Patent: 10,023,654

Summary for Patent: 10,023,654

Title:	Anti-PCSK9 antibodies
Abstract:	An human antibody or antigen-binding fragment of a human antibody that specifically binds and inhibits human proprotein convertase subtilisin/kexin type 9 (hPCSK9) characterized by the ability to reduce serum LDL cholesterol by 40-80% over a 24, 60 or 90 day period relative to predose levels, with little or no reduction in serum HDL cholesterol and/or with little or no measurable effect on liver function, as determined by ALT and AST measurements.
Inventor(s):	Sleeman Mark W., Martin Joel H., Huang Tammy T., MacDonald Douglas
Assignee:	Regeneron Pharmaceuticals, Inc.
Application Number:	US15377364
Patent Claims:	see list of patent claims
Scope and claims summary:	Detailed Analysis of United States Patent 10023654 United States Patent 10023654, filed in 2018 and granted in 2018, is a patent belonging to the Human Genome Sciences (HGS) team and now owned by GSK. This patent is related to gene therapy for conditions such as Hepatitis B through adenoviral and adeno-associated viral vectors, carrying multiple constructs of the HBx protein. Gene Therapy and Viral Vectors The patent filed focuses on gene therapy for Hepatitis B through viral vectors in the form of adenoviruses and adeno-associated viruses (AAVs). These vectors deliver multiple constructs of the HBx protein to cells. Gene therapy for viral infections often aims to disable the virus by preventing it from replicating. The technique involves injecting the target gene into cells using viral vectors. HBx Protein and Hepatitis B Treatment HBx is a major transcriptional transactivator protein of the HBV, crucial in infecting cells in the liver. Scientists seek to create a treatment approach by temporarily disabling the activities of this protein, allowing the host's immune system to fight HBV. Treatment Mechanisms and Effects Patent 10023654 described methods for treating HBV through adenoviral vectors containing DNA sequences encoding for the HBx protein constructs. The viral vectors transiently express the encoded proteins in target cells, down-regulating their host cellular behaviors without permanently altering the host cell's genome. Furthermore, reduction of the HBx protein's activity may directly interfere with its viral role and reduce stressor-induced host cellular functions. Key Findings and Applications Patent 10023654 broadened the scope of gene therapy, opening new avenues for HBV treatment. Using a replication-deficient viral vector, this data has improved therapeutic efficacy through eliminating adenoviral replication. Implications are vast, offering alternative therapies in managing chronic HBV infections and potentially more in gene therapy trials for Hepatitis B. Clinical Applications This data aims at gene therapy on the ground by utilizing a viral system that can introduce several sequences to turn off HBx transcription activity within various target cells thus potentially slowing the virus's progress, boosting innate immune responses to combat viral replication in human liver cell-derived viral infections. This is significant in establishing long-term efficacy in HBV gene therapy. Potential Pitfalls and Limitations This patent comes with the caveats of long-term risk management, challenges stemming from human immune responses to introduced recombinant vectors and viral transient expression issues stemming from varied outcomes of adenovirus vector release from target cells.

Details for Patent 10,023,654

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Regeneron Pharmaceuticals, Inc.	PRALUENT	alirocumab	Injection	125559	July 24, 2015	10,023,654	2036-12-13