A cell-based gene therapy for multiple myeloma has been recommended by the European Medicines Agency for patients with multiple myeloma, an incurable blood cancer that causes plasma cells to grow out of control. Symptoms can include bone fracture or even kidney failure. The drug, Carvykti (ciltacabtagene autoleucel), is a chimeric antigen receptor (CAR) T cell medicine consisting of a patient’s own immune cells which have been engineered to target and kill cancer cells.
Novartis is to explore new ways of delivering gene therapies to the brain under a licence option agreement with Voyager Therapeutics Inc, a US company with adeno-associated virus (AAV) capsid technology. Under the agreement, the Swiss multinational may elect to license novel capsids for use in directing therapies against three undisclosed central nervous system (CNS) targets. Further out, it may exercise options for two additional targets. The goal is to develop gene therapies for neurological diseases that manifest in deep regions of the brain.
An allogeneic cell therapy has been successfully used to treat patients 10 years and older with Duchenne muscular dystrophy, a rare genetic disease that affects both skeletal and cardiac muscle. The therapy, CAP-1002, was tested in a Phase 2, placebo-controlled trial and effectively reduced the deterioration of upper limb function in the patients as well as improved cardiac function. The results of the study, called HOPE-2, were published in The Lancet on 12 March 2022.
BioNTech SE has entered a three-year collaboration with Medigene AG to develop immunotherapies against solid tumours using Medigene’s proprietary T cell receptor (TCR) technology. This technology involves engineering a patient’s own T cells in order to display receptors that can recognise specific cancer antigens. Under the agreement, Medigene’s TCR discovery platform will be used in the development of cancer therapies against tumour targets nominated by BioNTech.
UK-based Oxford Biomedica Plc is to establish a gene therapy manufacturing presence in the US under a new agreement with Homology Medicines Inc, a clinical-stage genetics medicines company located near Boston, US. Homology has experience in the development and use of adeno-associated virus (AAV) vectors while Oxford Biomedica has a lentiviral vector delivery platform.
Intellia Therapeutics Inc has taken steps to advance its genome editing technology with the acquisition of Rewrite Therapeutics Inc. Intellia will pay Rewrite shareholders $45 million upfront and an additional $155 million and research and regulatory milestones through a mix of Intellia stock and cash. Further financial details were not disclosed.
Indapta Therapeutics Inc of San Francisco, US, has raised $50 million in a Series A financing round to advance its technology for allogeneic natural killer (NK) cell therapies. This involves isolating and expanding a subset of NK cells known as G-NK cells, suitable for administration with a monoclonal antibody.
Hookipa Pharma Inc is to take responsibility for advancing an antiviral drug candidate for HIV through early clinical development under an amended agreement with its partner Gilead Sciences Inc. Hookipa announced the recasting of the agreement on 15 February.
A woman who was diagnosed with HIV in 2013 and subsequently developed acute myeloid leukaemia, has reportedly seen her HIV go into remission four years after receiving a transplant of stem cells obtained from an infant’s umbilical cord blood. The case, reported by US media on 15 February, was described by scientists at the annual meeting of the Conference on Retroviruses and Opportunistic Infection. It is the third time a patient with HIV has been reportedly cured of an HIV infection.
Hal Barron, the chief scientific officer at GlaxoSmithKline Plc, is to become chief executive of a new regenerative medicine company that will seek to understand the process of cellular rejuvenation with the goal of slowing or even reversing the effects of disease. The company, Altos Labs Inc, will combine basic science with translational medicine across two institutes in the US and one in the UK. It is being launched with $3 billion of start-up capital, according to a statement issued on 19 January.