🧬 LCA16 causes severe vision loss in children due to KCNJ13 gene mutations.
🚀 Hubble plans Phase I/II trials for HUB-101, aiming to show safety and effectiveness in adults.
🤝 Both companies express commitment to advancing gene therapy for affected families.
Introduction:
Hubble Therapeutics has appointed Andelyn Biosciences as its partner for the production of clinical-grade adeno-associated virus (AAV) tailored for the gene therapy of Leber Congenital Amaurosis 16 (LCA16), a hereditary retinal disorder. This collaboration aims to harness Andelyn’s technological expertise to facilitate the development of therapeutic solutions for affected patients.
- Hubble Therapeutics has selected Andelyn Biosciences to produce AAV using the AAV Curator™ platform for LCA16 treatment.
- LCA16 is caused by mutations in the KCNJ13 gene, leading to severe visual impairment and eventual blindness in patients.
- The FDA has granted HUB-101, the candidate gene therapy, both Rare Pediatric Disease and Orphan Disease Designations.
- Andelyn is tasked with optimizing the production process for HUB-101 and generating GMP-grade materials for clinical trials.
- Phase I/II clinical trials are planned to evaluate the safety and efficacy of the treatment in adult populations.
Conclusion:
The partnership between Hubble Therapeutics and Andelyn Biosciences represents a significant step towards addressing the unmet needs of patients with LCA16. By leveraging Andelyn’s capabilities in gene therapy production, the initiative seeks to create a reliable pathway for the clinical application of HUB-101, highlighting the ongoing commitment to develop effective treatments for rare genetic disorders.
