Syncona Ltd had spun-out the ophtalmology specialist Beacon Therapeutics Holdings Ltd with a £96m Series A financing.
Tag Archive for: CGT
The world 3rd largest gene and cell therapy (CGT) cluster, the UK CGT Catapult has expanded its operations opening a subsidiary in Edinburgh.
Bayer boosts its gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Inc.
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.
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.
Biovian Oy’s second generation AAV platform will include Remedium Bio Inc.’s lead treatment candidate against Osteoarthritis.
CDMO Minaris Regenerative Medicine GmbH is to manufacture the leukocyte infusion therapy N-LIfT for LIFT BioSciences.
Wth a Series B €40m financing, Novadip Biosciences SA has raised investements to a total of €88m. It will use the proceeds to push two single-use cell therapies designed to accelerate healing.