A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...
The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...
A miracle baby who was treated with a pioneering gene-therapy at CHOP has reached a major milestone as he took his first steps.
A baby born with a rare genetic disorder has taken his first steps after receiving a groundbreaking, personalized gene editing treatment at CHOP.
The boy was born with the rare disease that would gradually take away his cognitive and motor function without treatment.
At ASH 2025, investigators reported early results from the first-in-human phase 1 inMMyCAR study evaluating KLN-1010, an ...
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 ...
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA), ...
X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...
While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...
A long-term, trust-based CDMO partnership can support cell and gene therapy developers from early process development through GMP manufacturing and commercial supply.
AMD Phase 3 timing, Otsuka funding to 2028, and mixed 4D-710 data with financial risk. See why FDMT stock is a hold.
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