Elpida Therapeutics Licenses Ionis ION283 to Advance Treatment for Lafora Disease
Elpida Therapeutics Acquires ION283 Program Rights to Ionis’ Investigational ASO Therapy for Lafora Disease, for Patients Living with this Devastating Ultra-Rare Disorder
Elpida Therapeutics, a nonprofit biotechnology company dedicated to developing treatments for ultra-rare pediatric neurological diseases, today announced that it has entered into an assignment and license agreement with Ionis Pharmaceuticals for ION283, an investigational antisense oligonucleotide (ASO) therapy for Lafora disease.
The agreement represents a major milestone for the global Lafora disease community and reflects Elpida Therapeutics’ commitment to advancing the clinical development of this investigational therapy with the goal of pursuing regulatory approval.
Lafora disease is a rare, progressive, and fatal neurodegenerative disorder that typically begins during adolescence, leading to severe epilepsy, cognitive decline, loss of independence, and premature death. Despite decades of research, there are currently no approved disease-modifying therapies.
Elpida plans to work with investigators, hospitals, regulators and patient advocacy organizations to advance this investigational medicine for patients living with this devastating ultra-rare disorder.
ION283 represents the kind of promising science that must not be left behind simply because a disease is too rare. Fundación Columbus is proud to work alongside Elpida and its international partners to advancing this therapy towards potential approval and Making it accessible to families affected by Lafora disease.”
— Javier García, Co-founder and Secretary, Fundación Columbus
Under Elpida’s leadership, the program will build upon the clinical foundation established to date while expanding development across additional international centers. The organization intends to collaborate with leading epilepsy and rare disease institutions throughout North America and Europe to accelerate patient access and generate the data needed to support future regulatory submissions.
“For families living with Lafora disease, every day matters. We are incredibly grateful to Ionis for their years of scientific innovation and for entrusting Elpida with the next chapter of this program. Our commitment is simple: move as quickly as possible, work collaboratively with the global clinical community, and do everything we can to bring this therapy to every patient.
— Terry Pirovolakis, Founder & Chief Executive Officer, Elpida Therapeutics
The addition of ION283 further expands Elpida’s growing pipeline of therapies targeting devastating ultra-rare neurological diseases. The organization is currently advancing CMT4J, CLN7 and SPG50 clinical-stage programs while pioneering a nonprofit biotechnology model designed to accelerate development, reduce costs, and aims to maximize patient access
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More information about the Lafora disease program and future clinical development plans will be shared as regulatory activities progress. Elpida also looks forward to working closely with Lafora patient organizations and the broader community to help inform and develop a thoughtful path forward for patients and families.
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About Elpida Therapeutics
Elpida Therapeutics is a nonprofit biotechnology company dedicated to developing transformative therapies for patients living with ultra-rare diseases. By leveraging scientific innovation, established therapeutic platforms, and strategic partnerships, Elpida is working to make life-changing treatments accessible to families who have historically been left without options.
The organization's unique, nonprofit development model focuses on advancing programs that traditional biotechnology companies are often unable to pursue due to limited commercial markets. By partnering with academic institutions, hospitals, foundations, and industry leaders, Elpida supports clinical trials while reducing costs and maximizes patient access.
Elpida's growing pipeline includes gene therapies and antisense oligonucleotide (ASO) programs targeting devastating pediatric neurological diseases, including SPG50, CLN7, CMT4J, and Lafora disease, with additional ultra-rare CNS programs expected to join its portfolio through collaborations with academic institutions and nonprofit organizations.
For more information, visit www.elpidatx.com.
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About Fundación Columbus
Fundación Columbus is a nonprofit organization founded in Spain in 2017 to accelerate the development of and access to advanced therapies for children with ultrarare diseases that lack viable commercial pathways. Through international collaborations with patient organizations, hospitals, researchers, biotechnology companies and institutions, the Foundation coordinates the clinical, regulatory, manufacturing and financial capabilities needed to turn scientific advances into real treatments. It also uses culture to connect science with society, raise awareness and mobilize philanthropic support—so that no child is left without treatment simply because their disease is too rare.
For more information, visit www.fundacioncolumbus.org​
Forward-Looking Statements
This press release contains forward-looking statements regarding the development, regulatory advancement, and potential commercialization of ION283 for Lafora disease. These statements are based on current expectations and are subject to risks and uncertainties, including regulatory decisions, clinical trial outcomes, manufacturing activities, financing, and other factors that could cause actual results to differ materially. Elpida Therapeutics undertakes no obligation to update these forward-looking statements except as required by applicable law"