Sponsors & Exhibitors

Janssen is a leading pharmaceutical company dedicated to addressing unmet medical needs in rare diseases and orphan drug development. With a strong focus on research and innovation, Janssen is committed to improving the lives of patients with rare...

About ParexelParexel is a leading global clinical research organization (CRO) providing insights-driven Clinical and Consulting solutions to the world’s life sciences industry. Leveraging deep local knowledge and a global breadth of...

Premier Research is a leading clinical research organization specializing in the design and execution of complex clinical trials for orphan drugs and rare diseases. With a focus on delivering innovative solutions and personalized services, Premier...

Sciensus Pharma Services Limited is a leading global provider of specialized services for the orphan drug and rare disease industry. With a focus on patient-centric solutions, Sciensus offers a comprehensive range of services including market access ...

ZS Associates is a global consulting firm specializing in sales and marketing solutions for healthcare and life sciences industries. With a focus on orphan drugs and rare diseases, ZS leverages data-driven insights and innovative strategies to help...

Ambit is an analytics and technology-enabled solutions company that allows biopharma to adapt to the challenges and opportunities in the rare and specialty disease markets. We bring best-in-class talent together with cutting-edge technology and data ...

Biomedica is the EU-headquartered commercialization partner of choice for small and mid-sized biopharma innovators expanding into ex-US markets. With local teams, established in-market infrastructure, and strong compliance standards across 30+...

Clinipace Inc., doing business as Caidya, is a leading global clinical research organization specializing in rare diseases and orphan drugs. With a focus on accelerating the development of innovative therapies for underserved patient...

At KJT, we bridge medical innovation with human understanding to help healthcare companies act with confidence and purpose. Our seasoned experts partner with you from insight to impact—bringing therapeutic depth, real-world evidence, and strategic...

ERGOMED is a global company specializing in providing clinical development services to the biotechnology and pharmaceutical industries. With a focus on orphan drugs and rare diseases, ERGOMED offers expertise in clinical trial management, regulatory ...

Genome Medical is a leading telehealth company specializing in genetic testing and precision medicine for rare diseases. Their innovative platform connects patients with genetic specialists to provide personalized care and treatment plans. With a...

Founded in 2003 by Dr Graeme Ladds, PharSafer® is a specialist Contract Research Organisation (CRO) in Global Clinical and Post Marketing Drug Safety, and Medical Services, with a wealth of experience in Pharmacovigilance, Auditing and Medical...

RTI Health Solutions is a leading research organization specializing in providing innovative solutions to complex health challenges. With a focus on orphan drugs and rare diseases, RTI offers expertise in health economics, outcomes research, market...

With patients at the forefront of everything we do, subrosa Health & Capital, a global consulting and execution services group, enables Rare Disease and Biotech originators to achieve accelerated access to any commercialization pathway, any...

AnovoRx is a leading pharmaceutical company dedicated to developing innovative orphan drugs and treatments for rare diseases. With a focus on precision medicine and personalized therapies, AnovoRx is committed to improving the lives of patients with ...

Bionical Emas is a global Early Access Programs (EAP) specialist bringing life-changing medicines to patients around the world.  Their comprehensive suite of services and capabilities benefit many of the world’s leading pharma and biotech...

Curant Rare delivers fully integrated, end-to-end specialty pharmacy solutions purpose-built for rare and ultra-rare diseases, where complexity is high and traditional models fall short. We go beyond dispensing to provide a deeply...

Lumanity is a leading global strategic partner for biopharmaceutical companies, engineering breakthrough value to transform lives. We combine deep scientific, clinical, medical, regulatory, and commercial expertise with advanced technology and...

Clinical Trial Enrollment Accelarator - When Every Patient Counts At Adnexi, we specialize in solving one of biopharma's toughest challenges: enrolling clinical trials in rare and ultra-rare diseases, fully and as fast as possible. Our...

Minoryx is a clinical-stage biotech company focused on the discovery and development of novel therapies for severe, orphan genetic diseases of the central nervous system (CNS) with high unmet medical need. Our lead compound (leriglitazone) is a...

Paradigm Therapeutics is a Charleston-based biotechnology company clinical-stage company advancing SD-101-6.0 (Zorblesia), a first-in-class topical therapy for Epidermolysis Bullosa (EB) across all subtypes, a devastating condition characterized by...

Zeal Specialty Pharmacy is a leading provider of specialized pharmaceutical services dedicated to improving the lives of patients with rare diseases. With a focus on orphan drugs, Zeal offers personalized medication management, patient support...

OPTEL is a global traceability partner helping clinical teams move toward first commercial launch with less risk and less complexity. Through VerifyBrand, OPTEL delivers a true one-stop-shop serialization solution that combines fixed...

