The FDA has granted both Orphan Drug Designation and Rare Pediatric Disease Designation for Tikun Therapeutics Inc.’s rAAV2-U1a-hELP1, a recombinant adeno-associated virus expressing the human ELP1 gene for the treatment of optic neuropathy in familial dysautonomia (FD), and for BPN-36964, a small molecule splicing modulator for systemic treatment of familial dysautonomia. FD is a severe orphan disease that is present at birth and characterized by sensory and autonomic dysfunction. Classic ha...