Editas partners to bring CRISPR to gene therapy in inherited retinal diseases


CRISPR gene editing player Editas Medicine ($EDIT) has done a deal with gene therapy small cap Adverum Biotechnologies ($ADVM) to introduce genome editing into diseased retina via gene therapy vectors. The deal is for up to 5 inherited retinal diseases that have poor existing treatments.

Ophthalmology is already core for Editas; its lead program is in Leber congenital amaurosis 10 (LCA10), a rare form of blindness. It presented data earlier this year on mediated editing of primary fibroblasts from LCA10 patients that eliminated the target CEP290 gene mutation and restored full-length protein expression with no detected off-target effects.

“Adverum brings a distinctive technology and experience base, and this collaboration aligns highly with our broader, multi-faceted delivery strategy,” said Editas President and CEO Katrine Bosley in a statement.

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The deal brings together Menlo Park, CA-based Adverum’s next-gen adeno-associated virus gene therapy vectors to be used in combination with Editas’ CRISPR-based genome editing approach.

In the deal, the Cambridge, MA-based company will pay Adverum $1 million up front to evaluate its vectors for use in clinical development. It will also pay for all preclinical activity, partially via the upfront payment. Editas will pay another $1 million option exercise fee for each indication it chooses to pursue under the partnership. Adverum is eligible for other undisclosed development and commercial milestone payments and royalties.

“Our innovative vectors have the potential to deliver Editas’ genome editing components efficiently to the retina,” said Adverum CEO Paul Cleveland. “This collaboration expands our opportunities to capitalize on our science, ophthalmology expertise and vector development know-how.”

In addition to LCA10, Editas also has programs in Duchenne muscular dystrophy and in hematopoietic stem/progenitor cells.

This deal with Adverum could help to further diversify Editas’ means for introducing and perpetuating its genome editing to treat genetic diseases; this can be done via various viral, or even nonviral, means that depend on the cell type.

- here is the release

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