Regenxbio Inc. v. Sarepta Therapeutics, Inc.

CourtCourt of Appeals for the Federal Circuit
DecidedFebruary 20, 2026
Docket24-1408
StatusPublished

This text of Regenxbio Inc. v. Sarepta Therapeutics, Inc. (Regenxbio Inc. v. Sarepta Therapeutics, Inc.) is published on Counsel Stack Legal Research, covering Court of Appeals for the Federal Circuit primary law. Counsel Stack provides free access to over 12 million legal documents including statutes, case law, regulations, and constitutions.

Bluebook
Regenxbio Inc. v. Sarepta Therapeutics, Inc., (Fed. Cir. 2026).

Opinion

Case: 24-1408 Document: 77 Page: 1 Filed: 02/20/2026

United States Court of Appeals for the Federal Circuit ______________________

REGENXBIO INC., TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA, Plaintiffs-Appellants

v.

SAREPTA THERAPEUTICS, INC., SAREPTA THERAPEUTICS THREE, LLC, Defendants-Appellees ______________________

2024-1408 ______________________

Appeal from the United States District Court for the District of Delaware in No. 1:20-cv-01226-RGA, Judge Richard G. Andrews. ______________________

Decided: February 20, 2026 ______________________

SUSAN E. MORRISON, Fish & Richardson PC, Wilming- ton, DE, argued for all plaintiffs-appellants. Plaintiff-ap- pellant REGENXBIO Inc. also represented by DEANNA JEAN REICHEL, Minneapolis, MN.

JULIE S. GOLDEMBERG, Morgan, Lewis & Bockius LLP, Philadelphia, PA, for plaintiff-appellant Trustees of the University of Pennsylvania. Also represented by AMY M. DUDASH, Wilmington, DE. Case: 24-1408 Document: 77 Page: 2 Filed: 02/20/2026

ROBERT B. WILSON, Quinn Emanuel Urquhart & Sulli- van, LLP, New York, NY, argued for defendants-appellees. Also represented by JAMES BAKER, ANASTASIA M. FERNANDS. ______________________

Before DYK, HUGHES, and STOLL, Circuit Judges. STOLL, Circuit Judge. REGENXBIO Inc. and The Trustees of the University of Pennsylvania (collectively, “REGENXBIO”) filed a pa- tent infringement suit in the United States District Court for the District of Delaware against Sarepta Therapeutics, Inc. and Sarepta Therapeutics Three, LLC for infringing claims 1–9, 12, 15, and 18–25 of U.S. Patent No. 10,526,617. Both parties moved for summary judg- ment of patent eligibility under 35 U.S.C. § 101. The dis- trict court granted Sarepta’s motion and held the claims ineligible under § 101 as directed to a natural phenome- non. Because we hold the claims are not directed to a nat- ural phenomenon, we reverse the district court’s decision and remand the case for further proceedings. BACKGROUND I Genetic disorders—like cystic fibrosis, hemophilia, and sickle cell anemia—are caused by mutations or deletions in the sequences of nucleotides in one’s DNA. Gene therapy allows the use of modified virus “vectors” to deliver a new therapeutic gene (a “transgene”) that replaces the defective or missing gene, treating or possibly even curing the dis- ease by addressing the underlying genetic disorder. Host cells can be engineered to contain plasmids, where a plas- mid is a circular piece of DNA that is separate from the chromosomes of the host cell, and the plasmid has the de- sired transgene within it. A host cell can make multiple copies of the plasmid and also proliferate to make more Case: 24-1408 Document: 77 Page: 3 Filed: 02/20/2026

