The present invention is related to variants of fungal serine protease enzyme, which have serine protease activity of Malbranchea protease. Also disclosed are isolated nucleic acid molecules, comprising polynucleotide sequences which encode variants of fungal serine protease enzyme, nucleic acid sequences encoding said protease variants, a host cell and a process of producing polypeptides having serine protease activity. Said protease variants are useful as enzyme preparations applicable in detergent compositions and for treating fibers, wool, hair, leather, or silk, for treating food or feed, or for any applications involving modification, degradation or removal of proteinaceous material.

Disclosed herein, inter alia, are compounds and methods for inhibiting Taspase1 and the treatment of cancer.

Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5′ inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3′ ITR.

Provided are methods of identifying, visualizing and purifying proteases from a complex biological sample.

Provided herein are compositions and methods for adoptive cell therapy comprising engineered immune cells that express an antigen-targeted chimeric antigen receptor and a prodrug converting enzyme for the treatment of inflammation, inflammatory diseases, or pathogenic infections.

The modified polypeptides include at least one amino acid substitution that allows the polypeptide to bind better to the S surface glycoprotein of coronaviruses that use ACE2 as a cell entry receptor, either through direct increases in affinity or through improved folding and expression of ACE2. Use of the modified ACE2 polypeptides for inhibiting CoV entry, replication and/or spread, for pre-exposure and post-exposure CoV prophylaxis, and for treating a CoV infection (e.g. COVTD-19), is also described.

Provided are methods for and compounds for modulating the complement system. In particular, compounds are provided that inhibit complement activation and compounds are provided that promote complement activation. The compounds are therapeutics by virtue of their effects on the complement system. Hence, the compounds that inhibit complement activation can be used for treatment of ischemic and reperfusion disorders, including myocardial infarction and stroke, sepsis, autoimmune diseases, inflammatory diseases and diseases with an inflammatory component, including Alzheimer's Disease and other neurodegenerative disorders.

A bone delivery conjugate having a structure selected from the group consisting of: A) X-D.sub.n-Y-protein-Z; and B) Z-protein-Y-D.sub.n-X, wherein X is absent or is an amino acid sequence of at least one amino acid; Y is absent or is an amino acid sequence of at least one amino acid; Z is absent or is an amino acid sequence of at least one amino acid; and D.sub.n is a poly aspartate wherein n=10 to 16. Compositions comprising same and methods of use thereof.

The present invention relates to dual-site BACE1 inhibitors, their manufacture, pharmaceutical compositions containing them and their use as therapeutically active substances. The active compounds of the present invention are useful in the therapeutic and/or prophylactic treatment of e.g. Alzheimer's disease.

Rett syndrome (RTT) is a neurodevelopmental disorder that affects girls almost exclusively (about 1 in 10 000 females). It is characterized by normal early growth and development followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, slowed brain and head growth, problems with walking, seizures, and intellectual disability. Herein, the inventors demonstrated that delivering a vector expressing a cholesterol 24-hydroxylase (CYP46A1) gene intravenously in a mouse model of RTT is able to prevent/correct the development of motor impairment, in a mild and an aggravated model of the disease both in male and female mice. In addition in males, the inventors demonstrated a prevention of the loss of Purkinje cells and an improvement of astrogliosis and microgliosis in the mild KO MECP2 model. Thus, the present invention relates to a vector for use in the treatment of Rett syndrome or related autism spectrum disorder, which vector comprises cholesterol 24-hydroxylase encoding nucleic acid.