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Gene-editing therapies are regulated in the United States by FDA's Center for Biologics Evaluation and Research as human gene therapy products, reviewed through the Investigational New Drug pathway under 21 CFR 312.23. The operative document is FDA's January 2024 guidance on editing of somatic…
Genetic testing oversight in the United States has returned to its pre-2024 framework: on September 19, 2025, the FDA reverted 21 CFR 809.3(a) to its prior text, after a federal district court vacated the 2024 laboratory-developed-test rule on March 31, 2025, per the FDA’s policy page. Oversight…
RNA therapeutics are drugs whose active substance is nucleic acid — small interfering RNA, antisense oligonucleotides, or messenger RNA — acting on the instructions a cell reads rather than blocking a finished protein. The category earned its standing one approval at a time: FDA approved Qfitlia…
The FDA has cleared GenEditBio's investigational new drug application for GEB-101, an in vivo genome-editing therapy for TGFBI corneal dystrophy, the company announced on January 5, 2026. The clearance enables the Phase 1/2 CLARITY trial, in which participants receive a single intrastromal…
Vertex Pharmaceuticals has presented the first clinical data for Casgevy in children ages 5-11 at the American Society of Hematology annual meeting, on December 6, 2025, in Orlando. All evaluated children in the sickle cell cohort remained free of vaso-occlusive crises for up to two years after…