CSL Acquires Gene Therapy Program from uniQure
As of the 25th of June, biotechnology giant CSL Limited (ASX: CSL) has announced that it will acquire the exclusive global rights for a new gene therapy program to treat haemophilia B disorder. The adeno associated virus (AAV) therapy called AMT – 061, or etranacogene dezaparvovec, is currently in phase three clinical trials. AMT-061 could potentially be the first gene therapies to deliver long term benefits for sufferers of the disease.
uniQure (NASDAQ: QURE) stands to receive a cash payment of US450 million for the exclusive rights, with ensuing regulatory and commercial sales milestones, as well as royalties. The agreement terms are that QURE will complete the trials and scale-up manufacture for early commercial access. AMT-061 will complement the haemophilic treatment portfolio that CSL has and is a signal for the positive direction in future genetic treatments. Concurrently, CSL is currently developing stem cell genetic therapies for sickle cell disease with Seattle Children’s Research Institute. In 1H20, haemophilic treatment Idelvion grew 21 per cent, and Afstyla rose 30 per cent. Additionally, over the past five years, CSL has invested US3.3 billion in research and development, with US832 million invested through 2018 and 2019.
Haemophilia B is a genetic disorder where patients have a deficient Factor IX (FIX) that affects the plasma levels in the blood, stopping clotting around cuts forming. Normal levels occur at around 50 to 150 per cent FIX, with haemophilia B patients suffering symptoms of internal bleeding into joints, muscles, dental sensitivity, and excessive bleeding after trauma. Current treatments include periodic intravenous FIX replacement therapies to restore levels to normal. Should AMT-061 be successful, appropriate candidates could be saved a lifetime of preventive maintenance through a one-time genetic therapy, freeing them of the burden of treatment and dangers of the illness.
The data surrounding AMT-061 shows that it could be profitable through volume. AMT-061 utilises viral vectors that carry the genes to fix the FIX deficient. The gene cassette is a patent-protected variant of Factor IX which turns on the right genes to create FIX in the body. Presently, AAV5 treatments are considered safe with no patient experiencing cytotoxic T-cell mediated immune responses to the capsid, which if experienced blocks gene transfer. Some patients have shown pre-existing antibodies to the therapy.
However, pre-clinical and clinical data shows AAV5 could be useful even in these cases, thereby increasing the volume of appropriate candidates. CSL is due to report their 2H20 results on the 19th of August, which follows their strong 1H20 performance that saw an 11 per cent jump in revenue and net profit after tax. This therapy acquisition is an exciting development in the future of haemophilic treatments, and even though it is currently in development, shows excellent promise for the future.
By Caroline Wong
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