The present invention pertains to an inventive release mechanism for extracellular vesicle (EV)-mediated intracellular and intramembrane delivery of therapeutic polypeptides. More specifically, the invention relates to EVs comprising polypeptide constructs which comprise a therapeutic polypeptide releasably attached to an exosomal polypeptide. Furthermore, the present invention pertains to manufacturing methods, pharmaceutical compositions, medical uses and applications, and various other embodiments related to the inventive EVs.

Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).

The invention relates to methods of treating multiple osteochondroma and anemia resulting from iron imbalance using small molecule ALK2 inhibitors.

The invention relates to methods of treating multiple osteochondroma and anemia resulting from iron imbalance using small molecule ALK2 inhibitors.

The present disclosure provides methods for treating hematologic malignancies in a recipient subject in need thereof comprising administering to the recipient subject an effective amount of donor myeloid progenitor cells, and an effective amount of donor umbilical cord blood (UCB) cells, wherein the UCB cells and the myeloid progenitor cells are HLA matched. In some embodiments, the donor for the myeloid progenitor cells is not related to the recipient subject and/or the donor for the UCB cells. Also disclosed herein are methods for promoting early myeloid recovery in a recipient subject following UCB transplantation.

The present disclosure provides methods for treating hematologic malignancies in a recipient subject in need thereof comprising administering to the recipient subject an effective amount of donor myeloid progenitor cells, and an effective amount of donor umbilical cord blood (UCB) cells, wherein the UCB cells and the myeloid progenitor cells are HLA matched. In some embodiments, the donor for the myeloid progenitor cells is not related to the recipient subject and/or the donor for the UCB cells. Also disclosed herein are methods for promoting early myeloid recovery in a recipient subject following UCB transplantation.

The present invention relates to methods and materials for modulating the complement alternative pathway (CAP), the complement classical pathway (CCP), the complement lectin/mannose pathway (CMP), or combinations thereof, as well as methods and materials for targeting diagnostic, prophylactic and therapeutic agents to localized areas of tissue within the body where they may more directly exert their effects upon the intended target cells or tissue, with reduced, associated systemic effects compared with administration of the same or similar agents in an untargeted, systemic manner. The methods and materials of the present invention may therefore allow for increased efficacy, lower threshold effective dosages and/or lower effective maintenance doses, and/or reduced associated undesired or adverse effects in terms of frequency or severity of occurrence, or both. The present invention also relates to methods and materials for modulating a host humoral immune response, especially reducing, inhibiting, or preventing a host humoral immune response.

The invention provides compositions and methods useful for the treatment and prevention of conditions associated with short telomere length.

The present invention relates to antibodies and antigen-binding fragments thereof to human BMP6 and compositions and methods of use thereof.

The present invention provides monovalent antibodies with a long half-life when administered in vivo, methods of making such monovalent antibodies, pharmaceutical compositions comprising such antibodies, and uses of the monovalent antibodies.