Author Archives: Patricia Inacio PhD

FDA Grants Rare Pediatric Disease Designation to ABO-202 for Infantile Batten Disease

The U.S. Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Abeona Therapeutics’ investigational ABO-202 program being developed for Batten disease caused by mutations in the CLN1 gene, also known as infantile neuronal ceroid lipofuscinosis. ABO-202 is an adeno-associated virus (AAV)-based gene therapy (AAV-CLN1) where weakened…

Photosensitivity May Indicate CLN Type 2 Disease in Youngsters, Study Suggests

Early photosensitivity may be a hallmark of ceroid lipofuscinosis type 2 (CLN2) disease, according to the results of a small study. The study “Photosensitivity is an early marker of neuronal ceroid lipofuscinosis type 2 disease” was published in the journal Epilepsia. The neuronal ceroid lipofuscinoses (NCLs), or Batten disease, are a heterogeneous group of lysosomal storage disorders that…