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Albert Maguire received the 2026 Breakthrough Prize in Life Sciences for developing a therapy for inherited retinal degeneration that became the first FDA-approved gene therapy for a genetic disease. Maguire recalled that the project began nearly four decades ago during conversations with his now wife (and co-awardee) Jean Bennett.... show more

Breakthrough

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Breakthrough Update: Today, Vertex announced that they are beginning their Phase 3, or pivotal trial for VX-880, a stem cell-derived islet therapy. A pivotal trial gathers the data required for an FDA submission. This therapy is for people with type 1 diabetes (T1D) and hypoglycemia unawareness and severe hypoglycemic episodes. This is a HUGE first for the #T1D community as it is the first time a scalable cure for some people with T1D is entering this phase. Breakthrough T1D and the T1D Fund are incredibly proud to have supported this work in cell therapies—a top priority for our organization. We want to congratulate our partners at Vertex and thank all the patients who have participated in clinical trials. Your courage and commitment are inspiring. Learn more here:

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.Crémieux dismantles the myth that China only makes "me too" drugs, and what it means for the future of gene therapy: "One of the things China's been accused of for a long time is just doing 'Me Too' drugs... where you basically see a new drug get approved and then you copy it immediately. "Are these Chinese drugs just Me Too? No. These are cell and gene therapy trials." "In the US we are banned from offering gene therapies that do germline changes, meaning that they affect sperm or eggs." "If we can cure a family of like a horrible, debilitating disease for all time, why don't we?"

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