Sarepta is best known for its RNA technology platform, which has led to two approved — though also controversial — drugs for Duchenne muscular dystrophy.
Yet, Sarepta is also deeply invested in gene therapy, having developed an extensive list of more than two dozen experimental treatments, six of which have reached human testing.
Rare diseases have been an early target in this rapidly growing field. The two gene therapies approved in the U.S., Roche’s Luxturna and Novartis’ Zolgensma, are respectively used to treat an uncommon form of blindness and a muscle disease that occurs in about 1 in every 10,000 births.
Sarepta’s therapies target a wide variety of rare diseases, including Duchenne muscular dystrophy, Pompe disease, and types of Limb-girdle muscular dystrophy. Taking a stake in AavantiBio, with its work in Friedreich’s ataxia, could expand Sarepta’s reach even further. The disease affects approximately 1 in every 40,000 people, according to the National Organization for Rare Disorders, which would equate to around 8,200 patients in the U.S.
“Our equity participation in AavantiBio serves our strategy to build our gene therapy engine through targeted investment in potentially life-enhancing therapies,” Doug Ingram, Sarepta’s CEO, said in a statement Thursday.
AavantiBio joins a couple large, powerful companies in the hunt for a gene therapy to treat Friedreich’s ataxia. Pfizer and Novartis are each working on their own programs.
Outside of gene therapy, Reata Pharmaceuticals disclosed last year positive data from a study that tested an oral drug, known as omaveloxolone, in patients with Friedreich’s ataxia. Reata said it intends to file the drug for approval based on those results.
AavantiBio will be headquartered in the greater Boston area, putting it close by Cambridge, Massachusetts-based Sarepta. Cumbo, along with his role as CEO, will take one of AavantiBio’s eight board of directors seats. Cumbo has, during the span of his career, helped launch 11 specialty products across multiple drug companies, including Sarepta, Gilead and Vertex, where he built a sales team for the hepatitis C drug Incivek.
Co-founders Byrne and Corti will be on the board too, alongside two independent directors and representatives from the investor group of Perceptive, Bain and RA. Louise Rodino-Klapac, senior vice president of gene therapy for Sarepta, will serve as a board observer.
