Sponsors & Exhibitors

Orphan Drugs & Rare Diseases 2018 East Coast

Orphan

Horizon Pharma plc is focused on researching, developing and commercializing innovative medicines that address unmet treatment needs for rare and rheumatic diseases. By fostering a growing pipeline of medicines in development and exploring all potential uses for currently marketed medicines, we strive to make a powerful difference for patients, their caregivers and physicians. For us, it’s personal: by living up to our own potential, we are helping others live up to theirs. For more information, please visit www.horizonpharma.com, follow us @HZNPplc on Twitter or like us on Facebook.

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Orphan

Eiger is a clinical-stage biopharmaceutical company focused on the development and commercialization of targeted therapies for rare diseases. We innovate by developing well-characterized drugs acting on newly identified or novel targets in rare diseases. Our mission is to systematically reduce the time and cost of the drug development process to more rapidly deliver important medicines to patients with rare diseases. We plan to initiate D-LIVR, a Phase 3 study to investigate ritonavir-boosted lonafarnib in a single, pivotal trial in hepatitis delta virus-infected patients by end of 2018. We are preparing an NDA for lonafarnib in the treatment of Progeria in 2019. For additional information about Eiger and its clinical programs, please visit www.eigerbio.com.

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Orphan

CENTOGENE is the worldwide leader in understanding hereditary rare diseases. Using a combination of genetic knowledge and newly developed techniques in proteomics and metabolomics profiling, CENTOGENE is improving the lives of patients living with rare diseases. CENTOGENE is accelerating orphan drug development and transforming global genetic data into more informed medical decisions for physicians and our industrial partners globally. We achieve this through knowledge created by CentoMD®, the world’s largest curated genetic database of rare disease clinical cases, our worldwide diagnostic testing services incorporating global diversity and epidemiological expertise, and biomarker development expertise for disease monitoring and treatment.

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Orphan

We are the leading and most reputable strategy consultancy in the German health care sector. SKC was founded in 2005 as a spin-off of the Boston Consulting Group and the Hannover Medical School (MHH) with focus on health care companies, organizations and institutions in Germany, Europe and overseas. Our managing directors and colleagues are experienced and reliable advisors to pharmaceutical, medical and biotechnological companies as well as health insurances.
With our profound knowledge of the German and the European health care systems we illuminate your individual way to a successful Market Access. We at SKC think that every activity in the health care industry should focus on the improvement of the patient’s situation. We are convinced that the best solution for a health care problem can only be found in a competitive landscape, shaped by innovation and empowered by trust. We believe a better, more effective, efficient and fair health care system is possible due to digital technologies, interdisciplinary networks and highest professional standards.

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Orphan

A full-service global medical communications agency. We bring 400 years of insights and experience across healthcare sectors in to our award winning work which has changed the lives of those suffering with rare diseases. We believe that medical communications that are transformative require new ways of thinking – combining scientific credibility and boundless creativity. We lead thinking in the rare disease sector by placing behavioural science at the heart of our innovative multichannel programmes to ensure we make a tangible positive difference in healthcare.

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Orphan

Pennside Partners is a leading healthcare consulting firm serving the pharmaceutical, biotechnology, and medical device sectors across the world for more than 25 years. We offer a valuable range of strategic services including launch planning, competitive benchmarking, market assessment, business development, reimbursement, generics and biosimilars studies, licensing and acquisition support, competitive intelligence, qualitative market research, medical congress coverage, and technology / pipeline analysis. We have vast project experience and expertise in all major disease areas including oncology, respiratory, cardiovascular, CNS, rheumatology, orphan/rare diseases, and dermatology. We have extensive in-house knowledge of new drug development in chemotherapy, targeted therapies, monoclonal antibodies, vaccines, and drug delivery. Pennside's multidisciplinary teams conduct in-depth primary field research supported by extensive review and analysis of existing data, information and knowledge to provide custom projects that meet our client's specific needs.

Pennside has offices in the United States, United Kingdom, and Switzerland.

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Orphan

Invitae Corporation (NYSE: NVTA) is a genetics company whose mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. Invitae’s goal is to aggregate most of the world’s genetic tests into a single service with higher quality, faster turnaround time and lower prices.

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Orphan

Rarity Health is a division of ENA Healthcare Communications, located in Shrewsbury, NJ. We are a rare disease-focused organization seeking to improve the unique bond between you and your patients in the complex world of rare disease. Our proprietary approach builds trusted relationships between patients, advocates, and brand teams, creating a unified voice that brings awareness and solutions for patients.

Rarity Health was founded in 1985 and is dedicated to connecting patients living with rare disease to stakeholders in advocacy and pharma. Rarity Health: Rare, Well Done.

