Compounds for the Treatment of Jak2-V617f-Associated Cmn

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent introduces pharmaceutical compositions containing inhibitors of beta1- and beta2-integrins, as well as their associated alpha subunits or molecules affecting intracellular signaling (such as Rap1-GTPase inhibitors), for treating chronic myeloproliferative neoplasia (CMN) driven by the JAK2-V617F mutation. These compounds specifically target the abnormal activation and adhesion of certain blood cells (mainly granulocytes and monocytes) to prevent symptoms such as splenomegaly (enlarged spleen), increased blood clotting (thrombosis), and inflammation commonly associated with JAK2-V617F-linked diseases like polycythemia vera, essential thrombocythemia, and primary myelofibrosis.

Use CasesContent extracted from patent full text and abstract with AI.

• Treatment of patients with JAK2-V617F-associated chronic myeloproliferative neoplasia (CMN), including polycythemia vera, essential thrombocythemia, and primary myelofibrosis.
• Prevention and reduction of splenomegaly in patients with JAK2-V617F-linked CMN.
• Reduction of thrombotic risk (prevention of blood clots) in affected patients.
• Management of inflammatory response syndrome related to JAK2-V617F mutations.
• Potential ex vivo or in vitro treatment of blood or tissue samples for research or disease modulation.

BenefitsContent extracted from patent full text and abstract with AI.

• Offers a more targeted therapeutic approach by inhibiting specific integrins associated with disease progression, potentially leading to fewer side effects than broader JAK kinase inhibitors.
• May help overcome incomplete or short-lived responses observed with existing JAK inhibitor therapies.
• Addresses major complications of CMN such as thrombosis and splenomegaly, which are leading causes of morbidity and mortality in these patients.
• Potentially reduces the abnormal trafficking and adhesion of myeloid cells, addressing disease mechanisms at their root.
• Provides options for combinatorial or adjunct therapies to enhance disease control.

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Inventors & Applicants

Patent Abstract

The present disclosure provides novel compounds for use in therapy of JAK2-V617F-associated chronic myeloproliferative neoplasia (CMN) and for use in therapy or prophylaxis of disorders or syndromes associated therewith, in particular splenomegaly and increased risk of thrombosis.