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RARE Drug Development Symposium Marks 10th Anniversary with Dedicated Pitch Sessions and The Theme and Call to Action to Align Advocates, Science and Industry

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BIIB While not explicitly mentioned, the context of rare disease drug development and advocacy symposium suggests potential involvement or interest from companies like Biogen, a major player in the biotech and rare disease space. However, without direct mention, this is speculative. REGN Similar to Biogen, Regeneron operates in the biotech and rare disease sector. Their participation or interest in such a symposium is plausible given the industry focus, but they are not directly named in the article. GILD Gilead Sciences is a large biopharmaceutical company that could be involved in rare disease research. The symposium's focus on drug development and industry partnerships makes it a relevant event for companies like Gilead, though they are not explicitly mentioned. BTE Baytex Energy is an oil and gas company and is not relevant to the content of the article, which focuses on rare disease drug development. Therefore, no sentiment can be derived. BROS Dutch Bros Inc. is a coffee company and is not related to the subject matter of rare disease drug development discussed in the article. No sentiment can be assigned. BFLY Butterfly Network focuses on medical imaging technology. While related to healthcare, the article's specific focus on rare disease drug development and advocacy does not directly mention or imply sentiment for this company. BCTX Celsion Corporation is a biotechnology company focused on cancer therapies. While in the biotech space, the article's specific focus on rare diseases and the companies mentioned do not directly include Celsion, thus no specific sentiment can be derived. BCRX BioCryst Pharmaceuticals is listed as a sponsor of the symposium, indicating their active participation and interest in rare disease drug development. This suggests a positive or at least neutral engagement with the field. BTAI BioXcel Therapeutics is a clinical-stage biopharmaceutical company. While not explicitly mentioned as a sponsor or participant, the symposium's theme of accelerating rare disease progress and aligning industry could be relevant to their work. ALRS Alerus Financial Corporation is a financial services company and is not relevant to the article's focus on rare disease drug development. No sentiment can be derived. CAPR Capricor Therapeutics is a biotechnology company focused on rare diseases. While not explicitly listed as a sponsor, their work aligns with the symposium's theme, suggesting potential interest or involvement. CRIS CRISPR Therapeutics is a leading gene-editing company. Their technology is highly relevant to rare disease research, and while not explicitly mentioned, their involvement in such a symposium would be logical. CYCN Cyclerion Therapeutics is a biopharmaceutical company. The article does not mention them specifically, and their focus areas may not directly align with the symposium's emphasis on rare diseases. EXAS Exact Sciences focuses on cancer diagnostics. While healthcare-related, the article's specific focus on rare disease drug development and the listed sponsors do not directly include Exact Sciences, so no sentiment can be assigned. FATE Fate Therapeutics is a clinical-stage biopharmaceutical company developing cell therapies. Their work in rare diseases makes them a potentially relevant entity, though not explicitly mentioned. GNTX Gentex Corporation manufactures dimming mirrors and electrochromic glass for automobiles. This is unrelated to the article's topic of rare disease drug development. HRMY Harmony Biosciences focuses on treatments for rare neurological diseases. Their work aligns with the symposium's theme, suggesting potential interest or involvement, although they are not explicitly mentioned. INCY Incyte Corporation is a biopharmaceutical company focused on oncology and inflammation. Their work in these areas could intersect with rare diseases, but they are not explicitly mentioned in the article. IONS Ionis Pharmaceuticals is a leader in RNA-targeted therapeutics, a field highly relevant to rare disease treatment. While not explicitly mentioned, their work aligns with the symposium's goals. KODK Eastman Kodak Company is primarily involved in imaging and printing technology, which is unrelated to the article's focus on rare disease drug development. LAKE Lake Shore Bancorp, Inc. is a bank holding company. This is unrelated to the article's topic of rare disease drug development. LULU Lululemon Athletica Inc. is an athletic apparel company and is not relevant to the article's focus on rare disease drug development. MRNA Moderna, Inc. is a biotechnology company known for its mRNA technology. While their platform is applicable to various diseases, including potentially rare ones, they are not explicitly mentioned in the article. NBIX Neurocrine Biosciences focuses on neurological and endocrine-related diseases. Their work aligns with the symposium's theme of rare disease progress, but they are not explicitly mentioned. NTRA Inter$-(NTRA) is a healthcare company. The article does not mention them specifically, and their focus areas may not directly align with the symposium's emphasis on rare diseases. OCGN Ocugen, Inc. is a biotechnology company focused on developing therapies for rare eye diseases. Their work directly aligns with the symposium's theme, suggesting potential interest, though not explicitly mentioned. ORGN Origin Enterprises PLC is an agricultural services company and is not relevant to the article's focus on rare disease drug development. PGEN Precigen, Inc. is a biopharmaceutical company developing therapies for cancer and autoimmune diseases. While their work is in the biotech space, the article does not specifically mention them or their relevance to rare diseases. PLRX Pliant Therapeutics is a clinical-stage biopharmaceutical company focused on rare diseases. Their work aligns with the symposium's theme, suggesting potential interest, though they are not explicitly mentioned. RARE Amicus Therapeutics is a biotechnology company focused on rare metabolic diseases. Their work aligns with the symposium's theme, suggesting potential interest, though they are not explicitly mentioned. RGNX REGENXBIO Inc. is a leading clinical-stage biotechnology company focused on gene therapy, a critical area for rare disease treatment. Their work aligns with the symposium's goals, though they are not explicitly mentioned. RXRX Recursion Pharmaceuticals uses AI to discover treatments for rare diseases. Their focus aligns perfectly with the symposium's theme, suggesting potential interest, although they are not explicitly mentioned. SAVA Cassava Sciences is a clinical-stage biotechnology company focused on neurodegenerative diseases. While some rare diseases fall into this category, they are not explicitly mentioned in the article. SGMO Sangamo Therapeutics is a gene therapy company focused on rare diseases and other serious conditions. Their work aligns with the symposium's theme, suggesting potential interest, though they are not explicitly mentioned. SNOW Snowflake Inc. is a cloud-based data warehousing company. While data is crucial for research, their direct involvement or mention in the context of rare disease drug development is not present in the article. SRPT Sarepta Therapeutics is a biotechnology company focused on developing genetic therapies for rare diseases. Their work aligns with the symposium's theme, suggesting potential interest, though they are not explicitly mentioned. STEM Stem, Inc. is an artificial intelligence company focused on energy storage. This is unrelated to the article's focus on rare disease drug development. TGTX TG Therapeutics is a biopharmaceutical company focused on cancer. While some rare cancers exist, the article's specific focus on rare diseases and the listed sponsors do not directly include TG Therapeutics. TLSA Tessera Technologies (now Xperi) is involved in semiconductor and intellectual property solutions. This is unrelated to the article's focus on rare disease drug development. TXG 10x Genomics provides tools for biological research, including genomics. Their technology is relevant to understanding rare diseases, but they are not explicitly mentioned in the article. VRTX Vertex Pharmaceuticals is a leading biotechnology company with a strong focus on rare diseases, particularly cystic fibrosis. While not explicitly mentioned as a sponsor, their work aligns closely with the symposium's theme. ZETA Zeta Global Holdings Corp. is a marketing technology company. This is unrelated to the article's focus on rare disease drug development.

