Atsena Therapeutics Inc.’s IND application for ATSN-201 has been cleared by the FDA enabling the company to initiate a phase I/II trial in patients with X-linked retinoschisis (XLRS) caused by pathogenic or likely pathogenic mutations in RS1.
Therini Bio Inc. has closed a US$36 million series A financing round that will support its work on developing fibrin-targeted therapies for diseases driven by chronic inflammation, including Alzheimer’s disease, multiple sclerosis and retinal diseases such as diabetic macular edema.
Ocugen Inc.’s OCU-410ST (AAV5-hRORA) has been awarded orphan drug designation by the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19) and cone-rod dystrophy 3 (CORD3) diseases.
In research presented this week at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, investigators from Life Biosciences Inc. reported using epigenetic reprogramming in a nonarteritic anterior ischemic optic neuropathy (NAION) model in nonhuman primates (NHPs).
Stoke Therapeutics Inc. has received clinical trial application (CTA) clearance from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study (OSPREY) of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA).
Nearly everyone with diabetes for >20 years will develop diabetic retinopathy or uveitis, which are collectively among the most common causes of premature blindness. Melanocortin is a short peptide that not only controls pigmentation, but that is most importantly expressed constitutively in the intraocular space where it serves essential functions suppressing inflammation in the retina, ultimately preventing blindness.
Perfuse Therapeutics Inc. has received FDA approval of its IND application for a first-in-human phase I/IIa study of PER-001 intravitreal implant in patients with glaucoma.
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.
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