Centogene is a leading company in the field of genetic diagnostics and precision medicine, dedicated to transforming clinical, genetic, and biochemical data into medical solutions for patients. Based in Rostock, Germany, the company operates...

CheckRare is the only publisher and learning platform focused exclusively on rare diseases and rare cancers. We bring physicians and patients the best science, research, and clinical practice information and present it in understandable and...

Experic is a contract development and manufacturing organization (CDMO) and clinical trial supply services company dedicated to revolutionizing pharmaceutical development, manufacturing, and clinical trial supply. Established in 2018, Experic offers ...

At Rare Rebels™, we use photojournalism, filmmaking, and podcasting to share the deeply personal stories of rare disease patients and the rebels standing beside them to make a difference. By capturing these stories, we raise awareness to show the...

Only Orphans Cote LLC is a pioneering biopharmaceutical company dedicated to developing innovative therapies for orphan diseases and rare disorders. With a focus on addressing unmet medical needs in underserved patient populations, the company...

OPTEL is a global traceability partner helping clinical teams move toward first commercial launch with less risk and less complexity. Through VerifyBrand, OPTEL delivers a true one-stop-shop serialization solution that combines fixed...

Pathways for Rare and Orphan Solutions PROS Foundation is a 501(c)(3) non-profit organization dedicated to helping bring treatments to patients living with rare diseases worldwide. PROS supports rare disease patient organizations with: ...

Pathways To Trust is a non-profit organization building trust in healthcare for rare disease patients facing high hurdles to care by bringing stakeholders together to develop healthcare provider and patient communication training.

For over 25 years, ProPharma has partnered with biotech, pharmaceutical, and medical device organizations to bring groundbreaking therapies to market. Offering a fully customizable, end-to-end suite of services, we empower our clients to overcome...

Rare Disease Research, LLC is a leading research organization dedicated to advancing the understanding and treatment of rare diseases. With a focus on innovative research and collaboration with key stakeholders in the rare disease community, Rare...

Founded in 2018 as the philanthropic arm of RTW Investments, LP, RTW Foundation powers rare disease research, medical innovation, and local community collaborations to improve the health of underserved communities. We provide capital, expertise, and ...

Sharp Sterile Manufacturing is a leading contract development and manufacturing organization (CDMO) specializing in the production of sterile pharmaceuticals for rare diseases and orphan drugs. With state-of-the-art facilities and a team of experts...

At the Society for Participatory Medicine, they connect everyone who has a stake in healthcare — patients, families, clinicians, payers, researchers, and policy leaders — to work together as partners. They believe collaboration, clear...

The Rare & Ready Coalition advocates for policies that ensure those living with rare or genetic conditions get the care they need, when they need it. The rare disease community deserves access to FDA-approved therapies as soon as they are...

And Scene delivers rare disease patient voice from the patients you're not hearing. Ninety percent of rare disease patients never post in online health communities. Your surveys, focus groups, and conference panels are hearing from the other...

Canary Concierge provides high touch clinical trial concierge services designed by people who deeply understand the challenges of study participation. Most existing concierge programs leave patients feeling under-cared...

Donor X is a small, woman-owned biospecimen sourcing and technology company that supports life science organizations with reliable access to high-quality human primary samples for research, development, and translational applications. Through a...

Humigent builds AI-powered operational agents purpose-built for biopharma companies developing and commercializing rare disease therapies. From clinical-stage biotechs preparing for first launch to commercial teams scaling with lean resources, our...

Panome Bio provides biomarker and mechanism discovery through Next-Generation metabolomics, proteomics, transcriptomics, exposomics, and integrated multi-omics analysis. Powered by a proprietary bioinformatics pipeline, we unlock complex biological...

pRxTrialPort is an AI-first, human-centred platform built to help patients become partners in research. In rare disease and oncology, too many people are still expected to navigate complexity, jargon, and life-changing decisions with far too little ...

RIVIA is the first Trial Intelligence platform with Agentic AI tools for modern sponsors. It brings all clinical and operational data into one single view, giving sponsors real-time control of the clinical development and saving up to 30% of the...

Unboxed Psychology is a behaviour science consultancy specialising in health psychology and behaviour change principles. Providing strategic services across healthcare working with pharmaceutical and biotech companies to infuse behaviour science...

Unravel Biosciences is a clinical-stage therapeutics company that integrates AI systems biology computation with rapid clinical validation of discovered targets, leading to four clinical trials starting in 2026. Unravel leverages its proprietary...

Workomics partners with biotechs to bring life-changing therapies to patients. The core of our work is deep collaboration with patients, caregivers, advocacy, healthcare providers, biotech and pharma. We create patient-centered advocacy engagement...