CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...

Gene therapy has the potential to dramatically benefit patients with genetic blood disorders, but a small study on participants in a clinical trial for sickle cell disease suggests that a key process ...

With the FDA expecting to approve 10–20 Cell and Gene Therapies (CGTs) annually by 2025, it is evident that the field will continue to hold immense commercial and medical opportunities. To harness ...

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

Our viewpoint is that in cases of ultra rare diseases where there is no commercial model or opportunity for traditional forms of investment, access to gene therapies could be provided via a clinical ...

Despite innovative new research, many cell and gene therapies do not make it all the way to the patients. Researchers and ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...

With 300 million people living with rare diseases, experts say new gene and cell therapies are giving families in Wisconsin hope for lasting treatment options.