Lenti-D investigational gene therapy in boys under 18 years old results.

[monkeybus]

About the Starbeam (ALD-102) Study

The Starbeam Study is assessing the efficacy and safety of an investigational gene therapy in boys up to 17 years of age with CALD. It involves transplantation with a patient’s own stem cells, which are modified to contain functional copies of the ABCD1 gene. This gene addition should result in the production of functional adrenoleukodystrophy protein (ALDP), a protein critical for the breakdown of very long chain fatty acids (VLCFAs). Buildup of VLCFAs in the central nervous system contributes to neurodegeneration in CALD.

Here is the pertinent bit...

“In June, we announced topline data that showed that 15/17 patients who had completed two years of follow-up post treatment with Lenti-D were free of major functional disabilities, the primary endpoint for this study,”

myelin.org/2017/09/bluebird...

businesswire.com/news/home/...

What is good news for our little brothers with ALD will surely benefit us with AMN somewhere down the line.

[Aaron98]

"What is good news for our little brothers with ALD will surely benefit us with AMN somewhere down the line."

Absolutely.