Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...

Vertex executives warned that Casgevy, its curative treatment for sickle cell disease, would be slow to reach patients. But ...

Personalized Gene Editing Saves Baby in Record Time In early 2025, researchers successfully treated a baby boy with a rare, ...

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial.

Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has been challenging. In the latest case, the scientists developed a CRISPR ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. A group of scientists successfully made a bespoke gene ...

CRISPR Therapeutics (NasdaqGM:CRSP) has set new 2026 milestones, including planned regulatory submissions for Casgevy in patients aged 5 to 11. The company outlined progress in several clinical ...

Zugo-cel, targeting CD19, is in an ongoing Phase 1 basket trial in autoimmune rheumatologic diseases. Preliminary clinical data from the Phase 1 study have been encouraging, and zugo-cel has been well ...

Developing a gene therapy typically takes years, but when Baby KJ was diagnosed with a deadly genetic condition, scientists had only months. What followed was a successful collaboration among ...

This report provides an in-depth analysis of the Cell and Gene Therapy industry, highlighting key company revenues, regulatory approvals, and market trends. It has been compiled by Towards Healthcare, ...

Muscles make up nearly 40% of the human body and power every move we make, from a child's first steps to recovery after ...