# US 2025/0368705 A1 · Gene-based medicines and cellular therapy for disease > Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt - Number: US 2025/0368705 A1 - Document ID: US20250368705A1 - Status: Published application - Kind: A1 - Application number: 19/226,097 - Filed: 2025-06-02 - Published: 2025-12-04 - Earliest claimed: 2024-06-04 - Assignees: CarryGenes Bioengineering, LLC; Artax Invest AB - Applicants: CarryGenes Bioengineering, LLC; Artax Invest AB - Inventors: Edward PERKINS; Amy L GREENE; Dominique BROCCOLI - Web page: https://patentgrep.com/patent/US20250368705A1 ## CPC - C07K 14/4708: {Duchenne dystrophy} - A61K 48/005: {characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered} - C12N 15/85: for animal cells - A01K 2227/105: Murine - A61K 38/00: Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00) - C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00) - A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D) - C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00) - A01K: ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS ## Abstract Herein disclosed are compositions comprising synthetic chromosomes and methods of their use to treat diseases and disorders (e.g., cancers, genetic and autoimmune diseases). Specifically described are methods of constructing synthetic chromosome compositions bearing one or multiple genes as well as regulatory sequences that control expression of the gene(s) such that, when these are expressed from the synthetic chromosome in an animal cell, at least one medicinal gene product is reliably, faithfully and indefinitely produced by the animal cells. As an example, the presently disclosed compositions and methods are used to bioengineer cells to enable them to express the entire dystrophin gene and additional regulatory nucleic acid sequences under tightly controlled conditions, allowing the present methods and compositions to be used as cellular medicines for treatment of diseases such as muscular dystrophies. ## Claims ### Claim 1 An autonomously replicating, stably inherited, non-integrating, non-native mammalian synthetic chromosome (mSynC) comprising: an rDNA-amplified centromere region; at least two telomeres; multiple copies of at least one type of unidirectional site-specific integration site; at least one irreversibly integrated genetic cassette greater than 5 kbp in size, wherein the integrated cassette comprises at least one therapeutic gene; a safety switch under tight expression control; and a marker allowing for identification of mSynC-bearing cells. ### Claim 2 (depends on 1) The mSynC of claim 1, further comprising at least one additional element selected from: a second therapeutic gene; a lineage-specific cellular differentiation gene and/or regulatory sequence; an enhancer of expression; a sequence encoding a cell-surface protein; a cellular growth factor; and a cytokine. ### Claim 3 (depends on 1) The mSynC of claim 1, wherein the therapeutic gene is present in multiple copies on the mSynC. ### Claim 4 (depends on 1) The mSynC of claim 1, comprising at least one therapeutic gene involved in muscle function. ### Claim 5 (depends on 4) The mSynC of claim 4 wherein the therapeutic gene encodes a gene product that treats a muscular dystrophy. ### Claim 6 (depends on 5) The mSynC of claim 5, wherein the muscular dystrophy is selected from Duchenne Muscular Dystrophy (DMD), Limb-girdle Muscular Dystrophy (LGMD), myotonic dystrophy, Facioscapulohumeral Dystrophy (FSHD), Oculopharyngeal Muscular Dystrophy (CPMD), Oculopharyngeal Muscular Dystrophy (CPMD) and congenital muscular dystrophy. ### Claim 7 (depends on 1) The mSynC of claim 1, wherein at least one gene encodes the entire dystrophin protein. ### Claim 8 (depends on 2) The mSynC of claim 2, wherein the mSynC comprises at least the second therapeutic gene, and the second therapeutic gene is selected from: another variant of the first therapeutic gene different from the first therapeutic gene, DP71ab, utrophin, dysferlin, acetylgalactosaminyltransferase, GALGT2, PAX7, nestin, calpain 3, alpha-, beta-, delta-, or gamma-sarcoglycan, desmin, and caveolin 3. ### Claim 9 (depends on 8) The mSynC of claim 8, comprising both the full-length DMD cDNA and the isoform Dp71ab. ### Claim 10 (depends on 8) The mSync of claim 8, wherein the mSynC further comprises at least one regulatory element that specifically regulates the second therapeutic gene. ### Claim 11 (depends on 1) The mSynC of claim 1, wherein multiple and different genes are present on the mSynC and, when inside host mammalian cells, express gene products that treat a complex disease having multiple causes. ### Claim 12 (depends on 11) The mSynC of claim 11, wherein the gene products are components of a multi-protein complex. ### Claim 13 (depends on 11) The mSynC of claim 11, wherein the gene products are expressed in the host cells at different levels. ### Claim 14 (depends on 1) A method of controlling expression of a therapeutic gene in a host cell employing the mSynC of claim 1. ### Claim 15 (depends on 1) A method of making a therapeutic cellular medicine by transferring the mSynC of claim 1 into a mammalian cell. ### Claim 16 (depends on 1) A method of cell-based therapy comprising: transferring, ex vivo, the mSynC of claim 1 into a mammalian cell; and administering the mSynC-carrying cells to a mammal in need of treatment. ### Claim 17 (depends on 15) The method of claim 15, wherein the mammalian cell is a progenitor cell, a satellite cell, a smooth muscle cell, a cardiac muscle cell, a skeletal muscle cell, a myoblast, a myotube, a syncytium, or a sarcomere. ### Claim 18 (depends on 1) A method of cell-based therapy comprising: isolating autologous somatic cells from a patient, reprogramming the patient-autologous cells to generate stem cells; transferring, ex vivo, the mSynC of claim 1 into the stem cells to generate transgenic patient-autologous stem cells; administering the transgenic patient-autologous stem cells carrying the mSynC to the patient. ### Claim 19 (depends on 18) The method of claim 18, further comprising reprogramming the patient-autologous cells to generate cells selected from: induced pluripotent stem cells (ipSCs), mesenchymal stem cells (MSCs), MSCs derived from umbilical cord (ucMSCs), myoblasts, mesoangioblasts (MABs), and human iPSC-derived MAB-like cells (HIDEMs). ### Claim 20 (depends on 18) The method of claim 18, wherein the autologous somatic cells are myoblasts, vessel cells, myotubes, muscle cells, adipose cells, bone marrow cells, cells from synovium, etc. ### Claim 21 (depends on 1) A cellular medicine for treating a disease, comprising mammalian cells carrying the mSynC of claim 1. ### Claim 22 (depends on 21) The cellular medicine of claim 21, wherein the disease is a Muscular Dystrophy. ### Claim 23 (depends on 21) The cellular medicine of claim 21, wherein the disease is DMD. ### Claim 24 (depends on 1) A host cell comprising the mSynC of claim 1. ### Claim 25 (depends on 24) The host cell comprising the mSynC of claim 24, wherein the host cell is a muscle cell. ### Claim 26 (depends on 24) The host cell comprising the mSynC of claim 24, wherein the host cell is a stem cell. ### Claim 27 (depends on 24) A cellular composition comprising the host cell of claim 24 and a pharmaceutically acceptable carrier.