Provided herein are compositions that include one or more adeno-associated virus (AAVs) vectors and methods of inducing differentiation of a hair cell using these vector(s).

The invention relates to methods for treating COVID-19 by targeting the inflammasome/caspase1/pyroptosis axis as a key inflammatory pathway. In particular, the invention relates to treating a patient infected with SARS-CoV-2 with an effective amount of one or more compounds that directly or indirectly inhibit one or more pathways of the inflammasome/caspase1/pyroptosis axis.

This invention relates to a topical gel drug product preparation containing a composition comprising an isolated polypeptide having a carboxy-terminal amino acid sequence of an alpha connexin (ACT peptide), peptide stabilizers, excipients, buffering agents, and the like. A formulation and preparation steps are disclosed for the manufacturing of a stable, elegant, and pourable topical gel. The resulting formulation possesses long term stability suitable for aesthetic as well as therapeutic applications including the prevention of scaring and accelerated healing of wounds. Methods for treatment of chronic wounds, including chronic ulcers, are also provided.

Methods for treating neurological and other disorders, including autoimmune disorders are described. Also described is a method of treating a disorder in which Toll-like Receptor 2 (TLR2) activation by binding to myeloid differentiation primary response 88 (MyD88) plays a role in disease pathogenesis. Further a method is described that includes the administration of a composition, including a peptide sequence, that inhibits the activation of TLR2 by MyD88.

The present invention includes compositions and methods comprising viral vector systems for multiplexed activation of endogenous genes as immunotherapy and viral-based immune-gene therapy.

Compositions and methods are provided for decreasing processing of chondroitin sulfate proteoglycan 4 (CSPG) in treatment of gliomas, which treatment may be combined with administration of an immune-oncology agent.

The present disclosure relates to methods of targeting specific cell types within the cochlea using optimized gene therapy vectors. In particular, the disclosure provides gene therapy vectors to specifically target cochlear cells and methods of treating hearing impairment and hearing-loss related disorders.

Provided herein are methods and agents for modulating the signaling pathway and components thereof that are responsible for assembly and disassembly of synapses in neurons, including amyloid beta (Aβ) mediated synaptotoxicity and synapse loss. Also provided herein are methods for screening and identifying candidate agents capable of modulating synapse formation and (Aβ) mediated synaptotoxicity.

The present invention provides compositions comprising phase separated nanoparticles, as well as methods of making the nanoparticles and uses thereof. The nanoparticles can be conjugated to therapeutics and used to treat diseases or to screen compounds.

The present disclosure generally relates to, among other things, a new class of receptors engineered to modulate transcription in a ligand-dependent manner. The new receptors provide a selectable degree of noise, expression level, and signal to noise ratio. The disclosure also provides compositions and methods useful for producing such receptors, nucleic acids encoding same, host cells genetically modified with the nucleic acids, as well as methods for modulating an activity of a cell and/or for the treatment of various health conditions or diseases, such as cancers.