Regeneron, Intellia target neurological diseases in expanded gene editing deal
03 Oct 2023
AcquisitionGene TherapyLicense out/in
Regeneron Pharmaceuticals and Intellia Therapeutics are again expanding a longstanding research partnership, this time with a focus on creating genetic medicines for neurological and muscular diseases.
Announced Tuesday, the new agreement initially has the companies studying two therapeutic targets that are not in the liver — a popular area of interest for gene therapy developers, as the liver is where infused medicines tend to end up. Any resulting drugs will also be “in vivo,” meaning they’re designed to be directly inserted into patients and, through various methods, deliver helpful genetic material to certain cells.
The deal tasks Intellia, a biotechnology built around CRISPR gene editing technology, with leading the “editing methodology” that underpins these medicines. Meanwhile, Regeneron, famous for its work with antibody drugs, will be in charge of designing the “viral vectors” that act like a shipping service, shuttling genetic cargo to the appropriate cells.
Financial terms weren’t disclosed, but the companies said they each have the opportunity to spearhead the development and commercialization of any potential products related to one of the two targets. The partner not in the lead will still have the option to enter into a co-development and co-commercialization agreement for the target.
“To date, the widespread use of genetic medicines has generally been limited by the inability to deliver a genetic payload to cells of interest in the body beyond the liver,” said Aris Baras, co-head of Regeneron’s genetic medicines unit, in a statement.
Baras added that the expanded deal with Intellia is “taking advantage of new technology and innovations to unlock these opportunities.” Christos Kyratsous, the other co-head of Regeneron’s genetic medicines unit, noted in the same statement how the company has invented and, through non-human testing, validated a new antibody-directed approach to gene therapy delivery.
The approach specifically revolves around AAVs, or adeno-associated viruses, which are common tools used by gene therapy makers. And, according to Kyratsous, it takes advantage of new AAV engineering technologies as well as Regeneron’s experience developing antibodies.
Regeneron and Intellia first teamed up in 2016, just two years after the latter company was founded. Their initial agreement revolved around using Intellia’s technology to advance in vivo gene therapies. It also allowed Regeneron to share in expenses and potential profits tied to NTLA-2001, an experimental treatment for a rare disease called transthyretin amyloidosis.
In late 2020, NTLA-2001 became the first in vivo CRISPR medicine to be infused directly into a patient’s blood — a landmark moment for the field.
That same year, Regeneron and Intellia expanded their collaboration for the first time, agreeing to co-develop possible treatments for the rare bleeding disorders hemophilia A and Bhemophilia A and B. Per the new deal terms, Regeneron gave Intellia $70 million upfront in addition to making a $30 million equity investment in its partner.
Regeneron has since inked more deals directed at genetic medicines. In 2022, it partnered with Germany-based ViGeneron, hoping to develop a gene therapy for an inherited eye disease. And this year, the big biotech agreed to buy Decibel Therapeutics for $109 million, giving it ownership of a potential one-time treatment for a certain kind of hearing loss.
Regeneron also recently entered a collaboration with Sonoma Biotherapeutics. Together, the companies want to create so-called “Treg” cell therapies for a range of autoimmune diseases, including ulcerative colitis and Crohn’s disease.
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