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 International LLC (Premier) is a leading global clinical research organization (CRO) and consulting partner with expertise in driving an efficient and effective path to market for the life sciences industry. Built with the needs of...

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 ...

AXIS Reimbursement is a specialist market access and HTA advisory firm supporting pharmaceutical and biotechnology companies to successfully bring innovative therapies to market across Europe and the UK. Founded in Ireland in 2012, AXIS has built a ...

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 ...

Farma Mondo is a leading pharmaceutical company dedicated to developing innovative treatments for rare diseases and orphan drugs. With a focus on improving the lives of patients with unmet medical needs, Farma Mondo combines cutting-edge...

As the leading provider of tech-enabled genetic services, we bridge critical gaps in access, ensuring that individuals and families affected by rare conditions receive the expert guidance they need. Through timely genetic counseling, streamlined...

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...

Sanofi is a global pharmaceutical company dedicated to discovering, developing, and distributing innovative medicines for patients worldwide. With a focus on rare diseases and orphan drugs, Sanofi is committed to addressing unmet medical needs and...

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 ...

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...

Forian delivers patient-level healthcare data and analytics that help life sciences teams make more informed commercial and clinical decisions. By combining closed and open claims into a hybrid dataset, Forian provides a more complete view of...

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...

LEO Pharma is a global pharmaceutical company dedicated to improving the lives of people with skin diseases and rare genetic disorders. With a focus on dermatology and critical care, LEO Pharma develops innovative treatments and therapies to address ...

Commercialization has entered an age of opportunity and connectivity - but also unprecedented complexity. Life sciences leaders are expected to make faster, higher‑stakes decisions with fewer resources and less clarity, while traditional, fragmented ...

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...

JW Pharmaceutical, a top-tier pharmaceutical company in Korea and a core affiliate of JW Holdings, is advancing a high-impact pipeline of differentiated therapeutics across key strategic areas, including rare CNS indications, immune and inflammatory ...

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...

MMS is an award-winning, data focused CRO, supporting pharmaceutical and biotech sponsors across the clinical development lifecycle. Founded in 2006 and winner of 2025 Fierce CRO Excellence in Client Service and Partnership Award, MMS is a global...

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 ...

Harvest Integrated Research Organization (HiRO) is a modern, full-service global CRO delivering disciplined, end-to-end clinical development solutions for biopharma innovators. With integrated teams, advanced clinical data science, and globally...

Know Rare is an AI Patient Intelligence Engine for rare disease that helps continuously find, engage, and understand rare disease patients to better power research, development, and commercialization. What started as our family's rare disease...

OMNI CRS is the leading performance-based patient recruitment partner that delivers pre-qualified, protocol-matched patients at scale, enabling study teams to hit enrollment targets faster while reducing screen failure rates and recruitment costs.

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...

OPIS is a full-service clinical research organization providing end-to-end support for pharmaceutical, biotechnology, and medical device clinical trials, including clinical operations, regulatory support, data management, biostatistics, and medical...

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 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...

Identifying at-risk individuals through family networks. Quoiden is a digital health platform focused on identifying individuals at risk for inherited conditions who remain undetected through traditional healthcare pathways. By enabling...

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...

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...

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...

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...

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...

Curestack is an AI-powered patient intelligence company transforming patient, caregiver and physician voice into decision-grade evidence for life sciences.In rare disease and oncologic, patients are scattered, underdiagnosed, and rarely heard at the ...

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...

E9 Genomics builds scientific reasoning systems that help therapeutics teams systematically explore biological search spaces to uncover non-obvious, high-value opportunities hidden outside the center of the distribution. Founded by the team behind...

Heme Soluions - Solving the world's blood shortage Patent pending Universal Oxygen Carrier for use in the artificial blood substitute market. + universal for all blood types  + safe  + stores at room temperature with 3 year shelf life  + 15 ...

Established in 2014, HiComp Microtech is a leading original design manufacturer (ODM) specializing in advanced microfluidic products and laboratory consumables. Our ISO-certified and FDA-registered facilities span 100,000 sq. ft., including...

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...

Make the right cells, consistently, at scale. iOrganBio is transforming human cell production with CellForge, an intelligent, automated manufacturing platform that monitors digitized cell states and uses AI to steer differentiation toward true in...

Miramoon Pharma is a biotechnology company developing first-in-class neuroprotective therapies for rare diseases. Leveraging a proprietary platform of small molecules targeting disrupted intracellular calcium homeostasis, the company is advancing...

NanoZymeX is a Basel-based biotech startup developing next-generation enzyme replacement therapies for lysosomal storage disorders, with Pompe disease as its lead indication. The company’s lipid nanoparticle platform is designed to improve...

Novel Bio is a lean, technology-differentiated CDMO providing a secure, domestic alternative for your critical raw materials. By combining optimized traditional E. coli platforms with agile operations, we deliver R&D, High-Quality (HQ), and...

ODDIFACT is a purpose driven company whose mission is to enable the emergence of treatments by showcasing the untapped medical research. It obtained from the FDA, 14 Orphan Drug Designations over the past two years and is seeking : 1/ closer...

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...

PRONAV Clinical is a global leader in early-phase clinical trial supply operations, specializing in the seamless delivery of investigational medicinal products to clinical trial sites worldwide. We specialize in supporting emerging pharma and...

Rhizome helps you know what your regulator thinks. Ask any regulatory or clincial question and we'll answer it, hallucination-free and citing up to 1,000 sources.  Used by companies from preseed biotechs to >$50b public companies. 

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...

Understanding comes first. Decisions follow. trialport is an AI-native clinical trial finder, built for the rare and orphan landscape. WHAT WE DO Find. We surface relevant studies for people living with rare and orphan conditions, fast. ...

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...

Winsights is an AI-powered digital health venture philanthropy platform that brings families living with rare conditions together and transforms their lived experiences into structured data they own, enabling doctors and researchers to develop...

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...