2026 Conference Sponsors
Azafaros
Azafaros is a clinical-stage biotech company founded in 2018. Our focus is on developing a pipeline of disease-modifying therapies for patients with rare genetic lysosomal storage disorders including, among others, GM1 and GM2 gangliosidoses and Niemann-Pick Type C disease (NPC). By applying its knowledge, network and courage, the Azafaros team challenges traditional development pathways to bring new drugs to the rare disease patients who need them. Azafaros is supported by leading healthcare investors.
azafaros.com
Beren Therapeutics
Beren Therapeutics P.B.C. is a founder-led biotechnology company pioneering the discovery, development, and commercialization of cyclodextrin-based therapeutics for conditions characterized by defective cholesterol trafficking. Beren’s first program, adrabetadex, is under U.S. Food and Drug Administration review with a Prescription Drug User Fee Act target action date of November 17, 2026, for the treatment of infantile-onset Niemann-Pick disease, type C (I-NPC). Beren and its subsidiary Mandos LLC have supported the NPC community since 2021 through an Expanded Access Program and are preparing for the U.S. commercial launch of adrabetadex, if approved, with a comprehensive set of patient access and family support programs. Beren’s public benefit purpose is to discover, develop, and deliver novel therapies that provide optimal benefit for patients, and to do so by integrating the needs of patients, caregivers, clinicians, and health systems from the beginning of the development process and maintaining a long-term focus on delivering meaningful therapies and access. Beren is headquartered in Thousand Oaks, Calif. To learn more about Beren, the adrabetadex program, and Beren’s cholesterol-trafficking focused therapeutic strategy, visit the company’s website or follow Beren on LinkedIn.
berentx.com
Cyclo Therapeutics
We at Cyclo Therapeutics, a Rafael company, are committed to collaborating with patients and families, as well as advocacy organizations around the world. Together, we share a common goal to improve the lives of patients, their families and caregivers affected by rare and neurological disorders. Our most advanced drug discovery program is focused on NPC. With 94 patients studied across 27 sites in 13 countries, the TransportNPC™ study is the most comprehensive, controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes of an investigational therapy for NPC. Additionally, ten patients were enrolled in a single-arm sub-study for newborns to 3 years of age. This achievement is a testament to our collective commitment to advance our knowledge of NPC, and to the unmet need that exists for this rare and underserved community. Please reach out to [email protected] with questions.
cyclotherapeutics.com
Intrabio
Intrabio is a biopharmaceutical company focused on discovering, developing, and commercializing therapies for neurodegenerative diseases with high unmet medical needs. Our clinical programs leverage the expertise of our scientific founders from the University of Oxford and University of Munich, the preeminent experts and pioneers in discovering and developing small molecule drugs that modulate lysosomal function and intracellular calcium signaling. Our management team and business consultants have vast commercial and regulatory experience in drug product development, including all stages from small molecule manufacturing, clinical studies, and regulatory approvals in the USA and Europe. With their successful track record of drug development and commercialization, IntraBio’s team translates research in the fields of lysosomal biology, autophagy, and neurology into orphan drugs and treatments that can significantly improve the lives of patients, their caregivers, and families.
intrabio.com
Sanofi
Sanofi is a leading global biopharmaceutical and healthcare company headquartered in Paris, France. It researches, develops, and manufactures prescription medicines, vaccines, and consumer healthcare products, with a major focus on immunology, rare diseases, and infectious diseases.
sanofi.com
Zevra Therapeutics
Zevra Therapeutics, Inc. is a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. The Company is focused on broadening access through geographic expansion opportunities, progressing its pipeline toward key milestones, and delivering meaningful therapeutics. The commercialization of its lead product, marketed in the U.S. for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disease, provides a strong corporate foundation and validates its ability to advance therapies from development to market. Zevra’s vision is realized through disciplined execution of its strategic plan and core values – patient centricity, integrity, accountability, innovation, and courage – which guide its efforts to deliver long-term value.
zevra.com
Curant Rare
At Curant Rare, we believe that every person living with a rare disease deserves compassionate, personalized support throughout their healthcare journey. As a specialty pharmacy and patient services partner, we are dedicated to helping individuals and families navigate the unique challenges that come with rare and complex conditions. We know that living with a rare disease often means facing uncertainty, overcoming barriers to
care, and coordinating support across many parts of the healthcare system. That’s why we believe through clinical expertise, education, unwavering advocacy and compassionate support, we can empower patients and families to focus on what matters most. We are honored to partner with the rare disease community and support continued advances in care, research, and treatment that bring hope for a brighter future. Together, we are building a future where every rare disease patient and caregiver feels seen, supported, and empowered – because no diagnosis should define what’s possible.
curantrare.com
Orsini
Providing patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers, and payors to support patients and their families in accessing revolutionary treatments for rare diseases. With access to four Niemann-Pick disease type C and acid sphingomyelinase deficiency therapies, Orsini supports the community through pharmacy distribution, patient services, clinical management, and convenient home infusion services. Orsini’s high-touch care model centers on experienced and trained therapy care teams that provide personalized care to ensure that No Patient is Left Behind™.
orsini.com
