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Analysis

1 / 477
1.WO/2025/259931COMPOSITIONS AND METHODS FOR RNA CIRCULARIZATION
WO - 18.12.2025
Int.Class C12N 15/63
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
 Appl.No PCT/US2025/033446 Applicant ORBITAL THERAPEUTICS, INC. Inventor VENKATARAMANAN, Srivats
The present invention provides nucleic acids and methods for making circular RNAs (circRNAs), and circular RNAs, compositions and methods of use thereof. The present invention uses selected self-splicing intron sequences (e.g., intronic sequences derived from self-splicing Group I or Group II introns) to produce circular RNAs. These self-splicing intron sequences described herein mediate efficient circularization of a linear RNA sequence.
2.WO/2025/259503BIOMARKERS FOR FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
WO - 18.12.2025
Int.Class G01N 33/68
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48Biological material, e.g. blood, urine; Haemocytometers
50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
68involving proteins, peptides or amino acids
 Appl.No PCT/US2025/032355 Applicant DYNE THERAPEUTICS, INC. Inventor WEEDEN, Timothy
The present disclosure, in some aspects, provides one or more biomarkers (e.g., plasma biomarkers or circulating biomarkers) for Facioscapulohumeral muscular dystrophy (FSHD). In some embodiments, a biomarker (e.g., plasma biomarkers or circulating biomarkers) described herein is regulated by DUX4. Methods (e.g., non-invasive methods) of using the biomarkers, e.g., for detection (e.g., early detection) and monitoring patient as well as treatment efficacy are also provided. The present disclosure, in other aspects, provides one or more biomarkers (e.g., transcriptome biomarkers) for Facioscapulohumeral muscular dystrophy (FSHD). In some embodiments, a biomarker (e.g., transcriptome biomarkers) described herein is regulated by DUX4. Methods of using the biomarkers, e.g., for detection (e.g., early detection) and monitoring patient as well as treatment efficacy are also provided.
3.WO/2025/257329FUSION POLYPEPTIDE AND LIPONANOPARTICLE COMPRISING THE SAME
WO - 18.12.2025
Int.Class A61K 38/17
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
 Appl.No PCT/EP2025/066426 Applicant STRIKE PHARMA AB Inventor MANGSBO, Sara
The present disclosure relates to fusion polypeptide comprising a tag moiety and a polypeptide comprising an apolipoprotein or a fragment thereof, for example for delivery of a loaded decorated liponanoparticle attached via the apolipoprotein or a fragment thereof to a cell of interest, wherein said loaded decorated liponanopaticle comprises a payload. Also disclosed are loaded decorated liponanoparticles comprising said fusion polypeptide and a complex comprising a bispecific binding molecule and the loaded decorated liponanoparticle. Medical uses of the fusion polypeptide, loaded decorated liponanoparticle and complex as disclosed herein are also provided.
4.20250375390Method of Lyophilisation
US - 11.12.2025
Int.Class A61K 9/51
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules
51Nanocapsules
 Appl.No 19298524 Applicant GlaxoSmithKline Biologicals SA Inventor Frédéric MATHOT

The present invention provides lyophilised pharmaceutical compositions and methods of making said lyophilised pharmaceutical compositions. More particularly, the present invention provides lyophilised pharmaceutical compositions comprising nucleic acid and lipid carrier particles and methods of making said lyophilised pharmaceutical compositions. The provided lyophilised compositions have improved critical quality attributes (CQAs) and the provided methods prevent the need for a deep-freeze cold chain. The present invention further provides the use of said lyophilised pharmaceutical compositions in medicine.

5.WO/2025/255393HERPES SIMPLEX VIRUS ANTIGEN BINDING AGENTS FOR MITIGATION OF HERPES SIMPLEX VIRUS ASSOCIATED DISEASE
WO - 11.12.2025
Int.Class C07K 16/08
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
08against material from viruses
 Appl.No PCT/US2025/032531 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor COHEN, Gary H.
Provided herein are compositions for the prevention and treatment of genital herpes, comprising antigen binding agents to herpes simplex virus (HSV) glycoproteins, including those involved in virus entry and immune evasion, ribonucleic acids (RNAs) encoding these antigen binding agents, and methods of use thereof.
6.20250375391LIPID NANOPARTICLE COMPOSITIONS COMPRISING SURFACE LIPID DERIVATIVES AND RELATES USES
US - 11.12.2025
Int.Class A61K 9/51
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules
51Nanocapsules
 Appl.No 19099108 Applicant ModernaTX, Inc. Inventor Yue HUI

The present disclosure provides lipid assemblies suitable for delivery of therapeutic agents to hematopoietic stem and progenitor cells (HSPCs), wherein the lipid assemblies comprise a neutral polymer surface lipid. The present disclosure also provides therapeutic and diagnostic uses related to the lipid assemblies.

7.20250376534CIRCULAR RNA ENCODING CHIMERIC ANTIGEN RECEPTORS TARGETING BCMA
US - 11.12.2025
Int.Class C07K 16/28
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
28against receptors, cell surface antigens or cell surface determinants
 Appl.No 18876542 Applicant Orna Therapeutics, Inc. Inventor Thomas Barnes

Circular RNA, along with related compositions and methods are described herein. In some embodiments, the inventive circular RNA comprises group I intron fragments, spacers, an IRES, duplex forming regions, and an expression sequence. In some embodiments, the expression sequence encodes an antigen. In some embodiments, circular RNA of the invention has improved expression, functional stability, immunogenicity, ease of manufacturing, and/or half-life when compared to linear RNA. In some embodiments, inventive methods and constructs result in improved circularization efficiency, splicing efficiency, and/or purity when compared to existing RNA circularization approaches.

8.20250375505COMPOSITIONS AND METHODS FOR MODIFYING BILE ACIDS TO REGULATE LIPID AND STEROID METABOLISM
US - 11.12.2025
Int.Class A61K 38/44
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
44Oxidoreductases (1)
 Appl.No 18719756 Applicant The Regents of the University of California Inventor Jonathan Lynch

Provided herein are methods and compositions related to treating or preventing metabolic disorder.

9.WO/2025/255283DOSING OF MUSCLE TARGETING COMPLEXES FOR TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
WO - 11.12.2025
Int.Class C07K 14/47
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
 Appl.No PCT/US2025/032352 Applicant DYNE THERAPEUTICS, INC. Inventor WEEDEN, Timothy
Aspects of the disclosure relate to methods of reducing expression or activity of DUX4 (e.g., DUX4 protein and/or mRNA) and/or methods of treating facioscapulohumeral muscular dystrophy (FSHD) in a subject. In some embodiments, the methods comprise administering to the subject a composition comprising complexes (e.g., muscle targeting complexes) comprising an oligonucleotide (e.g., an RNAi oligonucleotide such as an siRNA) covalently linked to an antibody (e.g., anti-TfRl antibody).
10.20250375389LIPID NANOPARTICLE COMPOSITIONS COMPRISING PHOSPHOLIPID DERIVATIVES AND RELATED USES
US - 11.12.2025
Int.Class A61K 9/51
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules
51Nanocapsules
 Appl.No 19099092 Applicant ModernaTX, Inc. Inventor Jaclyn MILTON

The present disclosure provides lipid assemblies suitable for delivery of therapeutic agents to hematopoietic stem and progenitor cells (HSPCs), wherein the lipid assemblies comprise a phosphatidylserine phospholipid. The present disclosure also provides therapeutic and diagnostic uses related to the lipid assemblies.

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