REGENXBIO INC. v. SAREPTA THERAPEUTICS, INC. 3

host cells. Plasmids can contain a capsid sequence, where a capsid is the outer shell of a vector. These plasmids can be purified, collected, and introduced into a mammalian host cell, along with other plasmid DNA, to generate a gene therapy vector. The ’617 patent is titled “Method of Detecting and/or Identifying Adeno-Associated Virus (AAV) Sequences and Isolating Novel Sequences Identified Thereby,” and is di- rected to genetically engineered host cells that contain adeno-associated virus rh.10 sequences. U.S. Patent No. 10,526,617 Title. The Background of the Invention ex- plains that AAVs are nonenveloped viruses with single- stranded DNA. It further explains that “AAV’s life cycle includes a latent phase at which AAV genomes, after infec- tion, are site specifically integrated into host chromosomes and an infectious phase in which, following either adenovi- rus or herpes simplex virus infection, the integrated ge- nomes are subsequently rescued, replicated, and packaged into infectious viruses.” Id. at col. 1 ll. 31–36. These “prop- erties of non-pathogenicity, broad host range infectiv- ity, . . . and potential site-specific chromosomal integration make AAV an attractive tool for gene transfer.” Id. at col. 1 ll. 36–40. The Background of the Invention recognizes that “[w]hat are desirable are AAV-based constructs for gene delivery.” Id. at col. 1 ll. 52–53. The inventors of the ’617 patent sought to develop such constructs and specifically developed “molecules which uti- lize the novel AAV sequences of the invention, including fragments thereof, for production of molecules useful in de- livery of a [transgene comprising a] heterologous gene or other nucleic acid sequences to a target cell.” Id. at col. 17 ll. 15–19. In pursuit of this, the inventors created host cells that “contain sequences encoding a novel AAV capsid”— and a heterologous non-AAV sequence. Id. at col. 17 ll. 36– 37; see also id. at col. 17 ll. 42–45, col. 18 ll. 19–26. Case: 24-1408 Document: 77 Page: 4 Filed: 02/20/2026

Claim 1 is representative for the purposes of this ap- peal: 1. A cultured host cell containing a recombinant nucleic acid molecule encoding an AAV vp1 capsid protein having a se- quence comprising amino acids 1 to 738 of SEQ ID NO: 81 (AAVrh.10) or a sequence at least 95% iden- tical to the full length of amino acids 1 to 738 of SEQ ID NO: 81, wherein the recombinant nucleic acid molecule further comprises a heterologous non-AAV sequence. Id. at col. 437 ll. 55–63. The cultured host cells required by the claims are un- disputedly human made. They do not exist in nature. No- tably, a recombinant nucleic acid molecule is created by chemically splicing together nucleic acid sequences from two different organisms. Appellants’ Br. 5; see also Parker Br. 11 (defining recombinant nucleic acids as “combining genetic material from two different sources,” which are used “to create new, human-made sequences” (citation omitted)). 1 And, as noted above, the claim term “heterolo- gous” means coming from a different species.

1 Amicus brief filed by the Parker Institute for Can- cer Immunotherapy, The J. David Gladstone Institutes, and the Dana-Farber Cancer Institute. See ECF No. 27. The amici further explain that creating recombinant DNA is a multi-step process: (1) fragments of DNA are created, for example, by using restriction enzymes chosen to cleave DNA at specific sites in order to get the desired fragments; (2) the desired DNA fragments are joined by DNA ligases; (3) the recombinant nucleic acid sequence with the desired DNA fragments is ligated into a plasmid that is used to Case: 24-1408 Document: 77 Page: 5 Filed: 02/20/2026

REGENXBIO INC. v. SAREPTA THERAPEUTICS, INC. 5

II REGENXBIO accused Sarepta of infringing claims 1– 9, 12, 15, and 18–25 of the ’617 patent based on Sarepta’s use of the AAV variant rh.74 in cultured host cells to make a gene therapy product referred to as SRP-9001, which treats Duchenne muscular dystrophy. See REGENXBIO Inc. v. Sarepta Therapeutics, Inc., No. 20-cv-1226-RGA, 2024 WL 68278, at *1 (D. Del. Jan. 5, 2024). Both parties moved for summary judgment on whether the asserted claims were eligible under 35 U.S.C. § 101. The district court noted that there were no underlying factual disputes pertaining to eligibility in this case and the parties agreed. REGENXBIO, 2024 WL 68278, at *3. The parties debated whether the claims disclose natu- ral products and both analogized this case to Association for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013), in which the Supreme Court considered the el- igibility of composition claims under the markedly differ- ent characteristics test from Diamond v. Chakrabarty, 447 U.S. 303 (1980). Neither party asserted that the claims are ineligible as an abstract idea or on any other grounds.

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