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Analysis

1 / 154
1.WO/2026/006483SNCA VECTORS AND ENGINEERED GUIDE RNAS
WO - 02.01.2026
Int.Class C12N 15/864
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
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
864Parvoviral vectors
 Appl.No PCT/US2025/035296 Applicant SHAPE THERAPEUTICS INC. Inventor BURLEIGH, Stephen
Disclosed herein are plasmids encoding engineered guide RNAs and compositions comprising the same. Also disclosed herein are AAVs containing engineered guide RNAs. Also disclosed herein are methods of treating diseases or conditions in a subject by administering engineered guide RNAs or pharmaceutical compositions described herein.
2.WO/2026/006334DUX4 VECTORS AND ENGINEERED GUIDE RNAS
WO - 02.01.2026
Int.Class C12N 15/864
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
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
864Parvoviral vectors
 Appl.No PCT/US2025/035079 Applicant SHAPE THERAPEUTICS INC. Inventor BURLEIGH, Stephen
Disclosed herein are plasmids encoding engineered guide RNAs and compositions comprising the same. Also disclosed herein are AAVs containing engineered guide RNAs. Also disclosed herein are methods of treating diseases or conditions in a subject by administering engineered guide RNAs or pharmaceutical compositions described herein.
3.WO/2026/006203COMPOSITIONS AND METHODS FOR MAKING CIRCULAR RNA
WO - 02.01.2026
Int.Class C12N 15/85
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
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
 Appl.No PCT/US2025/034866 Applicant ORBITAL THERAPEUTICS, INC. Inventor VENKATARAMANAN, Srivats
The present invention provides nucleic acids and methods for making circular RNAs (circRNAs), compositions comprising circular RNAs, and methods of use thereof. The present invention uses optimized self-splicing T4 bacteriophage td gene (T4td) intron sequences to produce circular RNAs. The optimized T4td intron sequences described herein mediate efficient circularization of a linear RNA sequence.
4.WO/2026/006593SYNTHETIC INTERNAL RIBOSOME ENTRY SITES
WO - 02.01.2026
Int.Class C12N 15/85
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
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
 Appl.No PCT/US2025/035485 Applicant ORNA THERAPEUTICS, INC. Inventor VUNGUTUR, Rahul
Circular RNA and precursor RNA polynucleotides comprising synthetic, non-naturally occurring, Internal Ribosome Entry Sites (IRES), along with related compositions and methods are described herein. In some embodiments, the synthetic IRESs comprise at least one addition, deletion, or substitution of a nucleotide, domain, or motif, as compared to a naturally occurring IRES. In some embodiments, the synthetic IRESs have improved function and/or expression and/or stability as compared to a naturally occurring IRES and/or are capable of increasing expression of operably linked expression sequences as compared to naturally occurring IRESs. Also presented herein are methods and compositions for the manufacture and preparation of the circularized RNAs comprising the synthetic IRESs, along with methods of administering said circular RNAs and related compositions to a subject in need thereof for treatment or prevention purposes.
5.WO/2026/006476ABCA4 VECTORS AND ENGINEERED GUIDE RNAS
WO - 02.01.2026
Int.Class C12N 15/113
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
 Appl.No PCT/US2025/035288 Applicant SHAPE THERAPEUTICS INC. Inventor BURLEIGH, Stephen
Disclosed herein are plasmids encoding engineered guide RNAs and compositions comprising the same. Also disclosed herein are AAV vectors containing engineered guide RNAs. Also disclosed herein are methods of treating diseases or conditions in a subject by administering engineered guide RNAs or pharmaceutical compositions described herein.
6.WO/2026/003582LIPIDS AND LIPID NANOPARTICLES
WO - 02.01.2026
Int.Class C07C 219/06
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
CACYCLIC OR CARBOCYCLIC COMPOUNDS
219Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
02having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
04the carbon skeleton being acyclic and saturated
06having the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
 Appl.No PCT/IB2025/000348 Applicant AXELYF EHF. Inventor CORNEBISE, Mark
The disclosure provides for lipids that may be formulated in a delivery vehicle to facilitate the encapsulation of a wide range of single or multiple payloads including therapeutic, theragnostic, preventive, prophylactic, pre-emptive, and diagnostic agents, such as, without limitation, nucleic acids (e.g., RNA or DNA), proteins, peptides, and small molecule active pharmaceutical ingredients (APIs). Methods of delivering and/or producing a polypeptide of interest in a cell are also provided.
7.WO/2026/006687CIRCULAR RNA COMPOSITIONS AND USES THEREOF FOR DISEASES CAUSED BY VARICELLA-ZOSTER VIRUS
WO - 02.01.2026
Int.Class A61K 39/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
 Appl.No PCT/US2025/035631 Applicant ORBITAL THERAPEUTICS, INC. Inventor BESIN, Gilles
The present disclosure relates to circular RNA sequences encoding an antigen from varicella zoster virus (VZV). The circular RNA sequences described herein are used as vaccines and compositions for preventing or treating diseases resulting from VZV, including shingles. Methods of use, including methods of treating or preventing a disease caused by VZV and methods for formulating and preparing the VZV vaccine compositions, are also described.
8.WO/2025/264930COMPOSITIONS AND METHODS FOR IMMUNE CELL TRAFFICKING MODULATION
WO - 26.12.2025
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 PCT/US2025/034383 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor BETTS, Michael, R.
The present invention relates to methods and compositions comprising a delivery vehicle for delivery of tissue trafficking modulators to modulate the trafficking of immune cell to a specific locus. The invention also relates to methods for treating or preventing a disease and disorder, including cancer, an infectious disease and an immunological disorder.
9.WO/2025/265034TRANSITORY MODULATION OF GENE TRANSCRIPTION THROUGH TARGETED MRNA-LNP DELIVERY OF MODIFIED TRANSCRIPTION ACTIVATOR-LIKE EFFECTORS (TALE)
WO - 26.12.2025
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/US2025/034552 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor BETTS, Michael, R.
Compositions are provided for transient transcriptional modulation of genes through delivery of RNA molecules encoding site-specific DNA binding molecules linked to transcriptional modulation domains, as well as methods for treating or preventing a disease or disorder, including a cancer, infectious disease, or immunological disease.
10.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.
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