & Partner

A bis-benzylisoquinoline alkaloid for use with chemotherapy for refractory and relapsed acute myeloid leukemia (AML)

Martin Welschof
Pitch & Partner

OPN-305, A first in class toll like receptor 2 (TLR2) antibody inhibitor

Philippe Guedat
Pitch & Partner

IFB-088 a potential new therapeutic option to treat demyelinating charcot-marie-tooth diseases

Jay Madan
Pitch & Partner

5-(4-carboxymethyl-phenylazo)-2-hydroxybenzoic acid disodium salt (INN-108) for treatment of ulcerative colitis in pediatric/adult patients


Laxminarayan Bhat
Pitch & Partner

RP5063: A novel investigational drug to treat pulmonary arterial hypertension (PAH)

Emerging Markets

Ray Takigiku
Pitch & Partner

BXQ-350, a novel approach for the potential treatment of glioblastoma multiforme

Alan Robertson
Pitch & Partner

ALS205 for the treatment of amyotrophic lateral sclerosis (ALS)

Lynnet Koh
Pitch & Partner

TZ 101/TZ 102: Transforming cancer patient survival and quality of life outcomes by improving the delivery of therapeutic cells to tumors and sites of inflammation

Erik Richardson
Pitch & Partner

Human rhodospin optogenetics for treatment of retinitis pigmentosa

Mehran F. Moghaddam
Pitch & Partner

A novel mechanism for treating ipf using small molecules

Matt Ritter
Pitch & Partner

Hepcidin for treatment of iron overload disorders



Douglas Ingram

Keynote panel: How rare disease companies are ramping up innovation to boost orphan drug development and patient centricity

  • Incorporating patients in the drug development process and working towards commercial viability- how to fully engage? 
  • How do you view disruptive therapies such as (gene therapy/gene editing) impacting the broader orphan disease development paradigm? How are you innovating within your company?
  • Responding to the changes in the political, regulatory and reimbursement landscape while guiding the direction of rare disease companies 
  • How to achieve industry collaboration and idea exchanges that can better serve the patient community? 

6:20 NETWORKING COCKTAIL PARTY – Reserved for Gold Sponsor

last published: 23/Mar/18 19:55