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Orphan

RareMoon Consulting is an orphan drug regulatory services firm focused on translating science into concise regulatory documents in preparation for key clinical trials both in the US and Europe. Our team possess a comprehensive knowledge of orphan drug development and a wealth of regulatory strategy from the hundreds of global agency interactions and work on successful orphan programs. Our sole focus in orphan drugs maintains our resourceful mindset and requires us to always consider novel strategies.
Based on client objectives, we help prioritize, define and accomplish regulatory milestones by offering strategic and full operational support, authoring through to submission.
We use a cross-functional ``eyes on - hands-on`` approach. The same senior-level regulatory scientists who strategize, also author and review every document we produce. This approach harmonizes strategies and enhances the knowledge of knowing when and how to interact, with a global perspective in mind.
To date, we have a 100% track record in achieving designations and navigating the Agencies for our Clients, ultimately driving successful programs to pivotal milestones, benefiting Patients and Stakeholders.
Your success is our success and we go to work for it.

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Orphan

MK&A enables pharmaceutical, biotech, device and diagnostic companies around the world to deliver quality care through innovative and effective partnerships with both patients and caregivers and patient, consumer and professional groups. Among its specialties is pre-approval drug access. MK&A has conceived and designed more than 30 early access programs for rare and more common diseases.

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Event Exhibitors

Orphan Drugs & Rare Diseases

Orphan

RareChannels is a DISRUPTIVE model, global, unaffiliated, web-based reference for pharma manufacturers in rare disease to find niche vendors directly, eliminating the in-efficient ``vendor-brokering``. New science in rare disease therapies is causing hundreds of unique content-expert firms to manifest, globally. We are harnessing these vendors in a category, or relevant term-searchable, website. Annual fee for the vendor, free-access to the users. Our Founder comes from rare manufacturer and consulting, sees the HOLE and has built the solution. Big consulting is charging TOO MUCH FOR VENDOR-BROKERING because there's currently no other way to find new and specific outsourced solutions in rare disease pharma manufacturing. Tiny manufacturers need help finding help; content experts cannot be found without paying consulting large ``finders fees``. We are only beginning with rare disease niche vendors, our future is a globally searchable niche-consulting reference, one new marketplace at a time, with our efficient marketing channel and search-engine for niche vendors, consulting, content experts, (ie solution partners) to be found. We also handle marketing material distribution at the International Rare conferences for our PIRs while they’re busy building their business. We get to rare EARLIER in a manufacturer’s lifecycle.

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Orphan

MNG Laboratories is an internationally recognized clinical diagnostic leader specializing in neurogenetic and complex biochemical testing. Our portfolio includes movement disorders, epilepsy, muscular dystrophies, intellectual disabilities, metabolic and other inherited disorders.

Our extensive offering of genetic and biochemical services, proprietary Genome MaNaGer® database and our Neurogenetic Answers™ reporting platform is recognized by genetic experts around the world. We are proud to offer first-in-class reporting and fast turnaround times.

With over 15 years of neurogenetic experience, and powered by a culture of discovery and advancement, MNG Laboratories delivers results that make a difference for patients and their families.

MNG Laboratories offers CLIA and CAP certified reports and adheres to current ACMG guidelines and recommendations.”

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Orphan

DefiniGEN was founded in April 2013 to industrialize the OptiDIFF stem cell production platform developed at the University of Cambridge, UK. The company has world-leading expertise in the area of iPSC production and metabolic disease modelling. The application of these technologies in drug discovery provides pharmaceutical companies with more predictive in vitro cell products enabling the development of safer and more effective treatments. In addition the technology platform utilises fully defined and humanized conditions required for the development of regenerative medicine cellular therapies.

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Orphan

Bexion Pharmaceuticals, a clinical-stage biopharmaceutical company is developing BXQ-350, a first-in-class agent composed of the multifunctional, lysosomal activator protein Saposin C and phosphatidylserine, that has demonstrated antitumor effects in vitro and in vivo, particularly in glioma and other solid tumors, including those that may lead to brain metastases. In 2013 the NCI awarded Bexion a prestigious ``Bridge Award`` of $3MM to support testing of BXQ-350 in the clinic. In February 2015, the FDA granted Bexion Orphan Drug status for Saposin C, the active ingredient in its proprietary drug BXQ-350, for the potential treatment of glioblastoma multiforme (GBM), a type of brain cancer. In June 2015, Bexion won a Tibbett's Award by the Small Business Administration for exemplifying the very best in innovation. Bexion has completed a multi-site first-in-human, Phase 1a study of BXQ-350 for solid tumors and gliomas, and is currently enrolling patients in an expansion phase.

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