Sangamo Therapeutics has lofty aims for its gene therapy programs — but at least one Big Pharma company thinks they’re achievable.
On Wednesday, the Richmond, California-based company announced a global collaboration and licensing agreement with Pfizer. The deal centers around Sangamo’s lead gene therapy candidate SB-525, which it hopes to eventually offer as a one-time treatment for hemophilia A.
In return for global commercialization rights, Pfizer will pay $70 million upfront and up to $475 million in milestone payments that encourage both the commercialization of SB-525 and the development of new hemophilia A therapies.
With fresh cash in hand and an orphan drug designation, Sangamo expects to move SB-525 into the clinic later this quarter.
Hemophilia A is an inherited disorder that arises from a deficiency in factor VIII, an important clotting factor. According to the National Hemophilia Foundation, it’s around four times as common as hemophilia B. Combined, the disorders affect around 20,000 Americans and thousands more overseas.
With the well-characterized deficiency in specific clotting factors, both forms of hemophilia are considered good candidates for gene therapies.
Sangamo’s approach seeks to correct the defective genes in vivo. In the case of SB-525, a recombinant adeno-associated virus (rAAV) vector is used to carry a functional copy of the factor VIII gene into the patient’s liver cells. From there, the gene fragments could theoretically increase blood levels of the deficient clotting factor. By just how much remains to be seen, but the hope is that the treatment would be long term.
There will no doubt be many complex problems to solve along the way. And even after commercialization, success is not guaranteed. The world’s first gene therapy, UniQure’s Glybera, was officially withdrawn from the European market last month due to a lack of demand. That’s not surprising given the therapy’s $1 million price tag.
Pfizer is undeterred.
Back in 2014, the New York City-based pharma giant inked a similar deal with another orphan disease/gene therapy specialist, Spark Therapeutics. The object of that affair, SPK-9001, is currently being evaluated in a Phase 1/2 trial in hemophilia B. The program appears to be advancing, with Pfizer paying a further $15 million in January as part of a $230 million milestones package.
Mikael Dolsten, president of worldwide research and development at Pfizer, confirmed the company’s long-term emphasis on gene therapies.
“Pfizer has made significant investments in gene therapy over the last few years and we are building an industry-leading expertise in recombinant adeno-associated virus (rAAV) vector design and manufacturing,” Dolsten said in a prepared statement announcing the new Sangamo deal.
Sangamo shares shot up close to 45 percent in after-hours trading following the news.
Photo: virusowy, Getty Images
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