CRISPR Therapeutics takes a swing at ALS gene editing in discovery deal with AAV upstart Capsida
When small biotech Capsida launched just a few months ago, it hit the scene with backing from drug giant AbbVie to chase AAV-delivered drugs for CNS. Now, the team has earned the support of one of the biggest names in gene editing — and it’s taking on a big challenge right away.
CRISPR Therapeutics has signed a deal with Capsida Biotherapeutics, a gene therapy player specializing in AAV engineering, to carve out a delivery mechanism for the company’s gene editing tech in ALS and rare neurodegenerative disorder Friedreich’s ataxia, the companies said Tuesday.
As part of their pact, Capsida will pick up R&D responsibilities for the nascent ALS program as well as chip away at capsid design for both programs. CRISPR, meanwhile will manage R&D for the FA program and develop gene editing candidates for both, the companies said.
In a release, CRISPR touted Capsida’s work in creating “high-throughput” adeno-associated viral vectors optimized to tissue target with limited off-target side effects. Both companies will hold options to co-development and -commercialization rights for the program which the partner company leads, CRISPR said.
The financial terms of the deal were not disclosed.
If one of the partners does opt-in, the two firms would share R&D and commercialization costs and profits on that given program. Capsida, with its experience in AAV production, will handle clinical and commercial manufacturing, if it reaches that point.
“The combination of Capsida’s AAV engineering platform and CRISPR Therapeutics’ gene-editing platform has the potential to enable transformative gene-edited therapies for patients with neurological diseases,” CRISPR CEO Samarth Kulkarni said in a statement. “This new partnership is one more step in our overall strategy of bringing together innovative and complementary technologies to unlock the full potential of our core platform.”
Kickstarted on work by CRISPR/Cas9 innovator Emmanuelle Charpentier back in 2014, CRISPR is perhaps best known for its sickle cell and beta thalassemia programs with Vertex, which recently read out winning data. Late last week, the partners read out data from two studies showing patients dosed with experimental drug CTX001 indicated a “consistent and sustained” response for at least three months after dosing. All 15 patients with transfusion-dependent beta thalassemia did not need further blood transfusions and all seven with severe sickle cell disease were pain free, the biotechs announced.
There was one patient not included in the data cutoff of March 30, however, who experienced a cerebellar hemorrhage less than three months after being treated. The serious side effect was related to the busulfan conditioning gene therapy patients undergo before receiving treatment, Vertex said, and has since resolved.
Capsida, meanwhile, only launched in April with a sizable $50 million check and $90 million in going-out money from AbbVie for its own discovery pact. The biotech is aiming initially at broad rare and neuro diseases, and AbbVie came on board with the goal of identifying three CNS targets.