GTX-101

GTX-101 refers to two distinct investigational drugs in development for different indications: 1. **Acasti Pharma's GTX-101** is a novel, non-narcotic, topical bio-adhesive film-forming spray formulation of bupivacaine hydrochloride. It is designed for the relief of pain associated with postherpetic neuralgia (PHN), a persistent neuropathic pain following shingles. The drug is administered via a metered-dose spray that forms a thin, touch-free, non-greasy film on the skin, potentially providing rapid onset and continuous pain relief for up to eight hours. Its biphasic delivery mechanism may offer advantages over oral gabapentin and lidocaine patches by enabling faster onset and longer duration of action[2][4][6][8][9]. The active ingredient is bupivacaine hydrochloride, which acts as a local anesthetic by blocking sodium channels in neuronal membranes. 2. **GeneTx Biotherapeutics' GTX-101** is an antisense oligonucleotide therapy developed for Angelman syndrome—a rare neurogenetic disorder caused by loss of function of the maternally inherited UBE3A gene. This version of GTX-101 inhibits transcription of UBE3A-AS across the normally silent paternal allele, aiming to restore expression from this allele and address the underlying cause rather than just symptoms[1][5][7]. It has received orphan drug designation from the FDA.

Other names

bupivacaine hydrochloride metered dose spray