During the drug development process, drug manufacturers must seek out an Investigational New Drug application (IND), which allows the treatment to be transported across state lines. IND clearance also allows for researchers to hold clinical trials to evaluate the potential treatment. According to a recent press release, the FDA recently cleared an IND for FBX-101, a gene therapy developed by Forge Biologics Inc. (“Forge”) for patients with Krabbe disease. To learn more about FBX-101 clinical trials, click here.
Developed by Forge, FBX-101 is an adeno-associated viral (AAV) gene therapy. It is administered following hematopoietic stem cell transplant (HSCT). AskBio explains that AAV gene therapy works by:
[transforming] a naturally occurring virus into a delivery mechanism for gene therapy. The viral DNA is replaced with new DNA, and it becomes a precisely coded vector and is no longer considered a virus, as most of the viral components have been replaced.
Next, the AAV vector delivers functional genes to the body. GALC gene mutations cause Krabbe disease. Thus, in this case, FBX-101 delivers a functional GALC gene. Specifically, FBX-101 addresses both the central and peripheral nervous system. In animal models of Krabbe disease, FBX-101 improved quality of life and overall survival rates. Additionally, the treatment reduced demyelination.
In addition to the IND clearance, FBX-101 also received Institutional Biosafety Committee and Institutional Review Board approval.
According to KrabbeConnect, Krabbe disease is:
a rare genetic disorder, also known as globoid cell leukodystrophy. Krabbe disease is described as a severe neurological condition that results from the loss of the protective covering (myelin sheath) surrounding nerve cells.
Overall, Krabbe disease is considered a lysosomal storage disorder and a leukodystrophy. Because of the GALC gene mutation, patients with Krabbe disease do not produce enough of the galactocerebrosidase enzyme. Normally, this enzyme breaks down complex fatty substances in the body. However, without this enzyme, these substances build up, causing myelin degeneration.
Krabbe disease usually occurs in either early onset (“infantile”) or late-onset forms. The latter form is more severe and is typically fatal within 2-4 years of life. Those with late-onset Krabbe disease typically have a more unique or delayed disease progression. Only 10-15% of Krabbe disease diagnoses are late-onset.
Symptoms of Krabbe disease include:
- Developmental delays
- Hearing and vision loss
- Feeding difficulties
- Muscle spasms
- Frequent vomiting
- Muscle rigidity
Learn more about Krabbe disease here.