18th Orphan Drugs & Rare Diseases 2022 Americas
Sponsors & Exhibitors
Early Access Care LLC
Early Access Care helps international clients with effective integrated solutions customized to their early access needs. Early Access Care specializes in pre-approval early access, managing the end-to-end (request intake to delivery at physician) process. Our goal is to Make Access More Accessible™.
We offer comprehensive high-touch services to manage requests globally.
Tanner Pharma Group is a specialist pharmaceutical services provider headquartered in Charlotte, NC USA with offices in the United Kingdom, Switzerland, Ireland and Brazil. With a focus on improving patient lives by increasing access to medicines around the world, the company has developed a portfolio of service offerings that provide international commercialization and patient access through licensing, managed access programs, global access programs and clinical trial services, as well as donation programs through biopharmaceutical and non-profit partnerships. By partnering with Tanner Pharma Group, companies are able to focus on their primary markets while ensuring that patients and clinicians receive the products they need through compliant and controlled channels. For more information, visit www.tannerpharma.com.
Founded in 2007, InformedDNA is the country’s largest applied genomics solutions company, helping more people harness the full power of the genomics revolution. With the largest, lab-independent staff of board-certified genetics specialists in the U.S., InformedDNA helps to ensure that pharmaceutical and biotech companies, health plans, clinicians, and patients all have access to the highest quality genetic services.
Key offerings for pharmaceutical and biotech companies include design support for genetics-based clinical trials and studies, patient engagement and recruitment, physician education and engagement, clinical genetic counseling, and test ordering and interpretation services.
For more information, visit https://informeddna.com/lifesciences/
Smartway designs, implements and manages global programs that empower access to innovative therapies.
We partner with pharma and biotech organizations to deliver customized programs that transform lives. These partnerships have made us a leader in the provision of Expanded Access, Early Access, Compassionate Use and Named Patient Programs across 98 countries. Over the last 20 years, we’ve gained the experience it takes to strategically advice, design, initiate and manage global or regional programs with you.
We provide end-to-end solutions to manage complex EAPs. Whatever your size and scale, we’re dedicated to empowering patient access to the right therapy
GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics’ lead product candidate, LUMEVOQ® (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
Pulse Infoframe Inc (Pulse) believes thatcollaboration cures disease and Pulse enablessuch collaboration. A new style of healthcareinformatics company, Pulsebuilds and supports global, healthcareecosystems. It’s solution, healthie™, empowersconsortia of pharmaceutical and medical devicecompanies, researchers, clinicians, advocacygroups and patients, by aggregating informationfrom a variety of sources and then, delivering keyhealthcare insights – real time — derived fromthese data. For patients with rare diseases, Pulse’sglobal coverage is critical. Already great strideshave been made in characterizing rare diseasesusing Pulse’s global and national registries. Theseinsights help professionals to advance medicalresearch, focus clinical trial recruitment, assistadvocacy groups in their dealings with policymakers and provide real-world evidence.
Please visit pulseinfoframe.com
Illingworth Research Group is a provider of patient-centric clinical research solutions. Services are tailored to clinical requirements but can include: mobile research nursing, patient concierge, and medical photography, for example. These services are operational in 45 countries and concentrate on taking the trail to the patient or making trial participation easier and more accessible to patients and their families.
Patient centricity is a vital part of our mission and has been so for 20 years. Our Research Nursing support focuses on bringing the trial to the patient in their home, school, or workplace. This can enhance both patient recruitment and retention. Our medical photography team offers an alternative approach by using imagery and video footage within the clinical trial. Imagery can be used in a range of applications concentrated within but not exclusive to dermatological trials.
Genomenon is a genomic health IT company that keeps pace with the constant advancements made in genomics and connects that research to patient DNA to help diagnose and treat patients with rare genetic diseases and cancer. Mastermind Genomic Landscapes inform pharmaceutical companies on precision medicine development, deliver genomic biomarkers for clinical trial target selection, and support CDx regulatory submissions with empirical evidence. Genomenon was named Company of the Year in Clinical Genomics Interpretation 2020 by Frost & Sullivan.