Elpida Therapeutics released a press release about acquiring ION283 and advancing this potential therapy. We are fortunate that Terry shared a letter with us earlier in the week.
For more information about the nonprofit biotech company and the license transfer from Ionis, you can read the press release that Elpida Therapeutics sent out here.
Please direct any questions about ION283, Elpida Therapeutics, or the Safety Study to ion283@chelseashope.org.
Exciting news for the Lafora community!
Ionis reached an agreement for the future of the ION283 asset with Elpida Therapeutics. Terry Pirovolakis, CEO of Elpida Therapeutics, asked us to share this letter with the Lafora community to introduce their company and their plans for ION283. You can read the letter here:
Chelsea’s Hope is committed to advocating for the Lafora community and helping accelerate the development of this potential treatment. Please direct any questions about ION283, Elpida Therapeutics, or the Safety Study to ion283@chelseashope.org, and we will get back to you.
As a reminder, Chelsea’s Hope will host a Lafora Therapy Q&A on August 11 at 1:30 PM EDT. This session will go over new resources we’ve recently shared about the Lafora therapeutic pipeline. Then, we will answer your questions submitted in advance of the meeting, ending with some time for questions and responses. We will use TransPerfect AI translation for captions. Please register in advance here.
Alessandra Camerini
Head of Relations with Patients and Patient Organisations
Dear Members of the International Lafora Patient Community,
Fondazione Telethon remains open to contributing to the development of ION283 and to collaborating with partners in Europe and in the United States, provided that the highest ethical and scientific standards are upheld and that any decision is taken exclusively in the best interest of patients.
Fondazione Telethon acknowledges the complexity of the ION283 case, also from the perspective of Ionis, which currently holds the license for the compound.
The essential authorizations required to set up a clinical study in Italy fall within Ionis’ decision-making space. Therefore, at this stage, Fondazione Telethon cannot serve as the reference point or source of information regarding a possible future development path for ION283. The decision concerning the continuation, redesign, or activation of a clinical program is not under our direct control. Should concrete and reliable updates become available, we will share them promptly.
We are aware that several research groups, in different Countries, are actively conducting studies on Lafora disease: this reflects the commitment of the international scientific community to advancing knowledge and identifying potential therapeutic strategies.
While dealing with the hard work to ensure progresses in identifying therapeutic solutions, we respectfully invite families, associations, and researchers to bear in mind that the scientific pathway required to transform science in therapies does not allow for shortcuts. The timelines necessary to ensure safety, efficacy and compliance with regulatory standards cannot be compressed beyond what scientific rigor and ethical standards allow.
We are aware that this message may sound demanding. However, clarity is necessary to preserve families who are already facing extraordinary challenges and a sense of unpostponable urgency every day.
Our commitment remains firm: to pursue research with responsibility, scientific soundness, transparency, and deep respect for patients and their loved ones.
The mission of Chelsea’s Hope is to improve the lives of those affected by Lafora disease and help accelerate the development of treatments.
Chelsea’s Hope Lafora Children Research Fund is an IRS 501(c)3 nonprofit organization. EIN: 27-1008382
Chelsea’s Hope c/o Dr. Donohue
976 Maywick Dr.
Lexington, KY 40504
Use ICD-10 code G40.C for Lafora.
