Introduction:
Rare disease specialist SynaptixBio Ltd has received its second FDA orphan drug designation (ODD) for a subtype of TUBB4A leukodystrophy. This ODD allows research and development of a therapy for isolated hypomyelination, a less severe form of the disease. SynaptixBio is using antisense oligonucleotide (ASO) technology to target the mutated TUBB4A gene and suppress the production of toxic proteins associated with leukodystrophies.
- SynaptixBio received its second FDA orphan drug designation for a form of TUBB4A leukodystrophy called isolated hypomyelination.
- Using antisense oligonucleotide (ASO) technology, SynaptixBio is targeting the mutated TUBB4A gene to suppress the production of toxic proteins.
- ASOs can alter gene expression and help build normal myelin sheaths surrounding nerve fibers in the brain.
- The ODD will provide tax credits, grants, exemption from some regulatory requirements, and potential market exclusivity for SynaptixBio.
- SynaptixBio has also received a BioMedical Catalyst grant and additional investment to support its research and development efforts.
Conclusion:
SynaptixBio Ltd has received its second FDA orphan drug designation, this time for isolated hypomyelination, a form of TUBB4A leukodystrophy. The company is using antisense oligonucleotide technology to target the mutated gene and suppress the production of toxic proteins associated with the disease. This designation will provide tax credits, grants, and regulatory exemptions, allowing SynaptixBio to continue its research and development efforts for this rare disease therapy.
