Alternative splicing aids in maintaining the complexity of the transcriptome in higher organisms, and small nuclear RNAs (snRNAs), which are major components of the spliceosome, are processed by the integrator complex. Dysfunctions of integrator complex lead to immature snRNA production and may result in secondary defects in mRNA splicing.
A modification of the CRISPR technique has made it possible to restore vision in mouse models with retinitis pigmentosa (RP). Scientists at the Institute of Visual Neuroscience and Stem Cell Engineering of Wuhan University of Science and Technology developed a new gene-editing tool called PE(SpRY) to edit in vivo a mutation of enzyme phosphodiesterase 6B (PDE6β) and return its function.
Abeona Therapeutics Inc. has announced three investigational preclinical gene therapy product candidates from its ophthalmology program. The new AAV-based therapies use novel AAV capsids from Abeona's in-licensed AIM capsid library. Abeona intends to submit its first pre-IND application meeting request this month.
Praetego Inc. has been granted a US$300,000 phase I Small Business Technology Transfer Research (STTR) award by the National Eye Institute (NEI) to advance the company's lead candidate, PTG-630, into preclinical proof of concept in diabetic retinopathy.
Akari Therapeutics plc has advanced its preclinical development program for long-acting PAS-nomacopan for geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). The company has produced new and promising versions and progressed from lab scale to pre-GMP optimization, and is targeting an IND application to the FDA in the first half of next year.