“Growing Focus on Gene Therapy”
The neuronal ceroid-lipofuscinoses (NCL) market is expanding due to increased awareness and advancements in treatment options for this group of rare neurodegenerative disorders. One emerging trend in the market is the growing focus on gene therapy, which aims to address the underlying genetic causes of NCL rather than just managing symptoms. Companies such as REGENXBIO and Abeona Therapeutics are developing gene therapies targeting specific forms of the disease, offering the potential for more effective, long-term treatment solutions. This shift toward precision medicine reflects a broader trend in the rare disease market, where therapies are increasingly tailored to individual genetic profiles. The orphan drug designation and regulatory support for NCL treatments are also encouraging the development of innovative therapies.
