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.

Whitepaper Download

Reducing the Complexity and Costs of Channel Planning and Logistics

How can you make the process of bringing your product to market less complex while also reducing costs? This Whitepaper identifies opportunities to simplify channel strategies for biopharma companies, their customers and patients. Discover how you can deliver savings and innovation to your business.

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

Bristol Myers Squibb may have bounced Jounce from its roster of inherited partners, but it’s hanging onto Anokion, a Swiss autoimmune-focused biotech.

The immunoassay will measure neurofilament light chain protein levels, found in the blood and cerebrospinal fluid, and linked to nerve damage.

The priority review action date sets Bristol Myers up to win approval for the bluebird-partnered anti-BCMA CAR-T therapy in late March.