Call for collaborations from academia and industry
This content is not under ERN-EYE responsibility*
Academic and ERN-EYE announcements
Call for Patient Referral: Hyperion Clinical Trial
Contact: Katarina Stingl
Institution: University of Tuebingen
Description: An international Phase 3 clinical trial is currently recruiting patients with LCA10 caused by the CEP290 c.2991+1655A>G mutation. The study is evaluating an antisense oligonucleotide therapy with previously demonstrated efficacy.
Key eligibility criteria include:
– Age ≥6 years
– Confirmed CEP290 c.2991+1655A>G mutation
– Residual visual function with relatively symmetric eyes
Due to the rarity of eligible cases, patient referrals are strongly encouraged.
https://clinicaltrials.gov/study/NCT06891443
Collaboration Request: If you are aware of any potential patients, please contact me or one of the participating sites.
Email: katarina.stingl@med.uni-tuebingen.de
RNA-based therapy for nonsense mutation with both structural and functional outcomes for ocular indications in blindness caused by ion channel disorder
Contact: Bikash R. Pattnaik
Institution: University of Wisconsin-Madison
We are developing an RNA-based therapy for nonsense mutation with both structural and functional outcomes for ocular indications in blindness caused by ion channel disorder. The two primary cell types targeted are Photoreceptors and RPE cells. Identified patients with nonsense mutations in any of the ion channel genes, such as KCNJ13, CACNA1F, KCNB1, KCNV, and BEST1, would be invaluable in assessing the feasibility of such therapeutic strategies. We would appreciate the opportunity to discuss any potential collaborations to obtain de-identified natural history and curated data. We are also happy to discuss this with patient groups to educate them.
Email: pattnaik@wisc.edu
Industrial announcements
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Disclaimer
*This page contains announcements on initiatives and activities related to rare eye diseases but not part of the ERN-EYE roadmap. The information shown on this page has been provided by ERN-EYE members or organisations with no professional relationship with ERN-EYE.
ERN-EYE is not responsible or liable in any manner for any content posted on this page. Although all announcement have to be approved for posting by the ERN-EYE Coordinating Committee prior to posting, ERN-EYE declines any responsibility or liability for the accuracy, content, completeness of the information contained.