Mayo Clinic joins $27.7 million gene-editing initiative for children with rare immune disorders
Mayo Clinic has joined a major multi-million-dollar initiative aiming to develop more affordable and scalable gene-editing therapies for children with rare inherited immune disorders.
The programme, known as AEGIS, has received an award of up to $27.7 million from the Advanced Research Projects Agency for Health (ARPA-H). Led by the Innovative Genomics Institute at the University of California, Berkeley, the five-year initiative brings together academic, clinical, nonprofit and industry partners.
The consortium will focus initially on inborn errors of immunity, a group of more than 500 rare genetic disorders that can leave children vulnerable to severe infections, autoimmune disease and other life-threatening complications. Researchers plan to use CRISPR-based technologies to repair disease-causing mutations in blood-forming stem cells, with the ambition of developing one-time treatments capable of restoring normal immune function.
Mayo Clinic will serve as one of three clinical sites supporting the programme's planned clinical trials, helping identify, enrol and care for children receiving the investigational therapies.
The AEGIS consortium will combine gene-editing technologies with new manufacturing and delivery approaches in the hope of creating a framework that can eventually support the development of therapies across hundreds of rare genetic conditions.
Read more via News Medical
Novartis pauses autoimmune CAR-T trials following three patient deaths
The rapidly growing use of CAR-T therapies in autoimmune disease has faced a significant setback this week after Novartis paused multiple clinical trials of its experimental therapy rapcabtagene autoleucel, also known as rap-cel or YTB323.
Novartis halted screening, randomisation and dosing across eight clinical trials after three patients died following severe cases of immune effector cell-associated haemophagocytic syndrome (IEC-HS), a potentially life-threatening inflammatory complication associated with immune-based therapies. Rap-cel is a CD19-directed autologous CAR-T therapy being investigated across a range of autoimmune and neurological conditions, including lupus, systemic sclerosis, rheumatoid arthritis, Sjögren's disease and multiple sclerosis.
The company has said the pause will allow for a comprehensive review of clinical and safety data across the programme. Its oncology studies of rap-cel are not affected.
The news was followed by Bristol Myers Squibb's decision to voluntarily pause enrolment in trials of its own CD19-directed CAR-T candidate in autoimmune diseases after observing inflammatory events, although the company said it had not recorded any deaths.
The pauses represent an important moment for the autoimmune CAR-T field. While the therapeutic potential remains significant, the latest events are likely to place even greater emphasis on patient selection, manufacturing consistency, safety monitoring and the management of inflammatory complications as programmes move into larger clinical trials.
Read more via Biopharma Dive and PharmaPhorum
Ocugen doses first patient in Phase 3 geographic atrophy gene therapy trial
Meanwhile, Ocugen has announced that the first patient has been dosed in its global Phase 3 registrational trial evaluating OCU410 for geographic atrophy secondary to dry age-related macular degeneration.
The ArMaDa3 trial is evaluating OCU410, an investigational AAV5-based modifier gene therapy designed to address multiple biological pathways involved in geographic atrophy through a single subretinal injection. According to Ocugen, the study is the first pivotal gene therapy trial in geographic atrophy. The global, multicentre Phase 3 trial will enrol 237 patients across the US, Canada, Europe and Latin America.
The programme recently received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration, following Phase 2 data. Ocugen has said its Phase 3 design is aligned with the FDA and that it anticipates filing a Biologics License Application in 2028.
As gene therapy developers increasingly look beyond rare monogenic diseases, the advancement of OCU410 into Phase 3 is another example of the industry's efforts to apply genetic medicines to larger patient populations and more complex diseases.
Read more via Clinical Trials Arena
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