Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during a surgical procedure, and replaces the human dopa decarboxylase ...

Currently, in the 2025-2027 "Fast, Forward" phase, ClearPoint aims to grow into a $500 million market opportunity, expand to 150 activated customers, and support the first commercial cell and gene ...

A significant highlight of ClearPoint’s presentation was the FDA approval of a partner’s gene therapy that is co-labeled with ClearPoint’s technology. The FDA authorized the SmartFlow Neuro Cannula to ...

It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.

Introduction & Market Context. ClearPoint Neuro Inc. (NASDAQ: CLPT) unveiled its corporate presentation on May 13, 2025, highlighting the company’s strategic vision as it enters what management calls ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...

Shares of Sarepta Therapeutics closed down as much as 42% to hit a nine-year low of $18.30 on Monday after a second death of ...

For patients with recessive dystrophic epidermolysis bullosa (RDEB), a one-time surgical application of prademagene ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns and their families. Now, we’re taking an even earlier step: treating ...

Gene Therapy For Inherited Disease In Infants. ByWilliam A. Haseltine, Contributor. Forbes contributors publish independent expert analyses and insights.