The US Food and Drug Administration has approved a new indication for Casgevy, a cell-based gene therapy for two blood disorders, making it available to individuals as young as two years. The disorders are sickle cell disease, a rare inherited blood disorder, and transfusion-dependent beta thalassaemia. Casgevy (exagamglogene autotemcel) was first approved by the FDA in 2023 for children with sickle cell disease from the age of 12 years.
uniQure NV has reached an agreement with the US Food and Drug Administration to proceed with a marketing authorisation application for its gene therapy for Huntington’s disease, a rare, inherited neurodegenerative disorder. The application is expected to be filed in the third quarter of this year, the company announced on 17 June.
Eli Lilly and Co’s agreement on 20 April to acquire Kelonia Therapeutics Inc in a deal worth up to $7 billion puts the company in place to exploit the emerging field of in vivo CAR-T therapy which has been described by analysts as a way to achieve therapeutic value without a complex production step. This compares with the traditional ex vivo approach in which T cells are extracted from a patient, modified in a laboratory, and then reinfused into the patient.
UCB SA announced on 3 May that it has signed an agreement to acquire Candid Therapeutics Inc of San Diego, US, broadening its portfolio to include biologics targeting autoimmune and inflammatory diseases. The Belgium-based biopharma company already has marketed products for epilepsy, plaque psoriasis, and Crohn’s disease. The acquisition will add preclinical and early clinical-stage products to UCB’s portfolio, the most advanced of which are bispecific T cell engagers. These are antibodies that can bind T cells to tumour cells to kill cancers.
The Belgian biotech Galapagos NV has announced a new strategy focused on the development of potential chimeric antigen receptor (CAR) T cells for the treatment of autoimmune diseases. If executed, the strategy would involve recalibrating a long-term relationship with Gilead Sciences Inc with whom it has been working since 2015. In separate statements on 23 March, Gilead announced the acquisition of the privately-held US biotech company Ouro Medicines Inc, and Galapagos said it is in discussions with Gilead to share the cost of developing the target company’s lead asset.
A German company developing cell therapies to treat autoimmune diseases has been accepted into the European Medicines Agency’s support scheme PRIME, a regulatory pathway for small and medium-sized enterprises developing drugs for serious medical needs. The Heidelberg-based company, TolerogenixX GmbH, has received PRIME designation for its lead cell therapy candidate MIC-Lx for the treatment of immuno-suppression in live donor kidney transplantation.
The gene therapy developer, uniQure NV, has asked for a second meeting with the US Food and Drug Administration to discuss the agency’s recommendation for further clinical development of its candidate therapy for Huntington’s disease AMT-130. The gene therapy is intended as a one-time treatment for Huntington’s, a rare neurodegenerative disorder. uniQure produced data from a Phase 1/2 trial in September 2025 which was intended as a pivotal study leading to a regulatory submission.
The US Food and Drug Administration has approved a new gene therapy for spinal muscular atrophy (SMA), a neurological disorder that can lead to progressive muscle weakness and in the most severe cases, death. The therapy, Itvisma (onasemnogene abeparvovec), has the same active ingredient as the gene therapy Zolgensma, but formulated at a different concentration. Zolgensma was approved by the FDA in 2019 to treat paediatric patients with SMA. The newest therapy extends the treatment group to older individuals with a confirmed mutation in the survival motor neuron 1 (SMN1) gene.
A venture capital syndicate has mobilised $141 million in Series B financing for an Italian biotech which is developing two gene therapies for retinal disorders. The company, AAVantgarde Bio Srl of Milan, is developing therapies for Stargardt disease and retinitis pigmentosa due to Usher syndrome type 1B. Both conditions are inherited retinal diseases for which there are no approved therapies. AAVantgarde is a spin-off of the Telethon Institute for Genetics and Medicine, an international research institute based in Naples.
Bristol Myers Squibb Co has stepped up its commitment to cell therapies with plans to spend $1.5 billion to acquire a Cambridge, Massachusetts biotech which is developing a medicine to reprogramme the immune system and treat autoimmune diseases. Orbital’s lead product, OTX-201, is an in vivo chimeric antigen receptor (CAR) T cell therapy that is being developed using ribonucleic acid (RNA) technology. The preclinical product consists of a circular RNA that encodes a CAR which targets cells expressing CD19, a B cell specific antigen.