Yet Another Collaboration of Editas’s CRISPR Technology for Inherited Eye Treatments


Editas Medicine collaborated with Adverum Biotechnologies to develop novel CRISPR-based therapies for up to five inherited eye diseases.

editas-Adverum-Biotechnologies collaborationThe companies did not describe about the retinal disease they were intended to treat with the therapies. Last month, Editas told about two eye disorders from their portfolio of ophthalmology for which they were intended to develop CRISPR-based medicines: Leber congenital amaurosis 10 (LCA10) – a rare eye disease that eventually leads to blindness and ocular herpes simplex virus.

According to the license agreement, Editas will pay a sum of $1 million upfront to Adverum for the clinical development and optimization of its vector technology for the development of CRISPR-based medicine.  Additionally, Editas will pay an option exercise fee of $1 million for each indication that it will choose under the deal. Adverum will also receive undisclosed amount of commercial and development milestone payments as well as royalties in future on products developed with the help of next generation AAV vectors.

Earlier this year in May, Editas presented study results of CRISPR technology based guide RNAs (gRNAs) which mediate editing and correction of CEP290 gene mutation in primary fibroblast cells in the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting.

CEP290 mutations is the primary cause of blindness in LCA10 patients. Editas designed gRNAs and paired them with Staphylococcus aureus Cas9 protein and delivered to the patient with the help of single AAV vector and corrected the CEP290 mutation in primary patient fibroblast cells.

Editas till date have entered into several research collaborations and this year alone has more than three collaborations under its wing. Few days back, Editas collaborated with Massachusetts General Hospital to access their intellectual property and technology related to high fidelity CRISPR technology variants CAS9 nuclease and CAS PAM.

We are pleased to bring together our gene therapy capabilities with Editas’ CRISPR based approach to genome editing,” said Paul Cleveland, chief executive officer of Adverum Biotechnologies. “Our innovative vectors have the potential to deliver Editas’ genome editing components efficiently to the retina. This collaboration expands our opportunities to capitalize on our science, ophthalmology expertise and vector development know-how.”

The latest collaboration of Editas Medicine will broaden the research horizon and will support the company to evaluate genome editing technology for retinal diseases with the help of gene therapy vectors.

Featured Image Credit: Pixabay

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