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

DNA

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.

FREE DAILY NEWSLETTER

Like this story? Subscribe to FierceBiotech!

Biopharma is a fast-growing world where big ideas come along every day. Our subscribers rely on FierceBiotech as their must-read source for the latest news, analysis and data in the world of biotech and pharma R&D. Sign up today to get biotech news and updates delivered to your inbox and read on the go.

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

Related Articles:
Editas bags CRISPR tech from MGH to add therapeutic breadth to pipeline
Groundbreaking gene editing player Caribou raises $30M B round
Editas signs genome-edited stem cell pact with GSK, Biogen biotech partner
Editas tests CRISPR/Cas9 enthusiasm--and a turbulent market--with $122M IPO
CAR-T powerhouse Juno allies with gene-editing upstart Editas in $737M deal
Gates backs a $120M breakout round for CRISPR/Cas9 pioneers at Editas

Suggested Articles

The Medicines Company announced long-term data for inclisiran, showing that the drug lowered “bad” LDL cholesterol by more than 50%.

On the heels of a $120 million verdict issued last month, J&J was ordered by a Philadelphia jury to pay $80 million to a woman whose mesh eroded.

Computing-enabled R&D shop Schrödinger will use its $110 million haul to advance its nascent pipeline of wholly owned drugs.