Daiichi Sankyo Company, Limited

Saitama Medical University

Kobe Gakuin Educational Foundation

Shinnosuke Tsuji

Takenobu Katagiri

Sho Tsukamoto

Kensuke Nakamura

Kenji Osawa

Yoshirou Kawaguchi

Takeshi Masuda

Makoto Koizumi

Kentaro Ito

Yoshiyuki Onishi

Masafumi Matsuo

Mitsuhiro Iwamoto

Yukiko Sekiguchi

Keigo Kumagai

The present invention establishes a molecular therapy for glycogen storage disease type Ia. The present invention provides an oligonucleotide of 15-30 bases comprising a nucleotide sequence complementary to die cDNA of G6PC gene with c.648G>T mutation, wherein the oligonucleotide comprises a sequence complementary to a region comprising any site between the 82to the 92nucleotide from the 5′ end of exon 5 of the G6PC gene with c.648C>T mutation, a pharmacologically acceptable salt or solvate thereof. Also provided is a pharmaceutical drug comprising the oligonucleotide, a pharmacologically acceptable salt or solvate thereof (e.g., therapeutic drug for glycogen storage disease type Ia).

Therapeutic agent for glycogen storage disease type IA

This application relates to a pharmaceutical composition for use in a method for treating and/or preventing a patient having ectopic ossification and/or brain tumor, wherein the patient has an active mutation in ALK2 protein which is responsible for ectopic ossification or brain tumor; an amino acid residue at position 330 of ALK2 is proline; and an active ingredient of this composition is an anti-ALK2 antibody or an antigen-binding fragment thereof comprising a property of binding to ALK2, a property of cross-linking ALK2, and a property of inhibiting BMP signal transduction.

Method of treating ectopic ossification or diffuse intrinsic pontine glioma in a subject by administering an anti-ALK2 antibody

This application provides: an antibody which specifically binds to an ALK2 protein and has an activity of inhibiting BMP signal transduction mediated by ALK2; a method for producing the antibody; and a pharmaceutical composition comprising the antibody, for treating and/or preventing ectopic ossification and/or bone dysplasia, anemia, or diffuse intrinsic pontine glioma (DIPG).

Anti-ALK2 antibody

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Tokyo, Japan

Shinnosuke Tsuji