Italian researchers have successfully ameliorated several IL-1-based autoimmune disorders by gene therapy into transplantable hematopoietic stem cells of mice.
Bristol-based eXmoor Pharma Ltd has completed a US$35m Series A financing to expand its cell and gene therapy manufacturing capabilities.
Leiden-based VarmX BV said it will use the €30m Series B2 money to complete the data required for IND approval of its lead candidate VMX-C001, a recombinante antidote to oral factor Xa inhibitors which are widely used to prevent blood clotting but can lead to dangerous bleeding episodes. The proceeds will also enable VarmX to scale manufacturing of VMX-C001 for its further clinical testing. The financing was led by Sound Bioventures the EIC Fund joining existing investors EQT Life Sciences (formerly LSP), Inkef, Lundbeckfonden BioCapital, Ysios Capital, BioGeneration Ventures, InnovationQuarter, and Libertatis Ergo Holding.The Dutch company had already completed a €32m Series B financing in 2020.
VarmX has completed enrollment of patients into a first-in-human study, to demonstrate safety and provide clinical proof of principle of the compound. The company will present initial data at the International Society on Thrombosis and Haemostasis conference in Montreal (June 24-28, 2023). Full study results of the study that is set to enroll 88 patients will be available later this year.
VMX-C001 is a modified recombinant human blood clotting factor X derived from the Australian brown snake venome that enables blood to clot normally in the presence of synthetic factor Xa blood clotting inhibitors. About 2-3% of the annually 10 million patients that receive Factor Xa blood clotting blockers suffer from severe bleeds. VMX-C001 is set to facilitate the fast, safe and effective treatment of severe spontaneous bleeds in patients that are taking direct oral anticoagulant blood thinners (DOACs) and, in addtion, to enable patients on this class of blood thinners to undergo emergency surgery without the risk of bleeding associated with FXa DOACs.
The compound has the potential for a strong and differentiated profile, including universal and single dose reversal, ease-of-use and safety that supports emergency care use and it shows promise for applications in other indications.
An anti-TNF-antibody-cytokine fusion combo with chemotherapy turns inoperable brain tumours hot in mice. A Phase I trial sponsored by Philogen SpA is underway.
GI specialist Ironwood Pharmaceuticals Inc. has announced to acquire Swiss VectivBio Holding AG for $17 per share in an all-cash transaction.
A new machine learning model can predict enzyme-substrate pairs, with an accuracy of over 90% and is ready to be used in pharmaceutical and industrial biotechnology.
Carbon-capture specialist LanzaTech Global and Plastipak Packaging Inc have produced the worlds first PET resin made from captured carbon emissions.
Roche’s brutons tyrosine kinas blocker fenebrutinib has met all endpoints in proof-of-concept study with patients suffering from relapsing-remitting multiple sclerosis.
uniQure N.V. is to sell a portion of its royalty rights from the net sales o its hemophilia B gene therapy etranacogene dezaparvovec-drbl to HealthCare Royalty and Sagard Healthcare for a gross purchase price of up to $400m in cash. This financing provides uniQure with immediate, non-equity-dilutive capital to continue to invest in our AAV gene therapy pipeline and platform and to advance potentially breakthrough gene therapies for patients, including AMT-130 in Huntingtons disease, AMT-260 for refractory temporal lobe epilepsy, AMT-162 for SOD-1 ALS and other product candidates, statedMatt Kapusta, CEO of uniQure.
Etranacogene dezaparvovec-drbl is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a persons own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.
Under the terms of the agreement, uniQure will receive an upfront cash payment of $375m in exchange for the lowest royalty tier on CSL Behrings worldwide net sales of etranacogene dezaparvovec-drbl up to 1.85 times the purchase price until June 30, 2032 or, if such cap is not met by June 30, 2032, up to 2.25x the purchase price through December 31, 2038.
Amsterdam-based uniQure NV led the multi-year development of etranacogene dezaparvovec-drbl and completed an exclusive global license and commercialization agreement with CSL Behring in May 2021. Etranacogene dezaparvovec-drbl HEis approved for the treatment of hemophilia B in the US, Europe and the United Kingdom. CSL Behring also is pursuing registration in additional countries.
uniQure expects the transaction will extend its cash runway into the second quarter of 2026, assuming receipt of the contractual milestone payment for the first sale of etranacogene dezaparvovec-drbl in the US.
Belgian T reg specialist Dualyx NV had raised €40m to progress its three preclinical Treg candidates for autoimmune diseases into the clinic.