RARE Drug Development Symposium Marks 10th Anniversary with Dedicated Pitch Sessions and The Theme and Call to Action to Align Advocates, Science and Industry BOSTON, July 28, 2026 /PRNewswire/ -- Global Genes, in partnership with The Rosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center (RSZ TNC) at Boston Children's Hospital and the Termeer Institute, is proud to announce the 10th Annual RARE Drug Development Symposium (RDDS) taking place September 9 – 11, 2026 in Boston. This landmark decennial event brings together the rare disease ecosystem to foster hands-on, collaborative learning designed to translate patient-lived experiences into research readiness and strategic therapeutic development.

This year's milestone symposium centers on the theme:

"Accelerating Rare Disease Progress: Aligning Advocates, Science & Industry"

To mark its decade of impact, the 2026 symposium is expanding its programming to include a dedicated day for Pitch Sessions. This new addition provides a critical platform for rare disease advocacy leaders to pitch their research portfolios, natural history data, and clinical trial readiness directly to biotech companies, clinical researchers, and venture philanthropists, de-risking pipelines and driving investment to underserved disease areas.

A Decade of Impact and Collaboration

For ten years, RDDS has served as a vital catalyst for rare disease advocates who are ready to lead, build vibrant ecosystems, partner with industry, and advance drug development pipelines.

"For a decade, the Rare Drug Development Symposia has brought together the patient community, academia and industry to advance advocate-partnered research, and this reflects the collaboration that underpins meaningful breakthroughs in rare disease," said Christophe Hotermans, SVP, Medical Affairs, Alexion, AstraZeneca Rare Disease, the Visionary Sponsor for this year's symposium. "We are committed to partnering with the rare disease community to advance research and enable innovation that reflects patient needs and real-world impact."

"Ten years ago, RDDS started as an experiment in bringing advocates and industry into the same room. Today it's a proven launchpad for patient-led and patient-partnered research," said Charlene Son Rigby, CEO of Global Genes. "Rare disease progress depends on advocates who know how to navigate research, build the right partnerships, and drive development forward. Ten years in, that's the commitment RDDS continues to deliver on."

Symposium Highlights & Interactive Programming

Building on a legacy where over 90% of past attendees reported significant increases in research knowledge and organizational confidence, the 2026 agenda is structured to deliver immediate, actionable value. Attendees will participate in:

This year's RDDS is made possible with the generous support of our sponsors: Alexion-AstraZeneca Rare Disease, BridgeBio, Travere Therapeutics, Astellas Gene Therapy, BioCryst, Catalyst Pharmaceuticals, Bial, Dyno Therapeutics, RTW Foundation, AlphaRose Therapeutics, Viralgen, and our Media Partner, RARE Revolution Magazine.

For more information regarding registration and the agenda for the 2026 RARE Drug Development Symposium, please visit the Global Genes website.

About Global Genes

Global Genes is a 501(c)(3) non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients, their families and disease communities globally. For nearly two decades, we've equipped rare disease patients and advocates with tools, training and support – to connect patients with needed resources, activate communities and advance research. Global Genes serves the more than 400 million people around the globe, and the nearly one in 10 Americans affected by rare diseases. With over 800 patient advocacy group members in our Global Advocacy Alliance, we work with patient advocates, industry partners and academia to build vital ecosystems to progress critical work in rare disease. Learn more at www.globalgenes.org.

SOURCE RARE-X