Compositions comprising mRNA, self-amplifying mRNA (sa-mRNA), modified mRNA, or circular RNA constructs comprising a gene-of-interest, and lipids and LNPs for use in therapy are disclosed. A method of delivering a payload to immune cells ex vivo by contacting immune cells with a lipid nanoparticle (LNP) encapsulating a payload encoding at least one polypeptide of interest, wherein the polypeptide of interest is an antigen receptor or an antibody.

Described herein is the Bruton's tyrosine kinase (Btk) inhibitor 1-((R)-3-(4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4-d]pyrimidin-1-yl)piperidin-1-yl)prop-2-en-1-one, including crystalline forms, solvates and pharmaceutically acceptable salts thereof. Also disclosed are pharmaceutical compositions that include the Btk inhibitor, as well as methods of using the Btk inhibitor, alone or in combination with other therapeutic agents, for the treatment of autoimmune diseases or conditions, heteroimmune diseases or conditions, cancer, including lymphoma, and inflammatory diseases or conditions.

The present invention relates to novel crystal forms of 4-(6-ethoxy-1-methoxy-thioxanthene-9-ylidene)-1-methyl piperidine salts and 4-(6-ethoxy-1-hydroxy-thioxanthene-9-ylidene)-1-methyl piperidine salts; especially to novel crystal forms of 4-(6-ethoxy-1-methoxy-thioxanthene-9-ylidene)-1-methyl piperidine hydrochloride and 4-(6-ethoxy-1-hydroxy-thioxanthene-9-ylidene)-1-methyl piperidine hydrochloride as well as to the use of these salts for preventing or treating migraine or pulmonary hypertension.

A diazabicyclooctane compound, which is a beta-lactame inhibitor, represented by the following formula (I): ##STR00001##
wherein A represents Ra(Rb)N or RcO; B represents NH or NC.sub.1-6 alkyl; C represents benzyl, H or SO.sub.3M, wherein M represents H, an inorganic or an organic cation; Ra and Rb represent H, C.sub.1-6 alkyl or acyl; Rc represents C.sub.1-6 alkyl or a heterocyclyl; A is unsubstituted or substituted with 0 to 4 substituents Fn1, wherein Fn1 represents C.sub.1-6 alkyl, O, or Rg-(CH.sub.2).sub.0-3, wherein Rg represents a heterocyclyl, phenyl, heteroaryl, acyl, RdO.sub.2S, Re(Rf)N, Re(Rf)NCO, ReO, ReOCO or a protective group, wherein Rd represents C.sub.1-6 alkyl or MO; Re and Rf represent H or C.sub.1-6 alkyl, and a heterocycle having at least one nitrogen atom may be formed between Ra and Rb, between Rc and B, or between Re and Rf.

The invention provides novel compounds having the general formula (I) ##STR00001##
wherein R.sup.1, R.sup.2, R.sup.3, R.sup.4, W, A and B are as described herein, compositions including the compounds and methods of using the compounds.

This invention is in the fields of immunology and autoimmunity. More particularly it concerns pharmaceutical compositions comprising compounds which are useful agents for inhibiting the functions of TIP60 and the use of such compounds in the treatment of an individual suffering, for example, from ulcerative colitis and other irritable bowel diseases.

The present invention relates to compounds and processes for preparing compounds of Formula (I), ##STR00001## including compounds such as trans-7-oxo-6-(sulphooxy)-1,6-diazabicyclo[3,2,1]octane-2-carboxamide and salts thereof (e.g., NXL-104).

The invention suggests amphoteric lipids wherein one or more amphoteric groups having an isoelectric point between 4 and 9 are substituted on a membranous or membrane-forming amphiphilic substance, as well as liposomes containing such compounds.

The invention suggests amphoteric lipids wherein one or more amphoteric groups having an isoelectric point between 4 and 9 are substituted on a membranous or membrane-forming amphiphilic substance, as well as liposomes containing such compounds.

The present invention describes a method for the synthesis of enantiomerically pure 3-amidinophenylalanine derivatives, which are used as pharmaceutically effective urokinase inhibitors, by starting from 3-cyanophenylalanine derivatives. The methods of manufacture comprising only one synthesis step lead to new intermediates, namely 3-hydroxyamidino- and 3-amidrazonophenylalanine derivatives. These intermediates or their acetyl derivatives can be reduced into the desired 3-amidino-phenylalanine derivatives under gentle conditions (H.sub.2 or ammonium .[.formiate.]. .Iadd.formate.Iaddend., Pd/C (approx. 10%), ethanol/water, room temperature, normal pressure or also H.sub.2, Pd/C, AcOH or HCl/ethanol, 1-3 bar) in excellent yields and in an enantiomeric excess of up to 99.9%.