Genzyme Corp., a wholly owned subsidiary of Sanofi, has filed a patent infringement lawsuit against Sarepta Therapeutics in the District Court for the District of Delaware, alleging that the biotech's Duchenne muscular dystrophy gene therapy Elevidys violates two key patents related to adeno-associated virus (AAV) vector technology.
The lawsuit, filed on July 26, 2024, claims that Sarepta's manufacture and sale of Elevidys (delandistrogene moxeparvovec-rokl) infringes U.S. Patent Nos. 9,051,542 and 7,704,721, both of which are set to expire on June 1, 2025.
Patent Technology at Issue
According to the complaint, the disputed patents address a critical manufacturing challenge in gene therapy production. Genzyme alleges that the manufacture of recombinant AAV (rAAV) vectors was historically plagued by a "major concern during production... that the rAAV vector particles will become insoluble and aggregate into clusters of viral particles, which can result in production difficulties and loss of vector functionality."
Genzyme's inventors allegedly solved this aggregation problem by discovering "that the use of certain high ionic strength solutions for preparing and storing rAAV vectors can prevent significant aggregation of virus particles at the concentrations needed for effective gene therapy."
The '542 patent claims cover compositions "for the storage of purified, recombinant adeno-associated virus (AAV) vector particles... wherein the purified AAV vector particles are stored in the composition without significant aggregation." The '721 patent claims are directed to "a method of preventing aggregation of recombinant adeno-associated virus (rAAV) virions in a purified preparation of rAAV virions."
Infringement Allegations
Genzyme alleges that Sarepta directly infringes the '542 patent by manufacturing, selling, offering for sale, and using the patented compositions in Elevidys. The lawsuit also claims that Sarepta infringes "by contracting with Catalent to manufacture Elevidys under the direction and under the control of Sarepta, and/or by forming a joint enterprise with manufacturers including Catalent for the manufacture of Elevidys." Notably, Catalent was not named as a defendant in the suit.
For the '721 patent, Genzyme alleges that Sarepta directly infringes by using the claimed methods in the manufacture of Elevidys and through its manufacturing arrangements with Catalent. The lawsuit further alleges indirect infringement through active inducement and contributory infringement.
Legal Remedies Sought
Genzyme is requesting that the Court enter judgments confirming patent infringement and finding that such infringement is "deliberate and willful." The company seeks compensatory damages, including potential treble damages under 35 U.S.C. § 284, and asks the court to find the case "exceptional" to award reasonable attorneys' fees, costs, and expenses under 35 U.S.C. § 285.
Elevidys Background
Elevidys addresses the underlying cause of Duchenne muscular dystrophy by delivering a functioning copy of the dystrophin gene, helping restore function and strength to muscles affected by the disease. The FDA granted Elevidys accelerated approval in June 2023 for ambulatory patients four to five years of age. In June 2024, the regulator awarded full approval and broadened the target population to include patients who are at least four years old, regardless of ambulation status.
Previous Patent Litigation
This lawsuit follows a separate patent dispute that Sarepta successfully defended. In January 2024, a Delaware judge ruled in favor of Sarepta in a patent challenge brought by Regenxbio and the University of Pennsylvania, with U.S. District Judge Richard Andrews dismissing the challenge after finding no evidence that Elevidys violated the plaintiffs' protected technologies.
The current case (1-24-cv-00882) has been assigned to Judge Andrews and represents another significant intellectual property battle in the competitive gene therapy landscape.
