Resumen de: WO2025030426A1
Provided is a pharmaceutical preparation for treating Bietti's crystalline dystrophy (BCD), comprising a recombinant AAV expressing CYP4V2, sodium chloride, poloxamer, phosphate, and water for injection, and having a pH between 7.0 to 7.6. The pharmaceutical preparation has an excellent virus titer stability, very few AAV aggregates, a high mRNA expression of the target gene after multiple times of freezing and thawing, and/or an excellent high temperature stability. Moreover, the animal experiments in vivo demonstrate that the preparations with the formulations have an excellent property in reducing local inflammatory response of the retina, and further clinical trials demonstrate that the pharmaceutical preparations conform to the relevant technical specifications for safety and effectiveness in the Chinese Pharmacopoeia and the United States Pharmacopoeia (USP).
Resumen de: WO2024182707A1
The present disclosure provides a first polynucleotide (e.g., a recombinant or isolated first polynucleotide) encoding an Interleukin (IL)-2 polypeptide and a second polynucleotide (e.g., a recombinant or isolated second polynucleotide) encoding an IL-12 polypeptide; recombinant nucleic acids and/or gene delivery vehicles comprising the same; compositions, formulations, and medicaments comprising the one or more of the polynucleotides, recombinant nucleic acids, and/or gene delivery vehicles; methods of their use (e.g., for the treatment of cancer); and articles of manufacture or kits thereof.
Resumen de: CN121099985A
The present disclosure provides the use of a compound of Formula I in lipid micron and nanoparticle compositions. The present disclosure also relates to microparticles or nanoparticles comprising a compound of Formula I and optionally a payload molecule, and medical uses thereof.
Resumen de: WO2024178113A1
Recombinant adeno-associated virus (AAV) vectors encoding a modified VP1 protein lacking an immunodominant T cell epitope, as well as AAV vector particles containing the modified VP1 protein, are described. Use of the recombinant AAV vectors and vector particles as improved gene therapy vectors with reduced immunogenicity is also described. Isolated VP1 peptides containing an immunodominant T cell epitope, and use thereof, is further described.
Resumen de: EP4582089A2
The invention provides a composition comprising a biphasic particle, the biphasic particle comprising an alginate gel encapsulating an inhibitory RNA for use in improving cardiac function in a mammal, wherein the inhibitory RNA reduces expression of a polypeptide in which reduced expression of the polypeptide improves cardiac function, thereby improving cardiac function of the mammal, wherein the polypeptide is eotaxin-3, cathepsin-S, DK -1, follistatin, ST-2, GRO-a, IL-21, NOV, transferrin, TIMP-2, TNFaRI, TNFaRII, angiostatin, CCL25, ANGPTL4, or MMP-3.
Resumen de: AU2025270991A1
The present disclosure relates to methods and compositions comprising derivatized-chitosan polyplexes reversibly coated with a polyanion-containing block co-polymer for the localized expression of IL-12 in mucosal tissues, preferably in combination with an IFN- 1 activator/inducer, for use in cancer immunotherapy. ov o v
Resumen de: JP2024042089A
To provide nanoparticle compositions comprising binding agents, carrier proteins and an amount of paclitaxel derivative, and optionally a therapeutic agent.SOLUTION: Described herein are nanoparticle compositions comprising carrier proteins and an amount of paclitaxel, and optionally binding agents and/or a therapeutic agent. The paclitaxel is present in an amount that is less than an amount that provides a therapeutic effect. Also disclosed herein are nanoparticles which contain (a) carrier protein, (b) a paclitaxel derivative having reduced toxicity compared to paclitaxel, and optionally (c) a binding agent and/or (d) a therapeutic agent. Further described are methods of making and using the same, in particular, as a cancer therapeutic.SELECTED DRAWING: Figure 1
Resumen de: KR20260031866A
본 발명은 ITO 나노입자를 이용한 광열 요법 치료용 조성물, 이를 이용한 광열 치료용 키트 및 치료방법에 관한 것으로, 상기 광열 요법 치료용 조성물은 인듐과 주석의 전구체를 혼합하여 반응시켜 제조되는 ITO 나노입자를 포함하고, 상기 ITO 나노입자는 적외선 흡광 가능한 것을 특징으로 한다.
Resumen de: KR20260031983A
본 발명은 돌말류에서 바이오실리카를 추출하고, 약물을 지질나노입자에 담지하여 약물중합체를 제조하며,상기 약물중합체와 상기 바이오실리카를 결합하여 약물전달체를 제조하되, 상기 제조단계는 상기 지질나노입자의 표면에 키토산을 코팅하여 상기 지질나노입자 또는 상기 약물중합체를 양전하로 대전시키는 것을 포함하고, 상기 제작단계는 상기 양전하로 대전된 상기 지질나노입자 또는 상기 약물중합체를 음전하를 띄는 상기 바이오실리카에 정전기적 방식으로 결합시키는 돌말류 기반 약물전달체의 제조방법에 관한 것이다.
Resumen de: KR20260032412A
본 발명은 기존 지질 나노입자(LNP)의 한계를 극복하기 위해 개발된 나노구조 지질 담체(NLC)에 DNA 압타머(aptamer)를 융합하여 표적 지향성 약물 전달 시스템을 개발하는 기술로서, 기존 지질 기반 나노입자의 장점을 유지하면서도 약물 전달 효율과 효능을 개선 하며, 항암 물질 외에도 다양한 소수성 생리활성 물질이나 저분자 화합물을 탑재하여 이용하기 위한 발명이다.
Resumen de: KR20260031928A
본 발명은 외부 자극에 반응하여 정밀한 위치 제어가 가능하고 약물 방출을 조절할 수 있는 약물전달시스템 플랫폼에 관한 것으로서, 보다 상세하게는 4D 프린팅 기반 약물전달시스템(DDS) 플랫폼에 있어서, 생분해성 하이드로겔로 이루어진 외벽; 상기 외벽 내부에 포함된 약학적 조성물; 상기 외벽에 포함되어 외부 전자기장에 의해 위치 및 방위 제어가 가능한 자성체 나노 입자; 및 상기 외벽에 형성되어 내부의 약학적 조성물을 외부로 방출하는, 4D 소재로 구성된 모세관 구조;를 포함하는 약물전달시스템 플랫폼에 관한 것이다.
Nº publicación: KR20260032546A 09/03/2026
Solicitante:
아퀴타스테라퓨틱스인크
Resumen de: US2024423914A1
Compounds are provided having the following Formula (I):or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein G1, R1, R2, R3, L1, and L2 are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.