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.
Complement Therapeutics GmbH raised €72 million (US$79.4 million) in a series A round to move into the clinic a novel gene therapy for treating geographic atrophy secondary to dry age-related macular degeneration. It’s the largest series A round completed in Europe so far this year.
PYC Therapeutics Ltd.’s lead candidate, VP-001, is entering the clinic, and the RNA therapy could be the first potential treatment for retinitis pigmentosa type 11, which causes blindness that begins in childhood and ultimately leads to legal blindness by middle age.
PYC Therapeutics Ltd.’s lead candidate, VP-001, is entering the clinic, and the RNA therapy could be the first potential treatment for retinitis pigmentosa type 11, which causes blindness that begins in childhood and ultimately leads to legal blindness by middle age.
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.
Japan’s PMDA has approved Aurion Biotech Inc.’s cell therapy, Vyznova, for the treatment of bullous keratopathy of the cornea, making it the first-ever approval of a cell therapy to treat corneal endothelial disease.
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